A Global Study to Assess the Drug Dynamics, Efficacy, and... | NCT02906020 | Trialant
NCT02906020
Sponsor
Genzyme, a Sanofi Company
Status
Terminated
Last Update Posted
May 24, 2022Actual
Enrollment
273Actual
Phase
Phase 2
Conditions
Parkinson's Disease
Interventions
venglustat GZ/SAR402671
Placebo
Countries
United States
Austria
Canada
France
Germany
Greece
Israel
Italy
Japan
Norway
Portugal
Singapore
Spain
Sweden
Taiwan
United Kingdom
Protocol Section
Identification Module
NCT ID
NCT02906020
Obsolete or Duplicate NCT IDs
Not provided
Organization Study
ACT14820
Secondary IDs
ID
Type
Description
Link
2016-000657-12
EudraCT Number
U1111-1180-6918
Registry Identifier
ICTRP
Brief Title
A Global Study to Assess the Drug Dynamics, Efficacy, and Safety of Venglustat (GZ/SAR402671) in Parkinson's Disease Patients Carrying a Glucocerebrosidase (GBA) Gene Mutation
Official Title
Multicenter, Randomized, Double-blind, Placebo Controlled Study to Assess the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of GZ/SAR402671 in Patients With Early-stage Parkinson's Disease Carrying a GBA Mutation or Other Pre-specified Variant
Acronym
MOVES-PD
Organization
SanofiINDUSTRY
Status Module
Record Verification Date
May 2022
Overall Recruitment Status or Expanded Access Status
Terminated
Last Known Status
Not provided
Delayed Posting
Not provided
Why Stopped
The topline results of the 52-week double-blind placebo-controlled period were analyzed. The study did not meet the primary or secondary endpoints. Based on these results, the decision was made to halt the long-term follow-up period of the study
Expanded Access Info
No
Start Date
Dec 15, 2016Actual
Primary Completion Date
Dec 18, 2020Actual
Completion Date
May 27, 2021Actual
First Submitted Date
Sep 14, 2016
First Submission Date that Met QC Criteria
Sep 14, 2016
First Posted Date
Sep 19, 2016Estimated
Results Waived
Not provided
Results First Submitted Date
Dec 16, 2021
Results First Submitted that Met QC Criteria
Feb 2, 2022
Results First Posted Date
Feb 28, 2022Actual
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
May 3, 2022
Last Update Posted Date
May 24, 2022Actual
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:
Part 1: To determine the safety and tolerability of 4, 8, and 15 milligrams of GZ/SAR402671 (venglustat) administered orally for 4 weeks, as compared to placebo in participants with early-stage Parkinson's disease (PD) carrying a glucocerebrosidase gene (GBA) mutation or other pre-specified variants.
Part 2: To determine the efficacy of GZ/SAR402671 administered orally daily, as compared to placebo in participants with early-stage PD carrying a GBA mutation or other pre-specified variants.
Secondary Objectives:
Part 1:
To assess the pharmacokinetic (PK) profile of oral dosing of GZ/SAR402671 in plasma when administered in early-stage PD participants carrying a GBA mutation.
To assess the exposure of GZ/SAR402671 in cerebrospinal fluid (CSF) when administered in early-stage PD participants carrying a GBA mutation.
Part 2:
To demonstrate overall safety and tolerability of GZ/SAR402671 administered orally for 52 weeks in early-stage PD participants carrying a GBA mutation as compared to placebo.
To assess the pharmacodynamic response to daily oral dosing of GZ/SAR402671 in plasma and CSF as measured by glucosylceramide (GL-1) when administered in early-stage PD participants carrying a GBA mutation over a 52-week period.
Detailed Description
Part 1: the total duration was as following:
i) Rest of the world (ROW): up to approximately 50 weeks (8.5 weeks of screening, maximum of 36 weeks of treatment and 6 weeks follow-up).
ii) Japan only: up to approximately 66 weeks (8.5 weeks of screening, maximum of 52 weeks of treatment and 6 weeks follow-up).
Part 2: the total duration was up to approximately 224 weeks that consisted of 8.5 weeks of screening period, 52 weeks of treatment period, 156 weeks of long-term follow-up (LTFU) period and 8 weeks of post-treatment period.
At the end of a 52-week main placebo-controlled treatment period, all participants were evaluated for possibility to transition to receive active treatment for 156 weeks plus 8-week post-treatment observation.
Conditions Module
Conditions
Parkinson's 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 2
Interventional Study Design
Allocation
Biospecimen
No data available
No data is available for this block.
Enrollment
273Actual
Arms/Interventions Module
Arm Groups
Label
Type
Description
Intervention Names
GZ/SAR402671
Experimental
Part 1: Increasing doses of GZ/SAR402671 were administered once per day. Part 2: A dose of GZ/SAR402671 (determined in Part 1) was administered once per day.
Drug: venglustat GZ/SAR402671
Placebo
Placebo Comparator
A matching placebo for Parts 1 and 2 was administered once per day.
Drug: Placebo
Interventions
Name
Type
Description
Arm Group Labels
Other Names
venglustat GZ/SAR402671
Drug
Pharmaceutical form: capsule Route of administration: oral
GZ/SAR402671
Outcomes Module
Primary Outcomes
Measure
Description
Time Frame
Part 1: Number of Participants With Treatment-Emergent Adverse Events (TEAEs), and Treatment-Emergent Serious Adverse Events (TESAEs)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug and does not necessarily had to have a causal relationship with the treatment. Serious AEs (SAEs) were any untoward medical occurrence that resulted in any of the following outcomes: death, life-threatening, required initial or prolonged in-patient hospitalization, persistent or significant disability/incapacity, congenital anomaly/birth defect, or considered as medically important event. TEAEs were the AEs that developed or worsened or became serious during the TEAE period (defined as the period from the time of first investigational medicinal product [IMP] administration up of 6 weeks after the last administration of the IMP).
From the first IMP administration up to 6 weeks after the last administration of the IMP (i.e., up to 42 weeks for ROW and up to 58 weeks for Japanese participants)
Part 1: Number of Participants With Abnormal Physical Examination Findings
Physical examination included following observations/measurements: general appearance; heart, skin, respiratory auscultation; head, eyes, ears, nose, and throat, extremities/joints, and abdomen. New onset of abnormal physical examination was defined as a normal physical examination at Baseline and an abnormal physical examination during the treatment-emergent (TE) period (defined as the period from the time of first IMP administration up of 6 weeks after the last administration of the IMP). Abnormalities in physical examination were based on investigator's evaluation.
From the first IMP administration up to 6 weeks after the last administration of the IMP (i.e., up to 42 weeks for ROW and up to 58 weeks for Japanese participants)
Part 1: Number of Participants With Abnormal Neurological Examination Findings
Neurological examination included at least assessments of the participant's cranial nerves, motor system (including muscle atrophy, tone, and power), mental status, deep tendon reflex, sensation, and cerebellar function. Abnormalities in neurological examination were based on investigator's evaluation.
Secondary Outcomes
Measure
Description
Time Frame
Part 2: Change From Baseline to Week 52 in Parkinson's Disease Cognitive Rating Scale (PD-CRS) Total Score
The PD-CRS detects early cognitive impairment in Parkinson's disease. It is composed of 2 scales, the fronto-subcortical scale (items: sustained attention, working memory, alternating and action verbal fluency, clock drawing, immediate, and delayed free recall verbal memory) and the posterior-cortical scale (items: confrontation naming and clock copying). The total score of the fronto-subcortical scale (sum of all items) ranged from 0 (worst) to 104 (maximum score indicates better) and the total score of the posterior-cortical scale (sum of all items) ranged from 0 (worst) to 30 (maximum score indicates better). The PD-CRS Total score = the sum of PD-CRS fronto-subcortical score and the PD-CRS posterior-cortical score, which ranged from 0 to 134, where higher score = less impairment.
Other Outcomes
Not provided
Eligibility Module
Eligibility Criteria
Inclusion criteria:
Male and female adults with a diagnosis of PD and who were heterozygous carriers of a GBA mutation associated with PD.
Participants carrying known sequence variants associated with GBA-PD must had rapid eye movement (REM) sleep behavior disorder (RBD) confirmed by historically documented polysomnography or by questionnaire.
Age greater than or equal to (>=) 18 years to 80 years inclusive at the time of informed consent signing (FOR JAPANESE PARTICIPANTS ONLY: Age >=20 years to 80 years, inclusive, at the time of signing the informed consent. Note: Japanese participants refers only to Japanese participants enrolled and living in Japan).
Had symptoms of PD >=2 years.
Hoehn and Yahr (H and Y) stage of 2 or lower at baseline.
Stable medication regimen of PD drugs for at least 30 days (at least 60 days for rasagiline) prior to randomization.
The participant was willing to abstain from grapefruit containing products for 72 hours prior to administration of the first dose of GZ/SAR402671 and for the duration of the entire treatment period (Part 1 and Part 2, Periods 2 and 3).
Signed written consent.
Exclusion criteria:
Parkinsonism due to drug(s) or toxin(s).
Participants carrying the LRRK2 G2019S mutation.
Participants with Gaucher disease (GD) as defined by clinical signs and symptoms (i.e., hepatosplenomegaly, cytopenia, skeletal disease) and/or marked deficiency of GCase activity compatible with GD.
Montreal Cognitive Assessment score less than 20.
Participants with prior surgical history of deep brain stimulation (DBS).
Participants with baseline brain MRI without contrast showing a structural abnormality that is a possible cause of their PD signs or symptoms.
Hepatic insufficiency with liver function tests (LFT) greater than (>) 2 times upper limit of normal at Screening Visit.
The participant had a documented diagnosis, as per local regulations, of any of the following infections: hepatitis B, hepatitis C, human immunodeficiency virus 1 or 2.
Renal insufficiency as defined by creatine >1.5 times normal at Screening Visit.
The participant had received strong or moderate inducers or inhibitors of CYP3A4 within 30 days or 5 half-lives prior to randomization, whichever is longer.
The participant had, according to World Health Organization (WHO) Grading, a cortical cataract > one-quarter the lens circumference (grade cortical catact-2 [COR-2]) or a posterior subcapsular cataract >2 millimeters (grade posterior subscapsular cataract [PSC-2]). Participant with nuclear cataracts would not be excluded.
The participant was currently receiving potentially cataractogenic medications, including chronic regimen (more frequently than every 2 weeks) of any dose or route of corticosteroids or any medication that could cause cataract or worsen the vision of participants with cataract (eg, glaucoma medications) according to the Prescribing Information.
If female, pregnant (defined as positive beta-human chorionic gonadotrophin [Beta-HCG] blood test) or lactating or breast-feeding.
Any medical disorders and/or clinically relevant findings that, in the opinion of the Investigator, could interfere with study-related procedures. This included condition(s) that precluded the safe performance of routine lumbar punctures, such as prohibitive spinal diseases, bleeding diasthesis, or clinically significant coagulopathy or thrombocytopenia.
Current participation in another investigational interventional study.
Any medications specifically used for treating memory dysfunction, such as, but not limited to cholinesterase inhibitors or memantine, within 30 days or 5 half-lives of these medications prior to randomization, whichever was longer.
The above information was not intended to contain all considerations relevant to a participant's potential participation in a clinical trial.
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
Post part 1 completion, 23 eligible & willing participants were re-randomized in Part 2 and were counted again in total enrollment number (273). i.e.,250 unique participants (29 in Part 1+221 in Part 2)+23 re-randomized in Part 2; these 23 participants are displayed only in 'participant flow' & 'adverse events' sections but not in any other section.
Recruitment Details
Study was conducted at 52 sites in 16 countries. A total of 273 participants were enrolled from 15-Dec-2016 to 18-Dec-2019. Study consisted of 2 Parts: Part 1 (dose escalation period) & Part 2 (double-blind [DB] treatment period + long-term follow-up [LTFU]) period.
Type of Units Analyzed
Not provided
Arm/Group Information
ID
Title
Description
FG000
Part 1: Placebo (ROW)
Participants from rest of world (ROW, except Japan), received placebo (matched to venglustat) capsule orally once daily (QD) for up to 36 weeks in Part 1.
FG001
Part 1: Venglustat 4 mg (ROW)
Periods
Title
Milestones
Reasons Not Completed
Part 1: Dose Escalation Period
Type
Comment
Milestone Data
STARTED
Baseline Characteristics Module
Baseline Analysis Population Description
Outcome Measures Module
Outcome Measures
Adverse Events Module
Frequency Threshold
5
More Info Module
Limitations and Caveats
Annotation Section
No data available
No data is available for this block.
Document Section
Large Document Module
Document Has No Statistical Analysis Plan (SAP)
Not provided
Uploaded Document Information
Type
Includes Protocol
Includes SAP
Includes ICF
Document Label
Document Date
Document Uploaded Date
Document File Name
Prot
Yes
No
No
Study Protocol
Feb 9, 2021
Dec 16, 2021
Derived Section
Miscellaneous Info Module
Version Holder
Jul 10, 2026
Removed Countries
Not provided
Submission Tracking
No data available
No data is available for this block.
Condition Browse Module
MeSH Terms
Intervention Browse Module
MeSH Terms
Randomized
Intervention Model
Parallel Assignment
Intervention Model Description
Not provided
Primary Purpose
Treatment
Observational Model
Not provided
Time Perspective
Not provided
Masking Info
Masking
Triple
Masking Description
Not provided
Who Masked
ParticipantInvestigatorOutcomes Assessor
Placebo
Drug
Pharmaceutical form: capsule Route of administration: oral
Placebo
From the first IMP administration up to 6 weeks after the last administration of the IMP (i.e., up to 42 weeks for ROW and up to 58 weeks for Japanese participants)
Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Hematology
Criteria for potentially clinically significant abnormalities (PCSA): Hemoglobin: less than or equal to (<=) 115 grams per liter (g/L) (Male[M]) or <=95 g/L (Female[F]), greater than or equal to (>=) 185 g/L (M) or >=165 g/L (F), Decrease from baseline (DFB) >=20 g/L; Hematocrit: <=0.37 volume/volume (v/v) (M) or <=0.32 v/v (F), >=0.55 v/v (M) or >=0.5 v/v (F); Red blood cells (RBC): >=6 Tera/L; Platelets: less than (<) 100 Giga/L, >=700 Giga/L; White blood cells (WBC): <3.0 Giga/L (Non-Black [NB]) or <2.0 Giga/L (Black [B]), >=16.0 Giga/L; Neutrophils: <1.5 Giga/L (NB) or <1.0 Giga/L (B); Lymphocytes: <lower limit of normal (LLN), greater than (>) 4.0 Giga/L; Monocytes: <LLN, >0.7 Giga/L; Basophils: >0.1 Giga/L; Eosinophils: >0.5 Giga/L or >upper limit of normal (ULN) (if ULN >=0.5 Giga/L).
From the first IMP administration up to 6 weeks after the last administration of the IMP (i.e., up to 42 weeks for ROW and up to 58 weeks for Japanese participants)
Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Liver Function Parameters
Criteria for PCSA: Alanine Aminotransferase (ALT): >3 ULN, >5 ULN; Aspartate aminotransferase (AST): >3 ULN; Alkaline phosphatase: >1.5 ULN; Total Bilirubin: >1.5 ULN; ALT and Bilirubin: >3 ULN and >2 ULN; Direct Bilirubin and Bilirubin: >35% and >1.5 ULN.
From the first IMP administration up to 6 weeks after the last administration of the IMP (i.e., up to 42 weeks for ROW and up to 58 weeks for Japanese participants)
Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Renal Parameters
Criteria for PCSA: Creatinine: >=150 micromoles per liter (mcmol/L) (adults), >=30% change from baseline, >=100% change from baseline; Blood urea nitrogen: >=17 millimoles (mmol)/L.
From the first IMP administration up to 6 weeks after the last administration of the IMP (i.e., up to 42 weeks for ROW and up to 58 weeks for Japanese participants)
Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Metabolic Parameters
Criteria for PCSA: Glucose: <=3.9 mmol/L and <LLN; >=11.1 mmol/L (unfasted [unfas]) or >=7 mmol/L (fasted [fas]); Albumin: <=25 g/L.
From the first IMP administration up to 6 weeks after the last administration of the IMP (i.e., up to 42 weeks for ROW and up to 58 weeks for Japanese participants)
Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Electrolytes Parameters
Criteria for PCSA: Sodium: <=129 mmol/L, >=160 mmol/L; Potassium: <3 mmol/L, >=5.5 mmol/L and Chloride: <80 mmol/L, >115 mmol/L.
From the first IMP administration up to 6 weeks after the last administration of the IMP (i.e., up to 42 weeks for ROW and up to 58 weeks for Japanese participants)
Part 1: Number of Participants With Potentially Clinically Significant Vital Signs Abnormalities
Criteria for PCSA: Systolic blood pressure (SBP) supine: <=95 millimeters of mercury (mmHg) and DFB >=20 mmHg; >=160 mmHg and increase from baseline (IFB) >=20 mmHg; Diastolic blood pressure (DBP) supine: <=45 mmHg and DFB >=10 mmHg; >=110 mmHg and IFB >=10 mmHg; SBP (Orthostatic): <=-20 mmHg; DBP (Orthostatic): <=-10 mmHg; Heart rate (HR) supine: <=50 beats per minute (bpm) and DFB >=20 bpm; >=120 bpm and IFB >=20 bpm; Weight: >=5% DFB; >=5% IFB.
From the first IMP administration up to 6 weeks after the last administration of the IMP (i.e., up to 42 weeks for ROW and up to 58 weeks for Japanese participants)
Part 1: Number of Participants With Potentially Clinically Significant Ophthalmological Abnormalities
Clinically significant observations in left eye, right eye and any eye (any eye among left and right) were assessed by the investigator based on methods like visual acuity, slit lamp examination, examination of the cornea, lens, and retina. Any abnormal clinically significant ophthalmological examination finding (which was present at screening or not) on any eye during the treatment-emergent period corresponded to cornea verticillata, cataract and abnormal overall evaluation
From the first IMP administration up to 6 weeks after the last administration of the IMP (i.e., up to 42 weeks for ROW and up to 58 weeks for Japanese participants)
Part 1: Number of Participants With Potentially Clinically Significant Electrocardiogram Abnormalities
From the first IMP administration up to 6 weeks after the last administration of the IMP (i.e., up to 42 weeks for ROW and up to 58 weeks for Japanese participants)
Part 2: Change From Baseline to Week 52 in Movement Disorder Society-Unified Parkinson's Disease Rating Scale (MDS-UPDRS) Part II+III Total Score
MDS-UPDRS is a multimodal scale consisting of 4 parts. Part II assessed motor experiences of daily living (total score range: 0 to 52). It contained 13 questions completed by the participant. Part III assessed the motor signs of PD and was administered by the rater (total score range: 0 to 132). Part III contained 33 scores based on 18 items. In both parts, higher score indicated more severe symptoms. For each question in both parts, numeric score was assigned between 0 to 4, where 0=Normal, 1=Slight, 2=Mild, 3=Moderate, 4=Severe. MDS-UPDRS Total Part II and III score = sum of Part II and III scores with score ranged from 0 (no symptom) to 184 (severe symptoms), where higher scores reflected more severe symptoms of PD. Data for this OM was not planned to be collected and analyzed for Part 1, Part 2: DB period re-randomized participants and Part 2 LTFU period, as pre-specified in protocol.
Baseline to Week 52
Baseline to Week 52
Part 2: Change From Baseline to Week 52 in Movement Disorder Society-Unified Parkinson's Disease Rating Scale (MDS-UPDRS) Part I+ II+III Score
MDS-UPDRS is a multimodal scale consisting of 4 parts. Part I assessed non-motor experiences of daily living and has 2 components (total score range: 0 to 52): Part IA contained 6 questions and was assessed by the examiner (total score range: 0 to 24). Part IB contained 7 questions on non-motor experiences of daily living which was completed by the participant (total score range: 0 to 28). Part II (13 questions completed by the participant) assessed motor experiences of daily living (total score range: 0 to 52). Part III assessed motor signs of PD and was administered by the rater (total score range: 0 to 132). Part III contained 33 scores based on 18 items. In all parts, higher score indicated more symptoms. For each question, numeric score was assigned between 0 to 4, where 0=Normal, 1=Slight, 2=Mild, 3=Moderate, 4=Severe. MDS-UPDRS total score = sum of Parts I, II, and III (Range: 0 to 236). Higher score = more severe symptoms of PD.
Baseline to Week 52
Part 2: Change From Baseline to Week 52 in Hoehn and Yahr (H and Y) Score
H and Y scale measured how Parkinson's symptoms progress and the level of disability. Scale allocated stage scores were from 0 to 5 to indicate relative level of disability as: Stage 0: no symptoms; Stage 1: symptoms on one side of the body only; Stage 2: symptoms on both sides of the body, without impairment of balance; Stage 3: Mild to moderate bilateral disease; some postural instability; physically independent; Stage 4: Severe disability; still able to walk or stand unassisted and Stage 5: Wheelchair bound or bedridden unless aided, where higher stage score described an increased severity of disease.
Baseline to Week 52
Scottsdale
Arizona
85259
United States
Investigational Site Number :8400004
La Jolla
California
92093
United States
Investigational Site Number :8400019
Palo Alto
California
94304
United States
Investigational Site Number :8400013
Sunnyvale
California
94085
United States
Investigational Site Number :8400015
New Haven
Connecticut
06510
United States
Investigational Site Number :8400008
Boca Raton
Florida
33486
United States
Investigational Site Number :8400016
Chicago
Illinois
60611
United States
Investigational Site Number :8400005
Chicago
Illinois
60612-3854
United States
Investigational Site Number :8400014
Boston
Massachusetts
02114
United States
Investigational Site Number :8400018
New York
New York
10003
United States
Investigational Site Number :8400010
New York
New York
10016
United States
Investigational Site Number :8400001
New York
New York
10032
United States
Investigational Site Number :8400021
Portland
Oregon
97210
United States
Investigational Site Number :8400020
Portland
Oregon
97225
United States
Investigational Site Number :8400009
Portland
Oregon
97239
United States
Investigational Site Number :8400002
Philadelphia
Pennsylvania
19107
United States
Investigational Site Number :8400006
Fairfax
Virginia
22030
United States
Investigational Site Number :8400012
Kirkland
Washington
98034
United States
Investigational Site Number :0400001
Innsbruck
6020
Austria
Investigational Site Number :1240003
Vancouver
British Columbia
V6T 2B5
Canada
Investigational Site Number :1240002
Ottawa
Ontario
K1Y 4E9
Canada
Investigational Site Number :1240001
Montreal
Quebec
H3A 2B4
Canada
Investigational Site Number :2500001
Paris
75013
France
Investigational Site Number :2760002
Kiel
24105
Germany
Investigational Site Number :2760001
Tübingen
72076
Germany
Investigational Site Number :3000002
Larissa
41110
Greece
Investigational Site Number :3760002
Haifa
3109601
Israel
Investigational Site Number :3760003
Petah Tikva
49100
Israel
Investigational Site Number :3760001
Tel Aviv
64239
Israel
Investigational Site Number :3760004
Tel Litwinsky
52621
Israel
Investigational Site Number :3800006
Rozzano
Milano
20089
Italy
Investigational Site Number :3800001
Catanzaro
88100
Italy
Investigational Site Number :3800004
Milan
20126
Italy
Investigational Site Number :3800003
Pavia
27100
Italy
Investigational Site Number :3800002
Salerno
84131
Italy
Investigational Site Number :3920005
Nagoya
Aichi-ken
466-8560
Japan
Investigational Site Number :3920002
Kyoto
Kyoto
606-8507
Japan
Investigational Site Number :3920004
Osaka
Osaka
530-8480
Japan
Investigational Site Number :3920001
Bunkyo-ku
Tokyo
113-8431
Japan
Investigational Site Number :3920003
Kodaira-shi
Tokyo
187-8551
Japan
Investigational Site Number :5780001
Trondheim
7006
Norway
Investigational Site Number :6200002
Coimbra
3000-075
Portugal
Investigational Site Number :6200003
Torres Vedras
2560-280
Portugal
Investigational Site Number :7020001
Singapore
169608
Singapore
Investigational Site Number :7020002
Singapore
308433
Singapore
Investigational Site Number :7240002
Barcelona
Barcelona [Barcelona]
08025
Spain
Investigational Site Number :7240001
Seville
41013
Spain
Investigational Site Number :7520001
Stockholm
14186
Sweden
Investigational Site Number :1580001
Taoyuan County
33305
Taiwan
Investigational Site Number :8260001
London
London, City of
NW1 2PG
United Kingdom
Investigational Site Number :8260002
Oxford
Oxfordshire
OX3 9DZ
United Kingdom
Derived
Schidlitzki A, Stanojlovic M, Fournier C, Kaufer C, Feja M, Gericke B, Garzotti M, Welford RWD, Steiner MA, Angot E, Richter F. Double-Edged Effects of Venglustat on Behavior and Pathology in Mice Overexpressing alpha-Synuclein. Mov Disord. 2023 Jun;38(6):1044-1055. doi: 10.1002/mds.29398. Epub 2023 Apr 12.
Peterschmitt MJ, Saiki H, Hatano T, Gasser T, Isaacson SH, Gaemers SJM, Minini P, Saubadu S, Sharma J, Walbillic S, Alcalay RN, Cutter G, Hattori N, Hoglinger GU, Marek K, Schapira AHV, Scherzer CR, Simuni T, Giladi N, Sardi SP, Fischer TZ; MOVES-PD Investigators. Safety, Pharmacokinetics, and Pharmacodynamics of Oral Venglustat in Patients with Parkinson's Disease and a GBA Mutation: Results from Part 1 of the Randomized, Double-Blinded, Placebo-Controlled MOVES-PD Trial. J Parkinsons Dis. 2022;12(2):557-570. doi: 10.3233/JPD-212714.
Participants from ROW (except Japan), received venglustat 4 milligrams (mg) capsule orally QD for up to 36 weeks in Part 1.
FG002
Part 1: Venglustat 8 mg (ROW)
Participants from ROW (except Japan), received venglustat 8 mg capsule orally QD for up to 36 weeks in Part 1.
FG003
Part 1: Venglustat 15 mg (ROW)
Participants from ROW (except Japan), received venglustat 15 mg capsule orally QD for up to 36 weeks in Part 1.
FG004
Part 1: Placebo (Japan Only)
Japanese participants received placebo (matched to venglustat) capsule orally QD for up to 52 weeks in Part 1.
FG005
Part 1: Venglustat 4 mg (Japan Only)
Japanese participants received venglustat 4 mg capsule orally QD for up to 52 weeks in Part 1.
FG006
Part 1: Venglustat 8 mg (Japan Only)
Japanese participants received venglustat 8 mg capsule orally QD for up to 52 weeks in Part 1.
FG007
Part 1: Venglustat 15 mg (Japan Only)
Japanese participants received venglustat 15 mg capsule orally QD for up to 52 weeks in Part 1.
FG008
Part 2, DB Period: Placebo
Participants received placebo (matched to venglustat) capsule orally QD for 52 weeks in Part 2 DB period.
FG009
Part 2, DB Period: Venglustat 15 mg
Participants received venglustat 15 mg capsule orally QD for 52 weeks in Part 2 DB period.
FG010
Part 2, DB Period: Placebo (Re-randomized From Part 1)
Participants who completed Part 1 with placebo (matched to venglustat) or venglustat 4/8/15 mg, met eligibility criteria for Part 2 and agreed to continue in the study, were re-randomized to receive placebo (matched to venglustat) capsule orally QD for 52 weeks in Part 2 DB period.
FG011
Part 2, DB Period: Venglustat 15 mg (Re-randomized From Part 1)
Participants who completed Part 1 with placebo (matched to venglustat) or venglustat 4/8/15 mg, met eligibility criteria for Part 2 and agreed to continue in the study, were re-randomized to receive venglustat 15 mg capsule orally QD for 52 weeks in Part 2 DB period.
FG012
Part 2, LTFU Period: Placebo Then Venglustat 15 mg
Participants who were enrolled in the study in Part 2, received placebo (matched to venglustat), completed Part 2 DB period, entered long-term follow-up (LTFU) period to receive venglustat 15 mg capsule orally QD for 156 weeks.
FG013
Part 2, LTFU Period: Venglustat 15 mg
Participants who were enrolled in the study in Part 2, received venglustat 15 mg, completed Part 2 DB period, entered LTFU period to receive venglustat 15 mg capsule orally QD for 156 weeks.
FG014
Part 2, LTFU: Placebo Then Veng 15 mg (Re-randomized From Part 1)
Participants from Part 1 who were re-randomized in the study in Part 2, received placebo (matched to venglustat) and completed Part 2 DB period, entered LTFU period to receive venglustat (Veng) 15 mg capsule orally QD for 156 weeks.
FG015
Part 2, LTFU: Venglustat 15 mg (Re-randomized From Part 1)
Participants from Part 1 who were re-randomized in the study in Part 2, received venglustat 15 mg and completed Part 2 DB period, entered LTFU period to receive venglustat 15 mg capsule orally QD for 156 weeks.
FG0004 subjects
FG0014 subjects
FG0025 subjects
FG0034 subjects
FG0043 subjects
FG0053 subjects
FG0063 subjects
FG0073 subjects
FG0080 subjects
FG0090 subjects
FG0100 subjects
FG0110 subjects
FG0120 subjects
FG0130 subjects
FG0140 subjects
FG0150 subjects
Treated
FG0004 subjects
FG0014 subjects
FG0025 subjects
FG0034 subjects
FG0043 subjects
FG0053 subjects
FG0063 subjects
FG0073 subjects
FG0080 subjects
FG0090 subjects
FG0100 subjects
FG0110 subjects
FG0120 subjects
FG0130 subjects
FG0140 subjects
FG0150 subjects
COMPLETED
FG0004 subjects
FG0013 subjects
FG0025 subjects
FG0033 subjects
FG0043 subjects
FG0053 subjects
FG0063 subjects
FG0073 subjects
FG0080 subjects
FG0090 subjects
FG0100 subjects
FG0110 subjects
FG0120 subjects
FG0130 subjects
FG0140 subjects
FG0150 subjects
NOT COMPLETED
FG0000 subjects
FG0011 subjects
FG0020 subjects
FG0031 subjects
FG0040 subjects
FG0050 subjects
FG0060 subjects
FG0070 subjects
FG0080 subjects
FG0090 subjects
FG0100 subjects
FG0110 subjects
FG0120 subjects
FG0130 subjects
FG0140 subjects
FG0150 subjects
Type
Comment
Reasons
Adverse Event
FG0000 subjects
FG0011 subjects
FG0020 subjects
FG0031 subjects
FG0040 subjects
FG0050 subjects
FG0060 subjects
FG0070 subjects
FG0080 subjects
FG0090 subjects
FG0100 subjects
FG0110 subjects
FG0120 subjects
FG0130 subjects
FG0140 subjects
FG0150 subjects
Part 2: Double-blind Treatment Period
Type
Comment
Milestone Data
STARTED
FG0000 subjects
FG0010 subjects
FG0020 subjects
FG0030 subjects
FG0040 subjects
FG0050 subjects
FG0060 subjects
FG0070 subjects
FG008111 subjects
FG009110 subjects
FG01010 subjects
FG01113 subjects
FG0120 subjects
FG0130 subjects
FG0140 subjects
FG0150 subjects
Treated
FG0000 subjects
FG0010 subjects
FG0020 subjects
FG0030 subjects
FG004
COMPLETED
FG0000 subjects
FG0010 subjects
FG0020 subjects
FG0030 subjects
FG004
NOT COMPLETED
FG0000 subjects
FG0010 subjects
FG0020 subjects
FG0030 subjects
FG004
Type
Comment
Reasons
Adverse Event
FG0000 subjects
FG0010 subjects
FG0020 subjects
FG003
Part 2: Long-term Follow-up Period
Type
Comment
Milestone Data
STARTED
FG0000 subjects
FG0010 subjects
FG0020 subjects
FG0030 subjects
FG0040 subjects
FG0050 subjects
FG0060 subjects
FG0070 subjects
FG0080 subjects
FG0090 subjects
FG0100 subjects
FG0110 subjects
FG01299 subjects
FG01385 subjects
FG01410 subjects
FG01511 subjectsOut of 13 completed participants in the previous part 2 DB period-arm, 2 didn't enter LTFU period.
Treated
FG0000 subjects
FG0010 subjects
FG0020 subjects
FG0030 subjects
FG004
COMPLETED
FG0000 subjects
FG0010 subjects
FG0020 subjects
FG0030 subjects
FG004
NOT COMPLETED
FG0000 subjects
FG0010 subjects
FG0020 subjects
FG0030 subjects
FG004
Type
Comment
Reasons
Adverse Event
FG0000 subjects
FG0010 subjects
FG0020 subjects
FG003
Analysis was performed on all randomized unique participants (N=250) only.
Type of Units Analyzed
Not provided
Arm/Group Information
ID
Title
Description
BG000
Part 1: Placebo (ROW)
Participants from rest of world (ROW, except Japan), received placebo (matched to venglustat) capsule orally once daily (QD) for up to 36 weeks in Part 1.
BG001
Part 1: Venglustat 4 mg (ROW)
Participants from ROW (except Japan), received venglustat 4 milligrams (mg) capsule orally QD for up to 36 weeks in Part 1.
BG002
Part 1: Venglustat 8 mg (ROW)
Participants from ROW (except Japan), received venglustat 8 mg capsule orally QD for up to 36 weeks in Part 1.
BG003
Part 1: Venglustat 15 mg (ROW)
Participants from ROW (except Japan), received venglustat 15 mg capsule orally QD for up to 36 weeks in Part 1.
BG004
Part 1: Placebo (Japan Only)
Japanese participants received placebo (matched to venglustat) capsule orally QD for up to 52 weeks in Part 1.
BG005
Part 1: Venglustat 4 mg (Japan Only)
Japanese participants received venglustat 4 mg capsule orally QD for up to 52 weeks in Part 1.
BG006
Part 1: Venglustat 8 mg (Japan Only)
Japanese participants received venglustat 8 mg capsule orally QD for up to 52 weeks in Part 1.
BG007
Part 1: Venglustat 15 mg (Japan Only)
Japanese participants received venglustat 15 mg capsule orally QD for up to 52 weeks in Part 1.
BG008
Part 2, DB Period: Placebo
Participants received placebo (matched to venglustat) capsule orally QD for 52 weeks in Part 2 DB period.
BG009
Part 2, DB Period: Venglustat 15 mg
Participants received venglustat 15 mg capsule orally QD for 52 weeks in Part 2 DB period.
BG010
Total Title
Denominators
Units
Counts
Participants
BG0004
BG0014
BG0025
BG0034
BG0043
BG0053
BG0063
BG0073
BG008111
BG009110
BG010250
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
Title
Measurements
BG00053.0± 8.8
BG00155.0± 3.9
BG00261.0± 6.9
BG003
Sex: Female, Male
Count of Participants
Participants
Title
Denominators
Categories
Title
Measurements
Female
BG0001
BG0011
BG002
Race (NIH/OMB)
Count of Participants
Participants
Title
Denominators
Categories
Title
Measurements
American Indian or Alaska Native
BG0000
BG0010
BG002
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
Part 1: Number of Participants With Treatment-Emergent Adverse Events (TEAEs), and Treatment-Emergent Serious Adverse Events (TESAEs)
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug and does not necessarily had to have a causal relationship with the treatment. Serious AEs (SAEs) were any untoward medical occurrence that resulted in any of the following outcomes: death, life-threatening, required initial or prolonged in-patient hospitalization, persistent or significant disability/incapacity, congenital anomaly/birth defect, or considered as medically important event. TEAEs were the AEs that developed or worsened or became serious during the TEAE period (defined as the period from the time of first investigational medicinal product [IMP] administration up of 6 weeks after the last administration of the IMP).
Analysis was performed on safety population which included both non-Japanese (ROW) and Japanese participants who received at least 1 dose of study medication in Part 1 of the study. This outcome measure (OM) was planned to be analyzed and reported for Part 1 only and not for Part 2, as pre-specified in protocol.
Posted
Count of Participants
Participants
From the first IMP administration up to 6 weeks after the last administration of the IMP (i.e., up to 42 weeks for ROW and up to 58 weeks for Japanese participants)
ID
Title
Description
OG000
Part 1: Placebo (ROW)
Participants from rest of world (ROW, except Japan), received placebo (matched to venglustat) capsule orally once daily (QD) for up to 36 weeks in Part 1.
OG001
Part 1: Venglustat 4 mg (ROW)
Participants from ROW (except Japan), received venglustat 4 milligrams (mg) capsule orally QD for up to 36 weeks in Part 1.
OG002
Part 1: Venglustat 8 mg (ROW)
Participants from ROW (except Japan), received venglustat 8 mg capsule orally QD for up to 36 weeks in Part 1.
OG003
Part 1: Venglustat 15 mg (ROW)
Participants from ROW (except Japan), received venglustat 15 mg capsule orally QD for up to 36 weeks in Part 1.
OG004
Part 1: Placebo (Japan Only)
Japanese participants received placebo (matched to venglustat) capsule orally QD for up to 52 weeks in Part 1.
OG005
Part 1: Venglustat 4 mg (Japan Only)
Japanese participants received venglustat 4 mg capsule orally QD for up to 52 weeks in Part 1.
OG006
Part 1: Venglustat 8 mg (Japan Only)
Japanese participants received venglustat 8 mg capsule orally QD for up to 52 weeks in Part 1.
Units
Counts
Participants
OG0004
OG0014
OG0025
OG003
Title
Denominators
Categories
Any TEAE
Title
Measurements
OG0004
OG0014
OG0024
OG003
Primary
Part 1: Number of Participants With Abnormal Physical Examination Findings
Physical examination included following observations/measurements: general appearance; heart, skin, respiratory auscultation; head, eyes, ears, nose, and throat, extremities/joints, and abdomen. New onset of abnormal physical examination was defined as a normal physical examination at Baseline and an abnormal physical examination during the treatment-emergent (TE) period (defined as the period from the time of first IMP administration up of 6 weeks after the last administration of the IMP). Abnormalities in physical examination were based on investigator's evaluation.
Analysis was performed on safety population. This OM was planned to be analyzed and reported for Part 1 only and not for Part 2, as pre-specified in protocol.
Posted
Count of Participants
Participants
From the first IMP administration up to 6 weeks after the last administration of the IMP (i.e., up to 42 weeks for ROW and up to 58 weeks for Japanese participants)
ID
Title
Description
OG000
Part 1: Placebo (ROW)
Participants from rest of world (ROW, except Japan), received placebo (matched to venglustat) capsule orally once daily (QD) for up to 36 weeks in Part 1.
OG001
Part 1: Venglustat 4 mg (ROW)
Participants from ROW (except Japan), received venglustat 4 milligrams (mg) capsule orally QD for up to 36 weeks in Part 1.
Primary
Part 1: Number of Participants With Abnormal Neurological Examination Findings
Neurological examination included at least assessments of the participant's cranial nerves, motor system (including muscle atrophy, tone, and power), mental status, deep tendon reflex, sensation, and cerebellar function. Abnormalities in neurological examination were based on investigator's evaluation.
Analysis was performed on safety population. This OM was planned to be analyzed and reported for Part 1 only and not for Part 2, as pre-specified in protocol.
Posted
Count of Participants
Participants
From the first IMP administration up to 6 weeks after the last administration of the IMP (i.e., up to 42 weeks for ROW and up to 58 weeks for Japanese participants)
ID
Title
Description
OG000
Part 1: Placebo (ROW)
Participants from rest of world (ROW, except Japan), received placebo (matched to venglustat) capsule orally once daily (QD) for up to 36 weeks in Part 1.
OG001
Part 1: Venglustat 4 mg (ROW)
Participants from ROW (except Japan), received venglustat 4 milligrams (mg) capsule orally QD for up to 36 weeks in Part 1.
OG002
Part 1: Venglustat 8 mg (ROW)
Participants from ROW (except Japan), received venglustat 8 mg capsule orally QD for up to 36 weeks in Part 1.
Primary
Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Hematology
Criteria for potentially clinically significant abnormalities (PCSA): Hemoglobin: less than or equal to (<=) 115 grams per liter (g/L) (Male[M]) or <=95 g/L (Female[F]), greater than or equal to (>=) 185 g/L (M) or >=165 g/L (F), Decrease from baseline (DFB) >=20 g/L; Hematocrit: <=0.37 volume/volume (v/v) (M) or <=0.32 v/v (F), >=0.55 v/v (M) or >=0.5 v/v (F); Red blood cells (RBC): >=6 Tera/L; Platelets: less than (<) 100 Giga/L, >=700 Giga/L; White blood cells (WBC): <3.0 Giga/L (Non-Black [NB]) or <2.0 Giga/L (Black [B]), >=16.0 Giga/L; Neutrophils: <1.5 Giga/L (NB) or <1.0 Giga/L (B); Lymphocytes: <lower limit of normal (LLN), greater than (>) 4.0 Giga/L; Monocytes: <LLN, >0.7 Giga/L; Basophils: >0.1 Giga/L; Eosinophils: >0.5 Giga/L or >upper limit of normal (ULN) (if ULN >=0.5 Giga/L).
Analysis was performed on safety population. This OM was planned to be analyzed and reported for Part 1 only and not for Part 2, as pre-specified in protocol.
Posted
Count of Participants
Participants
From the first IMP administration up to 6 weeks after the last administration of the IMP (i.e., up to 42 weeks for ROW and up to 58 weeks for Japanese participants)
ID
Title
Description
OG000
Part 1: Placebo (ROW)
Participants from rest of world (ROW, except Japan), received placebo (matched to venglustat) capsule orally once daily (QD) for up to 36 weeks in Part 1.
OG001
Part 1: Venglustat 4 mg (ROW)
Primary
Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Liver Function Parameters
Criteria for PCSA: Alanine Aminotransferase (ALT): >3 ULN, >5 ULN; Aspartate aminotransferase (AST): >3 ULN; Alkaline phosphatase: >1.5 ULN; Total Bilirubin: >1.5 ULN; ALT and Bilirubin: >3 ULN and >2 ULN; Direct Bilirubin and Bilirubin: >35% and >1.5 ULN.
Analysis was performed on safety population. This OM was planned to be analyzed and reported for Part 1 only and not for Part 2, as pre-specified in protocol.
Posted
Count of Participants
Participants
From the first IMP administration up to 6 weeks after the last administration of the IMP (i.e., up to 42 weeks for ROW and up to 58 weeks for Japanese participants)
ID
Title
Description
OG000
Part 1: Placebo (ROW)
Participants from rest of world (ROW, except Japan), received placebo (matched to venglustat) capsule orally once daily (QD) for up to 36 weeks in Part 1.
OG001
Part 1: Venglustat 4 mg (ROW)
Participants from ROW (except Japan), received venglustat 4 milligrams (mg) capsule orally QD for up to 36 weeks in Part 1.
OG002
Part 1: Venglustat 8 mg (ROW)
Participants from ROW (except Japan), received venglustat 8 mg capsule orally QD for up to 36 weeks in Part 1.
Primary
Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Renal Parameters
Criteria for PCSA: Creatinine: >=150 micromoles per liter (mcmol/L) (adults), >=30% change from baseline, >=100% change from baseline; Blood urea nitrogen: >=17 millimoles (mmol)/L.
Analysis was performed on safety population. This OM was planned to be analyzed and reported for Part 1 only and not for Part 2, as pre-specified in protocol.
Posted
Count of Participants
Participants
From the first IMP administration up to 6 weeks after the last administration of the IMP (i.e., up to 42 weeks for ROW and up to 58 weeks for Japanese participants)
ID
Title
Description
OG000
Part 1: Placebo (ROW)
Participants from rest of world (ROW, except Japan), received placebo (matched to venglustat) capsule orally once daily (QD) for up to 36 weeks in Part 1.
OG001
Part 1: Venglustat 4 mg (ROW)
Participants from ROW (except Japan), received venglustat 4 milligrams (mg) capsule orally QD for up to 36 weeks in Part 1.
OG002
Part 1: Venglustat 8 mg (ROW)
Participants from ROW (except Japan), received venglustat 8 mg capsule orally QD for up to 36 weeks in Part 1.
Primary
Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Metabolic Parameters
Criteria for PCSA: Glucose: <=3.9 mmol/L and <LLN; >=11.1 mmol/L (unfasted [unfas]) or >=7 mmol/L (fasted [fas]); Albumin: <=25 g/L.
Analysis was performed on safety population. This OM was planned to be analyzed and reported for Part 1 only and not for Part 2, as pre-specified in protocol.
Posted
Count of Participants
Participants
From the first IMP administration up to 6 weeks after the last administration of the IMP (i.e., up to 42 weeks for ROW and up to 58 weeks for Japanese participants)
ID
Title
Description
OG000
Part 1: Placebo (ROW)
Participants from rest of world (ROW, except Japan), received placebo (matched to venglustat) capsule orally once daily (QD) for up to 36 weeks in Part 1.
OG001
Part 1: Venglustat 4 mg (ROW)
Participants from ROW (except Japan), received venglustat 4 milligrams (mg) capsule orally QD for up to 36 weeks in Part 1.
OG002
Part 1: Venglustat 8 mg (ROW)
Participants from ROW (except Japan), received venglustat 8 mg capsule orally QD for up to 36 weeks in Part 1.
Primary
Part 1: Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Electrolytes Parameters
Criteria for PCSA: Sodium: <=129 mmol/L, >=160 mmol/L; Potassium: <3 mmol/L, >=5.5 mmol/L and Chloride: <80 mmol/L, >115 mmol/L.
Analysis was performed on safety population. This OM was planned to be analyzed and reported for Part 1 only and not for Part 2, as pre-specified in protocol.
Posted
Count of Participants
Participants
From the first IMP administration up to 6 weeks after the last administration of the IMP (i.e., up to 42 weeks for ROW and up to 58 weeks for Japanese participants)
ID
Title
Description
OG000
Part 1: Placebo (ROW)
Participants from rest of world (ROW, except Japan), received placebo (matched to venglustat) capsule orally once daily (QD) for up to 36 weeks in Part 1.
OG001
Part 1: Venglustat 4 mg (ROW)
Participants from ROW (except Japan), received venglustat 4 milligrams (mg) capsule orally QD for up to 36 weeks in Part 1.
OG002
Part 1: Venglustat 8 mg (ROW)
Participants from ROW (except Japan), received venglustat 8 mg capsule orally QD for up to 36 weeks in Part 1.
Primary
Part 1: Number of Participants With Potentially Clinically Significant Vital Signs Abnormalities
Criteria for PCSA: Systolic blood pressure (SBP) supine: <=95 millimeters of mercury (mmHg) and DFB >=20 mmHg; >=160 mmHg and increase from baseline (IFB) >=20 mmHg; Diastolic blood pressure (DBP) supine: <=45 mmHg and DFB >=10 mmHg; >=110 mmHg and IFB >=10 mmHg; SBP (Orthostatic): <=-20 mmHg; DBP (Orthostatic): <=-10 mmHg; Heart rate (HR) supine: <=50 beats per minute (bpm) and DFB >=20 bpm; >=120 bpm and IFB >=20 bpm; Weight: >=5% DFB; >=5% IFB.
Analysis was performed on safety population. This OM was planned to be analyzed and reported for Part 1 only and not for Part 2, as pre-specified in protocol.
Posted
Count of Participants
Participants
From the first IMP administration up to 6 weeks after the last administration of the IMP (i.e., up to 42 weeks for ROW and up to 58 weeks for Japanese participants)
ID
Title
Description
OG000
Part 1: Placebo (ROW)
Participants from rest of world (ROW, except Japan), received placebo (matched to venglustat) capsule orally once daily (QD) for up to 36 weeks in Part 1.
OG001
Part 1: Venglustat 4 mg (ROW)
Participants from ROW (except Japan), received venglustat 4 milligrams (mg) capsule orally QD for up to 36 weeks in Part 1.
OG002
Primary
Part 1: Number of Participants With Potentially Clinically Significant Ophthalmological Abnormalities
Clinically significant observations in left eye, right eye and any eye (any eye among left and right) were assessed by the investigator based on methods like visual acuity, slit lamp examination, examination of the cornea, lens, and retina. Any abnormal clinically significant ophthalmological examination finding (which was present at screening or not) on any eye during the treatment-emergent period corresponded to cornea verticillata, cataract and abnormal overall evaluation
Analysis was performed on safety population. This OM was planned to be analyzed and reported for Part 1 only and not for Part 2, as pre-specified in protocol.
Posted
Count of Participants
Participants
From the first IMP administration up to 6 weeks after the last administration of the IMP (i.e., up to 42 weeks for ROW and up to 58 weeks for Japanese participants)
ID
Title
Description
OG000
Part 1: Placebo (ROW)
Participants from rest of world (ROW, except Japan), received placebo (matched to venglustat) capsule orally once daily (QD) for up to 36 weeks in Part 1.
OG001
Part 1: Venglustat 4 mg (ROW)
Participants from ROW (except Japan), received venglustat 4 milligrams (mg) capsule orally QD for up to 36 weeks in Part 1.
OG002
Primary
Part 1: Number of Participants With Potentially Clinically Significant Electrocardiogram Abnormalities
Analysis was performed on safety population. This OM was planned to be analyzed and reported for Part 1 only and not for Part 2, as pre-specified in protocol.
Posted
Count of Participants
Participants
From the first IMP administration up to 6 weeks after the last administration of the IMP (i.e., up to 42 weeks for ROW and up to 58 weeks for Japanese participants)
ID
Title
Description
OG000
Part 1: Placebo (ROW)
Participants from rest of world (ROW, except Japan), received placebo (matched to venglustat) capsule orally once daily (QD) for up to 36 weeks in Part 1.
OG001
Part 1: Venglustat 4 mg (ROW)
Participants from ROW (except Japan), received venglustat 4 milligrams (mg) capsule orally QD for up to 36 weeks in Part 1.
OG002
Primary
Part 2: Change From Baseline to Week 52 in Movement Disorder Society-Unified Parkinson's Disease Rating Scale (MDS-UPDRS) Part II+III Total Score
MDS-UPDRS is a multimodal scale consisting of 4 parts. Part II assessed motor experiences of daily living (total score range: 0 to 52). It contained 13 questions completed by the participant. Part III assessed the motor signs of PD and was administered by the rater (total score range: 0 to 132). Part III contained 33 scores based on 18 items. In both parts, higher score indicated more severe symptoms. For each question in both parts, numeric score was assigned between 0 to 4, where 0=Normal, 1=Slight, 2=Mild, 3=Moderate, 4=Severe. MDS-UPDRS Total Part II and III score = sum of Part II and III scores with score ranged from 0 (no symptom) to 184 (severe symptoms), where higher scores reflected more severe symptoms of PD. Data for this OM was not planned to be collected and analyzed for Part 1, Part 2: DB period re-randomized participants and Part 2 LTFU period, as pre-specified in protocol.
Analyzed on intent-to-treat (ITT) population which included all randomized participants of Part 2 and analyzed in treatment group to which they were randomized. Here, overall number of participants analyzed = participants evaluable for this OM.
Posted
Least Squares Mean
Standard Error
units on a scale
Baseline to Week 52
ID
Title
Description
OG000
Part 2, DB Period: Placebo
Participants received placebo (matched to venglustat) capsule orally QD for 52 weeks in Part 2 DB period.
OG001
Part 2, DB Period: Venglustat 15 mg
Secondary
Part 2: Change From Baseline to Week 52 in Parkinson's Disease Cognitive Rating Scale (PD-CRS) Total Score
The PD-CRS detects early cognitive impairment in Parkinson's disease. It is composed of 2 scales, the fronto-subcortical scale (items: sustained attention, working memory, alternating and action verbal fluency, clock drawing, immediate, and delayed free recall verbal memory) and the posterior-cortical scale (items: confrontation naming and clock copying). The total score of the fronto-subcortical scale (sum of all items) ranged from 0 (worst) to 104 (maximum score indicates better) and the total score of the posterior-cortical scale (sum of all items) ranged from 0 (worst) to 30 (maximum score indicates better). The PD-CRS Total score = the sum of PD-CRS fronto-subcortical score and the PD-CRS posterior-cortical score, which ranged from 0 to 134, where higher score = less impairment.
Analysis was performed on ITT population. Here, overall number of participants analyzed = participants evaluable for this OM. Data for this OM was not planned to be collected and analyzed for Part 1, Part 2: DB period re-randomized participants and Part 2 LTFU period, as pre-specified in protocol.
Posted
Least Squares Mean
Standard Error
units on a scale
Baseline to Week 52
ID
Title
Description
OG000
Part 2, DB Period: Placebo
Participants received placebo (matched to venglustat) capsule orally QD for 52 weeks in Part 2 DB period.
OG001
Part 2, DB Period: Venglustat 15 mg
Secondary
Part 2: Change From Baseline to Week 52 in Movement Disorder Society-Unified Parkinson's Disease Rating Scale (MDS-UPDRS) Part I+ II+III Score
MDS-UPDRS is a multimodal scale consisting of 4 parts. Part I assessed non-motor experiences of daily living and has 2 components (total score range: 0 to 52): Part IA contained 6 questions and was assessed by the examiner (total score range: 0 to 24). Part IB contained 7 questions on non-motor experiences of daily living which was completed by the participant (total score range: 0 to 28). Part II (13 questions completed by the participant) assessed motor experiences of daily living (total score range: 0 to 52). Part III assessed motor signs of PD and was administered by the rater (total score range: 0 to 132). Part III contained 33 scores based on 18 items. In all parts, higher score indicated more symptoms. For each question, numeric score was assigned between 0 to 4, where 0=Normal, 1=Slight, 2=Mild, 3=Moderate, 4=Severe. MDS-UPDRS total score = sum of Parts I, II, and III (Range: 0 to 236). Higher score = more severe symptoms of PD.
Analysis was performed on ITT population. Here, overall number of participants analyzed = participants evaluable for this OM. Data for this OM was not planned to be collected and analyzed for Part 1, Part 2: DB period re-randomized participants and Part 2 LTFU period, as pre-specified in protocol.
Posted
Least Squares Mean
Standard Error
units on a scale
Baseline to Week 52
ID
Title
Description
OG000
Part 2, DB Period: Placebo
Participants received placebo (matched to venglustat) capsule orally QD for 52 weeks in Part 2 DB period.
Secondary
Part 2: Change From Baseline to Week 52 in Hoehn and Yahr (H and Y) Score
H and Y scale measured how Parkinson's symptoms progress and the level of disability. Scale allocated stage scores were from 0 to 5 to indicate relative level of disability as: Stage 0: no symptoms; Stage 1: symptoms on one side of the body only; Stage 2: symptoms on both sides of the body, without impairment of balance; Stage 3: Mild to moderate bilateral disease; some postural instability; physically independent; Stage 4: Severe disability; still able to walk or stand unassisted and Stage 5: Wheelchair bound or bedridden unless aided, where higher stage score described an increased severity of disease.
Analysis was performed on ITT population. Here, overall number of participants analyzed = participants evaluable for this OM. Data for this OM was not planned to be collected and analyzed for Part 1, Part 2: DB period re-randomized participants and Part 2 LTFU period, as pre-specified in protocol.
Posted
Least Squares Mean
Standard Error
units on a scale
Baseline to Week 52
ID
Title
Description
OG000
Part 2, DB Period: Placebo
Participants received placebo (matched to venglustat) capsule orally QD for 52 weeks in Part 2 DB period.
OG001
Part 2, DB Period: Venglustat 15 mg
Participants received venglustat 15 mg capsule orally QD for 52 weeks in Part 2 DB period.
Time Frame
From the first IMP administration up to 6 weeks after the last administration of the IMP (i.e., in Part 1: up to 42 weeks for ROW, up to 58 weeks for Japanese participants; in Part 2 DB period: up to 60 weeks; in Part 2 LTFU period: up to 164 weeks).
Description
Reported AEs and deaths were TEAEs that developed/worsened in grade or became serious during TEAE period (defined as the time from the first dose of the IMP to the last dose of IMP + 6 weeks). Analysis was performed on safety population.
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
Part 1: Placebo (ROW)
Participants from rest of world (ROW, except Japan), received placebo (matched to venglustat) capsule orally once daily (QD) for up to 36 weeks in Part 1.
0
4
0
4
4
4
EG001
Part 1: Venglustat 4 mg (ROW)
Participants from ROW (except Japan), received venglustat 4 milligrams (mg) capsule orally QD for up to 36 weeks in Part 1.
0
4
0
4
4
4
EG002
Part 1: Venglustat 8 mg (ROW)
Participants from ROW (except Japan), received venglustat 8 mg capsule orally QD for up to 36 weeks in Part 1.
0
5
0
5
4
5
EG003
Part 1: Venglustat 15 mg (ROW)
Participants from ROW (except Japan), received venglustat 15 mg capsule orally QD for up to 36 weeks in Part 1.
0
4
0
4
4
4
EG004
Part 1: Placebo (Japan Only)
Japanese participants received placebo (matched to venglustat) capsule orally QD for up to 52 weeks in Part 1.
0
3
0
3
2
3
EG005
Part 1: Venglustat 4 mg (Japan Only)
Japanese participants received venglustat 4 mg capsule orally QD for up to 52 weeks in Part 1.
0
3
0
3
3
3
EG006
Part 1: Venglustat 8 mg (Japan Only)
Japanese participants received venglustat 8 mg capsule orally QD for up to 52 weeks in Part 1.
0
3
0
3
3
3
EG007
Part 1: Venglustat 15 mg (Japan Only)
Japanese participants received venglustat 15 mg capsule orally QD for up to 52 weeks in Part 1.
0
3
0
3
2
3
EG008
Part 2, DB Period: Placebo
Participants received placebo (matched to venglustat) capsule orally QD for 52 weeks in Part 2 DB period.
0
111
12
111
87
111
EG009
Part 2, DB Period: Venglustat 15 mg
Participants received venglustat 15 mg capsule orally QD for 52 weeks in Part 2 DB period.
1
110
12
110
95
110
EG010
Part 2, DB Period: Placebo (Re-randomized From Part 1)
Participants who completed Part 1 with placebo (matched to venglustat) or venglustat 4/8/15 mg, met eligibility criteria for Part 2 and agreed to continue in the study, were re-randomized to receive placebo (matched to venglustat) capsule orally QD for 52 weeks in Part 2 DB period.
0
10
0
10
8
10
EG011
Part 2, DB Period: Venglustat 15 mg (Re-randomized From Part 1)
Participants who completed Part 1 with placebo (matched to venglustat) or venglustat 4/8/15 mg, met eligibility criteria for Part 2 and agreed to continue in the study, were re-randomized to receive venglustat 15 mg capsule orally QD for 52 weeks in Part 2 DB period.
0
13
3
13
11
13
EG012
Part 2, LTFU Period: Placebo Then Venglustat 15 mg
Participants who were enrolled in the study in Part 2, received placebo (matched to venglustat), completed Part 2 DB period, entered long-term follow-up (LTFU) period to receive venglustat 15 mg capsule orally QD for 156 weeks.
1
87
15
87
53
87
EG013
Part 2, LTFU Period: Venglustat 15 mg
Participants who were enrolled in the study in Part 2, received venglustat 15 mg, completed Part 2 DB period, entered LTFU period to receive venglustat 15 mg capsule orally QD for 156 weeks.
0
75
9
75
39
75
EG014
Part 2, LTFU: Placebo Then Veng 15 mg (Re-randomized From Part 1)
Participants from Part 1 who were re-randomized in the study in Part 2, received placebo (matched to venglustat) and completed Part 2 DB period, entered LTFU period to receive venglustat (Veng) 15 mg capsule orally QD for 156 weeks.
0
10
3
10
7
10
EG015
Part 2, LTFU: Venglustat 15 mg (Re-randomized From Part 1)
Participants from Part 1 who were re-randomized in the study in Part 2, received venglustat 15 mg and completed Part 2 DB period, entered LTFU period to receive venglustat 15 mg capsule orally QD for 156 weeks.
0
11
1
11
8
11
Serious Adverse Events
Term
Organ System
Source Vocabulary
Assessment Type
Notes
Statistical Information
Anaemia
Blood and lymphatic system disorders
MedDRA24.0
Systematic Assessment
EG0000 events0 affected4 at risk
EG0010 events0 affected4 at risk
EG0020 events0 affected5 at risk
EG0030 events0 affected4 at risk
EG0040 events0 affected3 at risk
EG0050 events0 affected3 at risk
EG0060 events0 affected3 at risk
EG0070 events0 affected3 at risk
EG0080 events0 affected111 at risk
EG0090 events0 affected110 at risk
EG0100 events0 affected10 at risk
EG0110 events0 affected13 at risk
EG0121 events1 affected87 at risk
EG0130 events0 affected75 at risk
EG0140 events0 affected10 at risk
EG0150 events0 affected11 at risk
Atrial Fibrillation
Cardiac disorders
MedDRA24.0
Systematic Assessment
EG0000 events0 affected4 at risk
EG0010 events0 affected4 at risk
EG0020 events0 affected5 at risk
EG003
Cardiac Arrest
Cardiac disorders
MedDRA24.0
Systematic Assessment
EG0000 events0 affected4 at risk
EG0010 events0 affected4 at risk
EG0020 events0 affected5 at risk
EG003
Cardio-Respiratory Arrest
Cardiac disorders
MedDRA24.0
Systematic Assessment
EG0000 events0 affected4 at risk
EG0010 events0 affected4 at risk
EG0020 events0 affected5 at risk
EG003
Abdominal Pain
Gastrointestinal disorders
MedDRA24.0
Systematic Assessment
EG0000 events0 affected4 at risk
EG0010 events0 affected4 at risk
EG0020 events0 affected5 at risk
EG003
Impaired Gastric Emptying
Gastrointestinal disorders
MedDRA24.0
Systematic Assessment
EG0000 events0 affected4 at risk
EG0010 events0 affected4 at risk
EG0020 events0 affected5 at risk
EG003
Inguinal Hernia
Gastrointestinal disorders
MedDRA24.0
Systematic Assessment
EG0000 events0 affected4 at risk
EG0010 events0 affected4 at risk
EG0020 events0 affected5 at risk
EG003
Intestinal Obstruction
Gastrointestinal disorders
MedDRA24.0
Systematic Assessment
EG0000 events0 affected4 at risk
EG0010 events0 affected4 at risk
EG0020 events0 affected5 at risk
EG003
Nausea
Gastrointestinal disorders
MedDRA24.0
Systematic Assessment
EG0000 events0 affected4 at risk
EG0010 events0 affected4 at risk
EG0020 events0 affected5 at risk
EG003
Volvulus
Gastrointestinal disorders
MedDRA24.0
Systematic Assessment
EG0000 events0 affected4 at risk
EG0010 events0 affected4 at risk
EG0020 events0 affected5 at risk
EG003
Abdominal Abscess
Infections and infestations
MedDRA24.0
Systematic Assessment
EG0000 events0 affected4 at risk
EG0010 events0 affected4 at risk
EG0020 events0 affected5 at risk
EG003
Covid-19
Infections and infestations
MedDRA24.0
Systematic Assessment
EG0000 events0 affected4 at risk
EG0010 events0 affected4 at risk
EG0020 events0 affected5 at risk
EG003
Colonic Abscess
Infections and infestations
MedDRA24.0
Systematic Assessment
EG0000 events0 affected4 at risk
EG0010 events0 affected4 at risk
EG0020 events0 affected5 at risk
EG003
Diverticulitis
Infections and infestations
MedDRA24.0
Systematic Assessment
EG0000 events0 affected4 at risk
EG0010 events0 affected4 at risk
EG0020 events0 affected5 at risk
EG003
Osteomyelitis
Infections and infestations
MedDRA24.0
Systematic Assessment
EG0000 events0 affected4 at risk
EG0010 events0 affected4 at risk
EG0020 events0 affected5 at risk
EG003
Pneumonia
Infections and infestations
MedDRA24.0
Systematic Assessment
EG0000 events0 affected4 at risk
EG0010 events0 affected4 at risk
EG0020 events0 affected5 at risk
EG003
Sepsis
Infections and infestations
MedDRA24.0
Systematic Assessment
EG0000 events0 affected4 at risk
EG0010 events0 affected4 at risk
EG0020 events0 affected5 at risk
EG003
Subacute Endocarditis
Infections and infestations
MedDRA24.0
Systematic Assessment
EG0000 events0 affected4 at risk
EG0010 events0 affected4 at risk
EG0020 events0 affected5 at risk
EG003
Urinary Tract Infection
Infections and infestations
MedDRA24.0
Systematic Assessment
EG0000 events0 affected4 at risk
EG0010 events0 affected4 at risk
EG0020 events0 affected5 at risk
EG003
Anastomotic Leak
Injury, poisoning and procedural complications
MedDRA24.0
Systematic Assessment
EG0000 events0 affected4 at risk
EG0010 events0 affected4 at risk
EG0020 events0 affected5 at risk
EG003
Fall
Injury, poisoning and procedural complications
MedDRA24.0
Systematic Assessment
EG0000 events0 affected4 at risk
EG0010 events0 affected4 at risk
EG0020 events0 affected5 at risk
EG003
Femoral Neck Fracture
Injury, poisoning and procedural complications
MedDRA24.0
Systematic Assessment
EG0000 events0 affected4 at risk
EG0010 events0 affected4 at risk
EG0020 events0 affected5 at risk
EG003
Intentional Overdose
Injury, poisoning and procedural complications
MedDRA24.0
Systematic Assessment
EG0000 events0 affected4 at risk
EG0010 events0 affected4 at risk
EG0020 events0 affected5 at risk
EG003
Post Lumbar Puncture Syndrome
Injury, poisoning and procedural complications
MedDRA24.0
Systematic Assessment
EG0000 events0 affected4 at risk
EG0010 events0 affected4 at risk
EG0020 events0 affected5 at risk
EG003
Postoperative Delirium
Injury, poisoning and procedural complications
MedDRA24.0
Systematic Assessment
EG0000 events0 affected4 at risk
EG0010 events0 affected4 at risk
EG0020 events0 affected5 at risk
EG003
Radius Fracture
Injury, poisoning and procedural complications
MedDRA24.0
Systematic Assessment
EG0000 events0 affected4 at risk
EG0010 events0 affected4 at risk
EG0020 events0 affected5 at risk
EG003
Rib Fracture
Injury, poisoning and procedural complications
MedDRA24.0
Systematic Assessment
EG0000 events0 affected4 at risk
EG0010 events0 affected4 at risk
EG0020 events0 affected5 at risk
EG003
Skin Laceration
Injury, poisoning and procedural complications
MedDRA24.0
Systematic Assessment
EG0000 events0 affected4 at risk
EG0010 events0 affected4 at risk
EG0020 events0 affected5 at risk
EG003
Subdural Haematoma
Injury, poisoning and procedural complications
MedDRA24.0
Systematic Assessment
EG0000 events0 affected4 at risk
EG0010 events0 affected4 at risk
EG0020 events0 affected5 at risk
EG003
Back Pain
Musculoskeletal and connective tissue disorders
MedDRA24.0
Systematic Assessment
EG0000 events0 affected4 at risk
EG0010 events0 affected4 at risk
EG0020 events0 affected5 at risk
EG003
Muscle Rigidity
Musculoskeletal and connective tissue disorders
MedDRA24.0
Systematic Assessment
EG0000 events0 affected4 at risk
EG0010 events0 affected4 at risk
EG0020 events0 affected5 at risk
EG003
Spinal Pain
Musculoskeletal and connective tissue disorders
MedDRA24.0
Systematic Assessment
EG0000 events0 affected4 at risk
EG0010 events0 affected4 at risk
EG0020 events0 affected5 at risk
EG003
Basal Cell Carcinoma
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
MedDRA24.0
Systematic Assessment
EG0000 events0 affected4 at risk
EG0010 events0 affected4 at risk
EG0020 events0 affected5 at risk
EG003
Breast Cancer Female
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
MedDRA24.0
Systematic Assessment
EG0000 events0 affected4 at risk
EG0010 events0 affected4 at risk
EG0020 events0 affected5 at risk
EG003
Malignant Melanoma
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
MedDRA24.0
Systematic Assessment
EG0000 events0 affected4 at risk
EG0010 events0 affected4 at risk
EG0020 events0 affected5 at risk
EG003
Malignant Melanoma In Situ
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
MedDRA24.0
Systematic Assessment
EG0000 events0 affected4 at risk
EG0010 events0 affected4 at risk
EG0020 events0 affected5 at risk
EG003
Non-Small Cell Lung Cancer
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
MedDRA24.0
Systematic Assessment
EG0000 events0 affected4 at risk
EG0010 events0 affected4 at risk
EG0020 events0 affected5 at risk
EG003
Ovarian Epithelial Cancer
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
MedDRA24.0
Systematic Assessment
EG0000 events0 affected4 at risk
EG0010 events0 affected4 at risk
EG0020 events0 affected5 at risk
EG003
Prostate Cancer
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
MedDRA24.0
Systematic Assessment
EG0000 events0 affected4 at risk
EG0010 events0 affected4 at risk
EG0020 events0 affected5 at risk
EG003
Cerebellar Haemorrhage
Nervous system disorders
MedDRA24.0
Systematic Assessment
EG0000 events0 affected4 at risk
EG0010 events0 affected4 at risk
EG0020 events0 affected5 at risk
EG003
Cognitive Disorder
Nervous system disorders
MedDRA24.0
Systematic Assessment
EG0000 events0 affected4 at risk
EG0010 events0 affected4 at risk
EG0020 events0 affected5 at risk
EG003
Freezing Phenomenon
Nervous system disorders
MedDRA24.0
Systematic Assessment
EG0000 events0 affected4 at risk
EG0010 events0 affected4 at risk
EG0020 events0 affected5 at risk
EG003
Lumbar Radiculopathy
Nervous system disorders
MedDRA24.0
Systematic Assessment
EG0000 events0 affected4 at risk
EG0010 events0 affected4 at risk
EG0020 events0 affected5 at risk
EG003
On And Off Phenomenon
Nervous system disorders
MedDRA24.0
Systematic Assessment
EG0000 events0 affected4 at risk
EG0010 events0 affected4 at risk
EG0020 events0 affected5 at risk
EG003
Parkinson's Disease
Nervous system disorders
MedDRA24.0
Systematic Assessment
EG0000 events0 affected4 at risk
EG0010 events0 affected4 at risk
EG0020 events0 affected5 at risk
EG003
Posterior Reversible Encephalopathy Syndrome
Nervous system disorders
MedDRA24.0
Systematic Assessment
EG0000 events0 affected4 at risk
EG0010 events0 affected4 at risk
EG0020 events0 affected5 at risk
EG003
Syncope
Nervous system disorders
MedDRA24.0
Systematic Assessment
EG0000 events0 affected4 at risk
EG0010 events0 affected4 at risk
EG0020 events0 affected5 at risk
EG003
Transient Ischaemic Attack
Nervous system disorders
MedDRA24.0
Systematic Assessment
EG0000 events0 affected4 at risk
EG0010 events0 affected4 at risk
EG0020 events0 affected5 at risk
EG003
Confusional State
Psychiatric disorders
MedDRA24.0
Systematic Assessment
EG0000 events0 affected4 at risk
EG0010 events0 affected4 at risk
EG0020 events0 affected5 at risk
EG003
Hallucination
Psychiatric disorders
MedDRA24.0
Systematic Assessment
EG0000 events0 affected4 at risk
EG0010 events0 affected4 at risk
EG0020 events0 affected5 at risk
EG003
Psychotic Disorder
Psychiatric disorders
MedDRA24.0
Systematic Assessment
EG0000 events0 affected4 at risk
EG0010 events0 affected4 at risk
EG0020 events0 affected5 at risk
EG003
Suicide Attempt
Psychiatric disorders
MedDRA24.0
Systematic Assessment
EG0000 events0 affected4 at risk
EG0010 events0 affected4 at risk
EG0020 events0 affected5 at risk
EG003
Acute Kidney Injury
Renal and urinary disorders
MedDRA24.0
Systematic Assessment
EG0000 events0 affected4 at risk
EG0010 events0 affected4 at risk
EG0020 events0 affected5 at risk
EG003
Micturition Urgency
Renal and urinary disorders
MedDRA24.0
Systematic Assessment
EG0000 events0 affected4 at risk
EG0010 events0 affected4 at risk
EG0020 events0 affected5 at risk
EG003
Ureterolithiasis
Renal and urinary disorders
MedDRA24.0
Systematic Assessment
EG0000 events0 affected4 at risk
EG0010 events0 affected4 at risk
EG0020 events0 affected5 at risk
EG003
Hypoxia
Respiratory, thoracic and mediastinal disorders
MedDRA24.0
Systematic Assessment
EG0000 events0 affected4 at risk
EG0010 events0 affected4 at risk
EG0020 events0 affected5 at risk
EG003
Pulmonary Embolism
Respiratory, thoracic and mediastinal disorders
MedDRA24.0
Systematic Assessment
EG0000 events0 affected4 at risk
EG0010 events0 affected4 at risk
EG0020 events0 affected5 at risk
EG003
Deep Vein Thrombosis
Vascular disorders
MedDRA24.0
Systematic Assessment
EG0000 events0 affected4 at risk
EG0010 events0 affected4 at risk
EG0020 events0 affected5 at risk
EG003
Hypertension
Vascular disorders
MedDRA24.0
Systematic Assessment
EG0000 events0 affected4 at risk
EG0010 events0 affected4 at risk
EG0020 events0 affected5 at risk
EG003
Hypertensive Urgency
Vascular disorders
MedDRA24.0
Systematic Assessment
EG0000 events0 affected4 at risk
EG0010 events0 affected4 at risk
EG0020 events0 affected5 at risk
EG003
Hypotension
Vascular disorders
MedDRA24.0
Systematic Assessment
EG0000 events0 affected4 at risk
EG0010 events0 affected4 at risk
EG0020 events0 affected5 at risk
EG003
Orthostatic Hypotension
Vascular disorders
MedDRA24.0
Systematic Assessment
EG0000 events0 affected4 at risk
EG0010 events0 affected4 at risk
EG0020 events0 affected5 at risk
EG003
Other Adverse Events
Term
Organ System
Source Vocabulary
Assessment Type
Notes
Statistical Information
Tinnitus
Ear and labyrinth disorders
MedDRA24.0
Systematic Assessment
EG0000 events0 affected4 at risk
EG0010 events0 affected4 at risk
EG0020 events0 affected5 at risk
EG0030 events0 affected4 at risk
EG0040 events0 affected3 at risk
EG0050 events0 affected3 at risk
EG0060 events0 affected3 at risk
EG0070 events0 affected3 at risk
EG0081 events1 affected111 at risk
EG0090 events0 affected110 at risk
EG0100 events0 affected10 at risk
EG0110 events0 affected13 at risk
EG0120 events0 affected87 at risk
EG0130 events0 affected75 at risk
EG0141 events1 affected10 at risk
EG0150 events0 affected11 at risk
Tympanic Membrane Perforation
Ear and labyrinth disorders
MedDRA24.0
Systematic Assessment
EG0000 events0 affected4 at risk
EG0010 events0 affected4 at risk
EG0020 events0 affected5 at risk
EG003
Vertigo
Ear and labyrinth disorders
MedDRA24.0
Systematic Assessment
EG0000 events0 affected4 at risk
EG0010 events0 affected4 at risk
EG0020 events0 affected5 at risk
EG003
Hypothyroidism
Endocrine disorders
MedDRA24.0
Systematic Assessment
EG0000 events0 affected4 at risk
EG0010 events0 affected4 at risk
EG0020 events0 affected5 at risk
EG003
Blepharitis
Eye disorders
MedDRA24.0
Systematic Assessment
EG0000 events0 affected4 at risk
EG0010 events0 affected4 at risk
EG0020 events0 affected5 at risk
EG003
Borderline Glaucoma
Eye disorders
MedDRA24.0
Systematic Assessment
EG0000 events0 affected4 at risk
EG0010 events0 affected4 at risk
EG0020 events0 affected5 at risk
EG003
Cataract Cortical
Eye disorders
MedDRA24.0
Systematic Assessment
EG0001 events1 affected4 at risk
EG0011 events1 affected4 at risk
EG0020 events0 affected5 at risk
EG003
Cataract Nuclear
Eye disorders
MedDRA24.0
Systematic Assessment
EG0000 events0 affected4 at risk
EG0011 events1 affected4 at risk
EG0020 events0 affected5 at risk
EG003
Corneal Infiltrates
Eye disorders
MedDRA24.0
Systematic Assessment
EG0000 events0 affected4 at risk
EG0010 events0 affected4 at risk
EG0020 events0 affected5 at risk
EG003
Dry Eye
Eye disorders
MedDRA24.0
Systematic Assessment
EG0000 events0 affected4 at risk
EG0011 events1 affected4 at risk
EG0020 events0 affected5 at risk
EG003
Eczema Eyelids
Eye disorders
MedDRA24.0
Systematic Assessment
EG0000 events0 affected4 at risk
EG0010 events0 affected4 at risk
EG0020 events0 affected5 at risk
EG003
Eye Irritation
Eye disorders
MedDRA24.0
Systematic Assessment
EG0000 events0 affected4 at risk
EG0010 events0 affected4 at risk
EG0020 events0 affected5 at risk
EG003
Glaucoma
Eye disorders
MedDRA24.0
Systematic Assessment
EG0000 events0 affected4 at risk
EG0010 events0 affected4 at risk
EG0020 events0 affected5 at risk
EG003
Hypermetropia
Eye disorders
MedDRA24.0
Systematic Assessment
EG0000 events0 affected4 at risk
EG0010 events0 affected4 at risk
EG0021 events1 affected5 at risk
EG003
Vision Blurred
Eye disorders
MedDRA24.0
Systematic Assessment
EG0000 events0 affected4 at risk
EG0010 events0 affected4 at risk
EG0020 events0 affected5 at risk
EG003
Abdominal Discomfort
Gastrointestinal disorders
MedDRA24.0
Systematic Assessment
EG0000 events0 affected4 at risk
EG0010 events0 affected4 at risk
EG0020 events0 affected5 at risk
EG003
Abdominal Distension
Gastrointestinal disorders
MedDRA24.0
Systematic Assessment
EG0000 events0 affected4 at risk
EG0010 events0 affected4 at risk
EG0020 events0 affected5 at risk
EG003
Anal Haemorrhage
Gastrointestinal disorders
MedDRA24.0
Systematic Assessment
EG0000 events0 affected4 at risk
EG0010 events0 affected4 at risk
EG0020 events0 affected5 at risk
EG003
Chronic Gastritis
Gastrointestinal disorders
MedDRA24.0
Systematic Assessment
EG0000 events0 affected4 at risk
EG0010 events0 affected4 at risk
EG0020 events0 affected5 at risk
EG003
Constipation
Gastrointestinal disorders
MedDRA24.0
Systematic Assessment
EG0000 events0 affected4 at risk
EG0012 events1 affected4 at risk
EG0020 events0 affected5 at risk
EG003
Cyclic Vomiting Syndrome
Gastrointestinal disorders
MedDRA24.0
Systematic Assessment
EG0000 events0 affected4 at risk
EG0010 events0 affected4 at risk
EG0020 events0 affected5 at risk
EG003
Dental Caries
Gastrointestinal disorders
MedDRA24.0
Systematic Assessment
EG0001 events1 affected4 at risk
EG0010 events0 affected4 at risk
EG0020 events0 affected5 at risk
EG003
Diarrhoea
Gastrointestinal disorders
MedDRA24.0
Systematic Assessment
EG0000 events0 affected4 at risk
EG0010 events0 affected4 at risk
EG0022 events2 affected5 at risk
EG003
Dry Mouth
Gastrointestinal disorders
MedDRA24.0
Systematic Assessment
EG0000 events0 affected4 at risk
EG0010 events0 affected4 at risk
EG0020 events0 affected5 at risk
EG003
Dysphagia
Gastrointestinal disorders
MedDRA24.0
Systematic Assessment
EG0000 events0 affected4 at risk
EG0010 events0 affected4 at risk
EG0020 events0 affected5 at risk
EG003
Eructation
Gastrointestinal disorders
MedDRA24.0
Systematic Assessment
EG0001 events1 affected4 at risk
EG0010 events0 affected4 at risk
EG0020 events0 affected5 at risk
EG003
Gastritis
Gastrointestinal disorders
MedDRA24.0
Systematic Assessment
EG0000 events0 affected4 at risk
EG0010 events0 affected4 at risk
EG0020 events0 affected5 at risk
EG003
Nausea
Gastrointestinal disorders
MedDRA24.0
Systematic Assessment
EG0001 events1 affected4 at risk
EG0011 events1 affected4 at risk
EG0020 events0 affected5 at risk
EG003
Periodontal Disease
Gastrointestinal disorders
MedDRA24.0
Systematic Assessment
EG0000 events0 affected4 at risk
EG0010 events0 affected4 at risk
EG0020 events0 affected5 at risk
EG003
Vomiting
Gastrointestinal disorders
MedDRA24.0
Systematic Assessment
EG0000 events0 affected4 at risk
EG0010 events0 affected4 at risk
EG0020 events0 affected5 at risk
EG003
Fatigue
General disorders
MedDRA24.0
Systematic Assessment
EG0000 events0 affected4 at risk
EG0010 events0 affected4 at risk
EG0021 events1 affected5 at risk
EG003
Oedema Peripheral
General disorders
MedDRA24.0
Systematic Assessment
EG0000 events0 affected4 at risk
EG0010 events0 affected4 at risk
EG0020 events0 affected5 at risk
EG003
Pain
General disorders
MedDRA24.0
Systematic Assessment
EG0000 events0 affected4 at risk
EG0010 events0 affected4 at risk
EG0020 events0 affected5 at risk
EG003
Covid-19
Infections and infestations
MedDRA24.0
Systematic Assessment
EG0000 events0 affected4 at risk
EG0010 events0 affected4 at risk
EG0020 events0 affected5 at risk
EG003
Conjunctivitis
Infections and infestations
MedDRA24.0
Systematic Assessment
EG0000 events0 affected4 at risk
EG0010 events0 affected4 at risk
EG0020 events0 affected5 at risk
EG003
Empyema
Infections and infestations
MedDRA24.0
Systematic Assessment
EG0000 events0 affected4 at risk
EG0010 events0 affected4 at risk
EG0020 events0 affected5 at risk
EG003
Fungal Skin Infection
Infections and infestations
MedDRA24.0
Systematic Assessment
EG0000 events0 affected4 at risk
EG0010 events0 affected4 at risk
EG0020 events0 affected5 at risk
EG003
Gastroenteritis
Infections and infestations
MedDRA24.0
Systematic Assessment
EG0000 events0 affected4 at risk
EG0010 events0 affected4 at risk
EG0020 events0 affected5 at risk
EG003
Nasopharyngitis
Infections and infestations
MedDRA24.0
Systematic Assessment
EG0000 events0 affected4 at risk
EG0010 events0 affected4 at risk
EG0020 events0 affected5 at risk
EG003
Pulpitis Dental
Infections and infestations
MedDRA24.0
Systematic Assessment
EG0000 events0 affected4 at risk
EG0010 events0 affected4 at risk
EG0020 events0 affected5 at risk
EG003
Retinitis
Infections and infestations
MedDRA24.0
Systematic Assessment
EG0000 events0 affected4 at risk
EG0010 events0 affected4 at risk
EG0020 events0 affected5 at risk
EG003
Tinea Pedis
Infections and infestations
MedDRA24.0
Systematic Assessment
EG0000 events0 affected4 at risk
EG0010 events0 affected4 at risk
EG0020 events0 affected5 at risk
EG003
Upper Respiratory Tract Infection
Infections and infestations
MedDRA24.0
Systematic Assessment
EG0000 events0 affected4 at risk
EG0010 events0 affected4 at risk
EG0020 events0 affected5 at risk
EG003
Urinary Tract Infection
Infections and infestations
MedDRA24.0
Systematic Assessment
EG0000 events0 affected4 at risk
EG0010 events0 affected4 at risk
EG0020 events0 affected5 at risk
EG003
Arthropod Bite
Injury, poisoning and procedural complications
MedDRA24.0
Systematic Assessment
EG0000 events0 affected4 at risk
EG0011 events1 affected4 at risk
EG0020 events0 affected5 at risk
EG003
Eyelid Injury
Injury, poisoning and procedural complications
MedDRA24.0
Systematic Assessment
EG0000 events0 affected4 at risk
EG0010 events0 affected4 at risk
EG0020 events0 affected5 at risk
EG003
Fall
Injury, poisoning and procedural complications
MedDRA24.0
Systematic Assessment
EG0000 events0 affected4 at risk
EG0010 events0 affected4 at risk
EG0020 events0 affected5 at risk
EG003
Ligament Rupture
Injury, poisoning and procedural complications
MedDRA24.0
Systematic Assessment
EG0000 events0 affected4 at risk
EG0010 events0 affected4 at risk
EG0020 events0 affected5 at risk
EG003
Limb Injury
Injury, poisoning and procedural complications
MedDRA24.0
Systematic Assessment
EG0000 events0 affected4 at risk
EG0010 events0 affected4 at risk
EG0020 events0 affected5 at risk
EG003
Post Lumbar Puncture Syndrome
Injury, poisoning and procedural complications
MedDRA24.0
Systematic Assessment
EG0000 events0 affected4 at risk
EG0010 events0 affected4 at risk
EG0020 events0 affected5 at risk
EG003
Procedural Nausea
Injury, poisoning and procedural complications
MedDRA24.0
Systematic Assessment
EG0000 events0 affected4 at risk
EG0010 events0 affected4 at risk
EG0020 events0 affected5 at risk
EG003
Procedural Pain
Injury, poisoning and procedural complications
MedDRA24.0
Systematic Assessment
EG0000 events0 affected4 at risk
EG0010 events0 affected4 at risk
EG0021 events1 affected5 at risk
EG003
Procedural Vomiting
Injury, poisoning and procedural complications
MedDRA24.0
Systematic Assessment
EG0000 events0 affected4 at risk
EG0010 events0 affected4 at risk
EG0020 events0 affected5 at risk
EG003
Rib Fracture
Injury, poisoning and procedural complications
MedDRA24.0
Systematic Assessment
EG0000 events0 affected4 at risk
EG0010 events0 affected4 at risk
EG0020 events0 affected5 at risk
EG003
Thermal Burn
Injury, poisoning and procedural complications
MedDRA24.0
Systematic Assessment
EG0000 events0 affected4 at risk
EG0010 events0 affected4 at risk
EG0020 events0 affected5 at risk
EG003
Alanine Aminotransferase Increased
Investigations
MedDRA24.0
Systematic Assessment
EG0000 events0 affected4 at risk
EG0010 events0 affected4 at risk
EG0020 events0 affected5 at risk
EG003
Blood Creatine Phosphokinase Increased
Investigations
MedDRA24.0
Systematic Assessment
EG0000 events0 affected4 at risk
EG0010 events0 affected4 at risk
EG0020 events0 affected5 at risk
EG003
Cardiac Murmur
Investigations
MedDRA24.0
Systematic Assessment
EG0000 events0 affected4 at risk
EG0010 events0 affected4 at risk
EG0021 events1 affected5 at risk
EG003
Hepatic Enzyme Increased
Investigations
MedDRA24.0
Systematic Assessment
EG0000 events0 affected4 at risk
EG0010 events0 affected4 at risk
EG0020 events0 affected5 at risk
EG003
Weight Decreased
Investigations
MedDRA24.0
Systematic Assessment
EG0000 events0 affected4 at risk
EG0010 events0 affected4 at risk
EG0020 events0 affected5 at risk
EG003
Decreased Appetite
Metabolism and nutrition disorders
MedDRA24.0
Systematic Assessment
EG0000 events0 affected4 at risk
EG0010 events0 affected4 at risk
EG0020 events0 affected5 at risk
EG003
Type 2 Diabetes Mellitus
Metabolism and nutrition disorders
MedDRA24.0
Systematic Assessment
EG0000 events0 affected4 at risk
EG0010 events0 affected4 at risk
EG0020 events0 affected5 at risk
EG003
Arthralgia
Musculoskeletal and connective tissue disorders
MedDRA24.0
Systematic Assessment
EG0000 events0 affected4 at risk
EG0012 events2 affected4 at risk
EG0020 events0 affected5 at risk
EG003
Back Pain
Musculoskeletal and connective tissue disorders
MedDRA24.0
Systematic Assessment
EG0000 events0 affected4 at risk
EG0010 events0 affected4 at risk
EG0021 events1 affected5 at risk
EG003
Extremity Contracture
Musculoskeletal and connective tissue disorders
MedDRA24.0
Systematic Assessment
EG0000 events0 affected4 at risk
EG0010 events0 affected4 at risk
EG0020 events0 affected5 at risk
EG003
Muscle Spasms
Musculoskeletal and connective tissue disorders
MedDRA24.0
Systematic Assessment
EG0000 events0 affected4 at risk
EG0010 events0 affected4 at risk
EG0020 events0 affected5 at risk
EG003
Pain In Extremity
Musculoskeletal and connective tissue disorders
MedDRA24.0
Systematic Assessment
EG0000 events0 affected4 at risk
EG0010 events0 affected4 at risk
EG0020 events0 affected5 at risk
EG003
Skin Papilloma
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
MedDRA24.0
Systematic Assessment
EG0000 events0 affected4 at risk
EG0010 events0 affected4 at risk
EG0020 events0 affected5 at risk
EG003
Amnesia
Nervous system disorders
MedDRA24.0
Systematic Assessment
EG0000 events0 affected4 at risk
EG0010 events0 affected4 at risk
EG0020 events0 affected5 at risk
EG003
Chorea
Nervous system disorders
MedDRA24.0
Systematic Assessment
EG0000 events0 affected4 at risk
EG0011 events1 affected4 at risk
EG0020 events0 affected5 at risk
EG003
Cognitive Disorder
Nervous system disorders
MedDRA24.0
Systematic Assessment
EG0000 events0 affected4 at risk
EG0010 events0 affected4 at risk
EG0020 events0 affected5 at risk
EG003
Dizziness
Nervous system disorders
MedDRA24.0
Systematic Assessment
EG0001 events1 affected4 at risk
EG0010 events0 affected4 at risk
EG0020 events0 affected5 at risk
EG003
Headache
Nervous system disorders
MedDRA24.0
Systematic Assessment
EG0002 events2 affected4 at risk
EG0010 events0 affected4 at risk
EG0021 events1 affected5 at risk
EG003
Hyperreflexia
Nervous system disorders
MedDRA24.0
Systematic Assessment
EG0000 events0 affected4 at risk
EG0010 events0 affected4 at risk
EG0020 events0 affected5 at risk
EG003
Hypoaesthesia
Nervous system disorders
MedDRA24.0
Systematic Assessment
EG0000 events0 affected4 at risk
EG0010 events0 affected4 at risk
EG0020 events0 affected5 at risk
EG003
Loss Of Consciousness
Nervous system disorders
MedDRA24.0
Systematic Assessment
EG0000 events0 affected4 at risk
EG0010 events0 affected4 at risk
EG0020 events0 affected5 at risk
EG003
Memory Impairment
Nervous system disorders
MedDRA24.0
Systematic Assessment
EG0000 events0 affected4 at risk
EG0011 events1 affected4 at risk
EG0020 events0 affected5 at risk
EG003
Paraesthesia
Nervous system disorders
MedDRA24.0
Systematic Assessment
EG0000 events0 affected4 at risk
EG0010 events0 affected4 at risk
EG0020 events0 affected5 at risk
EG003
Parkinson's Disease
Nervous system disorders
MedDRA24.0
Systematic Assessment
EG0000 events0 affected4 at risk
EG0010 events0 affected4 at risk
EG0021 events1 affected5 at risk
EG003
Peripheral Sensory Neuropathy
Nervous system disorders
MedDRA24.0
Systematic Assessment
EG0000 events0 affected4 at risk
EG0010 events0 affected4 at risk
EG0020 events0 affected5 at risk
EG003
Radial Nerve Palsy
Nervous system disorders
MedDRA24.0
Systematic Assessment
EG0000 events0 affected4 at risk
EG0010 events0 affected4 at risk
EG0020 events0 affected5 at risk
EG003
Resting Tremor
Nervous system disorders
MedDRA24.0
Systematic Assessment
EG0000 events0 affected4 at risk
EG0010 events0 affected4 at risk
EG0020 events0 affected5 at risk
EG003
Restless Legs Syndrome
Nervous system disorders
MedDRA24.0
Systematic Assessment
EG0000 events0 affected4 at risk
EG0010 events0 affected4 at risk
EG0020 events0 affected5 at risk
EG003
Sciatica
Nervous system disorders
MedDRA24.0
Systematic Assessment
EG0000 events0 affected4 at risk
EG0010 events0 affected4 at risk
EG0020 events0 affected5 at risk
EG003
Sleep Deficit
Nervous system disorders
MedDRA24.0
Systematic Assessment
EG0000 events0 affected4 at risk
EG0010 events0 affected4 at risk
EG0021 events1 affected5 at risk
EG003
Somnolence
Nervous system disorders
MedDRA24.0
Systematic Assessment
EG0001 events1 affected4 at risk
EG0010 events0 affected4 at risk
EG0020 events0 affected5 at risk
EG003
Tremor
Nervous system disorders
MedDRA24.0
Systematic Assessment
EG0000 events0 affected4 at risk
EG0010 events0 affected4 at risk
EG0020 events0 affected5 at risk
EG003
Anxiety
Psychiatric disorders
MedDRA24.0
Systematic Assessment
EG0000 events0 affected4 at risk
EG0010 events0 affected4 at risk
EG0020 events0 affected5 at risk
EG003
Confusional State
Psychiatric disorders
MedDRA24.0
Systematic Assessment
EG0000 events0 affected4 at risk
EG0011 events1 affected4 at risk
EG0020 events0 affected5 at risk
EG003
Delirium
Psychiatric disorders
MedDRA24.0
Systematic Assessment
EG0000 events0 affected4 at risk
EG0010 events0 affected4 at risk
EG0020 events0 affected5 at risk
EG003
Depressed Mood
Psychiatric disorders
MedDRA24.0
Systematic Assessment
EG0000 events0 affected4 at risk
EG0011 events1 affected4 at risk
EG0021 events1 affected5 at risk
EG003
Depression
Psychiatric disorders
MedDRA24.0
Systematic Assessment
EG0000 events0 affected4 at risk
EG0011 events1 affected4 at risk
EG0020 events0 affected5 at risk
EG003
Drug Abuse
Psychiatric disorders
MedDRA24.0
Systematic Assessment
EG0000 events0 affected4 at risk
EG0010 events0 affected4 at risk
EG0020 events0 affected5 at risk
EG003
Hallucination
Psychiatric disorders
MedDRA24.0
Systematic Assessment
EG0000 events0 affected4 at risk
EG0010 events0 affected4 at risk
EG0020 events0 affected5 at risk
EG003
Hallucination, Visual
Psychiatric disorders
MedDRA24.0
Systematic Assessment
EG0000 events0 affected4 at risk
EG0010 events0 affected4 at risk
EG0023 events2 affected5 at risk
EG003
Impulsive Behaviour
Psychiatric disorders
MedDRA24.0
Systematic Assessment
EG0000 events0 affected4 at risk
EG0010 events0 affected4 at risk
EG0020 events0 affected5 at risk
EG003
Insomnia
Psychiatric disorders
MedDRA24.0
Systematic Assessment
EG0000 events0 affected4 at risk
EG0010 events0 affected4 at risk
EG0020 events0 affected5 at risk
EG003
Panic Attack
Psychiatric disorders
MedDRA24.0
Systematic Assessment
EG0000 events0 affected4 at risk
EG0010 events0 affected4 at risk
EG0020 events0 affected5 at risk
EG003
Poor Quality Sleep
Psychiatric disorders
MedDRA24.0
Systematic Assessment
EG0000 events0 affected4 at risk
EG0010 events0 affected4 at risk
EG0021 events1 affected5 at risk
EG003
Rapid Eye Movement Sleep Behaviour Disorder
Psychiatric disorders
MedDRA24.0
Systematic Assessment
EG0000 events0 affected4 at risk
EG0010 events0 affected4 at risk
EG0020 events0 affected5 at risk
EG003
Dysuria
Renal and urinary disorders
MedDRA24.0
Systematic Assessment
EG0000 events0 affected4 at risk
EG0010 events0 affected4 at risk
EG0020 events0 affected5 at risk
EG003
Hypertonic Bladder
Renal and urinary disorders
MedDRA24.0
Systematic Assessment
EG0000 events0 affected4 at risk
EG0010 events0 affected4 at risk
EG0020 events0 affected5 at risk
EG003
Micturition Urgency
Renal and urinary disorders
MedDRA24.0
Systematic Assessment
EG0000 events0 affected4 at risk
EG0010 events0 affected4 at risk
EG0020 events0 affected5 at risk
EG003
Nocturia
Renal and urinary disorders
MedDRA24.0
Systematic Assessment
EG0000 events0 affected4 at risk
EG0010 events0 affected4 at risk
EG0020 events0 affected5 at risk
EG003
Pollakiuria
Renal and urinary disorders
MedDRA24.0
Systematic Assessment
EG0000 events0 affected4 at risk
EG0010 events0 affected4 at risk
EG0020 events0 affected5 at risk
EG003
Urinary Incontinence
Renal and urinary disorders
MedDRA24.0
Systematic Assessment
EG0000 events0 affected4 at risk
EG0010 events0 affected4 at risk
EG0020 events0 affected5 at risk
EG003
Ovarian Failure
Reproductive system and breast disorders
MedDRA24.0
Systematic Assessment
EG0000 events0 affected4 at risk
EG0010 events0 affected4 at risk
EG0020 events0 affected5 at risk
EG003
Uterine Haemorrhage
Reproductive system and breast disorders
MedDRA24.0
Systematic Assessment
EG0000 events0 affected4 at risk
EG0010 events0 affected4 at risk
EG0020 events0 affected5 at risk
EG003
Cough
Respiratory, thoracic and mediastinal disorders
MedDRA24.0
Systematic Assessment
EG0000 events0 affected4 at risk
EG0010 events0 affected4 at risk
EG0020 events0 affected5 at risk
EG003
Dyspnoea
Respiratory, thoracic and mediastinal disorders
MedDRA24.0
Systematic Assessment
EG0000 events0 affected4 at risk
EG0010 events0 affected4 at risk
EG0020 events0 affected5 at risk
EG003
Oropharyngeal Pain
Respiratory, thoracic and mediastinal disorders
MedDRA24.0
Systematic Assessment
EG0000 events0 affected4 at risk
EG0010 events0 affected4 at risk
EG0020 events0 affected5 at risk
EG003
Sleep Apnoea Syndrome
Respiratory, thoracic and mediastinal disorders
MedDRA24.0
Systematic Assessment
EG0000 events0 affected4 at risk
EG0010 events0 affected4 at risk
EG0020 events0 affected5 at risk
EG003
Tonsillar Cyst
Respiratory, thoracic and mediastinal disorders
MedDRA24.0
Systematic Assessment
EG0000 events0 affected4 at risk
EG0010 events0 affected4 at risk
EG0020 events0 affected5 at risk
EG003
Eczema
Skin and subcutaneous tissue disorders
MedDRA24.0
Systematic Assessment
EG0000 events0 affected4 at risk
EG0010 events0 affected4 at risk
EG0020 events0 affected5 at risk
EG003
Rash
Skin and subcutaneous tissue disorders
MedDRA24.0
Systematic Assessment
EG0000 events0 affected4 at risk
EG0010 events0 affected4 at risk
EG0020 events0 affected5 at risk
EG003
Rash Papular
Skin and subcutaneous tissue disorders
MedDRA24.0
Systematic Assessment
EG0000 events0 affected4 at risk
EG0010 events0 affected4 at risk
EG0020 events0 affected5 at risk
EG003
Skin Ulcer
Skin and subcutaneous tissue disorders
MedDRA24.0
Systematic Assessment
EG0000 events0 affected4 at risk
EG0010 events0 affected4 at risk
EG0020 events0 affected5 at risk
EG003
Urticaria
Skin and subcutaneous tissue disorders
MedDRA24.0
Systematic Assessment
EG0000 events0 affected4 at risk
EG0010 events0 affected4 at risk
EG0020 events0 affected5 at risk
EG003
Hypertension
Vascular disorders
MedDRA24.0
Systematic Assessment
EG0000 events0 affected4 at risk
EG0010 events0 affected4 at risk
EG0020 events0 affected5 at risk
EG003
Orthostatic Hypotension
Vascular disorders
MedDRA24.0
Systematic Assessment
EG0000 events0 affected4 at risk
EG0010 events0 affected4 at risk
EG0020 events0 affected5 at risk
EG003
This study did not achieve its primary or secondary endpoints at the time of the analysis of the double-blind 52-week treatment period of Part 2 and was terminated early.
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.
Japanese participants received venglustat 15 mg capsule orally QD for up to 52 weeks in Part 1.
4
OG0043
OG0053
OG0063
OG0073
4
OG0042
OG0053
OG0063
OG0072
Any TESAE
Title
Measurements
OG0000
OG0010
OG0020
OG0030
OG0040
OG0050
OG0060
OG0070
OG002
Part 1: Venglustat 8 mg (ROW)
Participants from ROW (except Japan), received venglustat 8 mg capsule orally QD for up to 36 weeks in Part 1.
OG003
Part 1: Venglustat 15 mg (ROW)
Participants from ROW (except Japan), received venglustat 15 mg capsule orally QD for up to 36 weeks in Part 1.
OG004
Part 1: Placebo (Japan Only)
Japanese participants received placebo (matched to venglustat) capsule orally QD for up to 52 weeks in Part 1.
OG005
Part 1: Venglustat 4 mg (Japan Only)
Japanese participants received venglustat 4 mg capsule orally QD for up to 52 weeks in Part 1.
OG006
Part 1: Venglustat 8 mg (Japan Only)
Japanese participants received venglustat 8 mg capsule orally QD for up to 52 weeks in Part 1.
OG007
Part 1: Venglustat 15 mg (Japan Only)
Japanese participants received venglustat 15 mg capsule orally QD for up to 52 weeks in Part 1.
Units
Counts
Participants
OG0004
OG0014
OG0025
OG0034
OG0043
OG0053
OG0063
OG0073
Title
Denominators
Categories
Title
Measurements
OG0000
OG0010
OG0021
OG0032
OG0040
OG0050
OG0060
OG0070
OG003
Part 1: Venglustat 15 mg (ROW)
Participants from ROW (except Japan), received venglustat 15 mg capsule orally QD for up to 36 weeks in Part 1.
OG004
Part 1: Placebo (Japan Only)
Japanese participants received placebo (matched to venglustat) capsule orally QD for up to 52 weeks in Part 1.
OG005
Part 1: Venglustat 4 mg (Japan Only)
Japanese participants received venglustat 4 mg capsule orally QD for up to 52 weeks in Part 1.
OG006
Part 1: Venglustat 8 mg (Japan Only)
Japanese participants received venglustat 8 mg capsule orally QD for up to 52 weeks in Part 1.
OG007
Part 1: Venglustat 15 mg (Japan Only)
Japanese participants received venglustat 15 mg capsule orally QD for up to 52 weeks in Part 1.
Units
Counts
Participants
OG0004
OG0014
OG0025
OG0034
OG0043
OG0053
OG0063
OG0073
Title
Denominators
Categories
Abnormal cranial nerve examination
Title
Measurements
OG0001
OG0011
OG0021
OG0032
OG0041
OG0053
OG0063
OG0072
Abnormal motor examination
Title
Measurements
OG0003
OG0014
OG0024
OG003
Abnormal strength examination
Title
Measurements
OG0001
OG0014
OG0023
OG003
Abnormal reflex examination
Title
Measurements
OG0000
OG0011
OG0021
OG003
Abnormal sensory examination
Title
Measurements
OG0000
OG0012
OG0020
OG003
Abnormal coordination examination
Title
Measurements
OG0002
OG0011
OG0021
OG003
Abnormal gait and coordination
Title
Measurements
OG0003
OG0014
OG0024
OG003
Participants from ROW (except Japan), received venglustat 4 milligrams (mg) capsule orally QD for up to 36 weeks in Part 1.
OG002
Part 1: Venglustat 8 mg (ROW)
Participants from ROW (except Japan), received venglustat 8 mg capsule orally QD for up to 36 weeks in Part 1.
OG003
Part 1: Venglustat 15 mg (ROW)
Participants from ROW (except Japan), received venglustat 15 mg capsule orally QD for up to 36 weeks in Part 1.
OG004
Part 1: Placebo (Japan Only)
Japanese participants received placebo (matched to venglustat) capsule orally QD for up to 52 weeks in Part 1.
OG005
Part 1: Venglustat 4 mg (Japan Only)
Japanese participants received venglustat 4 mg capsule orally QD for up to 52 weeks in Part 1.
OG006
Part 1: Venglustat 8 mg (Japan Only)
Japanese participants received venglustat 8 mg capsule orally QD for up to 52 weeks in Part 1.
OG007
Part 1: Venglustat 15 mg (Japan Only)
Japanese participants received venglustat 15 mg capsule orally QD for up to 52 weeks in Part 1.
Units
Counts
Participants
OG0004
OG0014
OG0025
OG0034
OG0043
OG0053
OG0063
OG0073
Title
Denominators
Categories
Hemoglobin <= 115 g/L (M); ≤ 95 g/L (F)
Title
Measurements
OG0000
OG0010
OG0020
OG0030
OG0040
OG0050
OG0060
OG0070
Hemoglobin >=185 g/L (M) or >=165 g/L (F)
Title
Measurements
OG0000
OG0010
OG0020
OG003
Hemoglobin DFB >=20 g/L
Title
Measurements
OG0001
OG0010
OG0021
OG003
Hematocrit: <=0.37 v/v (M) or <=0.32 v/v (F)
Title
Measurements
OG0000
OG0010
OG0021
OG003
Hematocrit: >=0.55 v/v (M); >=0.5 v/v (F)
Title
Measurements
OG0000
OG0010
OG0020
OG003
RBC: >=6 Tera/L
Title
Measurements
OG0000
OG0010
OG0020
OG003
Platelets: <100 Giga/L
Title
Measurements
OG0000
OG0010
OG0021
OG003
Platelets: >=700 Giga/L
Title
Measurements
OG0000
OG0010
OG0020
OG003
WBC: <3.0 Giga/L (NB) or <2.0 Giga/L (B)
Title
Measurements
OG0000
OG0010
OG0020
OG003
WBC: >=16.0 Giga/L
Title
Measurements
OG0000
OG0010
OG0020
OG003
Neutrophils <1.5 Giga/L (NB) or <1.0 Giga/L (B)
Title
Measurements
OG0000
OG0010
OG0020
OG003
Lymphocytes: <LLN
Title
Measurements
OG0000
OG0011
OG0020
OG003
Lymphocytes: >4.0 Giga/L
Title
Measurements
OG0000
OG0010
OG0020
OG003
Monocytes: <LLN
Title
Measurements
OG0000
OG0010
OG0020
OG003
Monocytes: >0.7 Giga/L
Title
Measurements
OG0000
OG0010
OG0022
OG003
Basophils: >0.1 Giga/L
Title
Measurements
OG0000
OG0010
OG0020
OG003
Eosinophils >0.5 Giga/L or >ULN (ULN >=0.5 Giga/L)
Title
Measurements
OG0000
OG0010
OG0020
OG003
OG003
Part 1: Venglustat 15 mg (ROW)
Participants from ROW (except Japan), received venglustat 15 mg capsule orally QD for up to 36 weeks in Part 1.
OG004
Part 1: Placebo (Japan Only)
Japanese participants received placebo (matched to venglustat) capsule orally QD for up to 52 weeks in Part 1.
OG005
Part 1: Venglustat 4 mg (Japan Only)
Japanese participants received venglustat 4 mg capsule orally QD for up to 52 weeks in Part 1.
OG006
Part 1: Venglustat 8 mg (Japan Only)
Japanese participants received venglustat 8 mg capsule orally QD for up to 52 weeks in Part 1.
OG007
Part 1: Venglustat 15 mg (Japan Only)
Japanese participants received venglustat 15 mg capsule orally QD for up to 52 weeks in Part 1.
Units
Counts
Participants
OG0004
OG0014
OG0025
OG0034
OG0043
OG0053
OG0063
OG0073
Title
Denominators
Categories
ALT >3 ULN
Title
Measurements
OG0000
OG0010
OG0020
OG0030
OG0041
OG0050
OG0061
OG0070
ALT >5 ULN
Title
Measurements
OG0000
OG0010
OG0020
OG003
AST >3 ULN
Title
Measurements
OG0000
OG0010
OG0020
OG003
AST >5 ULN
Title
Measurements
OG0000
OG0010
OG0020
OG003
Alkaline Phosphatase >1.5 ULN
Title
Measurements
OG0000
OG0010
OG0020
OG003
Total Bilirubin > 1.5 ULN
Title
Measurements
OG0000
OG0010
OG0020
OG003
ALT and Bilirubin: >3 ULN and >2 ULN
Title
Measurements
OG0000
OG0010
OG0020
OG003
Direct Bilirubin and Bilirubin: >35% and >1.5 ULN
Title
Measurements
OG0000
OG0010
OG0020
OG003
OG003
Part 1: Venglustat 15 mg (ROW)
Participants from ROW (except Japan), received venglustat 15 mg capsule orally QD for up to 36 weeks in Part 1.
OG004
Part 1: Placebo (Japan Only)
Japanese participants received placebo (matched to venglustat) capsule orally QD for up to 52 weeks in Part 1.
OG005
Part 1: Venglustat 4 mg (Japan Only)
Japanese participants received venglustat 4 mg capsule orally QD for up to 52 weeks in Part 1.
OG006
Part 1: Venglustat 8 mg (Japan Only)
Japanese participants received venglustat 8 mg capsule orally QD for up to 52 weeks in Part 1.
OG007
Part 1: Venglustat 15 mg (Japan Only)
Japanese participants received venglustat 15 mg capsule orally QD for up to 52 weeks in Part 1.
Units
Counts
Participants
OG0004
OG0014
OG0025
OG0034
OG0043
OG0053
OG0063
OG0073
Title
Denominators
Categories
Creatinine >=150 mcmol/L (Adults)
Title
Measurements
OG0000
OG0010
OG0020
OG0030
OG0040
OG0050
OG0060
OG0070
Creatinine >=30% change from baseline
Title
Measurements
OG0000
OG0010
OG0020
OG003
Creatinine >=100% change from baseline
Title
Measurements
OG0000
OG0010
OG0020
OG003
Blood Urea Nitrogen >=17 mmol/L
Title
Measurements
OG0000
OG0010
OG0020
OG003
OG003
Part 1: Venglustat 15 mg (ROW)
Participants from ROW (except Japan), received venglustat 15 mg capsule orally QD for up to 36 weeks in Part 1.
OG004
Part 1: Placebo (Japan Only)
Japanese participants received placebo (matched to venglustat) capsule orally QD for up to 52 weeks in Part 1.
OG005
Part 1: Venglustat 4 mg (Japan Only)
Japanese participants received venglustat 4 mg capsule orally QD for up to 52 weeks in Part 1.
OG006
Part 1: Venglustat 8 mg (Japan Only)
Japanese participants received venglustat 8 mg capsule orally QD for up to 52 weeks in Part 1.
OG007
Part 1: Venglustat 15 mg (Japan Only)
Japanese participants received venglustat 15 mg capsule orally QD for up to 52 weeks in Part 1.
Units
Counts
Participants
OG0004
OG0014
OG0025
OG0034
OG0043
OG0053
OG0063
OG0073
Title
Denominators
Categories
Glucose <=3.9 mmol/L and <LLN
Title
Measurements
OG0000
OG0010
OG0020
OG0030
OG0040
OG0050
OG0060
OG0070
Glucose >=11.1 mmol/L (unfas) or >=7 mmol/L (fas)
Title
Measurements
OG0000
OG0010
OG0020
OG003
Albumin <=25 g/L
Title
Measurements
OG0000
OG0010
OG0020
OG003
OG003
Part 1: Venglustat 15 mg (ROW)
Participants from ROW (except Japan), received venglustat 15 mg capsule orally QD for up to 36 weeks in Part 1.
OG004
Part 1: Placebo (Japan Only)
Japanese participants received placebo (matched to venglustat) capsule orally QD for up to 52 weeks in Part 1.
OG005
Part 1: Venglustat 4 mg (Japan Only)
Japanese participants received venglustat 4 mg capsule orally QD for up to 52 weeks in Part 1.
OG006
Part 1: Venglustat 8 mg (Japan Only)
Japanese participants received venglustat 8 mg capsule orally QD for up to 52 weeks in Part 1.
OG007
Part 1: Venglustat 15 mg (Japan Only)
Japanese participants received venglustat 15 mg capsule orally QD for up to 52 weeks in Part 1.
Units
Counts
Participants
OG0004
OG0014
OG0025
OG0034
OG0043
OG0053
OG0063
OG0073
Title
Denominators
Categories
Sodium <=129 mmol/L
Title
Measurements
OG0000
OG0010
OG0020
OG0030
OG0040
OG0050
OG0060
OG0070
Sodium >=160 mmol/L
Title
Measurements
OG0000
OG0010
OG0020
OG003
Potassium <3 mmol/L
Title
Measurements
OG0000
OG0010
OG0020
OG003
Potassium >=5.5 mmol/L
Title
Measurements
OG0000
OG0010
OG0020
OG003
Chloride <80 mmol/L
Title
Measurements
OG0000
OG0010
OG0020
OG003
Chloride >115 mmol/L
Title
Measurements
OG0000
OG0010
OG0020
OG003
Part 1: Venglustat 8 mg (ROW)
Participants from ROW (except Japan), received venglustat 8 mg capsule orally QD for up to 36 weeks in Part 1.
OG003
Part 1: Venglustat 15 mg (ROW)
Participants from ROW (except Japan), received venglustat 15 mg capsule orally QD for up to 36 weeks in Part 1.
OG004
Part 1: Placebo (Japan Only)
Japanese participants received placebo (matched to venglustat) capsule orally QD for up to 52 weeks in Part 1.
OG005
Part 1: Venglustat 4 mg (Japan Only)
Japanese participants received venglustat 4 mg capsule orally QD for up to 52 weeks in Part 1.
OG006
Part 1: Venglustat 8 mg (Japan Only)
Japanese participants received venglustat 8 mg capsule orally QD for up to 52 weeks in Part 1.
OG007
Part 1: Venglustat 15 mg (Japan Only)
Japanese participants received venglustat 15 mg capsule orally QD for up to 52 weeks in Part 1.
Units
Counts
Participants
OG0004
OG0014
OG0025
OG0034
OG0043
OG0053
OG0063
OG0073
Title
Denominators
Categories
SBP (supine) <=95 mmHg and DFB >=20 mmHg
Title
Measurements
OG0000
OG0010
OG0021
OG0030
OG0040
OG0050
OG0062
OG0070
SBP (supine) >=160 mmHg and IFB >=20 mmHg
Title
Measurements
OG0000
OG0010
OG0020
OG003
DBP (supine) <=45 mmHg and DFB >=10 mmHg
Title
Measurements
OG0000
OG0010
OG0020
OG003
DBP (supine) >=110 mmHg and IFB >=10 mmHg
Title
Measurements
OG0000
OG0010
OG0020
OG003
SBP (Orthostatic): <=-20 mmHg
Title
Measurements
OG0000
OG0010
OG0020
OG003
DBP (Orthostatic): <=-10 mmHg
Title
Measurements
OG0000
OG0010
OG0020
OG003
HR (supine) <=50 bpm and DFB >= 20 bpm
Title
Measurements
OG0000
OG0010
OG0020
OG003
HR (supine) >=120 bpm and IFB >=20 bpm
Title
Measurements
OG0000
OG0010
OG0020
OG003
Weight >=5% DFB
Title
Measurements
OG0000
OG0010
OG0021
OG003
Weight >=5% IFB
Title
Measurements
OG0001
OG0010
OG0021
OG003
Part 1: Venglustat 8 mg (ROW)
Participants from ROW (except Japan), received venglustat 8 mg capsule orally QD for up to 36 weeks in Part 1.
OG003
Part 1: Venglustat 15 mg (ROW)
Participants from ROW (except Japan), received venglustat 15 mg capsule orally QD for up to 36 weeks in Part 1.
OG004
Part 1: Placebo (Japan Only)
Japanese participants received placebo (matched to venglustat) capsule orally QD for up to 52 weeks in Part 1.
OG005
Part 1: Venglustat 4 mg (Japan Only)
Japanese participants received venglustat 4 mg capsule orally QD for up to 52 weeks in Part 1.
OG006
Part 1: Venglustat 8 mg (Japan Only)
Japanese participants received venglustat 8 mg capsule orally QD for up to 52 weeks in Part 1.
OG007
Part 1: Venglustat 15 mg (Japan Only)
Japanese participants received venglustat 15 mg capsule orally QD for up to 52 weeks in Part 1.
Units
Counts
Participants
OG0004
OG0014
OG0025
OG0034
OG0043
OG0053
OG0063
OG0073
Title
Denominators
Categories
Any eye
Title
Measurements
OG0003
OG0013
OG0024
OG0032
OG0042
OG0053
OG0063
OG0070
Right eye
Title
Measurements
OG0003
OG0013
OG0024
OG003
Left eye
Title
Measurements
OG0003
OG0013
OG0024
OG003
Part 1: Venglustat 8 mg (ROW)
Participants from ROW (except Japan), received venglustat 8 mg capsule orally QD for up to 36 weeks in Part 1.
OG003
Part 1: Venglustat 15 mg (ROW)
Participants from ROW (except Japan), received venglustat 15 mg capsule orally QD for up to 36 weeks in Part 1.
OG004
Part 1: Placebo (Japan Only)
Japanese participants received placebo (matched to venglustat) capsule orally QD for up to 52 weeks in Part 1.
OG005
Part 1: Venglustat 4 mg (Japan Only)
Japanese participants received venglustat 4 mg capsule orally QD for up to 52 weeks in Part 1.
OG006
Part 1: Venglustat 8 mg (Japan Only)
Japanese participants received venglustat 8 mg capsule orally QD for up to 52 weeks in Part 1.
OG007
Part 1: Venglustat 15 mg (Japan Only)
Japanese participants received venglustat 15 mg capsule orally QD for up to 52 weeks in Part 1.
Units
Counts
Participants
OG0004
OG0014
OG0025
OG0034
OG0043
OG0053
OG0063
OG0073
Title
Denominators
Categories
HR: <50 bpm
Title
Measurements
OG0000
OG0011
OG0021
OG0030
OG0041
OG0050
OG0060
OG0070
HR: <50 bpm and DFB >=20 bpm
Title
Measurements
OG0000
OG0010
OG0020
OG003
HR: <40 bpm
Title
Measurements
OG0000
OG0010
OG0020
OG003
HR: >90 bpm
Title
Measurements
OG0000
OG0010
OG0020
OG003
HR: >90 bpm and IFB >=20 bpm
Title
Measurements
OG0000
OG0010
OG0020
OG003
HR: >100 bpm
Title
Measurements
OG0000
OG0010
OG0020
OG003
PR Interval: >200 msec
Title
Measurements
OG0000
OG0010
OG0020
OG003
PR Interval: >200 msec and IFB >=25%
Title
Measurements
OG0000
OG0010
OG0020
OG003
PR Interval: >220 msec
Title
Measurements
OG0000
OG0010
OG0020
OG003
QRS Interval: >110 msec
Title
Measurements
OG0000
OG0011
OG0021
OG003
QRS Interval: >110 msec and IFB >=25%
Title
Measurements
OG0000
OG0010
OG0020
OG003
QRS Interval: >120 msec
Title
Measurements
OG0000
OG0010
OG0021
OG003
QT Interval: >500 msec
Title
Measurements
OG0000
OG0010
OG0020
OG003
QTcB interval: >450 msec
Title
Measurements
OG0000
OG0010
OG0021
OG003
QTcB interval: >480 msec
Title
Measurements
OG0000
OG0010
OG0020
OG003
QTcB interval: IFB >30 and <=60 msec
Title
Measurements
OG0000
OG0010
OG0021
OG003
QTcB interval: IFB >60 msec
Title
Measurements
OG0000
OG0010
OG0020
OG003
QTc F: >450 msec
Title
Measurements
OG0000
OG0010
OG0021
OG003
QTc F: >480 msec
Title
Measurements
OG0000
OG0010
OG0020
OG003
QTc F: IFB >30 and <=60 msec
Title
Measurements
OG0000
OG0010
OG0021
OG003
QTc F: IFB >60 msec
Title
Measurements
OG0000
OG0010
OG0020
OG003
Participants received venglustat 15 mg capsule orally QD for 52 weeks in Part 2 DB period.
Units
Counts
Participants
OG00095
OG00181
Title
Denominators
Categories
Title
Measurements
OG0004.71± 1.27
OG0017.29± 1.36
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
Least-squares (LS) mean, standard errors (SE) and p-value were estimated from mixed-effect model with repeated measures (MMRM) analysis which included fixed categorical effects of treatment group, randomization strata, time point, treatment-by-time point and strata-by-time point interaction and continuous fixed covariates Baseline value and Baseline value-by-time point interaction.
MMRM
=0.1679
The threshold for statistical significance was 0.05.
LS mean difference
2.58
Standard Error of the Mean
1.87
2-Sided
95
-1.10
6.27
Superiority
Participants received venglustat 15 mg capsule orally QD for 52 weeks in Part 2 DB period.
Units
Counts
Participants
OG00094
OG00182
Title
Denominators
Categories
Title
Measurements
OG0000.32± 1.27
OG001-0.65± 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
LS mean, SE and P-value were estimated from MMRM analysis which included fixed categorical effects of treatment group, randomization strata, time point, treatment-by-time point and strata-by-time point interaction and continuous fixed covariates Baseline value and Baseline value-by-time point interaction.
MMRM
=0.5996
The threshold for statistical significance was 0.05.
LS mean difference
-0.97
Standard Error of the Mean
1.85
2-Sided
95
-4.63
2.68
Superiority
OG001
Part 2, DB Period: Venglustat 15 mg
Participants received venglustat 15 mg capsule orally QD for 52 weeks in Part 2 DB period.
Units
Counts
Participants
OG00095
OG00181
Title
Denominators
Categories
Title
Measurements
OG0005.87± 1.45
OG0019.99± 1.55
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
LS mean, SE and P-value were estimated from MMRM analysis which included fixed categorical effects of treatment group, randomization strata, time point, treatment-by-time point and strata-by-time point interaction and continuous fixed covariates Baseline value and Baseline value-by-time-interaction
MMRM
=0.0535
The threshold for statistical significance was 0.05.
LS mean difference
4.13
Standard Error of the Mean
2.13
2-Sided
95
-0.06
8.32
Superiority
Units
Counts
Participants
OG00095
OG00180
Title
Denominators
Categories
Title
Measurements
OG0000.18± 0.05
OG0010.20± 0.06
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
LS mean, SE and P-value: estimated from MMRM analysis which included fixed categorical effects of treatment group, randomization strata, time point, treatment-by-time point and strata-by-time point interaction and continuous fixed covariates Baseline value and Baseline value-by-time-interaction.
MMRM
=0.7400
The threshold for statistical significance was 0.05.