A Multinational, Randomized, Double-blind, Placebo-contro... | NCT04221451 | Trialant
NCT04221451
Sponsor
Genzyme, a Sanofi Company
Status
Terminated
Last Update Posted
Jan 28, 2026Actual
Enrollment
75Actual
Phase
Phase 3
Conditions
Tay-Sachs Disease
Sandhoff Disease
Interventions
venglustat GZ402671
placebo
Countries
United States
Argentina
Brazil
Czechia
France
Germany
Italy
Japan
Portugal
Russia
Spain
Turkey (Türkiye)
United Kingdom
Protocol Section
Identification Module
NCT ID
NCT04221451
Obsolete or Duplicate NCT IDs
Not provided
Organization Study
EFC15299
Secondary IDs
ID
Type
Description
Link
U1111-1197-7905
Registry Identifier
ICTRP
EFC15299
Other Identifier
Sanofi Identifier
2019-002375-34
EudraCT Number
Brief Title
A Multinational, Randomized, Double-blind, Placebo-controlled Study to Assess the Efficacy, Pharmacodynamics, Pharmacokinetics, and Safety of Venglustat in Late-onset GM2
Official Title
A Multicenter, Multinational, Randomized, Double-blind, Placebo-controlled Study to Assess the Efficacy, Pharmacodynamics, Pharmacokinetics, Safety, and Tolerability of Venglustat in Late-onset GM2 Gangliosidosis (Tay-Sachs Disease and Sandhoff Disease) Together With a Separate Basket for Juvenile/Adolescent Late-onset GM2 Gangliosidosis and Ultra-rare Diseases Within the Same and Similar Glucosylceramide-based Sphingolipid Pathway
Acronym
AMETHIST
Organization
SanofiINDUSTRY
Status Module
Record Verification Date
Jan 2026
Overall Recruitment Status or Expanded Access Status
Terminated
Last Known Status
Not provided
Delayed Posting
Not provided
Why Stopped
Discontinued based on the absence of positive trends on clinical endpoints.
Expanded Access Info
No
Start Date
Jun 29, 2020Actual
Primary Completion Date
Dec 26, 2024Actual
Completion Date
Dec 26, 2024Actual
First Submitted Date
Jan 6, 2020
First Submission Date that Met QC Criteria
Jan 6, 2020
First Posted Date
Jan 9, 2020Actual
Results Waived
Not provided
Results First Submitted Date
Dec 3, 2025
Results First Submitted that Met QC Criteria
Jan 9, 2026
Results First Posted Date
Jan 28, 2026Actual
Certification/Extension (aka Delayed Results) First Submitted Date
Not provided
Certification/Extension First Submitted that Passed QC Review
Not provided
Certification/Extension First Posted Date
Not provided
Last Update Submitted Date
Jan 9, 2026
Last Update Posted Date
Jan 28, 2026Actual
Sponsor/Collaborators Module
Responsible Party, by Official Title
Sponsor
Lead Sponsor
Genzyme, a Sanofi CompanyINDUSTRY
Collaborators
Not provided
Oversight Module
Has Data Monitoring Committee (DMC)
Yes
Is FDA Regulated Drug
Yes
Is FDA Regulated Device
No
Is Unapproved Device
Not provided
Pediatric Postmarket Surveillance of a Device Product
Not provided
Product Exported from US
Not provided
FDAAA801 Violation
Not provided
Description Module
Brief Summary
Primary Objectives:
Primary population (adult participants with late-onset GM2 gangliosidosis): To assess the efficacy and pharmacodynamics (PD) of daily oral dosing of venglustat when administered over a 104-week period
Secondary population (participants with juvenile/adolescent late-onset GM2 gangliosidosis, GM1 gangliosidosis, saposin C deficiency, sialidosis type 1 or juvenile/adult galactosialidosis): To assess PD response (plasma and CSF GL-1 biomarker and disease specific biomarkers) of venglustat when administered once daily over a 104-week period
Secondary Objectives:
Primary population:
To assess the PD of daily oral dosing of venglustat and the effect of venglustat on selected performance test and scale over a 104-week period
To determine the safety and tolerability of venglustat when administered orally once daily over a 104-week period
To assess the pharmacokinetics (PK) of venglustat in plasma and cerebrospinal fluid (CSF)
Secondary population:
To assess the effect of venglustat on selected performance tests and scale over a 104-week period
To determine the safety and tolerability of venglustat when administered once daily over a 104-week period
To assess the PK of venglustat in plasma and CSF
To assess the acceptability and palatability of the venglustat tablet
Detailed Description
The total duration is up to approximately 223 weeks, including a 60-day screening period, a 104-week primary analysis treatment period, a 104-week open-label extension treatment period and a 6-week post-treatment safety observation period.
Conditions Module
Conditions
Tay-Sachs Disease
Sandhoff Disease
Keywords
Not provided
Design Module
Study Type
Interventional
Number of References to an Expanded Access Study
Not provided
Expanded Access Types
Not provided
Patient Registry
Not provided
Target Follow-Up Duration
Not provided
Phases
Phase 3
Interventional Study Design
Allocation
Biospecimen
No data available
No data is available for this block.
Enrollment
75Actual
Arms/Interventions Module
Arm Groups
Label
Type
Description
Intervention Names
GZ402671
Experimental
Primary population: participant will receive venglustat dose once daily during the primary analysis period (104 weeks) and the open-label extension period (104 weeks).
Secondary population: participant will receive venglustat at various doses once daily during the primary analysis period open-label (104 weeks) and the open-label extension period (104 weeks).
Drug: venglustat GZ402671
Placebo
Placebo Comparator
Primary population: participants will receive placebo once daily during the primary analysis period (104 weeks) and will receive venglustat dose once daily during the open-label extension period (104 weeks).
Drug: placebo
Interventions
Name
Type
Description
Arm Group Labels
Other Names
venglustat GZ402671
Drug
Pharmaceutical form: tablet
Route of administration: oral
GZ402671
Outcomes Module
Primary Outcomes
Measure
Description
Time Frame
PAP: PP: Percent Change From Baseline in CSF GM2 Biomarker to Week 104
Lumbar puncture was performed for obtaining CSF samples at specified timepoints to assess the presence of GM2 biomarker in PP in PAP. The baseline value was defined as the last available value before or equal to the first dose of study drug date in the PAP.
Baseline (Day 1) and Week 104
PAP: PP: Annualized Rate of Change From Baseline in the 9-HPT to Week 104
9-HPT is a test of upper extremity (arm and hand) function.Participant is seated at table with small, shallow container holding pegs and block containing 9 empty holes.On start command when stopwatch is started,participant picks up 9 pegs one at a time as quickly as possible, puts them in 9 holes and once they are in holes, removes them again as quickly as possible one at a time,replacing them into shallow container. Both dominant and non-dominant hands are tested twice (2 consecutive trials of dominant hand followed immediately by 2 consecutive trials of non-dominant hand).Total time (ranging up to 300 seconds):averaged, converted to reciprocals which were averaged and back-transformed to obtain global 9-HPT.Higher value on 9-HPT is indicative of higher disability.Mean annualized rate of change in 9-HPT was obtained from exponential transformation of mean slope of log-transformed 9-HPT.Baseline: last available value before or equal to the first dose of study drug date in the PAP.
Baseline (Day 1) and Week 104
PAP: SP: Percent Change From Baseline in Plasma and CSF Biomarkers (Glucosylceramide [GL-1] and GM2) to Week 104 in Juvenile/Adolescent Late-onset GM2 Gangliosidosis Participants
Plasma and CSF samples were collected at specified timepoints to assess the presence of GL-1 and GM2 biomarkers in juvenile/adolescent late-onset GM2 gangliosidosis participants of SP in PAP. The baseline value was defined as the last available value before or equal to the first dose of study drug date in the PAP.
Baseline (Day 1) and Week 104
PAP: SP: Percent Change From Baseline in Plasma and CSF GL-1 and GM1 Biomarkers to Week 104 in GM1 Gangliosidosis Participants
Secondary Outcomes
Measure
Description
Time Frame
PAP: PP: Absolute Change From Baseline in CSF GM2 Biomarker to Week 104
Lumbar puncture was performed for obtaining CSF samples at specified timepoints to assess the presence of GM2 biomarker in PP in PAP. The baseline value was defined as the last available value before or equal to the first dose of study drug date in the PAP.
Baseline (Day 1) and Week 104
Other Outcomes
Not provided
Eligibility Module
Eligibility Criteria
Inclusion criteria :
Primary population and adult secondary population: age ≥ 18 years
Juvenile/adolescent secondary population: 2 ≥ age < 18 years with weight ≥ 10 kg
Participants with a diagnosis of late onset GM2 gangliosidosis (Tay-Sachs disease and Sandhoff disease) caused by genetic β-hexosaminidase deficiency resulting from mutations in the HEXA or HEXB genes (primary population only); a secondary population will enroll patients with diagnosis of juvenile/adolescent GM2 gangliosidosis, GM1 gangliosidosis, saposin C deficiency, sialidosis type 1 or juvenile adult galactosialidosis
For primary population, the participant has the ability to perform the 9-HPT at the screening visit in < = 240 seconds for the 2 consecutive trials of the dominant hand and the 2 consecutive trials of the nondominant hand.
Participants with a history of seizures well controlled by medication other than strong or moderate inducer or inhibitor of CYP3A4
Participant is cooperative, able to ingest oral medication, willing to travel to a study site (if applicable), and able to comply with all aspects of the study, including all assessments, according to the Investigator's judgement
Signed written informed assent/consent
Contraception for sexually active male participants or female patient; not pregnant or breastfeeding; no sperm donating for male participant
Exclusion criteria:
Participant has clinical features of Tay-Sachs or Sandhoff disease, not caused by β-hexosaminidase deficiency resulting from mutations in the HEXA or HEXB genes and/or is without clinical features
For primary population and participants with juvenile/adolescent late onset GM2 gangliosidosis and GM1 gangliosidosis, the participant cannot understand and perform all age-appropriate study assessments with the exception of 25FWT and PROs.
Relevant medical disorders that would compromise his/her safety
Documented diagnosis of hepatitis B, C, human immunodeficiency virus 1 or 2
World Health Organization (WHO) grade >= 2 cortical cataract or a grade >= 2 posterior subcapsular cataract; patients with nuclear cataracts will be accepted
Participant who requires invasive ventilatory support
Current treatment by anticoagulants, cataractogenic medications or any medications that may worsen the vision of patient with cataract
Previous treatment with substrate reduction therapy (SRT) within 3 months prior to study enrollment, strong or moderate inducers or inhibitors of CYP3A4 within 14 days or 5 half-lives prior to enrollment. This also includes the consumption of grapefruit, grapefruit juice or grapefruit products within 72hrs prior to starting investigational medicinal product (IMP) administration.
Current participation in another study
Use of investigational medicinal product (IMP) within 3 months or 5 half-lives, whichever is longer, before study enrollment (for N-acetyl-leucine, within 5 half-lives before study enrollment).
Liver enzymes (alanine aminotransferase [ALT]/aspartate aminotransferase [AST]) or total bilirubin > 2 x the upper limite of normal (ULN) at the time of screening unless the participant has the diagnosis of Gilbert syndrome and maintains a level of bilirubin < 5 mg/dl and direct bilirubin < 20% (1 mg/dl) of total bilirubin level
Renal insufficiency is defined by estimated glomerular filtration rate (eGFR) < 30 mL/min/1.73m2 at the screening visit
The above information is not intended to contain all considerations relevant to a patient's potential participation in a clinical trial.
Accepts Healthy Volunteers
Yes
Sex
All
Sex/Gender Based
Not provided
Sex/Gender Description
Not provided
Minimum Age
2 Years
Maximum Age
Not provided
Standard Ages
ChildAdultOlder Adult
Study Population
Not provided
Sampling Method
Not provided
Contacts/Locations Module
Central Contacts
Not provided
Overall Officials
Name
Affiliation
Role
Clinical Sciences & Operations
Sanofi
Study Director
Locations
Facility
Status
City
State
ZIP
Country
Contacts
Ataxia Center and HD Center of Excellence, 300 UCLA Med Plaza, Suite B200 - Investigational site number 8400004
Nicoli ER, Annunziata I, d'Azzo A, Platt FM, Tifft CJ, Stepien KM. GM1 Gangliosidosis-A Mini-Review. Front Genet. 2021 Sep 3;12:734878. doi: 10.3389/fgene.2021.734878. eCollection 2021.
Qualified researchers may request access to patient level data and related study documents including the clinical study report, study protocol with any amendments, blank case report form, statistical analysis plan, and dataset specifications. Patient level data will be anonymized and study documents will be redacted to protect the privacy of trial participants. Further details on Sanofi's data sharing criteria, eligible studies, and process for requesting access can be found at: https://vivli.org
Types
Not provided
Time Frame
Not provided
Access Criteria
Not provided
URL
Not provided
Results Section
Participant Flow Module
Pre-assignment Details
In PAP, 59 participants in the PP (adult participants with diagnosis of late-onset disialotetrahexosylganglioside [GM2] gangliosidosis) and 16 participants in the SP (juvenile/adolescent participants with diagnosis of late-onset GM2 gangliosidosis, monosialotetrahexosylganglioside [GM1] gangliosidosis, saposin C deficiency, sialidosis type 1, or juvenile/adult galactosialidosis) were treated. Randomization for PP was stratified on participant's ability to walk at baseline visit (yes/no).
Recruitment Details
This study consisted of 2 periods: primary analysis period (PAP) & open-label extension (OLE) period. In PAP, primary population (PP) was randomized in a 2:1 ratio to receive either venglustat or placebo in double-blind manner. Secondary population (SP) received OL venglustat. Post completion of PAP, eligible participants entered OLE in which all participants received OL venglustat. The study was early terminated based on absence of positive trends on clinical endpoints; no safety concerns.
Type of Units Analyzed
Not provided
Arm/Group Information
ID
Title
Description
FG000
PAP: PP: Placebo
Participants in PP received placebo matched to venglustat oral tablet once daily for 104 weeks in a double-blind manner in PAP.
FG001
PAP: PP: Venglustat
Participants in PP received venglustat 15 milligrams (mg) oral tablet once daily for 104 weeks in a double-blind manner in PAP.
Plasma and CSF samples were collected at specified timepoints to assess the presence of GL-1 and GM1 biomarkers in GM1 gangliosidosis participants of SP in PAP. The baseline value was defined as the last available value before or equal to the first dose of study drug date in the PAP.
Baseline (Day 1) and Week 104
PAP: SP: Percent Change From Baseline in Plasma and CSF GL-1, GM2 and Monosialodihexosylganglioside (GM3) Biomarkers to Week 104 in Sialidosis Type 1 Participant
Plasma and CSF samples were collected at specified timepoints to assess the presence of GL-1, GM2 and GM3 biomarkers in sialidosis type 1 participant of SP in PAP. The baseline value was defined as the last available value before or equal to the first dose of study drug date in the PAP. Analysis was planned to be performed on sialidosis type 1 participant from secondary PD population (all enrolled participants with this clinical diagnosis who received at least 1 dose of study drug and who had baseline and post-baseline assessments of PD).
Baseline (Day 1) and Week 104
PAP: SP: Percent Change From Baseline in Plasma and CSF GL-1, GM1 and GM3 Biomarkers to Week 104 in Juvenile/Adult Galactosialidosis Participants
Plasma and CSF samples were collected at specified timepoints to assess the presence of GL-1, GM1 and GM3 biomarkers in juvenile/adult galactosialidosis participants of SP in PAP. The baseline value was defined as the last available value before or equal to the first dose of study drug date in the PAP. Analysis was planned to be performed on juvenile/adult galactosialidosis participant from secondary PD population which included all enrolled participants with this clinical diagnosis who received at least 1 dose of study drug and who had baseline and post-baseline assessments of PD.
Baseline (Day 1) and Week 104
PAP: SP: Percent Change From Baseline in Plasma and CSF GL-1 Biomarker to Week 104 in Saposin C Deficiency Participants
Plasma and CSF samples were planned to be collected at specified timepoints to assess the presence of GL-1 biomarkers in saposin C deficiency participants of SP in PAP. The baseline value was defined as the last available value before or equal to the first dose of study drug date in the PAP.
Baseline (Day 1) to Week 104
PAP: PP and SP: Change From Baseline in 25-foot Walk Test (25FWT) to Week 104
The 25FWT is a quantitative mobility and leg function performance test based on a timed 25-foot walk. The participant is directed to one end of a clearly marked 25-foot course and is instructed to walk 25 feet as quickly and safely as possible for 2 trials (can use assistive devices). The time is calculated from the initiation of the instruction to start and ends when the participant has reached the 25-foot mark. The amount of time (in seconds) to walk 25 feet is recorded (ranging up to 180 seconds). The 25FWT score is defined as the average of 2 trials. A higher value on the 25FWT is indicative of higher disability. The baseline value was defined as the last available value before or equal to the first dose of study drug date in the PAP.
Baseline (Day 1) and Week 104
PAP: PP and SP: Change From Baseline in the Neurological Examination of the Friedreich's Ataxia Rating Scale (FARS) (FARS-neuro) to Week 104
The FARS-neuro includes 23 items and is composed of 4 sections that assesses different neurological faculties: bulbar activity (4 items), upper limb coordination (5 items assessed right and left side), lower limb coordination (2 items assessed right and left side), peripheral nervous system (5 items assessed right and left side) and upright stability (7 items). Total score is calculated as the sum of scores on items of this section and ranges from 0 to 117; mean is presented. Higher value indicates higher disability. Baseline=last available value before or equal to first dose of study drug date in PAP. PP: primary efficacy population: all randomized participants with diagnosis of late-onset GM2 gangliosidosis aged >=18 years. SP: secondary efficacy population: all enrolled participants with diagnosis of juvenile/adolescent late-onset GM2 gangliosidosis, GM1 gangliosidosis, saposin C deficiency, sialidosis type 1, or juvenile/adult galactosialidosis.
Baseline (Day 1) and Week 104
PAP: PP and SP: Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Treatment-Emergent Serious Adverse Events (TESAEs)
An AE was any untoward medical occurrence in a participant or clinical study participant, temporally associated with the use of study drug, whether or not considered related to the study drug. SAE was any untoward medical occurrence that at any dose: resulted in death, was life-threatening, required inpatient hospitalization or prolongation of existing hospitalization, resulted in persistent disability/incapacity, was a congenital anomaly/birth defect or was an important medical event. TEAE were AEs that developed, worsened or became serious during the TE period.
From first dose of study drug (Day 1) up to end of PAP, 104 weeks
PAP: PP: Plasma Venglustat Concentration
Plasma samples were collected at specified timepoints to obtain venglustat concentrations for PP in PAP.
Pre-dose (0 hour) and 0.5, 3, 8, 12 and 24 hours post-dose at Week 12
PAP: SP: Plasma Venglustat Concentration
Plasma samples were collected at specified timepoints to obtain venglustat concentration for SP in PAP. Data is presented by dose level for all diseases combined for SP in PAP.
Pre-dose (0 hour) and 0.5, 3, 8, 12 and 24 hours post-dose at Week 12
PAP: PP and SP: CSF Venglustat Concentration
CSF samples were collected via lumbar puncture at Week 104 to obtain venglustat concentrations for PP and SP in PAP. For SP: data is presented by dose level for all diseases combined. Only those participants with data collected at specified timepoints are reported.
Week 104
PAP: PP and SP: Maximum Plasma Concentration Observed (Cmax) of Venglustat
Plasma samples were collected at specified timepoints to obtain Cmax of venglustat. The mean of Cmax, irrespective of the timepoint where the patient-individual Cmax value was observed is presented as opposed to endpoint 13 wherein mean of plasma venglustat concentration at each specified timepoint is presented. For SP: data is presented by dose level for all diseases combined. Only those participants with data collected at specified timepoints are reported.
Pre-dose (0 hour) and 0.5, 3, 8, 12 and 24 hours post-dose at Week 12
PAP: PP and SP: Time to Reach Maximum Plasma Concentration (Tmax) of Venglustat
Plasma samples were collected at specified timepoints to obtain tmax of venglustat. For SP: data is presented by dose level for all diseases combined. Only those participants with data collected at specified timepoints are reported.
Pre-dose (0 hour) and 0.5, 3, 8, 12 and 24 hours post-dose at Week 12
PAP: PP and SP: Area Under the Plasma Concentration Versus Time Curve Calculated Over a Predefined Time Period 0 to 24 Hours (AUC0-24) of Venglustat
Plasma samples were collected at specified timepoints to obtain AUC0-24 of venglustat. For SP: data is presented by dose level for all diseases combined. Only those participants with data collected at specified timepoints are reported.
Pre-dose (0 hour) and 0.5, 3, 8, 12 and 24 hours post-dose at Week 12
PAP: SP: Percent Change From Baseline in the 9-HPT to Week 104
9-HPT is a test of upper extremity (arm and hand) function.Participant is seated at table with small, shallow container holding pegs & block containing 9 empty holes.On start command (stopwatch started),participant picks up 9 pegs one at a time as quickly as possible, puts them in 9 holes & once they are in holes, removes them again as quickly as possible one at a time,replacing them into shallow container,2 consecutive trials of dominant hand followed immediately by 2 consecutive trials of non-dominant hand.Total time (ranging up to 300 seconds):averaged, converted to reciprocals which were averaged and back-transformed to obtain global 9-HPT.Higher value indicates higher disability.Baseline: last available value before or equal to first dose of study drug date in PAP.Secondary efficacy population:all enrolled participants with diagnosis of juvenile/adolescent late-onset GM2 gangliosidosis,GM1 gangliosidosis,saposin C deficiency,sialidosis type 1 or juvenile/adult galactosialidosis.
Baseline (Day 1) and Week 104
PAP: SP: Number of Participants With >=80% Compliance as Per Method of Intake
For the secondary pediatric population, the acceptability and palatability of venglustat tablets was assessed through the route of venglustat administration collected in the electronic case report form and study drug compliance throughout PAP. The assessment was based on tablet always swallowed as whole or chewed and swallowed at least once. Number of participants with >=80% compliance for each is presented here.
Baseline (Day 1) to Week 104
Emory Genetics - Investigational site number 8400006
Atlanta
Georgia
30322
United States
NIH National Human Genome Research Institute - 10 Center Dr - Bldg. 10 CRC3-2551 MSC 1205 - Investigational site number 8400005
Bethesda
Maryland
20892
United States
Massachusetts General Hospital - Charles River Plaza 175 Cambridge St. Ste 340 - Investigational site number 8400002
Boston
Massachusetts
02114
United States
NYU Langone - 550 First Avenue-Investigational site number 8400001
New York
New York
10016
United States
Hospital Universitario Austral Pilar, Buenos Aires_Unidad de Investigación Clínica_investigational site number 0320002
Pilar
Buenos Aires
B1629ODT
Argentina
Clínica Universitaria Reina Fabiola Córdoba_investigational site number 0320001
Córdoba
X5004FHP
Argentina
Hospital de Clinicas de Porto Alegre _investigational site number 0760001
Porto Alegre
Rio Grande do Sul
90035 003
Brazil
Vseobecna Fakultni Nemocnice V Praze Metabolicke centrum U Nemocnice 2_investigational site number 2030001
Prague
12808
Czechia
APHP - Centre Hospitalier la Pitié Salpetrière, Service de Neurologie, 47-83 Boulevard De l'Hôpital_Investigational site number 2500001
Paris
75013
France
Universitätsklinikum der Justus-Liebig-Universität Gießen Zentrum für Kinderheilkunde und Jugendmedizin Feulgenstraße 10-12_investigational site number 2760001
Giessen
35390
Germany
Investigational Site Number : 3800001
Milan
20133
Italy
Japanese Red Cross Akita Hospital 222-1 Nawashirosawa, Kamikitatesaruta_investigational site number 3920001
Akita
Akita
010-1495
Japan
Tohoku Medical and Pharmaceutical University Hospital_investigation site number 3920002
Sendai
Miyagi
983-8512
Japan
Centro Hospitalar Universitário Lisboa Norte- Hospital Santa Maria Avenida Professor Egas Moniz_investigational site number 6200002
Lisbon
1649-035
Portugal
Research Center Of Neurology 80, Volokolamskoye shosse_investigational site number 6430001
Moscow
125367
Russia
Hospital Universitari de Bellvitge. Feixa Llarga, S / N_investigational site number 7240004
L'Hospitalet de Llobregat
Barcelona [Barcelona]
08907
Spain
Hospital Maternoinfantil Sant Joan de Déu Paseo de Sant Joan de Déu, 2_investigational site number 7240001
Esplugues de Llobregat
Catalunya [Cataluña]
08950
Spain
Hospital Clínico Universitario de Santiago-CHUS. Travesia de Chopuana, s/n_investigational site number 7240002
Santiago de Compostela
Galicia [Galicia]
15706
Spain
Gazi Universitesi Tip Fakultesi Emniyet Street Mevlana Blv Besevler Yenimahalle_investigational site number 7920001
Ankara
06500
Turkey (Türkiye)
Cambridge University Hospitals NHS Foundation Trust Addenbrookes Hospital Hills Road_investigational site number 8260001
Cambridge
Cambridgeshire
CB2 OQQ
United Kingdom
Investigational Site Number : 8260003
Manchester
M13 9WL
United Kingdom
Salford Royal NHS Foundation Trust Salford Royal Hospital Stott Lane_investigational site number 8260002
Salford
M6 8HD
United Kingdom
FG002
PAP: SP: Venglustat
Participants in SP received venglustat oral tablet once daily based on the body weight for 104 weeks in an open-label manner in PAP as follows:
15 mg if >=50 kilograms [kg], 12 mg if 30 kg to <50 kg, 6 mg if 15 kg to <30 kg and 4 mg if 10 kg to <15 kg.
FG003
OLE: PP: Delayed Venglustat
Participants in PP who previously received placebo in PAP received venglustat 15 mg oral tablet once daily for 104 weeks in an open-label manner in OLE.
FG004
OLE: PP: Early Venglustat
Participants in PP who previously received venglustat in PAP continued to receive venglustat 15 mg oral tablet once daily for 104 weeks in an open-label manner in OLE.
FG005
OLE: SP: Venglustat
Participants in SP who previously received venglustat in PAP continued to receive venglustat oral tablet once daily based on the body weight for 104 weeks in an open-label manner in OLE as follows:
15 mg if >=50 kg, 12 mg if 30 kg to <50 kg, 6 mg if 15 kg to <30 kg and 4 mg if 10 kg to <15 kg.
FG00019 subjects
FG00140 subjects
FG00216 subjects
FG0030 subjects
FG0040 subjects
FG0050 subjects
COMPLETED
PAP period
FG00017 subjects
FG00135 subjects
FG00214 subjects
FG0030 subjects
FG0040 subjects
FG0050 subjects
NOT COMPLETED
FG0002 subjects
FG0015 subjects
FG0022 subjects
FG0030 subjects
FG0040 subjects
FG0050 subjects
Type
Comment
Reasons
Withdrawal by Subject
FG0002 subjects
FG0015 subjects
FG0021 subjects
FG0030 subjects
FG0040 subjects
FG0050 subjects
Other
FG0000 subjects
FG0010 subjects
FG0021 subjects
FG0030 subjects
FG004
OLE (104 Weeks): From 104 to 208 Weeks
Type
Comment
Milestone Data
STARTED
Only eligible participants entered the OLE period
FG0000 subjects
FG0010 subjects
FG0020 subjects
FG00316 subjects
FG00434 subjects
FG00514 subjects
COMPLETED
OLE period
FG0000 subjects
FG0010 subjects
FG0020 subjects
FG0030 subjects
NOT COMPLETED
FG0000 subjects
FG0010 subjects
FG0020 subjects
FG00316 subjects
FG004
Type
Comment
Reasons
Withdrawal by Subject
FG0000 subjects
FG0010 subjects
FG0020 subjects
FG003
Randomized population included all participants from screened population who were allocated to a randomized study drug by interactive response technology regardless of whether the study drug was received or not.
Type of Units Analyzed
Not provided
Arm/Group Information
ID
Title
Description
BG000
PAP: PP: Placebo
Participants in PP received placebo matched to venglustat oral tablet once daily for 104 weeks in a double-blind manner in PAP.
BG001
PAP: PP: Venglustat
Participants in PP received venglustat 15 mg oral tablet once daily for 104 weeks in a double-blind manner in PAP.
BG002
PAP: SP: Venglustat
Participants in SP received venglustat oral tablet once daily based on the body weight for 104 weeks in an open-label manner in PAP as follows:
15 mg if >=50 kg, 12 mg if 30 kg to <50 kg, 6 mg if 15 kg to <30 kg and 4 mg if 10 kg to <15 kg.
BG003
Total
Total of all reporting groups
Denominators
Units
Counts
Participants
BG00019
BG00140
BG00216
BG00375
Baseline Measures
Title
Description
Population Description
Parameter Type
Dispersion Type
Unit of Measure
Calculate Percentage
Denominator Units Selected
Denominators
Classes
Age, Continuous
Mean
Standard Deviation
years
Title
Denominators
Categories
ParticipantsBG00019
ParticipantsBG00140
ParticipantsBG00216
ParticipantsBG003
Sex: Female, Male
Count of Participants
Participants
Title
Denominators
Categories
ParticipantsBG00019
ParticipantsBG00140
ParticipantsBG002
Race (NIH/OMB)
Count of Participants
Participants
Title
Denominators
Categories
ParticipantsBG00019
ParticipantsBG00140
ParticipantsBG002
PP: Cerebrospinal fluid (CSF) GM2 biomarker
Lumbar puncture was performed for obtaining CSF samples at baseline to assess the presence of GM2 biomarker in PP in PAP.
PAP: Primary pharmacodynamic (PD) population included all randomized participants with diagnosis of late-onset GM2 gangliosidosis aged >=18 years who received at least 1 dose of study drug and who had baseline and post-baseline assessments of PD. Only those PP participants with data collected at baseline are reported.
Mean
Standard Deviation
nanogram/milliliter (ng/mL)
Title
Denominators
Categories
ParticipantsBG00018
ParticipantsBG001
PP: 9-hole peg Test (9-HPT)
9-HPT is a test of upper extremity (arm and hand) function.Participant is seated at table with small, shallow container holding pegs & block containing 9 empty holes.When stopwatch is started,participant picks up 9 pegs one at a time as quickly as possible,puts them in 9 holes and removes them again as quickly as possible one at a time,replacing them into shallow container; 2 trials for each hand. Total time (ranging up to 300 seconds):averaged, converted to reciprocals which were averaged and back-transformed to obtain global 9-HPT.Higher value on 9-HPT is indicative of higher disability.
Only PP participants are included for this analysis.
Mean
Standard Deviation
seconds
Title
Denominators
Categories
ParticipantsBG00019
ParticipantsBG001
Clinical diagnosis of SP
Participants with clinical diagnosis of juvenile/adolescent GM2 gangliosidosis, monosialotetrahexosylganglioside (GM1) gangliosidosis, sialidosis type 1, juvenile/adult galactosialidosis, or saposin C deficiency were referred to as SP.
Only SP participants are included for this analysis.
Count of Participants
Participants
Title
Denominators
Categories
ParticipantsBG0000
ParticipantsBG0010
ParticipantsBG002
Type
Title
Description
Population Description
Reporting Status
Anticipated Posting Date
Parameter Type
Dispersion Type
Unit of Measure
Calculate Percentage
Time Frame
Units Analyzed
Denominator Units Selected
Arm/Group Information
Denominators
Classes
Analyses
Primary
PAP: PP: Percent Change From Baseline in CSF GM2 Biomarker to Week 104
Lumbar puncture was performed for obtaining CSF samples at specified timepoints to assess the presence of GM2 biomarker in PP in PAP. The baseline value was defined as the last available value before or equal to the first dose of study drug date in the PAP.
PAP: Primary PD population included all randomized participants with diagnosis of late-onset GM2 gangliosidosis aged >=18 years who received at least 1 dose of study drug and who had baseline and post-baseline assessments of PD. Only those participants with data collected at specified timepoints are reported.
Posted
Mean
Standard Error
percent change
Baseline (Day 1) and Week 104
ID
Title
Description
OG000
PAP: PP: Placebo
Participants in PP received placebo matched to venglustat oral tablet once daily for 104 weeks in a double-blind manner in PAP.
OG001
PAP: PP: Venglustat
Participants in PP received venglustat 15 mg oral tablet once daily for 104 weeks in a double-blind manner in PAP.
Units
Counts
Participants
OG00016
OG00131
Title
Denominators
Categories
Title
Measurements
OG000-11.33± 4.23
OG001-47.57± 3.04
Group IDs
Group Description
Statistical Method
Statistical Comment
P-Value
P-Value Comment
Parameter Type
Parameter Value
Dispersion Type
Dispersion Value
Confidence Interval Sides
Confidence Interval %
CI Lower Limit
CI Upper Limit
CI Lower Limit Comment
CI Upper Limit Comment
Estimate Comment
Tested Non-Inferiority
Non-Inferiority Type
Non-Inferiority Comment
Other Analysis Description
OG000
OG001
Mean percent change from baseline obtained from robust analysis of covariance (ANCOVA) using Huber's M estimation, including the fixed categorical effects of treatment (venglustat versus placebo) and the continuous fixed covariate of baseline value.
Robust ANCOVA
<0.0001
Mean difference
-36.24
Standard Error of the Mean
5.20
2-Sided
90
-44.80
-27.68
Superiority
Primary
PAP: PP: Annualized Rate of Change From Baseline in the 9-HPT to Week 104
9-HPT is a test of upper extremity (arm and hand) function.Participant is seated at table with small, shallow container holding pegs and block containing 9 empty holes.On start command when stopwatch is started,participant picks up 9 pegs one at a time as quickly as possible, puts them in 9 holes and once they are in holes, removes them again as quickly as possible one at a time,replacing them into shallow container. Both dominant and non-dominant hands are tested twice (2 consecutive trials of dominant hand followed immediately by 2 consecutive trials of non-dominant hand).Total time (ranging up to 300 seconds):averaged, converted to reciprocals which were averaged and back-transformed to obtain global 9-HPT.Higher value on 9-HPT is indicative of higher disability.Mean annualized rate of change in 9-HPT was obtained from exponential transformation of mean slope of log-transformed 9-HPT.Baseline: last available value before or equal to the first dose of study drug date in the PAP.
PAP: Primary efficacy population included all randomized participants with diagnosis of late-onset GM2 gangliosidosis aged >=18 years. Only those participants with data collected at specified timepoints are reported.
Posted
Mean
Standard Error
percent per year
Baseline (Day 1) and Week 104
ID
Title
Description
OG000
PAP: PP: Placebo
Participants in PP received placebo matched to venglustat oral tablet once daily for 104 weeks in a double-blind manner in PAP.
OG001
PAP: PP: Venglustat
Primary
PAP: SP: Percent Change From Baseline in Plasma and CSF Biomarkers (Glucosylceramide [GL-1] and GM2) to Week 104 in Juvenile/Adolescent Late-onset GM2 Gangliosidosis Participants
Plasma and CSF samples were collected at specified timepoints to assess the presence of GL-1 and GM2 biomarkers in juvenile/adolescent late-onset GM2 gangliosidosis participants of SP in PAP. The baseline value was defined as the last available value before or equal to the first dose of study drug date in the PAP.
PAP: Analysis was performed on juvenile/adolescent late-onset GM2 gangliosidosis participants from secondary PD population which included all enrolled participants with this clinical diagnosis who received at least 1 dose of study drug and who had baseline and post-baseline assessments of PD.
Posted
Mean
Standard Deviation
percent change
Baseline (Day 1) and Week 104
ID
Title
Description
OG000
PAP: SP: Venglustat
Participants in SP received venglustat oral tablet once daily based on the body weight for 104 weeks in an open-label manner in PAP as follows:
15 mg if >=50 kg, 12 mg if 30 kg to <50 kg, 6 mg if 15 kg to <30 kg and 4 mg if 10 kg to <15 kg.
Units
Counts
Participants
Primary
PAP: SP: Percent Change From Baseline in Plasma and CSF GL-1 and GM1 Biomarkers to Week 104 in GM1 Gangliosidosis Participants
Plasma and CSF samples were collected at specified timepoints to assess the presence of GL-1 and GM1 biomarkers in GM1 gangliosidosis participants of SP in PAP. The baseline value was defined as the last available value before or equal to the first dose of study drug date in the PAP.
PAP: Analysis was performed on GM1 gangliosidosis participants from secondary PD population which included all enrolled participants with this clinical diagnosis who received at least 1 dose of study drug and who had baseline and post-baseline assessments of PD. Only those participants with data collected at specified timepoints are reported.
Posted
Mean
Standard Deviation
percent change
Baseline (Day 1) and Week 104
ID
Title
Description
OG000
PAP: SP: Venglustat
Participants in SP received venglustat oral tablet once daily based on the body weight for 104 weeks in an open-label manner in PAP as follows:
15 mg if >=50 kg, 12 mg if 30 kg to <50 kg, 6 mg if 15 kg to <30 kg and 4 mg if 10 kg to <15 kg.
Units
Counts
Participants
OG000
Primary
PAP: SP: Percent Change From Baseline in Plasma and CSF GL-1, GM2 and Monosialodihexosylganglioside (GM3) Biomarkers to Week 104 in Sialidosis Type 1 Participant
Plasma and CSF samples were collected at specified timepoints to assess the presence of GL-1, GM2 and GM3 biomarkers in sialidosis type 1 participant of SP in PAP. The baseline value was defined as the last available value before or equal to the first dose of study drug date in the PAP. Analysis was planned to be performed on sialidosis type 1 participant from secondary PD population (all enrolled participants with this clinical diagnosis who received at least 1 dose of study drug and who had baseline and post-baseline assessments of PD).
Due to the rarity of the disease and the specificity of the endpoint, reporting individual-level data would risk compromising participant privacy and confidentiality. As presented in baseline characteristics, only 1 participant with sialidosis type 1 was enrolled in the study.
Posted
Mean
Standard Deviation
percent change
Baseline (Day 1) and Week 104
ID
Title
Description
OG000
PAP: SP: Venglustat
Participants in SP received venglustat oral tablet once daily based on the body weight for 104 weeks in an open-label manner in PAP as follows:
15 mg if >=50 kg, 12 mg if 30 kg to <50 kg, 6 mg if 15 kg to <30 kg and 4 mg if 10 kg to <15 kg.
Units
Counts
Participants
Primary
PAP: SP: Percent Change From Baseline in Plasma and CSF GL-1, GM1 and GM3 Biomarkers to Week 104 in Juvenile/Adult Galactosialidosis Participants
Plasma and CSF samples were collected at specified timepoints to assess the presence of GL-1, GM1 and GM3 biomarkers in juvenile/adult galactosialidosis participants of SP in PAP. The baseline value was defined as the last available value before or equal to the first dose of study drug date in the PAP. Analysis was planned to be performed on juvenile/adult galactosialidosis participant from secondary PD population which included all enrolled participants with this clinical diagnosis who received at least 1 dose of study drug and who had baseline and post-baseline assessments of PD.
Due to the rarity of the disease and the specificity of the endpoint, reporting individual-level data would risk compromising participant privacy and confidentiality. As presented in baseline characteristics, only 1 participant with juvenile/adult galactosialidosis was enrolled in the study.
Posted
Mean
Standard Deviation
percent change
Baseline (Day 1) and Week 104
ID
Title
Description
OG000
PAP: SP: Venglustat
Participants in SP received venglustat oral tablet once daily based on the body weight for 104 weeks in an open-label manner in PAP as follows:
15 mg if >=50 kg, 12 mg if 30 kg to <50 kg, 6 mg if 15 kg to <30 kg and 4 mg if 10 kg to <15 kg.
Units
Counts
Primary
PAP: SP: Percent Change From Baseline in Plasma and CSF GL-1 Biomarker to Week 104 in Saposin C Deficiency Participants
Plasma and CSF samples were planned to be collected at specified timepoints to assess the presence of GL-1 biomarkers in saposin C deficiency participants of SP in PAP. The baseline value was defined as the last available value before or equal to the first dose of study drug date in the PAP.
PAP: Analysis was planned to be performed on saposin C deficiency participants from secondary PD population which included all enrolled participants with this clinical diagnosis who received at least 1 dose of study drug and who had baseline and post-baseline assessments of PD. As presented in baseline characteristics, there was no participant enrolled with Saposin C deficiency.
Posted
Baseline (Day 1) to Week 104
ID
Title
Description
OG000
PAP: SP: Venglustat
Participants in SP received venglustat oral tablet once daily based on the body weight for 104 weeks in an open-label manner in PAP as follows:
15 mg if >=50 kg, 12 mg if 30 kg to <50 kg, 6 mg if 15 kg to <30 kg and 4 mg if 10 kg to <15 kg.
Units
Counts
Participants
OG000
Secondary
PAP: PP: Absolute Change From Baseline in CSF GM2 Biomarker to Week 104
Lumbar puncture was performed for obtaining CSF samples at specified timepoints to assess the presence of GM2 biomarker in PP in PAP. The baseline value was defined as the last available value before or equal to the first dose of study drug date in the PAP.
PAP: Primary PD population included all randomized participants with diagnosis of late-onset GM2 gangliosidosis aged >=18 years who received at least 1 dose of study drug and who had baseline and post-baseline assessments of PD. Only those participants with data collected at specified timepoints are reported.
Posted
Mean
Standard Error
ng/mL
Baseline (Day 1) and Week 104
ID
Title
Description
OG000
PAP: PP: Placebo
Participants in PP received placebo matched to venglustat oral tablet once daily for 104 weeks in a double-blind manner in PAP.
OG001
PAP: PP: Venglustat
Participants in PP received venglustat 15 mg oral tablet once daily for 104 weeks in a double-blind manner in PAP.
Units
Counts
Participants
Secondary
PAP: PP and SP: Change From Baseline in 25-foot Walk Test (25FWT) to Week 104
The 25FWT is a quantitative mobility and leg function performance test based on a timed 25-foot walk. The participant is directed to one end of a clearly marked 25-foot course and is instructed to walk 25 feet as quickly and safely as possible for 2 trials (can use assistive devices). The time is calculated from the initiation of the instruction to start and ends when the participant has reached the 25-foot mark. The amount of time (in seconds) to walk 25 feet is recorded (ranging up to 180 seconds). The 25FWT score is defined as the average of 2 trials. A higher value on the 25FWT is indicative of higher disability. The baseline value was defined as the last available value before or equal to the first dose of study drug date in the PAP.
PAP PP:primary efficacy population included all randomized participants with diagnosis of late-onset GM2 gangliosidosis aged >=18 years.PAP SP: secondary efficacy population included all enrolled participants with a clinical diagnosis of juvenile/adolescent late-onset GM2 gangliosidosis, GM1 gangliosidosis, saposin C deficiency, sialidosis type 1, or juvenile/adult galactosialidosis.Only those participants with ability to walk at baseline and data collected at specified timepoints are reported.
Posted
Mean
Standard Deviation
seconds
Baseline (Day 1) and Week 104
ID
Title
Description
OG000
PAP: PP: Placebo
Participants in PP received placebo matched to venglustat oral tablet once daily for 104 weeks in a double-blind manner in PAP.
OG001
Secondary
PAP: PP and SP: Change From Baseline in the Neurological Examination of the Friedreich's Ataxia Rating Scale (FARS) (FARS-neuro) to Week 104
The FARS-neuro includes 23 items and is composed of 4 sections that assesses different neurological faculties: bulbar activity (4 items), upper limb coordination (5 items assessed right and left side), lower limb coordination (2 items assessed right and left side), peripheral nervous system (5 items assessed right and left side) and upright stability (7 items). Total score is calculated as the sum of scores on items of this section and ranges from 0 to 117; mean is presented. Higher value indicates higher disability. Baseline=last available value before or equal to first dose of study drug date in PAP. PP: primary efficacy population: all randomized participants with diagnosis of late-onset GM2 gangliosidosis aged >=18 years. SP: secondary efficacy population: all enrolled participants with diagnosis of juvenile/adolescent late-onset GM2 gangliosidosis, GM1 gangliosidosis, saposin C deficiency, sialidosis type 1, or juvenile/adult galactosialidosis.
Only those participants with data collected at specified timepoints are reported. For sialidosis type 1: due to the rarity of the disease and the specificity of the endpoint, reporting individual-level data would risk compromising participant privacy and confidentiality. As presented in baseline characteristics, only 1 participant with sialidosis type 1 was enrolled in the study.
Posted
Mean
Standard Deviation
score on a scale
Baseline (Day 1) and Week 104
ID
Title
Description
OG000
PAP: PP: Placebo
Participants in PP received placebo matched to venglustat oral tablet once daily for 104 weeks in a double-blind manner in PAP.
Secondary
PAP: PP and SP: Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Treatment-Emergent Serious Adverse Events (TESAEs)
An AE was any untoward medical occurrence in a participant or clinical study participant, temporally associated with the use of study drug, whether or not considered related to the study drug. SAE was any untoward medical occurrence that at any dose: resulted in death, was life-threatening, required inpatient hospitalization or prolongation of existing hospitalization, resulted in persistent disability/incapacity, was a congenital anomaly/birth defect or was an important medical event. TEAE were AEs that developed, worsened or became serious during the TE period.
PAP PP: primary safety population included all randomized participants with diagnosis of late-onset GM2 gangliosidosis aged >=18 years who received at least 1 dose of study drug.
PAP SP: secondary safety population included all enrolled participants with a clinical diagnosis of juvenile/adolescent late-onset GM2 gangliosidosis, GM1 gangliosidosis, saposin C deficiency, sialidosis type 1, or juvenile/adult galactosialidosis, who received at least 1 dose of study drug.
Posted
Count of Participants
Participants
From first dose of study drug (Day 1) up to end of PAP, 104 weeks
ID
Title
Description
OG000
PAP: PP: Placebo
Participants in PP received placebo matched to venglustat oral tablet once daily for 104 weeks in a double-blind manner in PAP.
OG001
PAP: PP: Venglustat
Secondary
PAP: PP: Plasma Venglustat Concentration
Plasma samples were collected at specified timepoints to obtain venglustat concentrations for PP in PAP.
PAP: Primary pharmacokinetic (PK) population included all randomized participants with diagnosis of late-onset GM2 gangliosidosis aged >=18 years who received at least 1 dose of study drug and had at least 1 PK assessment. Only those participants with data collected at specified timepoints are reported.
Posted
Mean
Standard Deviation
ng/mL
Pre-dose (0 hour) and 0.5, 3, 8, 12 and 24 hours post-dose at Week 12
ID
Title
Description
OG000
PAP: PP: Venglustat
Participants in PP received venglustat 15 mg oral tablet once daily for 104 weeks in a double-blind manner in PAP.
Units
Counts
Participants
OG000
Secondary
PAP: SP: Plasma Venglustat Concentration
Plasma samples were collected at specified timepoints to obtain venglustat concentration for SP in PAP. Data is presented by dose level for all diseases combined for SP in PAP.
PAP: Secondary PK population included all enrolled participants with a clinical diagnosis of juvenile/adolescent late-onset GM2 gangliosidosis, GM1 gangliosidosis, saposin C deficiency, sialidosis type 1, or juvenile/adult galactosialidosis, who received at least 1 dose of study drug and had at least 1 PK assessment. 'Overall Number of Participants Analyzed' indicates the total of maximum number of participants ('number analyzed') for each dose.
Posted
Mean
Standard Deviation
ng/mL
Pre-dose (0 hour) and 0.5, 3, 8, 12 and 24 hours post-dose at Week 12
ID
Title
Description
OG000
PAP: SP: Venglustat
Participants in SP received venglustat oral tablet once daily based on the body weight for 104 weeks in an open-label manner in PAP as follows:
15 mg if >=50 kg, 12 mg if 30 kg to <50 kg, 6 mg if 15 kg to <30 kg and 4 mg if 10 kg to <15 kg.
Units
Counts
Participants
OG000
Secondary
PAP: PP and SP: CSF Venglustat Concentration
CSF samples were collected via lumbar puncture at Week 104 to obtain venglustat concentrations for PP and SP in PAP. For SP: data is presented by dose level for all diseases combined. Only those participants with data collected at specified timepoints are reported.
PAP: Participants (PP: primary PK population=all randomized participants with diagnosis of late-onset GM2 gangliosidosis aged >=18 years and SP: secondary PK population=all enrolled participants with diagnosis of juvenile/adolescent late-onset GM2 gangliosidosis, GM1 gangliosidosis, saposin C deficiency, sialidosis type 1, or juvenile/adult galactosialidosis) who received at least 1 dose of study drug & had at least 1 PK assessment.
Posted
Mean
Standard Deviation
ng/mL
Week 104
ID
Title
Description
OG000
PAP: PP: Venglustat
Participants in PP received venglustat 15 mg oral tablet once daily for 104 weeks in a double-blind manner in PAP.
OG001
PAP: SP: Venglustat
Participants in SP received venglustat oral tablet once daily based on the body weight for 104 weeks in an open-label manner in PAP as follows:
15 mg if >=50 kg, 12 mg if 30 kg to <50 kg, 6 mg if 15 kg to <30 kg and 4 mg if 10 kg to <15 kg.
Secondary
PAP: PP and SP: Maximum Plasma Concentration Observed (Cmax) of Venglustat
Plasma samples were collected at specified timepoints to obtain Cmax of venglustat. The mean of Cmax, irrespective of the timepoint where the patient-individual Cmax value was observed is presented as opposed to endpoint 13 wherein mean of plasma venglustat concentration at each specified timepoint is presented. For SP: data is presented by dose level for all diseases combined. Only those participants with data collected at specified timepoints are reported.
PAP: Participants (PP: primary PK population=all randomized participants with diagnosis of late-onset GM2 gangliosidosis aged >=18 years and SP: secondary PK population=all enrolled participants with diagnosis of juvenile/adolescent late-onset GM2 gangliosidosis, GM1 gangliosidosis, saposin C deficiency, sialidosis type 1, or juvenile/adult galactosialidosis) who received at least 1 dose of study drug & had at least 1 PK assessment.
Posted
Mean
Standard Deviation
ng/mL
Pre-dose (0 hour) and 0.5, 3, 8, 12 and 24 hours post-dose at Week 12
ID
Title
Description
OG000
PAP: PP: Venglustat
Participants in PP received venglustat 15 mg oral tablet once daily for 104 weeks in a double-blind manner in PAP.
OG001
PAP: SP: Venglustat
Participants in SP received venglustat oral tablet once daily based on the body weight for 104 weeks in an open-label manner in PAP as follows:
15 mg if >=50 kg, 12 mg if 30 kg to <50 kg, 6 mg if 15 kg to <30 kg and 4 mg if 10 kg to <15 kg.
Secondary
PAP: PP and SP: Time to Reach Maximum Plasma Concentration (Tmax) of Venglustat
Plasma samples were collected at specified timepoints to obtain tmax of venglustat. For SP: data is presented by dose level for all diseases combined. Only those participants with data collected at specified timepoints are reported.
PAP: Participants (PP: primary PK population=all randomized participants with diagnosis of late-onset GM2 gangliosidosis aged >=18 years and SP: secondary PK population=all enrolled participants with diagnosis of juvenile/adolescent late-onset GM2 gangliosidosis, GM1 gangliosidosis, saposin C deficiency, sialidosis type 1, or juvenile/adult galactosialidosis) who received at least 1 dose of study drug & had at least 1 PK assessment.
Posted
Median
Full Range
hour
Pre-dose (0 hour) and 0.5, 3, 8, 12 and 24 hours post-dose at Week 12
ID
Title
Description
OG000
PAP: PP: Venglustat
Participants in PP received venglustat 15 mg oral tablet once daily for 104 weeks in a double-blind manner in PAP.
OG001
PAP: SP: Venglustat
Participants in SP received venglustat oral tablet once daily based on the body weight for 104 weeks in an open-label manner in PAP as follows:
15 mg if >=50 kg, 12 mg if 30 kg to <50 kg, 6 mg if 15 kg to <30 kg and 4 mg if 10 kg to <15 kg.
Secondary
PAP: PP and SP: Area Under the Plasma Concentration Versus Time Curve Calculated Over a Predefined Time Period 0 to 24 Hours (AUC0-24) of Venglustat
Plasma samples were collected at specified timepoints to obtain AUC0-24 of venglustat. For SP: data is presented by dose level for all diseases combined. Only those participants with data collected at specified timepoints are reported.
PAP: Participants (PP: primary PK population=all randomized participants with diagnosis of late-onset GM2 gangliosidosis aged >=18 years and SP: secondary PK population=all enrolled participants with diagnosis of juvenile/adolescent late-onset GM2 gangliosidosis, GM1 gangliosidosis, saposin C deficiency, sialidosis type 1, or juvenile/adult galactosialidosis) who received at least 1 dose of study drug & had at least 1 PK assessment.
Posted
Mean
Standard Deviation
hour*ng/mL
Pre-dose (0 hour) and 0.5, 3, 8, 12 and 24 hours post-dose at Week 12
ID
Title
Description
OG000
PAP: PP: Venglustat
Participants in PP received venglustat 15 mg oral tablet once daily for 104 weeks in a double-blind manner in PAP.
OG001
PAP: SP: Venglustat
Participants in SP received venglustat oral tablet once daily based on the body weight for 104 weeks in an open-label manner in PAP as follows:
15 mg if >=50 kg, 12 mg if 30 kg to <50 kg, 6 mg if 15 kg to <30 kg and 4 mg if 10 kg to <15 kg.
Secondary
PAP: SP: Percent Change From Baseline in the 9-HPT to Week 104
9-HPT is a test of upper extremity (arm and hand) function.Participant is seated at table with small, shallow container holding pegs & block containing 9 empty holes.On start command (stopwatch started),participant picks up 9 pegs one at a time as quickly as possible, puts them in 9 holes & once they are in holes, removes them again as quickly as possible one at a time,replacing them into shallow container,2 consecutive trials of dominant hand followed immediately by 2 consecutive trials of non-dominant hand.Total time (ranging up to 300 seconds):averaged, converted to reciprocals which were averaged and back-transformed to obtain global 9-HPT.Higher value indicates higher disability.Baseline: last available value before or equal to first dose of study drug date in PAP.Secondary efficacy population:all enrolled participants with diagnosis of juvenile/adolescent late-onset GM2 gangliosidosis,GM1 gangliosidosis,saposin C deficiency,sialidosis type 1 or juvenile/adult galactosialidosis.
Only those participants with data collected at specified timepoints are reported. For sialidosis type 1: due to the rarity of the disease and the specificity of the endpoint, reporting individual-level data would risk compromising participant privacy and confidentiality. As presented in baseline characteristics, only 1 participant with sialidosis type 1 was enrolled in the study.
Posted
Mean
Standard Error
percent change
Baseline (Day 1) and Week 104
ID
Title
Description
OG000
PAP: SP: Venglustat
Participants in SP received venglustat oral tablet once daily based on the body weight for 104 weeks in an open-label manner in PAP as follows:
15 mg if >=50 kg, 12 mg if 30 kg to <50 kg, 6 mg if 15 kg to <30 kg and 4 mg if 10 kg to <15 kg.
Secondary
PAP: SP: Number of Participants With >=80% Compliance as Per Method of Intake
For the secondary pediatric population, the acceptability and palatability of venglustat tablets was assessed through the route of venglustat administration collected in the electronic case report form and study drug compliance throughout PAP. The assessment was based on tablet always swallowed as whole or chewed and swallowed at least once. Number of participants with >=80% compliance for each is presented here.
Secondary safety population included all enrolled participants with a clinical diagnosis of juvenile/adolescent late-onset GM2 gangliosidosis, GM1 gangliosidosis, saposin C deficiency, sialidosis type 1, or juvenile/adult galactosialidosis, who received at least 1 dose of study drug. Only pediatric participants were evaluated for this endpoint.
Posted
Count of Participants
Participants
Baseline (Day 1) to Week 104
ID
Title
Description
OG000
PAP: SP: Venglustat
Participants in SP received venglustat oral tablet once daily based on the body weight for 104 weeks in an open-label manner in PAP as follows:
15 mg if >=50 kg, 12 mg if 30 kg to <50 kg, 6 mg if 15 kg to <30 kg and 4 mg if 10 kg to <15 kg.
Units
Counts
Participants
Time Frame
Adverse events:From first dose of study drug (Day 1) up to 104 weeks (PAP) and from 104 weeks up to 6 weeks post last drug in OLE, maximum duration 200 weeks due to study termination. Deaths were collected from first dose of study drug (Day 1) up to end of follow-up, maximum 200 weeks due to study termination.
Description
Analysis was performed on safety population (PAP: All enrolled participants who received at least 1 dose of study drug and OLE: all enrolled participants who received at least 1 venglustat dose after the 104-week visit).
All-Cause Mortality Comment
Not provided
Arm/Groups
ID
Title
Description
Deaths (Affected)
Deaths (At Risk)
Serious Events (Affected)
Serious Events (At Risk)
Other Events (Affected)
Other Events (At Risk)
EG000
PAP: PP: Placebo
Participants in PP received placebo matched to venglustat oral tablet once daily for 104 weeks in a double-blind manner in PAP.
0
19
4
19
19
19
EG001
PAP: PP: Venglustat
Participants in PP received venglustat 15 mg oral tablet once daily for 104 weeks in a double-blind manner in PAP.
0
40
8
40
38
40
EG002
PAP: SP: Venglustat
Participants in SP received venglustat oral tablet once daily based on the body weight for 104 weeks in an open-label manner in PAP as follows:
15 mg if >=50 kg, 12 mg if 30 kg to <50 kg, 6 mg if 15 kg to <30 kg and 4 mg if 10 kg to <15 kg.
1
16
6
16
14
16
EG003
OLE: PP: Delayed Venglustat
Participants in PP who previously received placebo in PAP received venglustat 15 mg oral tablet once daily for 104 weeks in an open-label manner in OLE.
1
16
4
16
11
16
EG004
OLE: PP: Early Venglustat
Participants in PP who previously received venglustat in PAP continued to receive venglustat 15 mg oral tablet once daily for 104 weeks in an open-label manner in OLE.
0
34
2
34
19
34
EG005
OLE: SP: Venglustat
Participants in SP who previously received venglustat in PAP continued to receive venglustat oral tablet once daily based on the body weight for 104 weeks in an open-label manner in OLE as follows:
15 mg if >=50 kg, 12 mg if 30 kg to <50 kg, 6 mg if 15 kg to <30 kg and 4 mg if 10 kg to <15 kg.
0
14
2
14
9
14
Serious Adverse Events
Term
Organ System
Source Vocabulary
Assessment Type
Notes
Statistical Information
Burn Infection
Infections and infestations
MedDra 27.1
Systematic Assessment
EG0000 events0 affected19 at risk
EG0011 events1 affected40 at risk
EG0020 events0 affected16 at risk
EG0030 events0 affected16 at risk
EG0040 events0 affected34 at risk
EG0050 events0 affected14 at risk
Cellulitis
Infections and infestations
MedDra 27.1
Systematic Assessment
EG0000 events0 affected19 at risk
EG0011 events1 affected40 at risk
EG0020 events0 affected16 at risk
EG003
Cystitis
Infections and infestations
MedDra 27.1
Systematic Assessment
EG0000 events0 affected19 at risk
EG0011 events1 affected40 at risk
EG0020 events0 affected16 at risk
EG003
Infected Bite
Infections and infestations
MedDra 27.1
Systematic Assessment
EG0000 events0 affected19 at risk
EG0010 events0 affected40 at risk
EG0020 events0 affected16 at risk
EG003
Respiratory Tract Infection Viral
Infections and infestations
MedDra 27.1
Systematic Assessment
EG0000 events0 affected19 at risk
EG0010 events0 affected40 at risk
EG0021 events1 affected16 at risk
EG003
Sepsis
Infections and infestations
MedDra 27.1
Systematic Assessment
EG0000 events0 affected19 at risk
EG0011 events1 affected40 at risk
EG0020 events0 affected16 at risk
EG003
Upper Respiratory Tract Infection
Infections and infestations
MedDra 27.1
Systematic Assessment
EG0000 events0 affected19 at risk
EG0010 events0 affected40 at risk
EG0020 events0 affected16 at risk
EG003
Urinary Tract Infection
Infections and infestations
MedDra 27.1
Systematic Assessment
EG0000 events0 affected19 at risk
EG0012 events2 affected40 at risk
EG0020 events0 affected16 at risk
EG003
Hypophagia
Metabolism and nutrition disorders
MedDra 27.1
Systematic Assessment
EG0001 events1 affected19 at risk
EG0010 events0 affected40 at risk
EG0020 events0 affected16 at risk
EG003
Acute Psychosis
Psychiatric disorders
MedDra 27.1
Systematic Assessment
EG0000 events0 affected19 at risk
EG0010 events0 affected40 at risk
EG0021 events1 affected16 at risk
EG003
Completed Suicide
Psychiatric disorders
MedDra 27.1
Systematic Assessment
EG0000 events0 affected19 at risk
EG0010 events0 affected40 at risk
EG0020 events0 affected16 at risk
EG003
Mania
Psychiatric disorders
MedDra 27.1
Systematic Assessment
EG0001 events1 affected19 at risk
EG0010 events0 affected40 at risk
EG0020 events0 affected16 at risk
EG003
Persecutory Delusion
Psychiatric disorders
MedDra 27.1
Systematic Assessment
EG0001 events1 affected19 at risk
EG0010 events0 affected40 at risk
EG0020 events0 affected16 at risk
EG003
Suicidal Ideation
Psychiatric disorders
MedDra 27.1
Systematic Assessment
EG0000 events0 affected19 at risk
EG0010 events0 affected40 at risk
EG0020 events0 affected16 at risk
EG003
Dyskinesia
Nervous system disorders
MedDra 27.1
Systematic Assessment
EG0000 events0 affected19 at risk
EG0010 events0 affected40 at risk
EG0021 events1 affected16 at risk
EG003
Respiratory Failure
Respiratory, thoracic and mediastinal disorders
MedDra 27.1
Systematic Assessment
EG0000 events0 affected19 at risk
EG0010 events0 affected40 at risk
EG0021 events1 affected16 at risk
EG003
Dysphagia
Gastrointestinal disorders
MedDra 27.1
Systematic Assessment
EG0000 events0 affected19 at risk
EG0010 events0 affected40 at risk
EG0021 events1 affected16 at risk
EG003
Faecaloma
Gastrointestinal disorders
MedDra 27.1
Systematic Assessment
EG0001 events1 affected19 at risk
EG0010 events0 affected40 at risk
EG0020 events0 affected16 at risk
EG003
Oesophageal Achalasia
Gastrointestinal disorders
MedDra 27.1
Systematic Assessment
EG0002 events1 affected19 at risk
EG0010 events0 affected40 at risk
EG0020 events0 affected16 at risk
EG003
Vomiting
Gastrointestinal disorders
MedDra 27.1
Systematic Assessment
EG0000 events0 affected19 at risk
EG0010 events0 affected40 at risk
EG0021 events1 affected16 at risk
EG003
Cholelithiasis
Hepatobiliary disorders
MedDra 27.1
Systematic Assessment
EG0000 events0 affected19 at risk
EG0011 events1 affected40 at risk
EG0020 events0 affected16 at risk
EG003
Hepatic Mass
Hepatobiliary disorders
MedDra 27.1
Systematic Assessment
EG0000 events0 affected19 at risk
EG0010 events0 affected40 at risk
EG0020 events0 affected16 at risk
EG003
Intervertebral Disc Protrusion
Musculoskeletal and connective tissue disorders
MedDra 27.1
Systematic Assessment
EG0000 events0 affected19 at risk
EG0010 events0 affected40 at risk
EG0020 events0 affected16 at risk
EG003
Urinary Retention
Renal and urinary disorders
MedDra 27.1
Systematic Assessment
EG0001 events1 affected19 at risk
EG0010 events0 affected40 at risk
EG0020 events0 affected16 at risk
EG003
Ankle Fracture
Injury, poisoning and procedural complications
MedDra 27.1
Systematic Assessment
EG0000 events0 affected19 at risk
EG0011 events1 affected40 at risk
EG0020 events0 affected16 at risk
EG003
Femur Fracture
Injury, poisoning and procedural complications
MedDra 27.1
Systematic Assessment
EG0000 events0 affected19 at risk
EG0010 events0 affected40 at risk
EG0021 events1 affected16 at risk
EG003
Hip Fracture
Injury, poisoning and procedural complications
MedDra 27.1
Systematic Assessment
EG0000 events0 affected19 at risk
EG0011 events1 affected40 at risk
EG0020 events0 affected16 at risk
EG003
Post Lumbar Puncture Syndrome
Injury, poisoning and procedural complications
MedDra 27.1
Systematic Assessment
EG0001 events1 affected19 at risk
EG0010 events0 affected40 at risk
EG0020 events0 affected16 at risk
EG003
Tibia Fracture
Injury, poisoning and procedural complications
MedDra 27.1
Systematic Assessment
EG0000 events0 affected19 at risk
EG0012 events2 affected40 at risk
EG0020 events0 affected16 at risk
EG003
Other Adverse Events
Term
Organ System
Source Vocabulary
Assessment Type
Notes
Statistical Information
Bacterial Infection
Infections and infestations
MedDra 27.1
Systematic Assessment
EG0000 events0 affected19 at risk
EG0010 events0 affected40 at risk
EG0021 events1 affected16 at risk
EG0030 events0 affected16 at risk
EG0040 events0 affected34 at risk
EG0050 events0 affected14 at risk
Bacterial Vaginosis
Infections and infestations
MedDra 27.1
Systematic Assessment
EG0000 events0 affected19 at risk
EG0010 events0 affected40 at risk
EG0020 events0 affected16 at risk
EG003
Breast Abscess
Infections and infestations
MedDra 27.1
Systematic Assessment
EG0000 events0 affected19 at risk
EG0010 events0 affected40 at risk
EG0021 events1 affected16 at risk
EG003
Bronchiolitis
Infections and infestations
MedDra 27.1
Systematic Assessment
EG0000 events0 affected19 at risk
EG0010 events0 affected40 at risk
EG0021 events1 affected16 at risk
EG003
Bronchitis
Infections and infestations
MedDra 27.1
Systematic Assessment
EG0000 events0 affected19 at risk
EG0011 events1 affected40 at risk
EG0020 events0 affected16 at risk
EG003
Covid-19
Infections and infestations
MedDra 27.1
Systematic Assessment
EG0004 events3 affected19 at risk
EG00114 events13 affected40 at risk
EG0028 events7 affected16 at risk
EG003
Conjunctivitis
Infections and infestations
MedDra 27.1
Systematic Assessment
EG0002 events2 affected19 at risk
EG0012 events1 affected40 at risk
EG0021 events1 affected16 at risk
EG003
Enterobiasis
Infections and infestations
MedDra 27.1
Systematic Assessment
EG0000 events0 affected19 at risk
EG0010 events0 affected40 at risk
EG0024 events1 affected16 at risk
EG003
Fungal Foot Infection
Infections and infestations
MedDra 27.1
Systematic Assessment
EG0001 events1 affected19 at risk
EG0010 events0 affected40 at risk
EG0020 events0 affected16 at risk
EG003
Gastroenteritis
Infections and infestations
MedDra 27.1
Systematic Assessment
EG0001 events1 affected19 at risk
EG0010 events0 affected40 at risk
EG0022 events2 affected16 at risk
EG003
Helicobacter Gastritis
Infections and infestations
MedDra 27.1
Systematic Assessment
EG0000 events0 affected19 at risk
EG0010 events0 affected40 at risk
EG0021 events1 affected16 at risk
EG003
Herpes Zoster
Infections and infestations
MedDra 27.1
Systematic Assessment
EG0000 events0 affected19 at risk
EG0010 events0 affected40 at risk
EG0020 events0 affected16 at risk
EG003
Hordeolum
Infections and infestations
MedDra 27.1
Systematic Assessment
EG0000 events0 affected19 at risk
EG0010 events0 affected40 at risk
EG0020 events0 affected16 at risk
EG003
Infected Bite
Infections and infestations
MedDra 27.1
Systematic Assessment
EG0000 events0 affected19 at risk
EG0010 events0 affected40 at risk
EG0021 events1 affected16 at risk
EG003
Influenza
Infections and infestations
MedDra 27.1
Systematic Assessment
EG0002 events2 affected19 at risk
EG0013 events3 affected40 at risk
EG0022 events2 affected16 at risk
EG003
Kidney Infection
Infections and infestations
MedDra 27.1
Systematic Assessment
EG0000 events0 affected19 at risk
EG0010 events0 affected40 at risk
EG0021 events1 affected16 at risk
EG003
Localised Infection
Infections and infestations
MedDra 27.1
Systematic Assessment
EG0000 events0 affected19 at risk
EG0011 events1 affected40 at risk
EG0020 events0 affected16 at risk
EG003
Nasopharyngitis
Infections and infestations
MedDra 27.1
Systematic Assessment
EG0001 events1 affected19 at risk
EG0012 events1 affected40 at risk
EG00213 events5 affected16 at risk
EG003
Oral Infection
Infections and infestations
MedDra 27.1
Systematic Assessment
EG0000 events0 affected19 at risk
EG0010 events0 affected40 at risk
EG0021 events1 affected16 at risk
EG003
Otitis Media
Infections and infestations
MedDra 27.1
Systematic Assessment
EG0000 events0 affected19 at risk
EG0010 events0 affected40 at risk
EG0021 events1 affected16 at risk
EG003
Perichondritis
Infections and infestations
MedDra 27.1
Systematic Assessment
EG0000 events0 affected19 at risk
EG0010 events0 affected40 at risk
EG0020 events0 affected16 at risk
EG003
Pharyngotonsillitis
Infections and infestations
MedDra 27.1
Systematic Assessment
EG0000 events0 affected19 at risk
EG0010 events0 affected40 at risk
EG0021 events1 affected16 at risk
EG003
Pneumonia
Infections and infestations
MedDra 27.1
Systematic Assessment
EG0000 events0 affected19 at risk
EG0010 events0 affected40 at risk
EG0021 events1 affected16 at risk
EG003
Respiratory Tract Infection
Infections and infestations
MedDra 27.1
Systematic Assessment
EG0000 events0 affected19 at risk
EG0016 events4 affected40 at risk
EG0021 events1 affected16 at risk
EG003
Sinusitis
Infections and infestations
MedDra 27.1
Systematic Assessment
EG0000 events0 affected19 at risk
EG0012 events2 affected40 at risk
EG0020 events0 affected16 at risk
EG003
Skin Infection
Infections and infestations
MedDra 27.1
Systematic Assessment
EG0000 events0 affected19 at risk
EG0010 events0 affected40 at risk
EG0021 events1 affected16 at risk
EG003
Upper Respiratory Tract Infection
Infections and infestations
MedDra 27.1
Systematic Assessment
EG0001 events1 affected19 at risk
EG0011 events1 affected40 at risk
EG0021 events1 affected16 at risk
EG003
Urinary Tract Infection
Infections and infestations
MedDra 27.1
Systematic Assessment
EG0002 events1 affected19 at risk
EG0019 events6 affected40 at risk
EG0022 events2 affected16 at risk
EG003
Urinary Tract Infection Bacterial
Infections and infestations
MedDra 27.1
Systematic Assessment
EG0001 events1 affected19 at risk
EG0010 events0 affected40 at risk
EG0020 events0 affected16 at risk
EG003
Vulvovaginal Candidiasis
Infections and infestations
MedDra 27.1
Systematic Assessment
EG0000 events0 affected19 at risk
EG0010 events0 affected40 at risk
EG0021 events1 affected16 at risk
EG003
Vulvovaginal Mycotic Infection
Infections and infestations
MedDra 27.1
Systematic Assessment
EG0000 events0 affected19 at risk
EG0010 events0 affected40 at risk
EG0020 events0 affected16 at risk
EG003
Drug Hypersensitivity
Immune system disorders
MedDra 27.1
Systematic Assessment
EG0000 events0 affected19 at risk
EG0010 events0 affected40 at risk
EG0021 events1 affected16 at risk
EG003
Seasonal Allergy
Immune system disorders
MedDra 27.1
Systematic Assessment
EG0000 events0 affected19 at risk
EG0010 events0 affected40 at risk
EG0020 events0 affected16 at risk
EG003
Cachexia
Metabolism and nutrition disorders
MedDra 27.1
Systematic Assessment
EG0000 events0 affected19 at risk
EG0010 events0 affected40 at risk
EG0021 events1 affected16 at risk
EG003
Decreased Appetite
Metabolism and nutrition disorders
MedDra 27.1
Systematic Assessment
EG0001 events1 affected19 at risk
EG0010 events0 affected40 at risk
EG0020 events0 affected16 at risk
EG003
Iron Deficiency
Metabolism and nutrition disorders
MedDra 27.1
Systematic Assessment
EG0000 events0 affected19 at risk
EG0010 events0 affected40 at risk
EG0020 events0 affected16 at risk
EG003
Vitamin D Deficiency
Metabolism and nutrition disorders
MedDra 27.1
Systematic Assessment
EG0000 events0 affected19 at risk
EG0013 events3 affected40 at risk
EG0020 events0 affected16 at risk
EG003
Anxiety
Psychiatric disorders
MedDra 27.1
Systematic Assessment
EG0002 events2 affected19 at risk
EG0019 events4 affected40 at risk
EG0020 events0 affected16 at risk
EG003
Apathy
Psychiatric disorders
MedDra 27.1
Systematic Assessment
EG0001 events1 affected19 at risk
EG0010 events0 affected40 at risk
EG0020 events0 affected16 at risk
EG003
Depression
Psychiatric disorders
MedDra 27.1
Systematic Assessment
EG0001 events1 affected19 at risk
EG0012 events2 affected40 at risk
EG0021 events1 affected16 at risk
EG003
Disorientation
Psychiatric disorders
MedDra 27.1
Systematic Assessment
EG0000 events0 affected19 at risk
EG0010 events0 affected40 at risk
EG0020 events0 affected16 at risk
EG003
Emotional Disorder
Psychiatric disorders
MedDra 27.1
Systematic Assessment
EG0000 events0 affected19 at risk
EG0010 events0 affected40 at risk
EG0020 events0 affected16 at risk
EG003
Grief Reaction
Psychiatric disorders
MedDra 27.1
Systematic Assessment
EG0001 events1 affected19 at risk
EG0011 events1 affected40 at risk
EG0020 events0 affected16 at risk
EG003
Insomnia
Psychiatric disorders
MedDra 27.1
Systematic Assessment
EG0000 events0 affected19 at risk
EG0010 events0 affected40 at risk
EG0021 events1 affected16 at risk
EG003
Irritability
Psychiatric disorders
MedDra 27.1
Systematic Assessment
EG0000 events0 affected19 at risk
EG0010 events0 affected40 at risk
EG0021 events1 affected16 at risk
EG003
Mania
Psychiatric disorders
MedDra 27.1
Systematic Assessment
EG0000 events0 affected19 at risk
EG0010 events0 affected40 at risk
EG0020 events0 affected16 at risk
EG003
Ataxia
Nervous system disorders
MedDra 27.1
Systematic Assessment
EG0002 events1 affected19 at risk
EG0010 events0 affected40 at risk
EG0020 events0 affected16 at risk
EG003
Balance Disorder
Nervous system disorders
MedDra 27.1
Systematic Assessment
EG0000 events0 affected19 at risk
EG0013 events2 affected40 at risk
EG0020 events0 affected16 at risk
EG003
Disturbance In Attention
Nervous system disorders
MedDra 27.1
Systematic Assessment
EG0000 events0 affected19 at risk
EG0012 events2 affected40 at risk
EG0020 events0 affected16 at risk
EG003
Dizziness
Nervous system disorders
MedDra 27.1
Systematic Assessment
EG0001 events1 affected19 at risk
EG0013 events3 affected40 at risk
EG0022 events2 affected16 at risk
EG003
Headache
Nervous system disorders
MedDra 27.1
Systematic Assessment
EG0004 events4 affected19 at risk
EG00119 events10 affected40 at risk
EG00210 events4 affected16 at risk
EG003
Hypoaesthesia
Nervous system disorders
MedDra 27.1
Systematic Assessment
EG0001 events1 affected19 at risk
EG0011 events1 affected40 at risk
EG0020 events0 affected16 at risk
EG003
Hyporeflexia
Nervous system disorders
MedDra 27.1
Systematic Assessment
EG0001 events1 affected19 at risk
EG0010 events0 affected40 at risk
EG0020 events0 affected16 at risk
EG003
Hypotonia
Nervous system disorders
MedDra 27.1
Systematic Assessment
EG0001 events1 affected19 at risk
EG0010 events0 affected40 at risk
EG0020 events0 affected16 at risk
EG003
Loss Of Consciousness
Nervous system disorders
MedDra 27.1
Systematic Assessment
EG0000 events0 affected19 at risk
EG0010 events0 affected40 at risk
EG0021 events1 affected16 at risk
EG003
Myasthenia Gravis
Nervous system disorders
MedDra 27.1
Systematic Assessment
EG0001 events1 affected19 at risk
EG0010 events0 affected40 at risk
EG0020 events0 affected16 at risk
EG003
Myoclonus
Nervous system disorders
MedDra 27.1
Systematic Assessment
EG0000 events0 affected19 at risk
EG0010 events0 affected40 at risk
EG0021 events1 affected16 at risk
EG003
Paraesthesia
Nervous system disorders
MedDra 27.1
Systematic Assessment
EG0002 events2 affected19 at risk
EG0013 events3 affected40 at risk
EG0020 events0 affected16 at risk
EG003
Seizure
Nervous system disorders
MedDra 27.1
Systematic Assessment
EG0000 events0 affected19 at risk
EG0010 events0 affected40 at risk
EG0021 events1 affected16 at risk
EG003
Speech Disorder
Nervous system disorders
MedDra 27.1
Systematic Assessment
EG0000 events0 affected19 at risk
EG0010 events0 affected40 at risk
EG0022 events2 affected16 at risk
EG003
Tremor
Nervous system disorders
MedDra 27.1
Systematic Assessment
EG0001 events1 affected19 at risk
EG0012 events2 affected40 at risk
EG0020 events0 affected16 at risk
EG003
Blepharitis
Eye disorders
MedDra 27.1
Systematic Assessment
EG0001 events1 affected19 at risk
EG0010 events0 affected40 at risk
EG0021 events1 affected16 at risk
EG003
Cataract
Eye disorders
MedDra 27.1
Systematic Assessment
EG0000 events0 affected19 at risk
EG0010 events0 affected40 at risk
EG0020 events0 affected16 at risk
EG003
Cataract Cortical
Eye disorders
MedDra 27.1
Systematic Assessment
EG0002 events2 affected19 at risk
EG0010 events0 affected40 at risk
EG0020 events0 affected16 at risk
EG003
Cataract Nuclear
Eye disorders
MedDra 27.1
Systematic Assessment
EG0001 events1 affected19 at risk
EG0012 events2 affected40 at risk
EG0020 events0 affected16 at risk
EG003
Cataract Subcapsular
Eye disorders
MedDra 27.1
Systematic Assessment
EG0002 events2 affected19 at risk
EG0010 events0 affected40 at risk
EG0020 events0 affected16 at risk
EG003
Dry Eye
Eye disorders
MedDra 27.1
Systematic Assessment
EG0002 events2 affected19 at risk
EG0011 events1 affected40 at risk
EG0020 events0 affected16 at risk
EG003
Eyelid Ptosis
Eye disorders
MedDra 27.1
Systematic Assessment
EG0001 events1 affected19 at risk
EG0010 events0 affected40 at risk
EG0020 events0 affected16 at risk
EG003
Lenticular Opacities
Eye disorders
MedDra 27.1
Systematic Assessment
EG0002 events2 affected19 at risk
EG0011 events1 affected40 at risk
EG0020 events0 affected16 at risk
EG003
Meibomianitis
Eye disorders
MedDra 27.1
Systematic Assessment
EG0001 events1 affected19 at risk
EG0010 events0 affected40 at risk
EG0021 events1 affected16 at risk
EG003
Strabismus
Eye disorders
MedDra 27.1
Systematic Assessment
EG0000 events0 affected19 at risk
EG0010 events0 affected40 at risk
EG0021 events1 affected16 at risk
EG003
Vision Blurred
Eye disorders
MedDra 27.1
Systematic Assessment
EG0002 events1 affected19 at risk
EG0012 events2 affected40 at risk
EG0020 events0 affected16 at risk
EG003
Visual Field Defect
Eye disorders
MedDra 27.1
Systematic Assessment
EG0001 events1 affected19 at risk
EG0010 events0 affected40 at risk
EG0020 events0 affected16 at risk
EG003
Visual Impairment
Eye disorders
MedDra 27.1
Systematic Assessment
EG0000 events0 affected19 at risk
EG0010 events0 affected40 at risk
EG0020 events0 affected16 at risk
EG003
Ear Pain
Ear and labyrinth disorders
MedDra 27.1
Systematic Assessment
EG0000 events0 affected19 at risk
EG0011 events1 affected40 at risk
EG0020 events0 affected16 at risk
EG003
Vertigo
Ear and labyrinth disorders
MedDra 27.1
Systematic Assessment
EG0000 events0 affected19 at risk
EG0014 events3 affected40 at risk
EG0020 events0 affected16 at risk
EG003
Tachycardia
Cardiac disorders
MedDra 27.1
Systematic Assessment
EG0001 events1 affected19 at risk
EG0010 events0 affected40 at risk
EG0020 events0 affected16 at risk
EG003
Atelectasis
Respiratory, thoracic and mediastinal disorders
MedDra 27.1
Systematic Assessment
EG0000 events0 affected19 at risk
EG0010 events0 affected40 at risk
EG0021 events1 affected16 at risk
EG003
Cough
Respiratory, thoracic and mediastinal disorders
MedDra 27.1
Systematic Assessment
EG0002 events2 affected19 at risk
EG0018 events8 affected40 at risk
EG0024 events2 affected16 at risk
EG003
Dyspnoea
Respiratory, thoracic and mediastinal disorders
MedDra 27.1
Systematic Assessment
EG0000 events0 affected19 at risk
EG0010 events0 affected40 at risk
EG0021 events1 affected16 at risk
EG003
Nasal Congestion
Respiratory, thoracic and mediastinal disorders
MedDra 27.1
Systematic Assessment
EG0000 events0 affected19 at risk
EG0011 events1 affected40 at risk
EG0021 events1 affected16 at risk
EG003
Obstructive Sleep Apnoea Syndrome
Respiratory, thoracic and mediastinal disorders
MedDra 27.1
Systematic Assessment
EG0001 events1 affected19 at risk
EG0010 events0 affected40 at risk
EG0020 events0 affected16 at risk
EG003
Oropharyngeal Pain
Respiratory, thoracic and mediastinal disorders
MedDra 27.1
Systematic Assessment
EG0001 events1 affected19 at risk
EG0013 events2 affected40 at risk
EG0020 events0 affected16 at risk
EG003
Rhinitis Allergic
Respiratory, thoracic and mediastinal disorders
MedDra 27.1
Systematic Assessment
EG0000 events0 affected19 at risk
EG0010 events0 affected40 at risk
EG0024 events1 affected16 at risk
EG003
Rhinorrhoea
Respiratory, thoracic and mediastinal disorders
MedDra 27.1
Systematic Assessment
EG0000 events0 affected19 at risk
EG0010 events0 affected40 at risk
EG0021 events1 affected16 at risk
EG003
Upper Respiratory Tract Congestion
Respiratory, thoracic and mediastinal disorders
MedDra 27.1
Systematic Assessment
EG0000 events0 affected19 at risk
EG0010 events0 affected40 at risk
EG0021 events1 affected16 at risk
EG003
Abdominal Pain
Gastrointestinal disorders
MedDra 27.1
Systematic Assessment
EG0000 events0 affected19 at risk
EG0010 events0 affected40 at risk
EG0022 events2 affected16 at risk
EG003
Abdominal Pain Upper
Gastrointestinal disorders
MedDra 27.1
Systematic Assessment
EG0001 events1 affected19 at risk
EG0010 events0 affected40 at risk
EG0022 events1 affected16 at risk
EG003
Colitis Ulcerative
Gastrointestinal disorders
MedDra 27.1
Systematic Assessment
EG0000 events0 affected19 at risk
EG0010 events0 affected40 at risk
EG0020 events0 affected16 at risk
EG003
Constipation
Gastrointestinal disorders
MedDra 27.1
Systematic Assessment
EG0003 events3 affected19 at risk
EG0016 events4 affected40 at risk
EG0021 events1 affected16 at risk
EG003
Dental Caries
Gastrointestinal disorders
MedDra 27.1
Systematic Assessment
EG0001 events1 affected19 at risk
EG0011 events1 affected40 at risk
EG0020 events0 affected16 at risk
EG003
Diarrhoea
Gastrointestinal disorders
MedDra 27.1
Systematic Assessment
EG0001 events1 affected19 at risk
EG0018 events5 affected40 at risk
EG0021 events1 affected16 at risk
EG003
Duodenogastric Reflux
Gastrointestinal disorders
MedDra 27.1
Systematic Assessment
EG0001 events1 affected19 at risk
EG0010 events0 affected40 at risk
EG0020 events0 affected16 at risk
EG003
Dysphagia
Gastrointestinal disorders
MedDra 27.1
Systematic Assessment
EG0002 events1 affected19 at risk
EG0011 events1 affected40 at risk
EG0021 events1 affected16 at risk
EG003
Gastritis Erosive
Gastrointestinal disorders
MedDra 27.1
Systematic Assessment
EG0000 events0 affected19 at risk
EG0010 events0 affected40 at risk
EG0021 events1 affected16 at risk
EG003
Gastrooesophageal Reflux Disease
Gastrointestinal disorders
MedDra 27.1
Systematic Assessment
EG0001 events1 affected19 at risk
EG0012 events2 affected40 at risk
EG0020 events0 affected16 at risk
EG003
Nausea
Gastrointestinal disorders
MedDra 27.1
Systematic Assessment
EG0003 events3 affected19 at risk
EG00110 events7 affected40 at risk
EG0021 events1 affected16 at risk
EG003
Tongue Ulceration
Gastrointestinal disorders
MedDra 27.1
Systematic Assessment
EG0001 events1 affected19 at risk
EG0010 events0 affected40 at risk
EG0020 events0 affected16 at risk
EG003
Tooth Impacted
Gastrointestinal disorders
MedDra 27.1
Systematic Assessment
EG0000 events0 affected19 at risk
EG0010 events0 affected40 at risk
EG0021 events1 affected16 at risk
EG003
Toothache
Gastrointestinal disorders
MedDra 27.1
Systematic Assessment
EG0000 events0 affected19 at risk
EG0012 events2 affected40 at risk
EG0021 events1 affected16 at risk
EG003
Vomiting
Gastrointestinal disorders
MedDra 27.1
Systematic Assessment
EG0001 events1 affected19 at risk
EG0015 events4 affected40 at risk
EG0025 events3 affected16 at risk
EG003
Dermatitis Allergic
Skin and subcutaneous tissue disorders
MedDra 27.1
Systematic Assessment
EG0001 events1 affected19 at risk
EG0010 events0 affected40 at risk
EG0020 events0 affected16 at risk
EG003
Eczema
Skin and subcutaneous tissue disorders
MedDra 27.1
Systematic Assessment
EG0002 events1 affected19 at risk
EG0010 events0 affected40 at risk
EG0020 events0 affected16 at risk
EG003
Hand Dermatitis
Skin and subcutaneous tissue disorders
MedDra 27.1
Systematic Assessment
EG0000 events0 affected19 at risk
EG0010 events0 affected40 at risk
EG0020 events0 affected16 at risk
EG003
Night Sweats
Skin and subcutaneous tissue disorders
MedDra 27.1
Systematic Assessment
EG0001 events1 affected19 at risk
EG0010 events0 affected40 at risk
EG0020 events0 affected16 at risk
EG003
Pruritus
Skin and subcutaneous tissue disorders
MedDra 27.1
Systematic Assessment
EG0002 events2 affected19 at risk
EG0012 events2 affected40 at risk
EG0020 events0 affected16 at risk
EG003
Pruritus Allergic
Skin and subcutaneous tissue disorders
MedDra 27.1
Systematic Assessment
EG0000 events0 affected19 at risk
EG0010 events0 affected40 at risk
EG0020 events0 affected16 at risk
EG003
Rash
Skin and subcutaneous tissue disorders
MedDra 27.1
Systematic Assessment
EG0001 events1 affected19 at risk
EG0011 events1 affected40 at risk
EG0020 events0 affected16 at risk
EG003
Seborrhoeic Dermatitis
Skin and subcutaneous tissue disorders
MedDra 27.1
Systematic Assessment
EG0001 events1 affected19 at risk
EG0010 events0 affected40 at risk
EG0020 events0 affected16 at risk
EG003
Skin Exfoliation
Skin and subcutaneous tissue disorders
MedDra 27.1
Systematic Assessment
EG0001 events1 affected19 at risk
EG0010 events0 affected40 at risk
EG0020 events0 affected16 at risk
EG003
Urticaria
Skin and subcutaneous tissue disorders
MedDra 27.1
Systematic Assessment
EG0000 events0 affected19 at risk
EG0010 events0 affected40 at risk
EG0020 events0 affected16 at risk
EG003
Arthralgia
Musculoskeletal and connective tissue disorders
MedDra 27.1
Systematic Assessment
EG0004 events3 affected19 at risk
EG0018 events7 affected40 at risk
EG0023 events2 affected16 at risk
EG003
Back Pain
Musculoskeletal and connective tissue disorders
MedDra 27.1
Systematic Assessment
EG0004 events3 affected19 at risk
EG0012 events2 affected40 at risk
EG0022 events2 affected16 at risk
EG003
Bursitis
Musculoskeletal and connective tissue disorders
MedDra 27.1
Systematic Assessment
EG0001 events1 affected19 at risk
EG0010 events0 affected40 at risk
EG0020 events0 affected16 at risk
EG003
Joint Swelling
Musculoskeletal and connective tissue disorders
MedDra 27.1
Systematic Assessment
EG0002 events1 affected19 at risk
EG0010 events0 affected40 at risk
EG0020 events0 affected16 at risk
EG003
Muscle Spasms
Musculoskeletal and connective tissue disorders
MedDra 27.1
Systematic Assessment
EG0001 events1 affected19 at risk
EG0010 events0 affected40 at risk
EG0020 events0 affected16 at risk
EG003
Muscular Weakness
Musculoskeletal and connective tissue disorders
MedDra 27.1
Systematic Assessment
EG0000 events0 affected19 at risk
EG0013 events3 affected40 at risk
EG0020 events0 affected16 at risk
EG003
Musculoskeletal Chest Pain
Musculoskeletal and connective tissue disorders
MedDra 27.1
Systematic Assessment
EG0001 events1 affected19 at risk
EG0010 events0 affected40 at risk
EG0020 events0 affected16 at risk
EG003
Myalgia
Musculoskeletal and connective tissue disorders
MedDra 27.1
Systematic Assessment
EG0001 events1 affected19 at risk
EG0012 events2 affected40 at risk
EG0020 events0 affected16 at risk
EG003
Neck Pain
Musculoskeletal and connective tissue disorders
MedDra 27.1
Systematic Assessment
EG0000 events0 affected19 at risk
EG0010 events0 affected40 at risk
EG0022 events2 affected16 at risk
EG003
Pain In Extremity
Musculoskeletal and connective tissue disorders
MedDra 27.1
Systematic Assessment
EG0000 events0 affected19 at risk
EG0015 events5 affected40 at risk
EG0022 events2 affected16 at risk
EG003
Crystalluria
Renal and urinary disorders
MedDra 27.1
Systematic Assessment
EG0001 events1 affected19 at risk
EG0010 events0 affected40 at risk
EG0020 events0 affected16 at risk
EG003
Haematuria
Renal and urinary disorders
MedDra 27.1
Systematic Assessment
EG0000 events0 affected19 at risk
EG0010 events0 affected40 at risk
EG0021 events1 affected16 at risk
EG003
Nephrolithiasis
Renal and urinary disorders
MedDra 27.1
Systematic Assessment
EG0001 events1 affected19 at risk
EG0010 events0 affected40 at risk
EG0020 events0 affected16 at risk
EG003
Pollakiuria
Renal and urinary disorders
MedDra 27.1
Systematic Assessment
EG0000 events0 affected19 at risk
EG0011 events1 affected40 at risk
EG0021 events1 affected16 at risk
EG003
Urinary Retention
Renal and urinary disorders
MedDra 27.1
Systematic Assessment
EG0001 events1 affected19 at risk
EG0014 events2 affected40 at risk
EG0020 events0 affected16 at risk
EG003
Amenorrhoea
Reproductive system and breast disorders
MedDra 27.1
Systematic Assessment
EG0000 events0 affected19 at risk
EG0011 events1 affected40 at risk
EG0021 events1 affected16 at risk
EG003
Cervical Polyp
Reproductive system and breast disorders
MedDra 27.1
Systematic Assessment
EG0001 events1 affected19 at risk
EG0010 events0 affected40 at risk
EG0020 events0 affected16 at risk
EG003
Dysmenorrhoea
Reproductive system and breast disorders
MedDra 27.1
Systematic Assessment
EG0000 events0 affected19 at risk
EG0011 events1 affected40 at risk
EG0020 events0 affected16 at risk
EG003
Chills
General disorders
MedDra 27.1
Systematic Assessment
EG0001 events1 affected19 at risk
EG0011 events1 affected40 at risk
EG0020 events0 affected16 at risk
EG003
Face Oedema
General disorders
MedDra 27.1
Systematic Assessment
EG0001 events1 affected19 at risk
EG0010 events0 affected40 at risk
EG0020 events0 affected16 at risk
EG003
Fatigue
General disorders
MedDra 27.1
Systematic Assessment
EG0003 events3 affected19 at risk
EG0013 events3 affected40 at risk
EG0021 events1 affected16 at risk
EG003
Gait Disturbance
General disorders
MedDra 27.1
Systematic Assessment
EG0001 events1 affected19 at risk
EG0011 events1 affected40 at risk
EG0021 events1 affected16 at risk
EG003
Injection Site Pain
General disorders
MedDra 27.1
Systematic Assessment
EG0000 events0 affected19 at risk
EG0010 events0 affected40 at risk
EG0020 events0 affected16 at risk
EG003
Medical Device Site Pain
General disorders
MedDra 27.1
Systematic Assessment
EG0000 events0 affected19 at risk
EG0010 events0 affected40 at risk
EG0021 events1 affected16 at risk
EG003
Pyrexia
General disorders
MedDra 27.1
Systematic Assessment
EG0003 events3 affected19 at risk
EG0014 events3 affected40 at risk
EG0023 events1 affected16 at risk
EG003
Vaccination Site Pain
General disorders
MedDra 27.1
Systematic Assessment
EG0000 events0 affected19 at risk
EG0010 events0 affected40 at risk
EG0020 events0 affected16 at risk
EG003
Alanine Aminotransferase Increased
Investigations
MedDra 27.1
Systematic Assessment
EG0000 events0 affected19 at risk
EG0011 events1 affected40 at risk
EG0020 events0 affected16 at risk
EG003
Blood Urine Present
Investigations
MedDra 27.1
Systematic Assessment
EG0000 events0 affected19 at risk
EG0010 events0 affected40 at risk
EG0021 events1 affected16 at risk
EG003
Depression Rating Scale Score Increased
Investigations
MedDra 27.1
Systematic Assessment
EG0001 events1 affected19 at risk
EG0010 events0 affected40 at risk
EG0020 events0 affected16 at risk
EG003
Electrocardiogram Qt Prolonged
Investigations
MedDra 27.1
Systematic Assessment
EG0000 events0 affected19 at risk
EG0010 events0 affected40 at risk
EG0021 events1 affected16 at risk
EG003
Gamma-Glutamyltransferase Increased
Investigations
MedDra 27.1
Systematic Assessment
EG0001 events1 affected19 at risk
EG0010 events0 affected40 at risk
EG0020 events0 affected16 at risk
EG003
Hepatic Enzyme Increased
Investigations
MedDra 27.1
Systematic Assessment
EG0000 events0 affected19 at risk
EG0010 events0 affected40 at risk
EG0020 events0 affected16 at risk
EG003
Lymphocyte Count Increased
Investigations
MedDra 27.1
Systematic Assessment
EG0001 events1 affected19 at risk
EG0010 events0 affected40 at risk
EG0020 events0 affected16 at risk
EG003
Muscle Strength Abnormal
Investigations
MedDra 27.1
Systematic Assessment
EG0008 events1 affected19 at risk
EG0014 events1 affected40 at risk
EG0020 events0 affected16 at risk
EG003
Platelet Count Decreased
Investigations
MedDra 27.1
Systematic Assessment
EG0002 events1 affected19 at risk
EG0010 events0 affected40 at risk
EG0020 events0 affected16 at risk
EG003
Weight Decreased
Investigations
MedDra 27.1
Systematic Assessment
EG0003 events3 affected19 at risk
EG0011 events1 affected40 at risk
EG0020 events0 affected16 at risk
EG003
White Blood Cell Count Increased
Investigations
MedDra 27.1
Systematic Assessment
EG0001 events1 affected19 at risk
EG0011 events1 affected40 at risk
EG0020 events0 affected16 at risk
EG003
Accidental Overdose
Injury, poisoning and procedural complications
MedDra 27.1
Systematic Assessment
EG0000 events0 affected19 at risk
EG0017 events4 affected40 at risk
EG0021 events1 affected16 at risk
EG003
Bone Contusion
Injury, poisoning and procedural complications
MedDra 27.1
Systematic Assessment
EG0001 events1 affected19 at risk
EG0010 events0 affected40 at risk
EG0020 events0 affected16 at risk
EG003
Contusion
Injury, poisoning and procedural complications
MedDra 27.1
Systematic Assessment
EG0006 events4 affected19 at risk
EG00110 events9 affected40 at risk
EG0020 events0 affected16 at risk
EG003
Face Injury
Injury, poisoning and procedural complications
MedDra 27.1
Systematic Assessment
EG0001 events1 affected19 at risk
EG0010 events0 affected40 at risk
EG0020 events0 affected16 at risk
EG003
Fall
Injury, poisoning and procedural complications
MedDra 27.1
Systematic Assessment
EG00031 events10 affected19 at risk
EG00161 events22 affected40 at risk
EG0022 events2 affected16 at risk
EG003
Fibula Fracture
Injury, poisoning and procedural complications
MedDra 27.1
Systematic Assessment
EG0001 events1 affected19 at risk
EG0011 events1 affected40 at risk
EG0020 events0 affected16 at risk
EG003
Foot Fracture
Injury, poisoning and procedural complications
MedDra 27.1
Systematic Assessment
EG0001 events1 affected19 at risk
EG0011 events1 affected40 at risk
EG0021 events1 affected16 at risk
EG003
Head Injury
Injury, poisoning and procedural complications
MedDra 27.1
Systematic Assessment
EG0002 events2 affected19 at risk
EG0010 events0 affected40 at risk
EG0020 events0 affected16 at risk
EG003
Immunisation Reaction
Injury, poisoning and procedural complications
MedDra 27.1
Systematic Assessment
EG0000 events0 affected19 at risk
EG0010 events0 affected40 at risk
EG0020 events0 affected16 at risk
EG003
Ligament Sprain
Injury, poisoning and procedural complications
MedDra 27.1
Systematic Assessment
EG0001 events1 affected19 at risk
EG0016 events4 affected40 at risk
EG0020 events0 affected16 at risk
EG003
Muscle Strain
Injury, poisoning and procedural complications
MedDra 27.1
Systematic Assessment
EG0000 events0 affected19 at risk
EG0010 events0 affected40 at risk
EG0021 events1 affected16 at risk
EG003
Post Lumbar Puncture Syndrome
Injury, poisoning and procedural complications
MedDra 27.1
Systematic Assessment
EG0001 events1 affected19 at risk
EG0014 events4 affected40 at risk
EG0022 events2 affected16 at risk
EG003
Procedural Pain
Injury, poisoning and procedural complications
MedDra 27.1
Systematic Assessment
EG0000 events0 affected19 at risk
EG0010 events0 affected40 at risk
EG0021 events1 affected16 at risk
EG003
Scratch
Injury, poisoning and procedural complications
MedDra 27.1
Systematic Assessment
EG0001 events1 affected19 at risk
EG0010 events0 affected40 at risk
EG0020 events0 affected16 at risk
EG003
Skin Laceration
Injury, poisoning and procedural complications
MedDra 27.1
Systematic Assessment
EG0000 events0 affected19 at risk
EG0011 events1 affected40 at risk
EG0021 events1 affected16 at risk
EG003
The study was early terminated based on the absence of positive trends on clinical endpoints; there were no safety concerns. For sialidosis type 1 and juvenile/adult galactosialidosis, due to the rarity of the disease and the specificity of the endpoint, reporting individual-level data would risk compromising participant privacy and confidentiality.
Certain Agreements
Are all PI(s) employees of the sponsor?
No
Restriction Type
OTHER
Results Disclosure Restriction on PI(s)?
Yes
Other Details
The Sponsor supports publication of clinical trial results but may request that investigators temporarily delay or alter publications in order to protect proprietary information. The Sponsor may also require that the results of multicenter studies be published only in their entirety and not as individual site data.
Congenital, Hereditary, and Neonatal Diseases and Abnormalities
D008064
Lipidoses
D008052
Lipid Metabolism, Inborn Errors
D016464
Lysosomal Storage Diseases
D008659
Metabolic Diseases
D009750
Nutritional and Metabolic Diseases
D052439
Lipid Metabolism Disorders
Browse Leaves
Not provided
Browse Branches
Not provided
0 subjects
FG0050 subjects
FG0040 subjects
FG0050 subjects
34 subjects
FG00514 subjects
2 subjects
FG0041 subjects
FG0052 subjects
Study terminated by sponsor
FG0000 subjects
FG0010 subjects
FG0020 subjects
FG00313 subjects
FG00433 subjects
FG00512 subjects
Adverse Event
FG0000 subjects
FG0010 subjects
FG0020 subjects
FG0031 subjects
FG0040 subjects
FG0050 subjects
75
Title
Measurements
BG00037.8± 15.4
BG00137.0± 13.3
BG00212.4± 7.3
BG00332.0± 16.3
16
ParticipantsBG00375
Title
Measurements
Female
BG00010
BG00119
BG00211
BG00340
Male
BG0009
BG00121
BG0025
BG00335
16
ParticipantsBG00375
Title
Measurements
American Indian or Alaska Native
BG0000
BG0010
BG0020
BG0030
Asian
BG0000
BG0013
BG0022
BG0035
Native Hawaiian or Other Pacific Islander
BG0000
BG0010
BG0020
BG0030
Black or African American
BG0000
BG0010
BG0021
BG0031
White
BG00017
BG00135
BG00212
BG00364
More than one race
BG0000
BG0010
BG0020
BG0030
Unknown or Not Reported
BG0002
BG0012
BG0021
BG0035
39
ParticipantsBG0020
ParticipantsBG00357
Title
Measurements
BG00067.11± 34.15
BG00163.00± 36.84
BG00364.30± 35.76
40
ParticipantsBG0020
ParticipantsBG00359
Title
Measurements
BG00045.89± 34.65
BG00141.93± 26.56
BG00343.20± 29.16
16
ParticipantsBG00316
Title
Measurements
Juvenile/adolescent GM2 gangliosidosis
BG0027
BG0037
GM1 gangliosidosis
BG0027
BG0037
Saposin C deficiency
BG0020
BG0030
Sialidosis type 1
BG0021
BG0031
Juvenile/adult galactosialidosis
BG0021
BG0031
Participants in PP received venglustat 15 mg oral tablet once daily for 104 weeks in a double-blind manner in PAP.
Units
Counts
Participants
OG00018
OG00139
Title
Denominators
Categories
Title
Measurements
OG0000.95± 1.92
OG0012.49± 1.35
Group IDs
Group Description
Statistical Method
Statistical Comment
P-Value
P-Value Comment
Parameter Type
Parameter Value
Dispersion Type
Dispersion Value
Confidence Interval Sides
Confidence Interval %
CI Lower Limit
CI Upper Limit
CI Lower Limit Comment
CI Upper Limit Comment
Estimate Comment
Tested Non-Inferiority
Non-Inferiority Type
Non-Inferiority Comment
Other Analysis Description
OG000
OG001
Mean slope of log-transformed 9-HPT obtained from a robust ANCOVA on individual slopes of log-transformed 9-HPT using Huber's M estimation.
Robust ANCOVA
The robust ANCOVA included fixed effect of treatment (venglustat versus placebo) and continuous fixed covariate of log-transformed baseline value.
0.7435
Mean difference
1.54
Standard Error of the Mean
2.35
2-Sided
90
-2.33
5.39
Superiority
OG000
7
Title
Denominators
Categories
GL-1: CSF
Title
Measurements
OG000-82.35± 9.94
GL-1: Plasma
Title
Measurements
OG000-79.28± 2.56
GM2: CSF
Title
Measurements
OG000-43.41± 23.14
GM2: Plasma
Title
Measurements
OG000-55.91± 11.76
6
Title
Denominators
Categories
GL-1: CSF
ParticipantsOG0004
Title
Measurements
OG00017.42± 188.41
GL-1: Plasma
ParticipantsOG0006
Title
Measurements
OG000-80.42± 8.17
GM1: CSF
ParticipantsOG0004
Title
Measurements
OG000-32.35± 17.46
GM1: Plasma
ParticipantsOG0006
Title
Measurements
OG000-52.32± 16.76
OG0001
Title
Denominators
Categories
GL-1: CSF
Title
Measurements
OG000NADue to the rarity of the disease and the specificity of the endpoint, reporting individual-level data would risk compromising participant privacy and confidentiality. As presented in baseline characteristics, only 1 participant with sialidosis type 1 was enrolled in the study.
GL-1: Plasma
Title
Measurements
OG000NADue to the rarity of the disease and the specificity of the endpoint, reporting individual-level data would risk compromising participant privacy and confidentiality. As presented in baseline characteristics, only 1 participant with sialidosis type 1 was enrolled in the study.
GM2: CSF
Title
Measurements
OG000NADue to the rarity of the disease and the specificity of the endpoint, reporting individual-level data would risk compromising participant privacy and confidentiality. As presented in baseline characteristics, only 1 participant with sialidosis type 1 was enrolled in the study.
GM2: Plasma
Title
Measurements
OG000NADue to the rarity of the disease and the specificity of the endpoint, reporting individual-level data would risk compromising participant privacy and confidentiality. As presented in baseline characteristics, only 1 participant with sialidosis type 1 was enrolled in the study.
GM3: CSF
Title
Measurements
OG000NADue to the rarity of the disease and the specificity of the endpoint, reporting individual-level data would risk compromising participant privacy and confidentiality. As presented in baseline characteristics, only 1 participant with sialidosis type 1 was enrolled in the study.
GM3: Plasma
Title
Measurements
OG000NADue to the rarity of the disease and the specificity of the endpoint, reporting individual-level data would risk compromising participant privacy and confidentiality. As presented in baseline characteristics, only 1 participant with sialidosis type 1 was enrolled in the study.
Participants
OG0001
Title
Denominators
Categories
GL-1: CSF
Title
Measurements
OG000NADue to the rarity of the disease and the specificity of the endpoint, reporting individual-level data would risk compromising participant privacy and confidentiality. As presented in baseline characteristics, only 1 participant with juvenile/adult galactosialidosis was enrolled in the study.
GL-1: Plasma
Title
Measurements
OG000NADue to the rarity of the disease and the specificity of the endpoint, reporting individual-level data would risk compromising participant privacy and confidentiality. As presented in baseline characteristics, only 1 participant with juvenile/adult galactosialidosis was enrolled in the study.
GM1: CSF
Title
Measurements
OG000NADue to the rarity of the disease and the specificity of the endpoint, reporting individual-level data would risk compromising participant privacy and confidentiality. As presented in baseline characteristics, only 1 participant with juvenile/adult galactosialidosis was enrolled in the study.
GM1: Plasma
Title
Measurements
OG000NADue to the rarity of the disease and the specificity of the endpoint, reporting individual-level data would risk compromising participant privacy and confidentiality. As presented in baseline characteristics, only 1 participant with juvenile/adult galactosialidosis was enrolled in the study.
GM3: CSF
Title
Measurements
OG000NADue to the rarity of the disease and the specificity of the endpoint, reporting individual-level data would risk compromising participant privacy and confidentiality. As presented in baseline characteristics, only 1 participant with juvenile/adult galactosialidosis was enrolled in the study.
GM3: Plasma
Title
Measurements
OG000NADue to the rarity of the disease and the specificity of the endpoint, reporting individual-level data would risk compromising participant privacy and confidentiality. As presented in baseline characteristics, only 1 participant with juvenile/adult galactosialidosis was enrolled in the study.
0
Title
Denominators
Categories
GL-1: CSF
GL-1: Plasma
OG00016
OG00131
Title
Denominators
Categories
Title
Measurements
OG000-17.64± 2.36
OG001-32.83± 1.70
PAP: PP: Venglustat
Participants in PP received venglustat 15 mg oral tablet once daily for 104 weeks in a double-blind manner in PAP.
OG002
PAP: SP: Venglustat
Participants in SP received venglustat oral tablet once daily based on the body weight for 104 weeks in an open-label manner in PAP as follows:
15 mg if >=50 kg, 12 mg if 30 kg to <50 kg, 6 mg if 15 kg to <30 kg and 4 mg if 10 kg to <15 kg.
Participants in PP received venglustat 15 mg oral tablet once daily for 104 weeks in a double-blind manner in PAP.
OG002
PAP: SP: Venglustat
Participants in SP received venglustat oral tablet once daily based on the body weight for 104 weeks in an open-label manner in PAP as follows:
15 mg if >=50 kg, 12 mg if 30 kg to <50 kg, 6 mg if 15 kg to <30 kg and 4 mg if 10 kg to <15 kg.
Units
Counts
Participants
OG00019
OG00140
OG00216
Title
Denominators
Categories
TEAEs
Title
Measurements
OG00019
OG00140
OG00214
TESAEs
Title
Measurements
OG0004
OG0018
OG0026
30
Title
Denominators
Categories
0 hour
ParticipantsOG00028
Title
Measurements
OG000112± 43.7
0.5 hours
ParticipantsOG00027
Title
Measurements
OG000125± 54.1
3 hours
ParticipantsOG00030
Title
Measurements
OG000188± 60.8
8 hours
ParticipantsOG00029
Title
Measurements
OG000149± 47.3
12 hours
ParticipantsOG00028
Title
Measurements
OG000132± 44.5
24 hours
ParticipantsOG00022
Title
Measurements
OG000105± 38.5
15
Title
Denominators
Categories
4 mg: 0 hour
ParticipantsOG0001
Title
Measurements
OG00056.4
4 mg: 0.5 hours
ParticipantsOG0000
4 mg: 3 hours
ParticipantsOG0001
Title
Measurements
OG000144
4 mg: 8 hours
ParticipantsOG0001
Title
Measurements
OG000101
4 mg: 12 hours
ParticipantsOG0001
Title
Measurements
OG00092.3
4 mg: 24 hours
ParticipantsOG0001
Title
Measurements
OG00058.1
6 mg: 0 hour
ParticipantsOG0007
Title
Measurements
OG00061.5± 34.1
6 mg: 0.5 hours
ParticipantsOG0006
Title
Measurements
OG00085.9± 56.8
6 mg: 3 hours
ParticipantsOG0007
Title
Measurements
OG000127± 72.4
6 mg: 8 hours
ParticipantsOG0007
Title
Measurements
OG00097.9± 55.6
6 mg: 12 hours
ParticipantsOG0007
Title
Measurements
OG00087.9± 55.9
6 mg: 24 hours
ParticipantsOG0005
Title
Measurements
OG00050.2± 25.5
12 mg: 0 hour
ParticipantsOG0004
Title
Measurements
OG00058.4± 40.8
12 mg: 0.5 hours
ParticipantsOG0004
Title
Measurements
OG00067.4± 36.4
12 mg: 3 hours
ParticipantsOG0004
Title
Measurements
OG000112± 40.8
12 mg: 8 hours
ParticipantsOG0004
Title
Measurements
OG00098.2± 22.0
12 mg: 12 hours
ParticipantsOG0004
Title
Measurements
OG00079.3± 35.8
12 mg: 24 hours
ParticipantsOG0003
Title
Measurements
OG00049.5± 17.4
15 mg: 0 hour
ParticipantsOG0003
Title
Measurements
OG00067.7± 7.05
15 mg: 1.5 hours
ParticipantsOG0003
Title
Measurements
OG00086.7± 35.0
15 mg: 3 hours
ParticipantsOG0003
Title
Measurements
OG000124± 16.0
15 mg: 8 hours
ParticipantsOG0003
Title
Measurements
OG00096.4± 10.2
15 mg: 12 hours
ParticipantsOG0003
Title
Measurements
OG00088.9± 6.05
15 mg: 24 hours
ParticipantsOG0003
Title
Measurements
OG00064.5± 13.5
Units
Counts
Participants
OG00018
OG0017
Title
Denominators
Categories
PP
ParticipantsOG00018
ParticipantsOG0010
Title
Measurements
OG0005.64± 2.05
SP: 4 mg
ParticipantsOG0000
ParticipantsOG0010
SP: 6 mg
ParticipantsOG0000
ParticipantsOG0012
Title
Measurements
OG0013.37± 2.19
SP: 12 mg
ParticipantsOG0000
ParticipantsOG0012
Title
Measurements
OG0013.22± 1.01
SP: 15 mg
ParticipantsOG0000
ParticipantsOG0013
Title
Measurements
OG0015.41± 0.57
Units
Counts
Participants
OG00030
OG00115
Title
Denominators
Categories
PP
ParticipantsOG00030
ParticipantsOG0010
Title
Measurements
OG000188± 60.8
SP: 4 mg
ParticipantsOG0000
ParticipantsOG0011
Title
Measurements
OG001144
SP: 6 mg
ParticipantsOG0000
ParticipantsOG0017
Title
Measurements
OG001127± 72.4
SP: 12 mg
ParticipantsOG0000
ParticipantsOG0014
Title
Measurements
OG001121± 39.5
SP: 15 mg
ParticipantsOG0000
ParticipantsOG0013
Title
Measurements
OG001124± 16.0
Units
Counts
Participants
OG00030
OG00115
Title
Denominators
Categories
PP
ParticipantsOG00030
ParticipantsOG0010
Title
Measurements
OG0003.00(2.77 to 7.05)
SP: 4 mg
ParticipantsOG0000
ParticipantsOG0011
Title
Measurements
OG0013.50
SP: 6 mg
ParticipantsOG0000
ParticipantsOG0017
Title
Measurements
OG0013.00(2.25 to 3.02)
SP: 12 mg
ParticipantsOG0000
ParticipantsOG0014
Title
Measurements
OG0015.50(3.00 to 12.00)
SP: 15 mg
ParticipantsOG0000
ParticipantsOG0013
Title
Measurements
OG0013.00(3.00 to 3.00)
Units
Counts
Participants
OG00022
OG00112
Title
Denominators
Categories
PP
ParticipantsOG00022
ParticipantsOG0010
Title
Measurements
OG0003230± 1110
SP: 4 mg
ParticipantsOG0000
ParticipantsOG0011
Title
Measurements
OG0012170
SP: 6 mg
ParticipantsOG0000
ParticipantsOG0015
Title
Measurements
OG0011600± 765
SP: 12 mg
ParticipantsOG0000
ParticipantsOG0013
Title
Measurements
OG0011840± 415
SP: 15 mg
ParticipantsOG0000
ParticipantsOG0013
Title
Measurements
OG0012150± 275
Units
Counts
Participants
OG00011
Title
Denominators
Categories
Juvenile/adolescent late-onset GM2 gangliodosis
ParticipantsOG0004
Title
Measurements
OG000-1.2± 24.5
GM1 gangliosidosis
ParticipantsOG0006
Title
Measurements
OG00011.7± 34.9
Sialidosis type 1
ParticipantsOG0001
Title
Measurements
OG000NADue to the rarity of the disease and the specificity of the endpoint, reporting individual-level data would risk compromising participant privacy and confidentiality.
Juvenile/adult galactosialidosis
ParticipantsOG0000
Saposin C deficiency
ParticipantsOG0000
OG00013
Title
Denominators
Categories
Participants always swallowed the tablet as whole
ParticipantsOG0008
Title
Measurements
OG0008
Participants chewed and swallowed the tablet at least once
ParticipantsOG0005
Title
Measurements
OG0004
0 events
0 affected
16 at risk
EG0040 events0 affected34 at risk
EG0050 events0 affected14 at risk
0 events
0 affected
16 at risk
EG0040 events0 affected34 at risk
EG0050 events0 affected14 at risk
0 events
0 affected
16 at risk
EG0040 events0 affected34 at risk
EG0051 events1 affected14 at risk
0 events
0 affected
16 at risk
EG0040 events0 affected34 at risk
EG0050 events0 affected14 at risk
0 events
0 affected
16 at risk
EG0040 events0 affected34 at risk
EG0050 events0 affected14 at risk
0 events
0 affected
16 at risk
EG0040 events0 affected34 at risk
EG0051 events1 affected14 at risk
0 events
0 affected
16 at risk
EG0040 events0 affected34 at risk
EG0050 events0 affected14 at risk
0 events
0 affected
16 at risk
EG0040 events0 affected34 at risk
EG0050 events0 affected14 at risk
0 events
0 affected
16 at risk
EG0040 events0 affected34 at risk
EG0050 events0 affected14 at risk
1 events
1 affected
16 at risk
EG0040 events0 affected34 at risk
EG0050 events0 affected14 at risk
0 events
0 affected
16 at risk
EG0041 events1 affected34 at risk
EG0050 events0 affected14 at risk
0 events
0 affected
16 at risk
EG0040 events0 affected34 at risk
EG0050 events0 affected14 at risk
1 events
1 affected
16 at risk
EG0040 events0 affected34 at risk
EG0050 events0 affected14 at risk
0 events
0 affected
16 at risk
EG0040 events0 affected34 at risk
EG0050 events0 affected14 at risk
0 events
0 affected
16 at risk
EG0040 events0 affected34 at risk
EG0050 events0 affected14 at risk
1 events
1 affected
16 at risk
EG0040 events0 affected34 at risk
EG0050 events0 affected14 at risk
0 events
0 affected
16 at risk
EG0040 events0 affected34 at risk
EG0050 events0 affected14 at risk
0 events
0 affected
16 at risk
EG0040 events0 affected34 at risk
EG0050 events0 affected14 at risk
0 events
0 affected
16 at risk
EG0040 events0 affected34 at risk
EG0050 events0 affected14 at risk
0 events
0 affected
16 at risk
EG0040 events0 affected34 at risk
EG0050 events0 affected14 at risk
1 events
1 affected
16 at risk
EG0040 events0 affected34 at risk
EG0050 events0 affected14 at risk
0 events
0 affected
16 at risk
EG0041 events1 affected34 at risk
EG0050 events0 affected14 at risk
0 events
0 affected
16 at risk
EG0040 events0 affected34 at risk
EG0050 events0 affected14 at risk
0 events
0 affected
16 at risk
EG0040 events0 affected34 at risk
EG0050 events0 affected14 at risk
0 events
0 affected
16 at risk
EG0040 events0 affected34 at risk
EG0050 events0 affected14 at risk
0 events
0 affected
16 at risk
EG0040 events0 affected34 at risk
EG0050 events0 affected14 at risk
0 events
0 affected
16 at risk
EG0040 events0 affected34 at risk
EG0050 events0 affected14 at risk
1 events
1 affected
16 at risk
EG0040 events0 affected34 at risk
EG0050 events0 affected14 at risk
0 events
0 affected
16 at risk
EG0040 events0 affected34 at risk
EG0051 events1 affected14 at risk
0 events
0 affected
16 at risk
EG0040 events0 affected34 at risk
EG0050 events0 affected14 at risk
0 events
0 affected
16 at risk
EG0040 events0 affected34 at risk
EG0050 events0 affected14 at risk
0 events
0 affected
16 at risk
EG0040 events0 affected34 at risk
EG0051 events1 affected14 at risk
2 events
2 affected
16 at risk
EG0042 events2 affected34 at risk
EG0051 events1 affected14 at risk
0 events
0 affected
16 at risk
EG0040 events0 affected34 at risk
EG0050 events0 affected14 at risk
0 events
0 affected
16 at risk
EG0040 events0 affected34 at risk
EG0057 events1 affected14 at risk
0 events
0 affected
16 at risk
EG0040 events0 affected34 at risk
EG0050 events0 affected14 at risk
0 events
0 affected
16 at risk
EG0040 events0 affected34 at risk
EG0050 events0 affected14 at risk
0 events
0 affected
16 at risk
EG0040 events0 affected34 at risk
EG0050 events0 affected14 at risk
1 events
1 affected
16 at risk
EG0040 events0 affected34 at risk
EG0050 events0 affected14 at risk
1 events
1 affected
16 at risk
EG0040 events0 affected34 at risk
EG0050 events0 affected14 at risk
0 events
0 affected
16 at risk
EG0040 events0 affected34 at risk
EG0050 events0 affected14 at risk
0 events
0 affected
16 at risk
EG0040 events0 affected34 at risk
EG0050 events0 affected14 at risk
0 events
0 affected
16 at risk
EG0040 events0 affected34 at risk
EG0050 events0 affected14 at risk
1 events
1 affected
16 at risk
EG0040 events0 affected34 at risk
EG0050 events0 affected14 at risk
2 events
2 affected
16 at risk
EG0043 events1 affected34 at risk
EG0052 events1 affected14 at risk
0 events
0 affected
16 at risk
EG0040 events0 affected34 at risk
EG0050 events0 affected14 at risk
0 events
0 affected
16 at risk
EG0041 events1 affected34 at risk
EG0050 events0 affected14 at risk
1 events
1 affected
16 at risk
EG0040 events0 affected34 at risk
EG0050 events0 affected14 at risk
0 events
0 affected
16 at risk
EG0040 events0 affected34 at risk
EG0050 events0 affected14 at risk
0 events
0 affected
16 at risk
EG0043 events3 affected34 at risk
EG0050 events0 affected14 at risk
0 events
0 affected
16 at risk
EG0040 events0 affected34 at risk
EG0050 events0 affected14 at risk
1 events
1 affected
16 at risk
EG0040 events0 affected34 at risk
EG0050 events0 affected14 at risk
0 events
0 affected
16 at risk
EG0040 events0 affected34 at risk
EG0050 events0 affected14 at risk
0 events
0 affected
16 at risk
EG0040 events0 affected34 at risk
EG0050 events0 affected14 at risk
0 events
0 affected
16 at risk
EG0044 events3 affected34 at risk
EG0052 events2 affected14 at risk
0 events
0 affected
16 at risk
EG0040 events0 affected34 at risk
EG0050 events0 affected14 at risk
0 events
0 affected
16 at risk
EG0040 events0 affected34 at risk
EG0050 events0 affected14 at risk
0 events
0 affected
16 at risk
EG0040 events0 affected34 at risk
EG0051 events1 affected14 at risk
0 events
0 affected
16 at risk
EG0040 events0 affected34 at risk
EG0050 events0 affected14 at risk
0 events
0 affected
16 at risk
EG0040 events0 affected34 at risk
EG0051 events1 affected14 at risk
0 events
0 affected
16 at risk
EG0040 events0 affected34 at risk
EG0050 events0 affected14 at risk
1 events
1 affected
16 at risk
EG0041 events1 affected34 at risk
EG0050 events0 affected14 at risk
1 events
1 affected
16 at risk
EG0040 events0 affected34 at risk
EG0050 events0 affected14 at risk
0 events
0 affected
16 at risk
EG0041 events1 affected34 at risk
EG0050 events0 affected14 at risk
0 events
0 affected
16 at risk
EG0040 events0 affected34 at risk
EG0051 events1 affected14 at risk
0 events
0 affected
16 at risk
EG0040 events0 affected34 at risk
EG0050 events0 affected14 at risk
0 events
0 affected
16 at risk
EG0041 events1 affected34 at risk
EG0051 events1 affected14 at risk
1 events
1 affected
16 at risk
EG0040 events0 affected34 at risk
EG0050 events0 affected14 at risk
1 events
1 affected
16 at risk
EG0040 events0 affected34 at risk
EG0050 events0 affected14 at risk
0 events
0 affected
16 at risk
EG0040 events0 affected34 at risk
EG0050 events0 affected14 at risk
0 events
0 affected
16 at risk
EG0040 events0 affected34 at risk
EG0050 events0 affected14 at risk
0 events
0 affected
16 at risk
EG0042 events2 affected34 at risk
EG0050 events0 affected14 at risk
1 events
1 affected
16 at risk
EG0040 events0 affected34 at risk
EG0050 events0 affected14 at risk
0 events
0 affected
16 at risk
EG0040 events0 affected34 at risk
EG0050 events0 affected14 at risk
1 events
1 affected
16 at risk
EG0041 events1 affected34 at risk
EG0050 events0 affected14 at risk
0 events
0 affected
16 at risk
EG0042 events2 affected34 at risk
EG0050 events0 affected14 at risk
1 events
1 affected
16 at risk
EG0040 events0 affected34 at risk
EG0050 events0 affected14 at risk
2 events
2 affected
16 at risk
EG0043 events3 affected34 at risk
EG0059 events1 affected14 at risk
0 events
0 affected
16 at risk
EG0040 events0 affected34 at risk
EG0050 events0 affected14 at risk
0 events
0 affected
16 at risk
EG0040 events0 affected34 at risk
EG0050 events0 affected14 at risk
0 events
0 affected
16 at risk
EG0040 events0 affected34 at risk
EG0050 events0 affected14 at risk
0 events
0 affected
16 at risk
EG0040 events0 affected34 at risk
EG0050 events0 affected14 at risk
0 events
0 affected
16 at risk
EG0040 events0 affected34 at risk
EG0050 events0 affected14 at risk
0 events
0 affected
16 at risk
EG0040 events0 affected34 at risk
EG0050 events0 affected14 at risk
0 events
0 affected
16 at risk
EG0040 events0 affected34 at risk
EG0050 events0 affected14 at risk
0 events
0 affected
16 at risk
EG0040 events0 affected34 at risk
EG0050 events0 affected14 at risk
0 events
0 affected
16 at risk
EG0040 events0 affected34 at risk
EG0050 events0 affected14 at risk
0 events
0 affected
16 at risk
EG0040 events0 affected34 at risk
EG0050 events0 affected14 at risk
0 events
0 affected
16 at risk
EG0040 events0 affected34 at risk
EG0050 events0 affected14 at risk
0 events
0 affected
16 at risk
EG0041 events1 affected34 at risk
EG0051 events1 affected14 at risk
0 events
0 affected
16 at risk
EG0040 events0 affected34 at risk
EG0050 events0 affected14 at risk
0 events
0 affected
16 at risk
EG0041 events1 affected34 at risk
EG0050 events0 affected14 at risk
0 events
0 affected
16 at risk
EG0040 events0 affected34 at risk
EG0050 events0 affected14 at risk
0 events
0 affected
16 at risk
EG0040 events0 affected34 at risk
EG0050 events0 affected14 at risk
0 events
0 affected
16 at risk
EG0040 events0 affected34 at risk
EG0050 events0 affected14 at risk
0 events
0 affected
16 at risk
EG0040 events0 affected34 at risk
EG0050 events0 affected14 at risk
0 events
0 affected
16 at risk
EG0040 events0 affected34 at risk
EG0050 events0 affected14 at risk
0 events
0 affected
16 at risk
EG0040 events0 affected34 at risk
EG0050 events0 affected14 at risk
0 events
0 affected
16 at risk
EG0040 events0 affected34 at risk
EG0050 events0 affected14 at risk
0 events
0 affected
16 at risk
EG0040 events0 affected34 at risk
EG0050 events0 affected14 at risk
0 events
0 affected
16 at risk
EG0040 events0 affected34 at risk
EG0051 events1 affected14 at risk
0 events
0 affected
16 at risk
EG0040 events0 affected34 at risk
EG0051 events1 affected14 at risk
0 events
0 affected
16 at risk
EG0040 events0 affected34 at risk
EG0050 events0 affected14 at risk
0 events
0 affected
16 at risk
EG0040 events0 affected34 at risk
EG0050 events0 affected14 at risk
0 events
0 affected
16 at risk
EG0040 events0 affected34 at risk
EG0050 events0 affected14 at risk
0 events
0 affected
16 at risk
EG0040 events0 affected34 at risk
EG0050 events0 affected14 at risk
0 events
0 affected
16 at risk
EG0040 events0 affected34 at risk
EG0050 events0 affected14 at risk
0 events
0 affected
16 at risk
EG0041 events1 affected34 at risk
EG0050 events0 affected14 at risk
0 events
0 affected
16 at risk
EG0040 events0 affected34 at risk
EG0050 events0 affected14 at risk
0 events
0 affected
16 at risk
EG0040 events0 affected34 at risk
EG0050 events0 affected14 at risk
0 events
0 affected
16 at risk
EG0040 events0 affected34 at risk
EG0051 events1 affected14 at risk
0 events
0 affected
16 at risk
EG0040 events0 affected34 at risk
EG0050 events0 affected14 at risk
0 events
0 affected
16 at risk
EG0040 events0 affected34 at risk
EG0050 events0 affected14 at risk
0 events
0 affected
16 at risk
EG0041 events1 affected34 at risk
EG0051 events1 affected14 at risk
0 events
0 affected
16 at risk
EG0040 events0 affected34 at risk
EG0050 events0 affected14 at risk
1 events
1 affected
16 at risk
EG0040 events0 affected34 at risk
EG0050 events0 affected14 at risk
1 events
1 affected
16 at risk
EG0042 events2 affected34 at risk
EG0051 events1 affected14 at risk
0 events
0 affected
16 at risk
EG0040 events0 affected34 at risk
EG0050 events0 affected14 at risk
0 events
0 affected
16 at risk
EG0041 events1 affected34 at risk
EG0050 events0 affected14 at risk
0 events
0 affected
16 at risk
EG0040 events0 affected34 at risk
EG0050 events0 affected14 at risk
0 events
0 affected
16 at risk
EG0041 events1 affected34 at risk
EG0050 events0 affected14 at risk
0 events
0 affected
16 at risk
EG0040 events0 affected34 at risk
EG0050 events0 affected14 at risk
0 events
0 affected
16 at risk
EG0041 events1 affected34 at risk
EG0050 events0 affected14 at risk
2 events
2 affected
16 at risk
EG0041 events1 affected34 at risk
EG0050 events0 affected14 at risk
0 events
0 affected
16 at risk
EG0040 events0 affected34 at risk
EG0050 events0 affected14 at risk
0 events
0 affected
16 at risk
EG0040 events0 affected34 at risk
EG0050 events0 affected14 at risk
0 events
0 affected
16 at risk
EG0040 events0 affected34 at risk
EG0050 events0 affected14 at risk
1 events
1 affected
16 at risk
EG0040 events0 affected34 at risk
EG0050 events0 affected14 at risk
0 events
0 affected
16 at risk
EG0040 events0 affected34 at risk
EG0050 events0 affected14 at risk
0 events
0 affected
16 at risk
EG0040 events0 affected34 at risk
EG0050 events0 affected14 at risk
0 events
0 affected
16 at risk
EG0040 events0 affected34 at risk
EG0051 events1 affected14 at risk
0 events
0 affected
16 at risk
EG0040 events0 affected34 at risk
EG0050 events0 affected14 at risk
0 events
0 affected
16 at risk
EG0040 events0 affected34 at risk
EG0050 events0 affected14 at risk
1 events
1 affected
16 at risk
EG0040 events0 affected34 at risk
EG0050 events0 affected14 at risk
0 events
0 affected
16 at risk
EG0040 events0 affected34 at risk
EG0050 events0 affected14 at risk
0 events
0 affected
16 at risk
EG0040 events0 affected34 at risk
EG0050 events0 affected14 at risk
0 events
0 affected
16 at risk
EG0040 events0 affected34 at risk
EG0050 events0 affected14 at risk
0 events
0 affected
16 at risk
EG0040 events0 affected34 at risk
EG0051 events1 affected14 at risk
3 events
3 affected
16 at risk
EG0040 events0 affected34 at risk
EG0050 events0 affected14 at risk
0 events
0 affected
16 at risk
EG0041 events1 affected34 at risk
EG0051 events1 affected14 at risk
0 events
0 affected
16 at risk
EG0040 events0 affected34 at risk
EG0050 events0 affected14 at risk
0 events
0 affected
16 at risk
EG0040 events0 affected34 at risk
EG0050 events0 affected14 at risk
1 events
1 affected
16 at risk
EG0040 events0 affected34 at risk
EG0050 events0 affected14 at risk
0 events
0 affected
16 at risk
EG0040 events0 affected34 at risk
EG0050 events0 affected14 at risk
0 events
0 affected
16 at risk
EG0040 events0 affected34 at risk
EG0050 events0 affected14 at risk
0 events
0 affected
16 at risk
EG0040 events0 affected34 at risk
EG0050 events0 affected14 at risk
0 events
0 affected
16 at risk
EG0040 events0 affected34 at risk
EG0051 events1 affected14 at risk
0 events
0 affected
16 at risk
EG0040 events0 affected34 at risk
EG0052 events1 affected14 at risk
0 events
0 affected
16 at risk
EG0040 events0 affected34 at risk
EG0050 events0 affected14 at risk
0 events
0 affected
16 at risk
EG0040 events0 affected34 at risk
EG0050 events0 affected14 at risk
0 events
0 affected
16 at risk
EG0040 events0 affected34 at risk
EG0050 events0 affected14 at risk
0 events
0 affected
16 at risk
EG0040 events0 affected34 at risk
EG0050 events0 affected14 at risk
1 events
1 affected
16 at risk
EG0040 events0 affected34 at risk
EG0050 events0 affected14 at risk
1 events
1 affected
16 at risk
EG0040 events0 affected34 at risk
EG0050 events0 affected14 at risk
0 events
0 affected
16 at risk
EG0040 events0 affected34 at risk
EG0050 events0 affected14 at risk
0 events
0 affected
16 at risk
EG0040 events0 affected34 at risk
EG0051 events1 affected14 at risk
0 events
0 affected
16 at risk
EG0040 events0 affected34 at risk
EG0050 events0 affected14 at risk
0 events
0 affected
16 at risk
EG0040 events0 affected34 at risk
EG0050 events0 affected14 at risk
1 events
1 affected
16 at risk
EG0040 events0 affected34 at risk
EG0050 events0 affected14 at risk
0 events
0 affected
16 at risk
EG0040 events0 affected34 at risk
EG0050 events0 affected14 at risk
0 events
0 affected
16 at risk
EG0040 events0 affected34 at risk
EG0052 events1 affected14 at risk
0 events
0 affected
16 at risk
EG0040 events0 affected34 at risk
EG0050 events0 affected14 at risk
0 events
0 affected
16 at risk
EG0040 events0 affected34 at risk
EG0051 events1 affected14 at risk
0 events
0 affected
16 at risk
EG0040 events0 affected34 at risk
EG0051 events1 affected14 at risk
0 events
0 affected
16 at risk
EG0042 events2 affected34 at risk
EG0050 events0 affected14 at risk
0 events
0 affected
16 at risk
EG0040 events0 affected34 at risk
EG0050 events0 affected14 at risk
0 events
0 affected
16 at risk
EG0040 events0 affected34 at risk
EG0050 events0 affected14 at risk
0 events
0 affected
16 at risk
EG0040 events0 affected34 at risk
EG0050 events0 affected14 at risk
0 events
0 affected
16 at risk
EG0040 events0 affected34 at risk
EG0050 events0 affected14 at risk
1 events
1 affected
16 at risk
EG0040 events0 affected34 at risk
EG0050 events0 affected14 at risk
0 events
0 affected
16 at risk
EG0040 events0 affected34 at risk
EG0050 events0 affected14 at risk
0 events
0 affected
16 at risk
EG0040 events0 affected34 at risk
EG0050 events0 affected14 at risk
0 events
0 affected
16 at risk
EG0040 events0 affected34 at risk
EG0050 events0 affected14 at risk
1 events
1 affected
16 at risk
EG0040 events0 affected34 at risk
EG0050 events0 affected14 at risk
0 events
0 affected
16 at risk
EG0040 events0 affected34 at risk
EG0050 events0 affected14 at risk
0 events
0 affected
16 at risk
EG0040 events0 affected34 at risk
EG0050 events0 affected14 at risk
0 events
0 affected
16 at risk
EG0040 events0 affected34 at risk
EG0050 events0 affected14 at risk
2 events
2 affected
16 at risk
EG0040 events0 affected34 at risk
EG0050 events0 affected14 at risk
0 events
0 affected
16 at risk
EG0040 events0 affected34 at risk
EG0050 events0 affected14 at risk
3 events
2 affected
16 at risk
EG0044 events4 affected34 at risk
EG0050 events0 affected14 at risk
0 events
0 affected
16 at risk
EG0040 events0 affected34 at risk
EG0050 events0 affected14 at risk
0 events
0 affected
16 at risk
EG0040 events0 affected34 at risk
EG0050 events0 affected14 at risk
0 events
0 affected
16 at risk
EG0040 events0 affected34 at risk
EG0050 events0 affected14 at risk
0 events
0 affected
16 at risk
EG0040 events0 affected34 at risk
EG0051 events1 affected14 at risk
1 events
1 affected
16 at risk
EG0040 events0 affected34 at risk
EG0050 events0 affected14 at risk
0 events
0 affected
16 at risk
EG0040 events0 affected34 at risk
EG0050 events0 affected14 at risk
0 events
0 affected
16 at risk
EG0040 events0 affected34 at risk
EG0050 events0 affected14 at risk
0 events
0 affected
16 at risk
EG0040 events0 affected34 at risk
EG0050 events0 affected14 at risk
0 events
0 affected
16 at risk
EG0040 events0 affected34 at risk
EG0050 events0 affected14 at risk
1 events
1 affected
16 at risk
EG0041 events1 affected34 at risk
EG0050 events0 affected14 at risk
29.6
± 66.5
0.4
± 0.8
2.8
± 10.6
-2.5
± 17.0
NA
Due to the rarity of the disease and the specificity of the endpoint, reporting individual-level data would risk compromising participant privacy and confidentiality.