A Study to Assess Safety and Efficacy of Avalglucosidase... | NCT03019406 | Trialant
NCT03019406
Sponsor
Genzyme, a Sanofi Company
Status
Active, not recruiting
Last Update Posted
Jan 30, 2026Actual
Enrollment
22Actual
Phase
Phase 2
Conditions
Glycogen Storage Disease Type II-Pompe's Disease
Interventions
Avalglucosidase alfa (GZ402666)
Alglucosidase alfa (GZ419829)
Countries
United States
France
Japan
Taiwan
United Kingdom
Protocol Section
Identification Module
NCT ID
NCT03019406
Obsolete or Duplicate NCT IDs
Not provided
Organization Study
ACT14132
Secondary IDs
ID
Type
Description
Link
U1111-1179-4616
Registry Identifier
ICTRP
2016-003475-21
EudraCT Number
Brief Title
A Study to Assess Safety and Efficacy of Avalglucosidase Alfa Administered Every Other Week in Pediatric Patients With Infantile-onset Pompe Disease Previously Treated With Alglucosidase Alfa
Official Title
An Open-label Ascending Dose Cohort Study to Assess the Safety, Pharmacokinetics, and Preliminary Efficacy of Avalglucosidase Alfa (NeoGAA, GZ402666) in Patients With Infantile-onset Pompe Disease Treated With Alglucosidase Alfa Who Demonstrate Clinical Decline or Sub-optimal Clinical Response
Acronym
Mini-COMET
Organization
SanofiINDUSTRY
Status Module
Record Verification Date
Jan 2026
Overall Recruitment Status or Expanded Access Status
Active, not recruiting
Last Known Status
Not provided
Delayed Posting
Not provided
Why Stopped
Not provided
Expanded Access Info
No
Start Date
Oct 12, 2017Actual
Primary Completion Date
Sep 30, 2019Actual
Completion Date
Aug 10, 2027Estimated
First Submitted Date
Dec 20, 2016
First Submission Date that Met QC Criteria
Jan 10, 2017
First Posted Date
Jan 12, 2017Estimated
Results Waived
Not provided
Results First Submitted Date
Aug 24, 2021
Results First Submitted that Met QC Criteria
Oct 22, 2021
Results First Posted Date
Nov 22, 2021Actual
Certification/Extension (aka Delayed Results) First Submitted Date
Sep 25, 2020
Certification/Extension First Submitted that Passed QC Review
Oct 22, 2021
Certification/Extension First Posted Date
Nov 22, 2021Actual
Last Update Submitted Date
Jan 13, 2026
Last Update Posted Date
Jan 30, 2026Actual
Sponsor/Collaborators Module
Responsible Party, by Official Title
Sponsor
Lead Sponsor
Genzyme, a Sanofi CompanyINDUSTRY
Collaborators
Not provided
Oversight Module
Has Data Monitoring Committee (DMC)
Yes
Is FDA Regulated Drug
Yes
Is FDA Regulated Device
No
Is Unapproved Device
Not provided
Pediatric Postmarket Surveillance of a Device Product
Not provided
Product Exported from US
Not provided
FDAAA801 Violation
Not provided
Description Module
Brief Summary
This is a multi-stage, phase 2, open-label, multicenter, multinational, ascending dose cohort, repeated intravenous (IV) infusion study of avalglucosidase alfa in pediatric patients with Infantile-Onset Pompe Disease (IOPD) who have been previously treated with alglucosidase alfa for a minimum of 6 months immediately prior to study entry and have demonstrated clinical decline or unsatisfactory clinical response.
Detailed Description
The duration of the study for each participant will be up to 9 years and 4 months that will consist of a 14-day screening period, that may be extended to up to 4 weeks in pre-specified situations. This is followed by a 25-week treatment period and an up to a 462-week treatment extension period and a 4-week post-treatment observation period. Cohort 1 and 2 (Cohort 1: avalglucosidase alfa 20 milligrams per kilogram [mg/kg], Cohort 2: avalglucosidase alfa 40 mg/kg) will be non-randomized and Cohort 3 (Cohort 3a: avalglucosidase alfa 40 mg/kg [maximum tolerated dose] and Cohort 3b: alglucosidase alfa) will be randomized.
Conditions Module
Conditions
Glycogen Storage Disease Type II-Pompe'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
22Actual
Arms/Interventions Module
Arm Groups
Label
Type
Description
Intervention Names
Cohort 1: Avalglucosidase Alfa 20 mg/kg
Experimental
Avalglucosidase alfa, 20 mg/kg intravenous (IV) infusion every other week (qow) for 25 weeks in the Primary Analysis Period (PAP), followed by same treatment from Week 26 up to Week 371 in extension treatment period (ETP).
Drug: Avalglucosidase alfa (GZ402666)
Cohort 2: Avalglucosidase Alfa 40 mg/kg
Experimental
Avalglucosidase alfa 40 mg/kg IV infusion qow for 25 weeks in the PAP, followed by same treatment from Week 26 up to Week 371 in ETP.
Drug: Avalglucosidase alfa (GZ402666)
Cohort 3a: Avalglucosidase Alfa 40 mg//kg
Experimental
After determination of the highest tolerated avalglucosidase alfa dose in Cohort 1 and Cohort 2 (after at least 5 participants in each Cohort 1 and Cohort 2 had received the 7th dose of avalglucosidase alfa or completed Week 13 with a minimum of 6 infusions), participants received avalglucosidase alfa 40 mg/kg (the highest tolerated dose) IV infusion qow for 25 weeks in PAP, followed by same treatment from Week 26 up to Week 371 in ETP.
Drug: Avalglucosidase alfa (GZ402666)
Cohort 3b: Alglucosidase Alfa in PAP
Experimental
After determination of the highest tolerated avalglucosidase alfa dose in Cohort 1 and Cohort 2 (after at least 5 participants in each Cohort 1 and Cohort 2 had received the 7th dose of avalglucosidase alfa or completed Week 13 with a minimum of 6 infusions), participants received alglucosidase alfa at their current stable dose (defined as dose [between 20 mg/kg qow and 40 mg/kg weekly as per physician] administered regularly for a minimum of 6 months immediately prior to entry in this study) IV infusion for 25 weeks in PAP. After PAP, participants received avalglucosidase alfa 40mg/kg IV infusion qow from Week 26 up to Week 371 in ETP.
Interventions
Name
Type
Description
Arm Group Labels
Other Names
Avalglucosidase alfa (GZ402666)
Drug
Pharmaceutical form: powder for concentrate for solution for infusion,
Route of administration: IV
Cohort 1: Avalglucosidase Alfa 20 mg/kg
Outcomes Module
Primary Outcomes
Measure
Description
Time Frame
PAP: Number of Participants With Treatment-emergent Adverse Events (TEAEs), Serious Treatment-emergent Adverse Events, and Adverse Event of Special Interest (AESI)
Adverse event (AE): any untoward medical occurrence in participant received study drug & did not necessarily had to have causal relationship with treatment. TEAEs: AEs developed/worsened in grade/become serious during PAP period (from the time of 1st study drug dose up to Week 25). Serious AE(SAE): any untoward medical occurrence at any dose resulted in death, was life-threatening, required inpatient hospitalization, prolongation of existing hospitalization, resulted in persistent or significant disability/incapacity, was congenital anomaly/birth defect or was medically important event. TEAEs included SAEs & non-SAEs. AESI:AE (serious/non-serious) of scientific & medical concern specific to Sponsor's product/program, for which ongoing monitoring & immediate notification by Investigator to Sponsor required.
From Baseline to Week 25
PAP: Number of Participants With Infusion-associated Reactions (IARs)
IARs were defined as AESIs that occurred during either the infusion or the observation period following the infusion which were deemed to be related or possibly related to the study drug. Protocol-defined IARs: An AESIs that occurred during either the infusion or the observation period following the infusion which were deemed to be related or possibly related to study drug. Algorithm-defined IARs: any TEAE meeting either 1 or 2 criteria: 1) event occurred from the start of infusion to the end of infusion plus 24 hours, and considered related to study drug, 2) If an AE time component was missing, compared AE Start date with infusion start date and infusion end date. If an AE Start date was between infusion start date and infusion end date plus one day, and it was related to study drug.
From Baseline to Week 25
Secondary Outcomes
Measure
Description
Time Frame
PAP: Number of Participants With Anti-drug Antibody (ADA) Response
Anti-drug antibody response was categorized as: Treatment induced ADAs: ADAs developed de novo (seroconversion) following administration of study drug. Treatment boosted ADAs: pre-existing ADAs that were boosted at least two titer steps from Baseline (i.e., 4-fold increase in titers) followed by administration of study drug.
From Baseline to Week 25
Other Outcomes
Not provided
Eligibility Module
Eligibility Criteria
Inclusion criteria:
The participants has confirmed acid alpha-glucosidase (GAA) enzyme deficiency from any tissue source.
The participants who has reached legal age of majority as defined by local regulation, or the participant's legal guardian(s) must provide signed informed consent prior to performing any study-related procedures. If the participant is legally minor per local regulations, assent shall be obtained from participants, if applicable.
The participants (and participant's legal guardian if participant is legally minor as defined by local regulation) must have the ability to comply with the clinical protocol.
The participants is less than 18 years old.
The participants, if female and of childbearing potential, must have a negative serum pregnancy test (beta-human chorionic gonadotropin) and must not breastfeeding at screening/Baseline.
The participant has cardiomyopathy at the time of diagnosis: i.e., left ventricular mass index (LVMI) equivalent to mean age specific LVMI plus 2 standard deviations.
The participant has been receiving a stable dose of alglucosidase alfa regularly for a minimum of 6 months immediately prior to study entry.
For participants in Stage 1: The participant has documented evidence of clinical decline in at least 1 of the following parameters related to Pompe Disease and not related to intercurrent illness as assessed by the Investigator: respiratory function, motor skills, and/or cardiac parameters.
For participants in Stage 2: The participant has documented evidence of suboptimal clinical response in at least 1 of the following parameters related to Pompe Disease and not related to intercurrent illness as assessed by the Investigator: respiratory function, motor skills, and/or new onset of ptosis.
Exclusion criteria:
Participants are excluded from the study if any of the following criteria apply:
The participant has high antibody titer to alglucosidase alfa.
The participant has a high risk for a severe allergic reaction to neoGAA (avalglucosidase alfa).
The participant requires any prohibited concomitant medications (e.g., immune modulatory treatment) for the duration of the study.
The participant has previously participated in any ACT14132 study cohort.
Female participant of childbearing potential not protected by highly effective contraceptive method of birth control and/or who is unwilling or unable to tested for pregnancy.
The above information is not intended to contain all considerations relevant to a participant's potential participation in a clinical trial.
Accepts Healthy Volunteers
Yes
Sex
All
Sex/Gender Based
Not provided
Sex/Gender Description
Not provided
Minimum Age
6 Months
Maximum Age
17 Years
Standard Ages
Child
Study Population
Not provided
Sampling Method
Not provided
Contacts/Locations Module
Central Contacts
Not provided
Overall Officials
Name
Affiliation
Role
Clinical Sciences & Operations
Sanofi
Study Director
Locations
Facility
Status
City
State
ZIP
Country
Contacts
Regional Medical Genetics Center of New York Site Number : 8400002
Kronn D, Davison J, Broomfield A, Brassier A, Labarthe F, Hahn SH, Kumada S, Ohki H, Prakalapakorn SG, Wilson C, Haack KA, Huynh-Ba O, Richards S, Sparks S, Tammireddy S, Zhou T, Chien YH, Kishnani PS; Mini-COMET investigators. The Mini-COMET Clinical Trial: Safety and Efficacy of Avalglucosidase Alfa after 97 Weeks of Treatment in Children with Infantile-Onset Pompe Disease Previously Treated with Alglucosidase Alfa. J Pediatr. 2025 Oct;285:114664. doi: 10.1016/j.jpeds.2025.114664. Epub 2025 May 29.
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
Participants in Cohorts 1 received avalglucosidase alfa 20 milligrams per kilogram (mg/kg) every other week (qow) and participants in Cohort 2 received avalglucosidase alfa 40 mg/kg qow. No randomization process was applied for Cohort 1 and 2. Participants in Cohort 3 were randomized (stratified by gender) to receive either avalglucosidase alfa 40 mg/kg qow (3a) or their current stable alglucosidase alfa dose regimen (3b). Data reported based on primary completion date, i.e., 30 September 2019.
Recruitment Details
The study was conducted at 10 active centers in 5 countries. Total of 22 participants were screened between 12 October 2017 and 03 April 2019. None of the participants were screen failed. Participants were assigned to treatment by using interactive response technology system.
Type of Units Analyzed
Not provided
Arm/Group Information
ID
Title
Description
FG000
Cohort 1: Avalglucosidase Alfa 20 mg/kg
Avalglucosidase alfa, 20 mg/kg intravenous (IV) infusion qow for 25 weeks in the Primary Analysis Period (PAP), followed by same treatment from Week 26 up to Week 371 in extension treatment period (ETP).
FG001
Cohort 2: Avalglucosidase Alfa 40 mg/kg
Periods
Title
Milestones
Reasons Not Completed
PAP: up to 25 Weeks
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
Not provided
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
Jan 5, 2021
Aug 24, 2021
Derived Section
Miscellaneous Info Module
Version Holder
Jul 10, 2026
Removed Countries
Not provided
Submission Tracking
Estimated Results First Submitted Date
Not provided
Condition Browse Module
No data available
No data is available for this block.
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
None (Open Label)
Masking Description
Not provided
Who Masked
Not provided
Drug: Avalglucosidase alfa (GZ402666)
Drug: Alglucosidase alfa (GZ419829)
Cohort 2: Avalglucosidase Alfa 40 mg/kg
Cohort 3a: Avalglucosidase Alfa 40 mg//kg
Cohort 3b: Alglucosidase Alfa in PAP
Nexviazyme
Alglucosidase alfa (GZ419829)
Drug
Pharmaceutical form: powder for concentrate for solution for infusion,
Route of administration: IV
Cohort 3b: Alglucosidase Alfa in PAP
Myozyme®
Lumizyme®
PAP: Pharmacokinetic (PK) Parameter: Maximum Observed Plasma Concentration (Cmax) of Avalglucosidase Alfa
Cmax is the maximum observed plasma concentration.
Cohort 1: pre-dose; at end of infusion; and at 2, 4, 6, and 8 hours after end of infusion on Day 1 (Week 1) and Week 25 Cohort 2 and 3: pre-dose; at end of infusion; and at 2, 4, 6, 8, and 12 to 16 hours after end of infusion on Day 1 (Week 1) and Week 25
PAP: Pharmacokinetic Parameter: Time to Achieve Maximum Observed Plasma Concentration (Tmax) of Avalglucosidase Alfa
Tmax is the time to achieve maximum plasma concentration.
Cohort 1: pre-dose; at end of infusion; and at 2, 4, 6, and 8 hours after end of infusion on Day 1 (Week 1) and Week 25 Cohort 2 and 3: pre-dose; at end of infusion; and at 2, 4, 6, 8, and 12 to 16 hours after end of infusion on Day 1 (Week 1) and Week 25
PAP: Pharmacokinetic Parameter: Area Under the Plasma Concentration Versus Time Curve From Time 0 to the Time of Last Quantifiable Concentration (AUC0-last) of Avalglucosidase Alfa
AUC0-last is the area under the plasma concentration versus time curve from time 0 to the time of last quantifiable concentration.
Cohort 1: pre-dose; at end of infusion; and at 2, 4, 6, and 8 hours after end of infusion on Day 1 (Week 1) and Week 25 Cohort 2 & 3: pre-dose; at end of infusion; and at 2, 4, 6, 8, and 12 to 16 hours after end of infusion on Day 1 (Week 1) and Week 25
PAP: Pharmacokinetic Parameter: Terminal Half-life (t1/2) of Avalglucosidase Alfa
t1/2 is the time required for the plasma concentration of a drug to decrease by half of its initial concentration.
Cohort 1: pre-dose; at end of infusion; and at 2, 4, 6, and 8 hours after end of infusion on Day 1 (Week 1) and Week 25 Cohort 2 & 3: pre-dose; at end of infusion; and at 2, 4, 6, 8, and 12 to 16 hours after end of infusion on Day 1 (Week 1) and Week 25
PAP: Pharmacokinetic Parameter: Clearance (CL) of Avalglucosidase Alfa
CL is defined as a quantitative measure of the rate at which a drug substance is removed from the body.
Cohort 1: pre-dose; at end of infusion; and at 2, 4, 6, and 8 hours after end of infusion on Day 1 (Week 1) and Week 25 Cohort 2 and 3: pre-dose; at end of infusion; and at 2, 4, 6, 8, and 12 to 16 hours after end of infusion on Day 1 (Week 1) and Week 25
PAP: Pharmacokinetic Parameter: Volume of Distribution at Steady State (Vss) of Avalglucosidase Alfa
Steady state volume of distribution (Vss) is defined as the theoretical volume in which the total amount of drug would need to be uniformly distributed to produce the desired plasma concentration of a drug.
Cohort 1: pre-dose; at end of infusion; and at 2, 4, 6, and 8 hours after end of infusion on Day 1 (Week 1) and Week 25 Cohort 2 & 3: pre-dose; at end of infusion; and at 2, 4, 6, 8, and 12 to 16 hours after end of infusion on Day 1 (Week 1) and Week 25
PAP: Change From Baseline in Gross Motor Function Measure-88 (GMFM-88) Test Scores at Week 25
GMFM-88 was developed specifically to detect quantitative changes in gross motor function. The GMFM-88 consisted of 88 items organized into 5 dimensions; lying and rolling (17 items); sitting (20 items); crawling and kneeling (14 items); standing (13 items) and walking, running and jumping (24 items). Each item was scored on a 4-point Likert scale with scores range: 0= cannot do; 1 = initiates less then [<] 10 percentage [%] of the task; 2 = partially completes [10% to <100% of the task] and 3 = task completion. The score for each dimension was expressed as a % of the maximum score for that dimension.Total percentage score was obtained by adding the percentage scores for each dimension and dividing the sum by the total number of dimensions. Total scores ranged from 0% to 100%; where higher scores indicated better motor functions.
Baseline, Week 25
PAP: Number of Participants in Gross Motor Function Classification System-Expanded and Revised (GMFCS-E and R) Scores at Baseline and Week 25
GMFCS-E&R was a 5 level classification system for specific age ranges; observations were performed on 5 levels based on self-initiated movement, with emphasis on sitting, transfers, and mobility: Level I (walks without limitations), Level II (walks with limitations), Level III (walks using a hand-held mobility device), Level IV (self-mobility with limitations; may use powered mobility) and level V (transported in a manual wheel chair) (I to V). The distinctions between levels were based on functional limitations, the need for assistive mobility devices, and to a much lesser extent, quality of movement, and were designed to be meaningful in daily life. The lower level represented good motor functioning and higher level represented low motor functioning. Number of participants in each level of classification at Baseline and Week 25 were reported.
Baseline, Week 25
PAP: Change From Baseline in Pompe Pediatric Evaluation of Disability Inventory (Pompe-PEDI) Functional Skills Scale: Mobility Domain Test Score-Scaled Score at Week 25
Pompe-PEDI: disease specific version to assess functional capabilities and performance in children with Pompe disease from 2 months through adolescence. It comprised of Functional Skills Scale and Caregiver Assistance Scale; both scales had 3 domains: Self Care, Mobility, and Social Function. Mobility domain was used to measure change in mobility due to changes in muscle strength; consisted of 160 mobility items for participant/legal guardian. The total number of mobility items the child was capable of, was converted to a scaled score with a range of 0 to 100, where scores near "0" indicated low capability and scores near "100" indicated high capability), where higher score was indicative of greater functional ability. Scaled scores were used to interpret individual function and progress over time.
Baseline, Week 25
PAP: Change From Baseline in Quick Motor Function Test (QMFT) Scores at Week 25
QMFT was observer administered test comprised of 16 items specifically difficult for participants with Pompe disease. Each item was scored on 5-point ordinal scale ranged from 0 to 4 (higher score indicated better outcome). Total QMFT score was obtained by adding the scores of all items and ranged from 0 (unable to perform motor function tests) to 64 (normal muscle function), where higher score indicated better outcome/greater motor function.
Baseline, Week 25
PAP: Echo-Left Ventricular Mass Z-Score (LVM Z-score) M-mode at Baseline, Week 25, and Change From Baseline at Week 25
Cardiac function was evaluated using LVM Z-score as assessed by echocardiogram in M-mode. Z-Scores indicated the number of standard deviations (SD) from the mean in a normal distribution. The normal range is -2 to 2 and greater than 2 may indicate left ventricular hypertrophy. A negative change from Baseline indicated a decrease and positive change from Baseline indicated an increase in LVM Z-score. In this OM, absolute scores at Baseline and Week 25 along with Change from Baseline at Week 25 in LVM Z-score were reported.
Baseline, Week 25
PAP: Change From Baseline in Eyelid Position Measurements: Interpalpebral Fissure Distance (IPFD) - Left Non-Flash and Right Non-Flash at Week 25
IPFD is the widest vertical distance (in millimeters) between the upper eyelid and the lower eyelid when the participant is looking in "primary gaze" (i.e. normal gaze when looking straight forward). Images were taken while the participants was wearing a pair of empty eyeglass frames with millimeters rulers attached as a standardized tool to measure eyelid position without camera flash. A negative change from Baseline indicated a decrease and positive change from Baseline indicated an increase in measured distance.
Baseline, Week 25
PAP: Change From Baseline in Eyelid Position Measurements: Left and Right Margin Reflex Distance (MRD) at Week 25
The MRD is the vertical distance (in millimeters) between the light reflex and the upper eyelid when the participant was looking in "primary gaze" while fixating on a light source. Images were taken while the participants was wearing a pair of empty eyeglass frames with millimeters rulers attached as a standardized tool to measure eyelid position. A negative change from Baseline indicated a decrease and positive change from Baseline indicated an increase in measured distance.
Baseline, Week 25
PAP: Change From Baseline in Eyelid Position Measurements Assessed by Margin Pupil Distance (MPD) - Left Non-Flash and Right Non-Flash at Week 25
The MPD is the vertical distance (in millimeters) between the center of the pupil and the upper eyelid margin. Images were taken while the participants was wearing a pair of empty eyeglass frames with millimeters rulers attached as a standardized tool to measure eyelid position without camera flash. A negative change from Baseline indicated a decrease and positive change from Baseline indicated an increase in measured distance.
Baseline, Week 25
PAP: Change From Baseline in Creatine Kinase Value at Week 25
Change from Baseline in Creatine kinase value (to assess muscle damage) at Week 25 were reported in this OM.
Baseline, Week 25
ETP: Number of Participants With Treatment Emergent Adverse Events, Serious Treatment Emergent Adverse Events, and Adverse Event of Special Interest
Data for this outcome measure will be reported at the time of anticipated last participant last visit results posting (August 2028).
Up to 9 years and 4 months
ETP: Number of Participants With Infusion-associated Reactions
Data for this outcome measure will be reported at the time of anticipated last participant last visit results posting (August 2028).
Up to 9 years and 4 months
Duke University Medical Center Site Number : 8400001
Durham
North Carolina
27710-4000
United States
Seattle Childrens Hospital and Regional Medical Center- Site Number : 8400005
Seattle
Washington
98040
United States
Investigational Site Number : 2500004
Nantes
44093
France
Investigational Site Number : 2500003
Paris
75015
France
Investigational Site Number : 2500002
Paris
75019
France
Investigational Site Number : 2500001
Tours
37044
France
Investigational Site Number : 3920001
Fuchu-shi
Tokyo
183-0042
Japan
Investigational Site Number : 3920002
Fuchu-shi
Tokyo
183-8561
Japan
Investigational Site Number : 1580001
Hsinchu
30059
Taiwan
Investigational Site Number : 8260001
London
London, City of
WC1N 3JH
United Kingdom
Investigational Site Number : 8260002
Manchester
M13 9WL
United Kingdom
Derived
Kishnani PS, Kronn D, Brassier A, Broomfield A, Davison J, Hahn SH, Kumada S, Labarthe F, Ohki H, Pichard S, Prakalapakorn SG, Haack KA, Kittner B, Meng X, Sparks S, Wilson C, Zaher A, Chien YH; Mini-COMET Investigators. Safety and efficacy of avalglucosidase alfa in individuals with infantile-onset Pompe disease enrolled in the phase 2, open-label Mini-COMET study: The 6-month primary analysis report. Genet Med. 2023 Feb;25(2):100328. doi: 10.1016/j.gim.2022.10.010. Epub 2022 Dec 21.
Avalglucosidase alfa 40 mg/kg IV infusion qow for 25 weeks in the PAP, followed by same treatment from Week 26 up to Week 371 in ETP.
FG002
Cohort 3a: Avalglucosidase Alfa 40 mg//kg
After determination of the highest tolerated avalglucosidase alfa dose in Cohort 1 and Cohort 2 (after at least 5 participants in each Cohort 1 and Cohort 2 had received the 7th dose of avalglucosidase alfa or completed Week 13 with a minimum of 6 infusions), participants received avalglucosidase alfa 40 mg/kg (the highest tolerated dose) IV infusion qow for 25 weeks in PAP, followed by same treatment from Week 26 up to Week 371 in ETP.
FG003
Cohort 3b: Alglucosidase Alfa in PAP
After determination of the highest tolerated avalglucosidase alfa dose in Cohort 1 and Cohort 2 (after at least 5 participants in each Cohort 1 and Cohort 2 had received the 7th dose of avalglucosidase alfa or completed Week 13 with a minimum of 6 infusions), participants received alglucosidase alfa at their current stable dose (defined as dose [between 20 mg/kg qow and 40 mg/kg weekly as per physician] administered regularly for a minimum of 6 months immediately prior to entry in this study) IV infusion for 25 weeks in PAP. After PAP, participants received avalglucosidase alfa 40mg/kg IV infusion qow from Week 26 up to Week 371 in ETP.
FG0006 subjects
FG0015 subjects
FG0025 subjects
FG0036 subjects
Treated
FG0006 subjects
FG0015 subjects
FG0025 subjects
FG0036 subjects
COMPLETED
FG0006 subjects
FG0015 subjects
FG0025 subjects
FG0036 subjects
NOT COMPLETED
FG0000 subjects
FG0010 subjects
FG0020 subjects
FG0030 subjects
ETP: Ongoing From Week 26
Type
Comment
Milestone Data
STARTED
FG0006 subjects
FG0015 subjects
FG0025 subjects
FG0033 subjectsParticipants who completed PAP and accepted to receive avalglucosidase alfa treatment in ETP.
COMPLETED
FG0000 subjects
FG0010 subjects
FG0020 subjects
FG0030 subjects
NOT COMPLETED
FG0006 subjects
FG0015 subjects
FG0025 subjects
FG0033 subjects
Type
Comment
Reasons
Ongoing
FG0006 subjects
FG0015 subjects
FG0025 subjects
FG003
Analysis was performed on safety population which included participants who had received at least 1 infusion (partial or total) and were analyzed according to treatment received.
Type of Units Analyzed
Not provided
Arm/Group Information
ID
Title
Description
BG000
Cohort 1: Avalglucosidase Alfa 20 mg/kg
Avalglucosidase alfa, 20 mg/kg IV infusion qow for 25 weeks in the PAP, followed by same treatment from Week 26 up to Week 371 in extension treatment period (ETP).
BG001
Cohort 2: Avalglucosidase Alfa 40 mg/kg
Avalglucosidase alfa 40 mg/kg IV infusion qow for 25 weeks in the PAP, followed by same treatment from Week 26 up to Week 371 in ETP.
BG002
Cohort 3a: Avalglucosidase Alfa 40 mg//kg
After determination of the highest tolerated avalglucosidase alfa dose in Cohort 1 and Cohort 2 (after at least 5 participants in each Cohort 1 and Cohort 2 had received the 7th dose of avalglucosidase alfa or completed Week 13 with a minimum of 6 infusions), participants received avalglucosidase alfa 40 mg/kg (the highest tolerated dose) IV infusion qow for 25 weeks in PAP, followed by same treatment from Week 26 up to Week 371 in ETP.
BG003
Cohort 3b: Alglucosidase Alfa in PAP
After determination of the highest tolerated avalglucosidase alfa dose in Cohort 1 and Cohort 2 (after at least 5 participants in each Cohort 1 and Cohort 2 had received the 7th dose of avalglucosidase alfa or completed Week 13 with a minimum of 6 infusions), participants received alglucosidase alfa at their current stable dose (defined as dose [between 20 mg/kg qow and 40 mg/kg weekly as per physician] administered regularly for a minimum of 6 months immediately prior to entry in this study) IV infusion for 25 weeks in PAP. After PAP, participants received avalglucosidase alfa 40mg/kg IV infusion qow from Week 26 up to Week 371 in ETP.
BG004
Total
Total of all reporting groups
Denominators
Units
Counts
Participants
BG0006
BG0015
BG0025
BG0036
BG00422
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
BG0007.6± 3.4
BG0018.1± 4.1
BG0026.9± 2.7
BG003
Sex: Female, Male
Count of Participants
Participants
Title
Denominators
Categories
Title
Measurements
Female
BG0001
BG0012
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
PAP: Number of Participants With Treatment-emergent Adverse Events (TEAEs), Serious Treatment-emergent Adverse Events, and Adverse Event of Special Interest (AESI)
Adverse event (AE): any untoward medical occurrence in participant received study drug & did not necessarily had to have causal relationship with treatment. TEAEs: AEs developed/worsened in grade/become serious during PAP period (from the time of 1st study drug dose up to Week 25). Serious AE(SAE): any untoward medical occurrence at any dose resulted in death, was life-threatening, required inpatient hospitalization, prolongation of existing hospitalization, resulted in persistent or significant disability/incapacity, was congenital anomaly/birth defect or was medically important event. TEAEs included SAEs & non-SAEs. AESI:AE (serious/non-serious) of scientific & medical concern specific to Sponsor's product/program, for which ongoing monitoring & immediate notification by Investigator to Sponsor required.
Analysis was performed on safety population.
Posted
Count of Participants
Participants
From Baseline to Week 25
ID
Title
Description
OG000
PAP: Cohort 1: Avalglucosidase Alfa 20 mg/kg
Avalglucosidase alfa 20 mg/kg IV infusion qow for 25 weeks in PAP.
OG001
PAP: Cohort 2: Avalglucosidase Alfa 40 mg/kg
Avalglucosidase alfa 40 mg/kg IV infusion qow for 25 weeks in PAP.
OG002
PAP: Cohort 3a: Avalglucosidase Alfa 40 mg/kg
After determination of the highest tolerated avalglucosidase alfa dose in Cohort 1 and Cohort 2 (after at least 5 participants in each Cohort 1 and Cohort 2 had received the 7th dose of avalglucosidase alfa or completed Week 13 with a minimum of 6 infusions), participants received avalglucosidase alfa 40 mg/kg (the highest tolerated dose) IV infusion qow for 25 weeks in PAP.
OG003
PAP: Cohort 3b: Alglucosidase Alfa
After determination of the highest tolerated avalglucosidase alfa dose in Cohort 1 and Cohort 2 (after at least 5 participants in each Cohort 1 and Cohort 2 had received the 7th dose of avalglucosidase alfa or completed Week 13 with a minimum of 6 infusions), participants received alglucosidase alfa at their current stable dose (defined as dose [between 20 mg/kg qow and 40 mg/kg weekly as per physician] administered regularly for a minimum of 6 months immediately prior to entry in this study) IV infusion for 25 weeks in PAP.
Units
Counts
Participants
OG0006
OG0015
OG0025
OG003
Title
Denominators
Categories
Any TEAEs
Title
Measurements
OG0005
OG0015
OG0025
OG003
Primary
PAP: Number of Participants With Infusion-associated Reactions (IARs)
IARs were defined as AESIs that occurred during either the infusion or the observation period following the infusion which were deemed to be related or possibly related to the study drug. Protocol-defined IARs: An AESIs that occurred during either the infusion or the observation period following the infusion which were deemed to be related or possibly related to study drug. Algorithm-defined IARs: any TEAE meeting either 1 or 2 criteria: 1) event occurred from the start of infusion to the end of infusion plus 24 hours, and considered related to study drug, 2) If an AE time component was missing, compared AE Start date with infusion start date and infusion end date. If an AE Start date was between infusion start date and infusion end date plus one day, and it was related to study drug.
Analysis was performed on safety population.
Posted
Count of Participants
Participants
From Baseline to Week 25
ID
Title
Description
OG000
PAP: Cohort 1: Avalglucosidase Alfa 20 mg/kg
Avalglucosidase alfa 20 mg/kg IV infusion qow for 25 weeks in PAP.
OG001
PAP: Cohort 2: Avalglucosidase Alfa 40 mg/kg
Avalglucosidase alfa 40 mg/kg IV infusion qow for 25 weeks in PAP.
OG002
PAP: Cohort 3a: Avalglucosidase Alfa 40 mg/kg
Secondary
PAP: Number of Participants With Anti-drug Antibody (ADA) Response
Anti-drug antibody response was categorized as: Treatment induced ADAs: ADAs developed de novo (seroconversion) following administration of study drug. Treatment boosted ADAs: pre-existing ADAs that were boosted at least two titer steps from Baseline (i.e., 4-fold increase in titers) followed by administration of study drug.
Analysis was performed on the ADA evaluable population which included participants who had received at least 1 infusion (partial or total) and had at least one ADA sample taken post-baseline after drug administration that was appropriate for ADA testing with a reportable result.
Posted
Count of Participants
Participants
From Baseline to Week 25
ID
Title
Description
OG000
PAP: Cohort 1: Avalglucosidase Alfa 20 mg/kg
Avalglucosidase alfa 20 mg/kg IV infusion qow for 25 weeks in PAP.
OG001
PAP: Cohort 2: Avalglucosidase Alfa 40 mg/kg
Avalglucosidase alfa 40 mg/kg IV infusion qow for 25 weeks in PAP.
OG002
PAP: Cohort 3a: Avalglucosidase Alfa 40 mg/kg
After determination of the highest tolerated avalglucosidase alfa dose in Cohort 1 and Cohort 2 (after at least 5 participants in each Cohort 1 and Cohort 2 had received the 7th dose of avalglucosidase alfa or completed Week 13 with a minimum of 6 infusions), participants received avalglucosidase alfa 40 mg/kg (the highest tolerated dose) IV infusion qow for 25 weeks in PAP.
Secondary
PAP: Pharmacokinetic (PK) Parameter: Maximum Observed Plasma Concentration (Cmax) of Avalglucosidase Alfa
Cmax is the maximum observed plasma concentration.
Analysis was performed on PK population which included PAP participants who had received at least 1 infusion (partial or total) and had evaluable drug concentration data. Here, 'number analyzed' = participants with available data for each specified category. Data for this OM was not planned to be collected and analyzed for PAP: Cohort 3b: Alglucosidase Alfa arm.
Posted
Mean
Standard Deviation
nanograms per milliliter
Cohort 1: pre-dose; at end of infusion; and at 2, 4, 6, and 8 hours after end of infusion on Day 1 (Week 1) and Week 25 Cohort 2 and 3: pre-dose; at end of infusion; and at 2, 4, 6, 8, and 12 to 16 hours after end of infusion on Day 1 (Week 1) and Week 25
ID
Title
Description
OG000
PAP: Cohort 1: Avalglucosidase Alfa 20 mg/kg
Avalglucosidase alfa 20 mg/kg IV infusion qow for 25 weeks in PAP.
OG001
PAP: Cohort 2: Avalglucosidase Alfa 40 mg/kg
Avalglucosidase alfa 40 mg/kg IV infusion qow for 25 weeks in PAP.
OG002
PAP: Cohort 3a: Avalglucosidase Alfa 40 mg/kg
After determination of the highest tolerated avalglucosidase alfa dose in Cohort 1 and Cohort 2 (after at least 5 participants in each Cohort 1 and Cohort 2 had received the 7th dose of avalglucosidase alfa or completed Week 13 with a minimum of 6 infusions), participants received avalglucosidase alfa 40 mg/kg (the highest tolerated dose) IV infusion qow for 25 weeks in PAP.
Secondary
PAP: Pharmacokinetic Parameter: Time to Achieve Maximum Observed Plasma Concentration (Tmax) of Avalglucosidase Alfa
Tmax is the time to achieve maximum plasma concentration.
Analysis was performed on PK population. Here, 'number analyzed' = participants with available data for each specified category. Data for this OM was not planned to be collected and analyzed for PAP: Cohort 3b: Alglucosidase Alfa arm.
Posted
Median
Full Range
hours
Cohort 1: pre-dose; at end of infusion; and at 2, 4, 6, and 8 hours after end of infusion on Day 1 (Week 1) and Week 25 Cohort 2 and 3: pre-dose; at end of infusion; and at 2, 4, 6, 8, and 12 to 16 hours after end of infusion on Day 1 (Week 1) and Week 25
ID
Title
Description
OG000
PAP: Cohort 1: Avalglucosidase Alfa 20 mg/kg
Avalglucosidase alfa 20 mg/kg IV infusion qow for 25 weeks in PAP.
OG001
PAP: Cohort 2: Avalglucosidase Alfa 40 mg/kg
Avalglucosidase alfa 40 mg/kg IV infusion qow for 25 weeks in PAP.
OG002
PAP: Cohort 3a: Avalglucosidase Alfa 40 mg/kg
After determination of the highest tolerated avalglucosidase alfa dose in Cohort 1 and Cohort 2 (after at least 5 participants in each Cohort 1 and Cohort 2 had received the 7th dose of avalglucosidase alfa or completed Week 13 with a minimum of 6 infusions), participants received avalglucosidase alfa 40 mg/kg (the highest tolerated dose) IV infusion qow for 25 weeks in PAP.
Secondary
PAP: Pharmacokinetic Parameter: Area Under the Plasma Concentration Versus Time Curve From Time 0 to the Time of Last Quantifiable Concentration (AUC0-last) of Avalglucosidase Alfa
AUC0-last is the area under the plasma concentration versus time curve from time 0 to the time of last quantifiable concentration.
Analysis was performed on PK population. Here, 'number analyzed' = participants with available data for each specified category. Data for this OM was not planned to be collected and analyzed for PAP: Cohort 3b: Alglucosidase Alfa arm.
Posted
Mean
Standard Deviation
nanograms*hours per milliliter
Cohort 1: pre-dose; at end of infusion; and at 2, 4, 6, and 8 hours after end of infusion on Day 1 (Week 1) and Week 25 Cohort 2 & 3: pre-dose; at end of infusion; and at 2, 4, 6, 8, and 12 to 16 hours after end of infusion on Day 1 (Week 1) and Week 25
ID
Title
Description
OG000
PAP: Cohort 1: Avalglucosidase Alfa 20 mg/kg
Avalglucosidase alfa 20 mg/kg IV infusion qow for 25 weeks in PAP.
OG001
PAP: Cohort 2: Avalglucosidase Alfa 40 mg/kg
Avalglucosidase alfa 40 mg/kg IV infusion qow for 25 weeks in PAP.
OG002
PAP: Cohort 3a: Avalglucosidase Alfa 40 mg/kg
After determination of the highest tolerated avalglucosidase alfa dose in Cohort 1 and Cohort 2 (after at least 5 participants in each Cohort 1 and Cohort 2 had received the 7th dose of avalglucosidase alfa or completed Week 13 with a minimum of 6 infusions), participants received avalglucosidase alfa 40 mg/kg (the highest tolerated dose) IV infusion qow for 25 weeks in PAP.
Secondary
PAP: Pharmacokinetic Parameter: Terminal Half-life (t1/2) of Avalglucosidase Alfa
t1/2 is the time required for the plasma concentration of a drug to decrease by half of its initial concentration.
Analysis was performed on PK population. Here, 'number analyzed' = participants with available data for each specified category. Data for this OM was not planned to be collected and analyzed for PAP: Cohort 3b: Alglucosidase Alfa arm.
Posted
Mean
Standard Deviation
hours
Cohort 1: pre-dose; at end of infusion; and at 2, 4, 6, and 8 hours after end of infusion on Day 1 (Week 1) and Week 25 Cohort 2 & 3: pre-dose; at end of infusion; and at 2, 4, 6, 8, and 12 to 16 hours after end of infusion on Day 1 (Week 1) and Week 25
ID
Title
Description
OG000
PAP: Cohort 1: Avalglucosidase Alfa 20 mg/kg
Avalglucosidase alfa 20 mg/kg IV infusion qow for 25 weeks in PAP.
OG001
PAP: Cohort 2: Avalglucosidase Alfa 40 mg/kg
Avalglucosidase alfa 40 mg/kg IV infusion qow for 25 weeks in PAP.
OG002
PAP: Cohort 3a: Avalglucosidase Alfa 40 mg/kg
After determination of the highest tolerated avalglucosidase alfa dose in Cohort 1 and Cohort 2 (after at least 5 participants in each Cohort 1 and Cohort 2 had received the 7th dose of avalglucosidase alfa or completed Week 13 with a minimum of 6 infusions), participants received avalglucosidase alfa 40 mg/kg (the highest tolerated dose) IV infusion qow for 25 weeks in PAP.
Secondary
PAP: Pharmacokinetic Parameter: Clearance (CL) of Avalglucosidase Alfa
CL is defined as a quantitative measure of the rate at which a drug substance is removed from the body.
Analysis was performed on PK population. Here, 'number analyzed' = participants with available data for each specified category. Data for this OM was not planned to be collected and analyzed for PAP: Cohort 3b: Alglucosidase Alfa arm.
Posted
Mean
Standard Deviation
milliliters per hours
Cohort 1: pre-dose; at end of infusion; and at 2, 4, 6, and 8 hours after end of infusion on Day 1 (Week 1) and Week 25 Cohort 2 and 3: pre-dose; at end of infusion; and at 2, 4, 6, 8, and 12 to 16 hours after end of infusion on Day 1 (Week 1) and Week 25
ID
Title
Description
OG000
PAP: Cohort 1: Avalglucosidase Alfa 20 mg/kg
Avalglucosidase alfa 20 mg/kg IV infusion qow for 25 weeks in PAP.
OG001
PAP: Cohort 2: Avalglucosidase Alfa 40 mg/kg
Avalglucosidase alfa 40 mg/kg IV infusion qow for 25 weeks in PAP.
OG002
PAP: Cohort 3a: Avalglucosidase Alfa 40 mg/kg
After determination of the highest tolerated avalglucosidase alfa dose in Cohort 1 and Cohort 2 (after at least 5 participants in each Cohort 1 and Cohort 2 had received the 7th dose of avalglucosidase alfa or completed Week 13 with a minimum of 6 infusions), participants received avalglucosidase alfa 40 mg/kg (the highest tolerated dose) IV infusion qow for 25 weeks in PAP.
Secondary
PAP: Pharmacokinetic Parameter: Volume of Distribution at Steady State (Vss) of Avalglucosidase Alfa
Steady state volume of distribution (Vss) is defined as the theoretical volume in which the total amount of drug would need to be uniformly distributed to produce the desired plasma concentration of a drug.
Analysis was performed on PK population. Here, 'number analyzed' = participants with available data for each specified category. Data for this OM was not planned to be collected and analyzed for PAP: Cohort 3b: Alglucosidase Alfa arm.
Posted
Mean
Standard Deviation
milliliters
Cohort 1: pre-dose; at end of infusion; and at 2, 4, 6, and 8 hours after end of infusion on Day 1 (Week 1) and Week 25 Cohort 2 & 3: pre-dose; at end of infusion; and at 2, 4, 6, 8, and 12 to 16 hours after end of infusion on Day 1 (Week 1) and Week 25
ID
Title
Description
OG000
PAP: Cohort 1: Avalglucosidase Alfa 20 mg/kg
Avalglucosidase alfa 20 mg/kg IV infusion qow for 25 weeks in PAP.
OG001
PAP: Cohort 2: Avalglucosidase Alfa 40 mg/kg
Avalglucosidase alfa 40 mg/kg IV infusion qow for 25 weeks in PAP.
OG002
PAP: Cohort 3a: Avalglucosidase Alfa 40 mg/kg
After determination of the highest tolerated avalglucosidase alfa dose in Cohort 1 and Cohort 2 (after at least 5 participants in each Cohort 1 and Cohort 2 had received the 7th dose of avalglucosidase alfa or completed Week 13 with a minimum of 6 infusions), participants received avalglucosidase alfa 40 mg/kg (the highest tolerated dose) IV infusion qow for 25 weeks in PAP.
Secondary
PAP: Change From Baseline in Gross Motor Function Measure-88 (GMFM-88) Test Scores at Week 25
GMFM-88 was developed specifically to detect quantitative changes in gross motor function. The GMFM-88 consisted of 88 items organized into 5 dimensions; lying and rolling (17 items); sitting (20 items); crawling and kneeling (14 items); standing (13 items) and walking, running and jumping (24 items). Each item was scored on a 4-point Likert scale with scores range: 0= cannot do; 1 = initiates less then [<] 10 percentage [%] of the task; 2 = partially completes [10% to <100% of the task] and 3 = task completion. The score for each dimension was expressed as a % of the maximum score for that dimension.Total percentage score was obtained by adding the percentage scores for each dimension and dividing the sum by the total number of dimensions. Total scores ranged from 0% to 100%; where higher scores indicated better motor functions.
Analysis was performed on safety population. Here, 'overall number of participants analyzed' = participants with available data for this OM.
Posted
Mean
Standard Deviation
percentage of total score
Baseline, Week 25
ID
Title
Description
OG000
PAP: Cohort 1: Avalglucosidase Alfa 20 mg/kg
Avalglucosidase alfa 20 mg/kg IV infusion qow for 25 weeks in PAP.
OG001
PAP: Cohort 2: Avalglucosidase Alfa 40 mg/kg
Avalglucosidase alfa 40 mg/kg IV infusion qow for 25 weeks in PAP.
Secondary
PAP: Number of Participants in Gross Motor Function Classification System-Expanded and Revised (GMFCS-E and R) Scores at Baseline and Week 25
GMFCS-E&R was a 5 level classification system for specific age ranges; observations were performed on 5 levels based on self-initiated movement, with emphasis on sitting, transfers, and mobility: Level I (walks without limitations), Level II (walks with limitations), Level III (walks using a hand-held mobility device), Level IV (self-mobility with limitations; may use powered mobility) and level V (transported in a manual wheel chair) (I to V). The distinctions between levels were based on functional limitations, the need for assistive mobility devices, and to a much lesser extent, quality of movement, and were designed to be meaningful in daily life. The lower level represented good motor functioning and higher level represented low motor functioning. Number of participants in each level of classification at Baseline and Week 25 were reported.
Analysis was performed on safety population.
Posted
Count of Participants
Participants
Baseline, Week 25
ID
Title
Description
OG000
PAP: Cohort 1: Avalglucosidase Alfa 20 mg/kg
Avalglucosidase alfa 20 mg/kg IV infusion qow for 25 weeks in PAP.
OG001
PAP: Cohort 2: Avalglucosidase Alfa 40 mg/kg
Avalglucosidase alfa 40 mg/kg IV infusion qow for 25 weeks in PAP.
Secondary
PAP: Change From Baseline in Pompe Pediatric Evaluation of Disability Inventory (Pompe-PEDI) Functional Skills Scale: Mobility Domain Test Score-Scaled Score at Week 25
Pompe-PEDI: disease specific version to assess functional capabilities and performance in children with Pompe disease from 2 months through adolescence. It comprised of Functional Skills Scale and Caregiver Assistance Scale; both scales had 3 domains: Self Care, Mobility, and Social Function. Mobility domain was used to measure change in mobility due to changes in muscle strength; consisted of 160 mobility items for participant/legal guardian. The total number of mobility items the child was capable of, was converted to a scaled score with a range of 0 to 100, where scores near "0" indicated low capability and scores near "100" indicated high capability), where higher score was indicative of greater functional ability. Scaled scores were used to interpret individual function and progress over time.
Analysis was performed on safety population. Here, 'overall number of participants analyzed' = participants with available data for this OM.
Posted
Mean
Standard Deviation
score on a scale
Baseline, Week 25
ID
Title
Description
OG000
PAP: Cohort 1: Avalglucosidase Alfa 20 mg/kg
Avalglucosidase alfa 20 mg/kg IV infusion qow for 25 weeks in PAP.
OG001
PAP: Cohort 2: Avalglucosidase Alfa 40 mg/kg
Avalglucosidase alfa 40 mg/kg IV infusion qow for 25 weeks in PAP.
Secondary
PAP: Change From Baseline in Quick Motor Function Test (QMFT) Scores at Week 25
QMFT was observer administered test comprised of 16 items specifically difficult for participants with Pompe disease. Each item was scored on 5-point ordinal scale ranged from 0 to 4 (higher score indicated better outcome). Total QMFT score was obtained by adding the scores of all items and ranged from 0 (unable to perform motor function tests) to 64 (normal muscle function), where higher score indicated better outcome/greater motor function.
Analysis was performed on safety population. Here, 'overall number of participants analyzed' = participants with available data for this OM.
Posted
Mean
Standard Deviation
score on a scale
Baseline, Week 25
ID
Title
Description
OG000
PAP: Cohort 1: Avalglucosidase Alfa 20 mg/kg
Avalglucosidase alfa 20 mg/kg IV infusion qow for 25 weeks in PAP.
OG001
PAP: Cohort 2: Avalglucosidase Alfa 40 mg/kg
Avalglucosidase alfa 40 mg/kg IV infusion qow for 25 weeks in PAP.
OG002
PAP: Cohort 3a: Avalglucosidase Alfa 40 mg/kg
After determination of the highest tolerated avalglucosidase alfa dose in Cohort 1 and Cohort 2 (after at least 5 participants in each Cohort 1 and Cohort 2 had received the 7th dose of avalglucosidase alfa or completed Week 13 with a minimum of 6 infusions), participants received avalglucosidase alfa 40 mg/kg (the highest tolerated dose) IV infusion qow for 25 weeks in PAP.
Secondary
PAP: Echo-Left Ventricular Mass Z-Score (LVM Z-score) M-mode at Baseline, Week 25, and Change From Baseline at Week 25
Cardiac function was evaluated using LVM Z-score as assessed by echocardiogram in M-mode. Z-Scores indicated the number of standard deviations (SD) from the mean in a normal distribution. The normal range is -2 to 2 and greater than 2 may indicate left ventricular hypertrophy. A negative change from Baseline indicated a decrease and positive change from Baseline indicated an increase in LVM Z-score. In this OM, absolute scores at Baseline and Week 25 along with Change from Baseline at Week 25 in LVM Z-score were reported.
Analysis was performed on safety population. Here, 'number analyzed' = participants with available data for each specified category.
Posted
Mean
Standard Deviation
Z-score
Baseline, Week 25
ID
Title
Description
OG000
PAP: Cohort 1: Avalglucosidase Alfa 20 mg/kg
Avalglucosidase alfa 20 mg/kg IV infusion qow for 25 weeks in PAP.
OG001
PAP: Cohort 2: Avalglucosidase Alfa 40 mg/kg
Avalglucosidase alfa 40 mg/kg IV infusion qow for 25 weeks in PAP.
OG002
PAP: Cohort 3a: Avalglucosidase Alfa 40 mg/kg
After determination of the highest tolerated avalglucosidase alfa dose in Cohort 1 and Cohort 2 (after at least 5 participants in each Cohort 1 and Cohort 2 had received the 7th dose of avalglucosidase alfa or completed Week 13 with a minimum of 6 infusions), participants received avalglucosidase alfa 40 mg/kg (the highest tolerated dose) IV infusion qow for 25 weeks in PAP.
Secondary
PAP: Change From Baseline in Eyelid Position Measurements: Interpalpebral Fissure Distance (IPFD) - Left Non-Flash and Right Non-Flash at Week 25
IPFD is the widest vertical distance (in millimeters) between the upper eyelid and the lower eyelid when the participant is looking in "primary gaze" (i.e. normal gaze when looking straight forward). Images were taken while the participants was wearing a pair of empty eyeglass frames with millimeters rulers attached as a standardized tool to measure eyelid position without camera flash. A negative change from Baseline indicated a decrease and positive change from Baseline indicated an increase in measured distance.
Analysis was performed on safety population.
Posted
Mean
Standard Deviation
millimeters
Baseline, Week 25
ID
Title
Description
OG000
PAP: Cohort 1: Avalglucosidase Alfa 20 mg/kg
Avalglucosidase alfa 20 mg/kg IV infusion qow for 25 weeks in PAP.
OG001
PAP: Cohort 2: Avalglucosidase Alfa 40 mg/kg
Avalglucosidase alfa 40 mg/kg IV infusion qow for 25 weeks in PAP.
OG002
PAP: Cohort 3a: Avalglucosidase Alfa 40 mg/kg
After determination of the highest tolerated avalglucosidase alfa dose in Cohort 1 and Cohort 2 (after at least 5 participants in each Cohort 1 and Cohort 2 had received the 7th dose of avalglucosidase alfa or completed Week 13 with a minimum of 6 infusions), participants received avalglucosidase alfa 40 mg/kg (the highest tolerated dose) IV infusion qow for 25 weeks in PAP.
Secondary
PAP: Change From Baseline in Eyelid Position Measurements: Left and Right Margin Reflex Distance (MRD) at Week 25
The MRD is the vertical distance (in millimeters) between the light reflex and the upper eyelid when the participant was looking in "primary gaze" while fixating on a light source. Images were taken while the participants was wearing a pair of empty eyeglass frames with millimeters rulers attached as a standardized tool to measure eyelid position. A negative change from Baseline indicated a decrease and positive change from Baseline indicated an increase in measured distance.
Analysis was performed on safety population. Here, 'number analyzed' = participants with available data for each specified category.
Posted
Mean
Standard Deviation
millimeters
Baseline, Week 25
ID
Title
Description
OG000
PAP: Cohort 1: Avalglucosidase Alfa 20 mg/kg
Avalglucosidase alfa 20 mg/kg IV infusion qow for 25 weeks in PAP.
OG001
PAP: Cohort 2: Avalglucosidase Alfa 40 mg/kg
Avalglucosidase alfa 40 mg/kg IV infusion qow for 25 weeks in PAP.
OG002
PAP: Cohort 3a: Avalglucosidase Alfa 40 mg/kg
After determination of the highest tolerated avalglucosidase alfa dose in Cohort 1 and Cohort 2 (after at least 5 participants in each Cohort 1 and Cohort 2 had received the 7th dose of avalglucosidase alfa or completed Week 13 with a minimum of 6 infusions), participants received avalglucosidase alfa 40 mg/kg (the highest tolerated dose) IV infusion qow for 25 weeks in PAP.
Secondary
PAP: Change From Baseline in Eyelid Position Measurements Assessed by Margin Pupil Distance (MPD) - Left Non-Flash and Right Non-Flash at Week 25
The MPD is the vertical distance (in millimeters) between the center of the pupil and the upper eyelid margin. Images were taken while the participants was wearing a pair of empty eyeglass frames with millimeters rulers attached as a standardized tool to measure eyelid position without camera flash. A negative change from Baseline indicated a decrease and positive change from Baseline indicated an increase in measured distance.
Analysis was performed on safety population. Here, 'number analyzed' = participants with available data for each specified category.
Posted
Mean
Standard Deviation
millimeters
Baseline, Week 25
ID
Title
Description
OG000
PAP: Cohort 1: Avalglucosidase Alfa 20 mg/kg
Avalglucosidase alfa 20 mg/kg IV infusion qow for 25 weeks in PAP.
OG001
PAP: Cohort 2: Avalglucosidase Alfa 40 mg/kg
Avalglucosidase alfa 40 mg/kg IV infusion qow for 25 weeks in PAP.
OG002
PAP: Cohort 3a: Avalglucosidase Alfa 40 mg/kg
After determination of the highest tolerated avalglucosidase alfa dose in Cohort 1 and Cohort 2 (after at least 5 participants in each Cohort 1 and Cohort 2 had received the 7th dose of avalglucosidase alfa or completed Week 13 with a minimum of 6 infusions), participants received avalglucosidase alfa 40 mg/kg (the highest tolerated dose) IV infusion qow for 25 weeks in PAP.
Secondary
PAP: Change From Baseline in Creatine Kinase Value at Week 25
Change from Baseline in Creatine kinase value (to assess muscle damage) at Week 25 were reported in this OM.
Analysis was performed on safety population.
Posted
Mean
Standard Deviation
international units per liters
Baseline, Week 25
ID
Title
Description
OG000
PAP: Cohort 1: Avalglucosidase Alfa 20 mg/kg
Avalglucosidase alfa 20 mg/kg IV infusion qow for 25 weeks in PAP.
OG001
PAP: Cohort 2: Avalglucosidase Alfa 40 mg/kg
Avalglucosidase alfa 40 mg/kg IV infusion qow for 25 weeks in PAP.
OG002
PAP: Cohort 3a: Avalglucosidase Alfa 40 mg/kg
After determination of the highest tolerated avalglucosidase alfa dose in Cohort 1 and Cohort 2 (after at least 5 participants in each Cohort 1 and Cohort 2 had received the 7th dose of avalglucosidase alfa or completed Week 13 with a minimum of 6 infusions), participants received avalglucosidase alfa 40 mg/kg (the highest tolerated dose) IV infusion qow for 25 weeks in PAP.
OG003
PAP: Cohort 3b: Alglucosidase Alfa
Secondary
ETP: Number of Participants With Treatment Emergent Adverse Events, Serious Treatment Emergent Adverse Events, and Adverse Event of Special Interest
Data for this outcome measure will be reported at the time of anticipated last participant last visit results posting (August 2028).
Not Posted
Aug 2028
Up to 9 years and 4 months
Participants
Secondary
ETP: Number of Participants With Infusion-associated Reactions
Data for this outcome measure will be reported at the time of anticipated last participant last visit results posting (August 2028).
Not Posted
Aug 2028
Up to 9 years and 4 months
Participants
Time Frame
From Day 1 to Week 25 in PAP and from Week 26 to 102 in ETP. Safety data were planned to be collected and analyzed for pooled population of participants who received avalglucosidase alfa 40 mg/kg (Cohorts 2 and 3a of PAP) in ETP. AE data collection is still ongoing and will be updated upon study completion.
Description
TEAEs: AEs that developed/worsened in grade/become serious during treatment epoch (for PAP:time just prior to 1st study drug dose in ETP or 4 weeks after last infusion for those who didn't get into ETP or 2 weeks after last infusion in PAP if participant enrolled in another study/received ERT) (for ETP:time from 1st administration of study drug in ETP to last administration of study drug +4 weeks /2 weeks if participant got enrolled in another study/received ERT). Analyzed 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
PAP: Cohort 1: Avalglucosidase Alfa 20 mg/kg
Avalglucosidase alfa 20 mg/kg IV infusion qow for 25 weeks in PAP.
0
6
1
6
5
6
EG001
PAP: Cohort 2: Avalglucosidase Alfa 40 mg/kg
Avalglucosidase alfa 40 mg/kg IV infusion qow for 25 weeks in PAP.
0
5
3
5
5
5
EG002
PAP: Cohort 3a: Avalglucosidase Alfa 40 mg/kg
After determination of the highest tolerated avalglucosidase alfa dose in Cohort 1 and Cohort 2 (after at least 5 participants in each Cohort 1 and Cohort 2 had received the 7th dose of avalglucosidase alfa or completed Week 13 with a minimum of 6 infusions), participants received avalglucosidase alfa 40 mg/kg (the highest tolerated dose) IV infusion qow for 25 weeks in PAP.
0
5
0
5
5
5
EG003
PAP: Cohort 3b: Alglucosidase Alfa
After determination of the highest tolerated avalglucosidase alfa dose in Cohort 1 and Cohort 2 (after at least 5 participants in each Cohort 1 and Cohort 2 had received the 7th dose of avalglucosidase alfa or completed Week 13 with a minimum of 6 infusions), participants received alglucosidase alfa at their current stable dose (defined as dose [between 20 mg/kg qow and 40 mg/kg weekly as per physician] administered regularly for a minimum of 6 months immediately prior to entry in this study) IV infusion for 25 weeks in PAP.
0
6
2
6
4
6
EG004
ETP: Avalglucosidase Alfa 20 mg/kg
Included all participants of Cohort 1 who received avalglucosidase alfa 20 mg/kg IV infusion qow for 25 weeks in PAP, followed by same treatment from Week 26 up to Week 371 in ETP.
0
6
4
6
6
6
EG005
Pooled Arm ETP: Avalglucosidase Alfa 40 mg/kg
Pooled arm included all participants of Cohorts 2 and 3a who received avalglucosidase alfa 40 mg/kg IV infusion qow for 25 weeks in PAP, followed by same treatment from Week 26 up to Week 371 in ETP.
0
10
2
10
7
10
EG006
Alglucosidase Alfa in PAP Then Avalglucosidase Alfa 40 mg/kg in ETP
Included all participants of Cohort 3b who received alglucosidase alfa at their current stable dose (defined as dose [between 20 mg/kg qow and 40 mg/kg weekly as per physician] administered regularly for a minimum of 6 months immediately prior to entry in this study) IV infusion for 25 weeks in the PAP and after PAP, switched to receive avalglucosidase alfa 40 mg/kg IV infusion qow from Week 26 up to Week 371 in ETP.
0
3
1
3
3
3
Serious Adverse Events
Term
Organ System
Source Vocabulary
Assessment Type
Notes
Statistical Information
Atrial Thrombosis
Cardiac disorders
MedDRA 22.0
Systematic Assessment
EG0000 events0 affected6 at risk
EG0010 events0 affected5 at risk
EG0020 events0 affected5 at risk
EG0030 events0 affected6 at risk
EG0041 events1 affected6 at risk
EG0050 events0 affected10 at risk
EG0060 events0 affected3 at risk
Tympanic Membrane Perforation
Ear and labyrinth disorders
MedDRA 22.0
Systematic Assessment
EG0000 events0 affected6 at risk
EG0010 events0 affected5 at risk
EG0020 events0 affected5 at risk
EG003
Eyelid Ptosis
Eye disorders
MedDRA 22.0
Systematic Assessment
EG0000 events0 affected6 at risk
EG0013 events2 affected5 at risk
EG0020 events0 affected5 at risk
EG003
Strabismus
Eye disorders
MedDRA 22.0
Systematic Assessment
EG0000 events0 affected6 at risk
EG0010 events0 affected5 at risk
EG0020 events0 affected5 at risk
EG003
Pyrexia
General disorders
MedDRA 22.0
Systematic Assessment
EG0000 events0 affected6 at risk
EG0013 events1 affected5 at risk
EG0020 events0 affected5 at risk
EG003
Gastroenteritis
Infections and infestations
MedDRA 22.0
Systematic Assessment
EG0000 events0 affected6 at risk
EG0010 events0 affected5 at risk
EG0020 events0 affected5 at risk
EG003
Influenza
Infections and infestations
MedDRA 22.0
Systematic Assessment
EG0000 events0 affected6 at risk
EG0010 events0 affected5 at risk
EG0020 events0 affected5 at risk
EG003
Lung Infection Pseudomonal
Infections and infestations
MedDRA 22.0
Systematic Assessment
EG0000 events0 affected6 at risk
EG0010 events0 affected5 at risk
EG0020 events0 affected5 at risk
EG003
Otitis Media
Infections and infestations
MedDRA 22.0
Systematic Assessment
EG0000 events0 affected6 at risk
EG0010 events0 affected5 at risk
EG0020 events0 affected5 at risk
EG003
Pneumonia
Infections and infestations
MedDRA 22.0
Systematic Assessment
EG0000 events0 affected6 at risk
EG0010 events0 affected5 at risk
EG0020 events0 affected5 at risk
EG003
Respiratory Tract Infection Viral
Infections and infestations
MedDRA 22.0
Systematic Assessment
EG0002 events1 affected6 at risk
EG0010 events0 affected5 at risk
EG0020 events0 affected5 at risk
EG003
Urinary Tract Infection
Infections and infestations
MedDRA 22.0
Systematic Assessment
EG0000 events0 affected6 at risk
EG0010 events0 affected5 at risk
EG0020 events0 affected5 at risk
EG003
Femur Fracture
Injury, poisoning and procedural complications
MedDRA 22.0
Systematic Assessment
EG0000 events0 affected6 at risk
EG0010 events0 affected5 at risk
EG0020 events0 affected5 at risk
EG003
Joint Dislocation
Injury, poisoning and procedural complications
MedDRA 22.0
Systematic Assessment
EG0000 events0 affected6 at risk
EG0010 events0 affected5 at risk
EG0020 events0 affected5 at risk
EG003
Post Procedural Haemorrhage
Injury, poisoning and procedural complications
MedDRA 22.0
Systematic Assessment
EG0000 events0 affected6 at risk
EG0010 events0 affected5 at risk
EG0020 events0 affected5 at risk
EG003
Device Malfunction
Product Issues
MedDRA 22.0
Systematic Assessment
EG0000 events0 affected6 at risk
EG0010 events0 affected5 at risk
EG0020 events0 affected5 at risk
EG003
Adenoidal Hypertrophy
Respiratory, thoracic and mediastinal disorders
MedDRA 22.0
Systematic Assessment
EG0000 events0 affected6 at risk
EG0010 events0 affected5 at risk
EG0020 events0 affected5 at risk
EG003
Lung Consolidation
Respiratory, thoracic and mediastinal disorders
MedDRA 22.0
Systematic Assessment
EG0000 events0 affected6 at risk
EG0010 events0 affected5 at risk
EG0020 events0 affected5 at risk
EG003
Respiratory Distress
Respiratory, thoracic and mediastinal disorders
MedDRA 22.0
Systematic Assessment
EG0000 events0 affected6 at risk
EG0011 events1 affected5 at risk
EG0020 events0 affected5 at risk
EG003
Tonsillar Hypertrophy
Respiratory, thoracic and mediastinal disorders
MedDRA 22.0
Systematic Assessment
EG0000 events0 affected6 at risk
EG0010 events0 affected5 at risk
EG0020 events0 affected5 at risk
EG003
Other Adverse Events
Term
Organ System
Source Vocabulary
Assessment Type
Notes
Statistical Information
Lymph Node Pain
Blood and lymphatic system disorders
MedDRA 22.0
Systematic Assessment
EG0000 events0 affected6 at risk
EG0010 events0 affected5 at risk
EG0020 events0 affected5 at risk
EG0030 events0 affected6 at risk
EG0041 events1 affected6 at risk
EG0050 events0 affected10 at risk
EG0060 events0 affected3 at risk
Neutropenia
Blood and lymphatic system disorders
MedDRA 22.0
Systematic Assessment
EG0000 events0 affected6 at risk
EG0011 events1 affected5 at risk
EG0020 events0 affected5 at risk
EG003
Pericardial Effusion
Cardiac disorders
MedDRA 22.0
Systematic Assessment
EG0001 events1 affected6 at risk
EG0010 events0 affected5 at risk
EG0020 events0 affected5 at risk
EG003
Cerumen Impaction
Ear and labyrinth disorders
MedDRA 22.0
Systematic Assessment
EG0000 events0 affected6 at risk
EG0010 events0 affected5 at risk
EG0020 events0 affected5 at risk
EG003
Deafness
Ear and labyrinth disorders
MedDRA 22.0
Systematic Assessment
EG0001 events1 affected6 at risk
EG0010 events0 affected5 at risk
EG0020 events0 affected5 at risk
EG003
Ear Pain
Ear and labyrinth disorders
MedDRA 22.0
Systematic Assessment
EG0000 events0 affected6 at risk
EG0011 events1 affected5 at risk
EG0020 events0 affected5 at risk
EG003
Excessive Cerumen Production
Ear and labyrinth disorders
MedDRA 22.0
Systematic Assessment
EG0001 events1 affected6 at risk
EG0010 events0 affected5 at risk
EG0020 events0 affected5 at risk
EG003
Middle Ear Effusion
Ear and labyrinth disorders
MedDRA 22.0
Systematic Assessment
EG0000 events0 affected6 at risk
EG0010 events0 affected5 at risk
EG0021 events1 affected5 at risk
EG003
Otorrhoea
Ear and labyrinth disorders
MedDRA 22.0
Systematic Assessment
EG0002 events1 affected6 at risk
EG0010 events0 affected5 at risk
EG0020 events0 affected5 at risk
EG003
Conjunctivochalasis
Eye disorders
MedDRA 22.0
Systematic Assessment
EG0000 events0 affected6 at risk
EG0011 events1 affected5 at risk
EG0020 events0 affected5 at risk
EG003
Eye Discharge
Eye disorders
MedDRA 22.0
Systematic Assessment
EG0000 events0 affected6 at risk
EG0010 events0 affected5 at risk
EG0021 events1 affected5 at risk
EG003
Eye Irritation
Eye disorders
MedDRA 22.0
Systematic Assessment
EG0000 events0 affected6 at risk
EG0010 events0 affected5 at risk
EG0022 events2 affected5 at risk
EG003
Keratopathy
Eye disorders
MedDRA 22.0
Systematic Assessment
EG0000 events0 affected6 at risk
EG0010 events0 affected5 at risk
EG0020 events0 affected5 at risk
EG003
Lacrimation Increased
Eye disorders
MedDRA 22.0
Systematic Assessment
EG0000 events0 affected6 at risk
EG0010 events0 affected5 at risk
EG0021 events1 affected5 at risk
EG003
Abdominal Hernia
Gastrointestinal disorders
MedDRA 22.0
Systematic Assessment
EG0000 events0 affected6 at risk
EG0010 events0 affected5 at risk
EG0020 events0 affected5 at risk
EG003
Abdominal Pain
Gastrointestinal disorders
MedDRA 22.0
Systematic Assessment
EG0000 events0 affected6 at risk
EG0011 events1 affected5 at risk
EG0021 events1 affected5 at risk
EG003
Aphthous Ulcer
Gastrointestinal disorders
MedDRA 22.0
Systematic Assessment
EG0000 events0 affected6 at risk
EG0010 events0 affected5 at risk
EG0021 events1 affected5 at risk
EG003
Constipation
Gastrointestinal disorders
MedDRA 22.0
Systematic Assessment
EG0000 events0 affected6 at risk
EG0011 events1 affected5 at risk
EG0020 events0 affected5 at risk
EG003
Dental Caries
Gastrointestinal disorders
MedDRA 22.0
Systematic Assessment
EG0000 events0 affected6 at risk
EG0010 events0 affected5 at risk
EG0020 events0 affected5 at risk
EG003
Diarrhoea
Gastrointestinal disorders
MedDRA 22.0
Systematic Assessment
EG0000 events0 affected6 at risk
EG0011 events1 affected5 at risk
EG0022 events2 affected5 at risk
EG003
Dysphagia
Gastrointestinal disorders
MedDRA 22.0
Systematic Assessment
EG0000 events0 affected6 at risk
EG0010 events0 affected5 at risk
EG0020 events0 affected5 at risk
EG003
Faeces Soft
Gastrointestinal disorders
MedDRA 22.0
Systematic Assessment
EG0000 events0 affected6 at risk
EG0010 events0 affected5 at risk
EG0020 events0 affected5 at risk
EG003
Loose Tooth
Gastrointestinal disorders
MedDRA 22.0
Systematic Assessment
EG0001 events1 affected6 at risk
EG0010 events0 affected5 at risk
EG0020 events0 affected5 at risk
EG003
Mucous Stools
Gastrointestinal disorders
MedDRA 22.0
Systematic Assessment
EG0000 events0 affected6 at risk
EG0010 events0 affected5 at risk
EG0020 events0 affected5 at risk
EG003
Nausea
Gastrointestinal disorders
MedDRA 22.0
Systematic Assessment
EG0000 events0 affected6 at risk
EG0010 events0 affected5 at risk
EG0021 events1 affected5 at risk
EG003
Regurgitation
Gastrointestinal disorders
MedDRA 22.0
Systematic Assessment
EG0000 events0 affected6 at risk
EG0011 events1 affected5 at risk
EG0020 events0 affected5 at risk
EG003
Teething
Gastrointestinal disorders
MedDRA 22.0
Systematic Assessment
EG0000 events0 affected6 at risk
EG0010 events0 affected5 at risk
EG0020 events0 affected5 at risk
EG003
Toothache
Gastrointestinal disorders
MedDRA 22.0
Systematic Assessment
EG0001 events1 affected6 at risk
EG0015 events1 affected5 at risk
EG0020 events0 affected5 at risk
EG003
Vomiting
Gastrointestinal disorders
MedDRA 22.0
Systematic Assessment
EG0000 events0 affected6 at risk
EG0013 events1 affected5 at risk
EG0022 events2 affected5 at risk
EG003
Asthenia
General disorders
MedDRA 22.0
Systematic Assessment
EG0000 events0 affected6 at risk
EG0010 events0 affected5 at risk
EG0021 events1 affected5 at risk
EG003
Catheter Site Oedema
General disorders
MedDRA 22.0
Systematic Assessment
EG0000 events0 affected6 at risk
EG0012 events1 affected5 at risk
EG0020 events0 affected5 at risk
EG003
Device Related Thrombosis
General disorders
MedDRA 22.0
Systematic Assessment
EG0000 events0 affected6 at risk
EG0010 events0 affected5 at risk
EG0020 events0 affected5 at risk
EG003
Fatigue
General disorders
MedDRA 22.0
Systematic Assessment
EG0000 events0 affected6 at risk
EG0010 events0 affected5 at risk
EG0021 events1 affected5 at risk
EG003
Feeling Hot
General disorders
MedDRA 22.0
Systematic Assessment
EG0000 events0 affected6 at risk
EG0010 events0 affected5 at risk
EG0021 events1 affected5 at risk
EG003
Infusion Site Swelling
General disorders
MedDRA 22.0
Systematic Assessment
EG0000 events0 affected6 at risk
EG0011 events1 affected5 at risk
EG0020 events0 affected5 at risk
EG003
Injection Site Pain
General disorders
MedDRA 22.0
Systematic Assessment
EG0001 events1 affected6 at risk
EG0010 events0 affected5 at risk
EG0020 events0 affected5 at risk
EG003
Malaise
General disorders
MedDRA 22.0
Systematic Assessment
EG0000 events0 affected6 at risk
EG0010 events0 affected5 at risk
EG0021 events1 affected5 at risk
EG003
Oedema Peripheral
General disorders
MedDRA 22.0
Systematic Assessment
EG0001 events1 affected6 at risk
EG0010 events0 affected5 at risk
EG0020 events0 affected5 at risk
EG003
Pyrexia
General disorders
MedDRA 22.0
Systematic Assessment
EG0004 events2 affected6 at risk
EG0014 events1 affected5 at risk
EG0022 events2 affected5 at risk
EG003
Bronchitis
Infections and infestations
MedDRA 22.0
Systematic Assessment
EG0001 events1 affected6 at risk
EG0010 events0 affected5 at risk
EG0020 events0 affected5 at risk
EG003
Bronchitis Viral
Infections and infestations
MedDRA 22.0
Systematic Assessment
EG0001 events1 affected6 at risk
EG0010 events0 affected5 at risk
EG0020 events0 affected5 at risk
EG003
Conjunctivitis
Infections and infestations
MedDRA 22.0
Systematic Assessment
EG0002 events1 affected6 at risk
EG0010 events0 affected5 at risk
EG0020 events0 affected5 at risk
EG003
Enterovirus Infection
Infections and infestations
MedDRA 22.0
Systematic Assessment
EG0001 events1 affected6 at risk
EG0010 events0 affected5 at risk
EG0020 events0 affected5 at risk
EG003
Fungal Skin Infection
Infections and infestations
MedDRA 22.0
Systematic Assessment
EG0000 events0 affected6 at risk
EG0010 events0 affected5 at risk
EG0020 events0 affected5 at risk
EG003
Gastroenteritis
Infections and infestations
MedDRA 22.0
Systematic Assessment
EG0000 events0 affected6 at risk
EG0011 events1 affected5 at risk
EG0020 events0 affected5 at risk
EG003
Hand-Foot-And-Mouth Disease
Infections and infestations
MedDRA 22.0
Systematic Assessment
EG0000 events0 affected6 at risk
EG0010 events0 affected5 at risk
EG0020 events0 affected5 at risk
EG003
Influenza
Infections and infestations
MedDRA 22.0
Systematic Assessment
EG0001 events1 affected6 at risk
EG0010 events0 affected5 at risk
EG0020 events0 affected5 at risk
EG003
Lower Respiratory Tract Infection
Infections and infestations
MedDRA 22.0
Systematic Assessment
EG0000 events0 affected6 at risk
EG0010 events0 affected5 at risk
EG0020 events0 affected5 at risk
EG003
Lung Infection
Infections and infestations
MedDRA 22.0
Systematic Assessment
EG0000 events0 affected6 at risk
EG0010 events0 affected5 at risk
EG0020 events0 affected5 at risk
EG003
Lung Infection Pseudomonal
Infections and infestations
MedDRA 22.0
Systematic Assessment
EG0000 events0 affected6 at risk
EG0010 events0 affected5 at risk
EG0020 events0 affected5 at risk
EG003
Nail Infection
Infections and infestations
MedDRA 22.0
Systematic Assessment
EG0000 events0 affected6 at risk
EG0010 events0 affected5 at risk
EG0020 events0 affected5 at risk
EG003
Nasopharyngitis
Infections and infestations
MedDRA 22.0
Systematic Assessment
EG0000 events0 affected6 at risk
EG0010 events0 affected5 at risk
EG0020 events0 affected5 at risk
EG003
Otitis Externa
Infections and infestations
MedDRA 22.0
Systematic Assessment
EG0000 events0 affected6 at risk
EG0010 events0 affected5 at risk
EG0020 events0 affected5 at risk
EG003
Otitis Media
Infections and infestations
MedDRA 22.0
Systematic Assessment
EG0001 events1 affected6 at risk
EG0010 events0 affected5 at risk
EG0020 events0 affected5 at risk
EG003
Parainfluenzae Virus Infection
Infections and infestations
MedDRA 22.0
Systematic Assessment
EG0000 events0 affected6 at risk
EG0010 events0 affected5 at risk
EG0020 events0 affected5 at risk
EG003
Pneumonia
Infections and infestations
MedDRA 22.0
Systematic Assessment
EG0001 events1 affected6 at risk
EG0010 events0 affected5 at risk
EG0020 events0 affected5 at risk
EG003
Pneumonia Pseudomonal
Infections and infestations
MedDRA 22.0
Systematic Assessment
EG0000 events0 affected6 at risk
EG0010 events0 affected5 at risk
EG0020 events0 affected5 at risk
EG003
Rhinitis
Infections and infestations
MedDRA 22.0
Systematic Assessment
EG0000 events0 affected6 at risk
EG0010 events0 affected5 at risk
EG0020 events0 affected5 at risk
EG003
Sepsis
Infections and infestations
MedDRA 22.0
Systematic Assessment
EG0000 events0 affected6 at risk
EG0010 events0 affected5 at risk
EG0020 events0 affected5 at risk
EG003
Stoma Site Infection
Infections and infestations
MedDRA 22.0
Systematic Assessment
EG0000 events0 affected6 at risk
EG0010 events0 affected5 at risk
EG0020 events0 affected5 at risk
EG003
Tinea Pedis
Infections and infestations
MedDRA 22.0
Systematic Assessment
EG0000 events0 affected6 at risk
EG0010 events0 affected5 at risk
EG0020 events0 affected5 at risk
EG003
Tonsillitis
Infections and infestations
MedDRA 22.0
Systematic Assessment
EG0002 events1 affected6 at risk
EG0010 events0 affected5 at risk
EG0020 events0 affected5 at risk
EG003
Upper Respiratory Tract Infection
Infections and infestations
MedDRA 22.0
Systematic Assessment
EG0005 events2 affected6 at risk
EG0010 events0 affected5 at risk
EG0023 events2 affected5 at risk
EG003
Urinary Tract Infection
Infections and infestations
MedDRA 22.0
Systematic Assessment
EG0002 events2 affected6 at risk
EG0010 events0 affected5 at risk
EG0020 events0 affected5 at risk
EG003
Urinary Tract Infection Bacterial
Infections and infestations
MedDRA 22.0
Systematic Assessment
EG0000 events0 affected6 at risk
EG0010 events0 affected5 at risk
EG0021 events1 affected5 at risk
EG003
Viral Infection
Infections and infestations
MedDRA 22.0
Systematic Assessment
EG0000 events0 affected6 at risk
EG0010 events0 affected5 at risk
EG0020 events0 affected5 at risk
EG003
Viral Rash
Infections and infestations
MedDRA 22.0
Systematic Assessment
EG0001 events1 affected6 at risk
EG0010 events0 affected5 at risk
EG0020 events0 affected5 at risk
EG003
Arthropod Bite
Injury, poisoning and procedural complications
MedDRA 22.0
Systematic Assessment
EG0000 events0 affected6 at risk
EG0010 events0 affected5 at risk
EG0021 events1 affected5 at risk
EG003
Arthropod Sting
Injury, poisoning and procedural complications
MedDRA 22.0
Systematic Assessment
EG0000 events0 affected6 at risk
EG0012 events1 affected5 at risk
EG0020 events0 affected5 at risk
EG003
Contusion
Injury, poisoning and procedural complications
MedDRA 22.0
Systematic Assessment
EG0000 events0 affected6 at risk
EG0011 events1 affected5 at risk
EG0020 events0 affected5 at risk
EG003
Craniocerebral Injury
Injury, poisoning and procedural complications
MedDRA 22.0
Systematic Assessment
EG0000 events0 affected6 at risk
EG0010 events0 affected5 at risk
EG0020 events0 affected5 at risk
EG003
Fall
Injury, poisoning and procedural complications
MedDRA 22.0
Systematic Assessment
EG0002 events2 affected6 at risk
EG0012 events1 affected5 at risk
EG0020 events0 affected5 at risk
EG003
Ligament Sprain
Injury, poisoning and procedural complications
MedDRA 22.0
Systematic Assessment
EG0000 events0 affected6 at risk
EG0011 events1 affected5 at risk
EG0020 events0 affected5 at risk
EG003
Muscle Strain
Injury, poisoning and procedural complications
MedDRA 22.0
Systematic Assessment
EG0000 events0 affected6 at risk
EG0010 events0 affected5 at risk
EG0021 events1 affected5 at risk
EG003
Procedural Pain
Injury, poisoning and procedural complications
MedDRA 22.0
Systematic Assessment
EG0000 events0 affected6 at risk
EG0010 events0 affected5 at risk
EG0021 events1 affected5 at risk
EG003
Skin Abrasion
Injury, poisoning and procedural complications
MedDRA 22.0
Systematic Assessment
EG0000 events0 affected6 at risk
EG0013 events1 affected5 at risk
EG0020 events0 affected5 at risk
EG003
Sunburn
Injury, poisoning and procedural complications
MedDRA 22.0
Systematic Assessment
EG0000 events0 affected6 at risk
EG0011 events1 affected5 at risk
EG0020 events0 affected5 at risk
EG003
Thermal Burn
Injury, poisoning and procedural complications
MedDRA 22.0
Systematic Assessment
EG0000 events0 affected6 at risk
EG0011 events1 affected5 at risk
EG0020 events0 affected5 at risk
EG003
Vascular Access Complication
Injury, poisoning and procedural complications
MedDRA 22.0
Systematic Assessment
EG0000 events0 affected6 at risk
EG0010 events0 affected5 at risk
EG0020 events0 affected5 at risk
EG003
Wound
Injury, poisoning and procedural complications
MedDRA 22.0
Systematic Assessment
EG0000 events0 affected6 at risk
EG0010 events0 affected5 at risk
EG0021 events1 affected5 at risk
EG003
Alpha-1 Acid Glycoprotein Increased
Investigations
MedDRA 22.0
Systematic Assessment
EG0000 events0 affected6 at risk
EG0010 events0 affected5 at risk
EG0020 events0 affected5 at risk
EG003
Faecal Calprotectin Increased
Investigations
MedDRA 22.0
Systematic Assessment
EG0000 events0 affected6 at risk
EG0010 events0 affected5 at risk
EG0020 events0 affected5 at risk
EG003
Gamma-Glutamyltransferase Increased
Investigations
MedDRA 22.0
Systematic Assessment
EG0000 events0 affected6 at risk
EG0010 events0 affected5 at risk
EG0020 events0 affected5 at risk
EG003
Haemoglobin Decreased
Investigations
MedDRA 22.0
Systematic Assessment
EG0001 events1 affected6 at risk
EG0010 events0 affected5 at risk
EG0020 events0 affected5 at risk
EG003
Heart Rate Increased
Investigations
MedDRA 22.0
Systematic Assessment
EG0000 events0 affected6 at risk
EG0010 events0 affected5 at risk
EG0020 events0 affected5 at risk
EG003
Arthralgia
Musculoskeletal and connective tissue disorders
MedDRA 22.0
Systematic Assessment
EG0000 events0 affected6 at risk
EG0011 events1 affected5 at risk
EG0020 events0 affected5 at risk
EG003
Back Pain
Musculoskeletal and connective tissue disorders
MedDRA 22.0
Systematic Assessment
EG0001 events1 affected6 at risk
EG0010 events0 affected5 at risk
EG0020 events0 affected5 at risk
EG003
Foot Deformity
Musculoskeletal and connective tissue disorders
MedDRA 22.0
Systematic Assessment
EG0000 events0 affected6 at risk
EG0010 events0 affected5 at risk
EG0020 events0 affected5 at risk
EG003
Musculoskeletal Chest Pain
Musculoskeletal and connective tissue disorders
MedDRA 22.0
Systematic Assessment
EG0000 events0 affected6 at risk
EG0010 events0 affected5 at risk
EG0020 events0 affected5 at risk
EG003
Musculoskeletal Pain
Musculoskeletal and connective tissue disorders
MedDRA 22.0
Systematic Assessment
EG0000 events0 affected6 at risk
EG0011 events1 affected5 at risk
EG0020 events0 affected5 at risk
EG003
Myalgia
Musculoskeletal and connective tissue disorders
MedDRA 22.0
Systematic Assessment
EG0000 events0 affected6 at risk
EG0010 events0 affected5 at risk
EG0020 events0 affected5 at risk
EG003
Osteopenia
Musculoskeletal and connective tissue disorders
MedDRA 22.0
Systematic Assessment
EG0000 events0 affected6 at risk
EG0010 events0 affected5 at risk
EG0020 events0 affected5 at risk
EG003
Pain In Extremity
Musculoskeletal and connective tissue disorders
MedDRA 22.0
Systematic Assessment
EG0003 events1 affected6 at risk
EG0010 events0 affected5 at risk
EG0021 events1 affected5 at risk
EG003
Headache
Nervous system disorders
MedDRA 22.0
Systematic Assessment
EG0001 events1 affected6 at risk
EG0010 events0 affected5 at risk
EG0022 events2 affected5 at risk
EG003
Presyncope
Nervous system disorders
MedDRA 22.0
Systematic Assessment
EG0000 events0 affected6 at risk
EG0010 events0 affected5 at risk
EG0020 events0 affected5 at risk
EG003
Seizure
Nervous system disorders
MedDRA 22.0
Systematic Assessment
EG0000 events0 affected6 at risk
EG0010 events0 affected5 at risk
EG0020 events0 affected5 at risk
EG003
Device Occlusion
Product Issues
MedDRA 22.0
Systematic Assessment
EG0000 events0 affected6 at risk
EG0010 events0 affected5 at risk
EG0022 events2 affected5 at risk
EG003
Enuresis
Psychiatric disorders
MedDRA 22.0
Systematic Assessment
EG0001 events1 affected6 at risk
EG0010 events0 affected5 at risk
EG0020 events0 affected5 at risk
EG003
Nightmare
Psychiatric disorders
MedDRA 22.0
Systematic Assessment
EG0000 events0 affected6 at risk
EG0010 events0 affected5 at risk
EG0020 events0 affected5 at risk
EG003
Staring
Psychiatric disorders
MedDRA 22.0
Systematic Assessment
EG0000 events0 affected6 at risk
EG0010 events0 affected5 at risk
EG0020 events0 affected5 at risk
EG003
Dysuria
Renal and urinary disorders
MedDRA 22.0
Systematic Assessment
EG0000 events0 affected6 at risk
EG0010 events0 affected5 at risk
EG0020 events0 affected5 at risk
EG003
Haematuria
Renal and urinary disorders
MedDRA 22.0
Systematic Assessment
EG0000 events0 affected6 at risk
EG0010 events0 affected5 at risk
EG0020 events0 affected5 at risk
EG003
Neurogenic Bladder
Renal and urinary disorders
MedDRA 22.0
Systematic Assessment
EG0000 events0 affected6 at risk
EG0010 events0 affected5 at risk
EG0020 events0 affected5 at risk
EG003
Genital Erosion
Reproductive system and breast disorders
MedDRA 22.0
Systematic Assessment
EG0001 events1 affected6 at risk
EG0010 events0 affected5 at risk
EG0020 events0 affected5 at risk
EG003
Asthma
Respiratory, thoracic and mediastinal disorders
MedDRA 22.0
Systematic Assessment
EG0000 events0 affected6 at risk
EG0010 events0 affected5 at risk
EG0020 events0 affected5 at risk
EG003
Atelectasis
Respiratory, thoracic and mediastinal disorders
MedDRA 22.0
Systematic Assessment
EG0000 events0 affected6 at risk
EG0010 events0 affected5 at risk
EG0020 events0 affected5 at risk
EG003
Bronchiectasis
Respiratory, thoracic and mediastinal disorders
MedDRA 22.0
Systematic Assessment
EG0000 events0 affected6 at risk
EG0010 events0 affected5 at risk
EG0020 events0 affected5 at risk
EG003
Cough
Respiratory, thoracic and mediastinal disorders
MedDRA 22.0
Systematic Assessment
EG0001 events1 affected6 at risk
EG0011 events1 affected5 at risk
EG0024 events2 affected5 at risk
EG003
Epistaxis
Respiratory, thoracic and mediastinal disorders
MedDRA 22.0
Systematic Assessment
EG0000 events0 affected6 at risk
EG0010 events0 affected5 at risk
EG0020 events0 affected5 at risk
EG003
Increased Upper Airway Secretion
Respiratory, thoracic and mediastinal disorders
MedDRA 22.0
Systematic Assessment
EG0000 events0 affected6 at risk
EG0010 events0 affected5 at risk
EG0021 events1 affected5 at risk
EG003
Laryngeal Oedema
Respiratory, thoracic and mediastinal disorders
MedDRA 22.0
Systematic Assessment
EG0000 events0 affected6 at risk
EG0010 events0 affected5 at risk
EG0020 events0 affected5 at risk
EG003
Lower Respiratory Tract Congestion
Respiratory, thoracic and mediastinal disorders
MedDRA 22.0
Systematic Assessment
EG0001 events1 affected6 at risk
EG0010 events0 affected5 at risk
EG0020 events0 affected5 at risk
EG003
Nasal Congestion
Respiratory, thoracic and mediastinal disorders
MedDRA 22.0
Systematic Assessment
EG0000 events0 affected6 at risk
EG0010 events0 affected5 at risk
EG0021 events1 affected5 at risk
EG003
Oropharyngeal Pain
Respiratory, thoracic and mediastinal disorders
MedDRA 22.0
Systematic Assessment
EG0000 events0 affected6 at risk
EG0013 events2 affected5 at risk
EG0020 events0 affected5 at risk
EG003
Productive Cough
Respiratory, thoracic and mediastinal disorders
MedDRA 22.0
Systematic Assessment
EG0000 events0 affected6 at risk
EG0010 events0 affected5 at risk
EG0020 events0 affected5 at risk
EG003
Pulmonary Pain
Respiratory, thoracic and mediastinal disorders
MedDRA 22.0
Systematic Assessment
EG0000 events0 affected6 at risk
EG0010 events0 affected5 at risk
EG0020 events0 affected5 at risk
EG003
Respiratory Tract Congestion
Respiratory, thoracic and mediastinal disorders
MedDRA 22.0
Systematic Assessment
EG0000 events0 affected6 at risk
EG0010 events0 affected5 at risk
EG0021 events1 affected5 at risk
EG003
Rhinorrhoea
Respiratory, thoracic and mediastinal disorders
MedDRA 22.0
Systematic Assessment
EG0000 events0 affected6 at risk
EG0010 events0 affected5 at risk
EG0022 events2 affected5 at risk
EG003
Tachypnoea
Respiratory, thoracic and mediastinal disorders
MedDRA 22.0
Systematic Assessment
EG0000 events0 affected6 at risk
EG0010 events0 affected5 at risk
EG0021 events1 affected5 at risk
EG003
Tonsillar Hypertrophy
Respiratory, thoracic and mediastinal disorders
MedDRA 22.0
Systematic Assessment
EG0000 events0 affected6 at risk
EG0010 events0 affected5 at risk
EG0020 events0 affected5 at risk
EG003
Upper Respiratory Tract Congestion
Respiratory, thoracic and mediastinal disorders
MedDRA 22.0
Systematic Assessment
EG0000 events0 affected6 at risk
EG0010 events0 affected5 at risk
EG0020 events0 affected5 at risk
EG003
Alopecia Areata
Skin and subcutaneous tissue disorders
MedDRA 22.0
Systematic Assessment
EG0000 events0 affected6 at risk
EG0010 events0 affected5 at risk
EG0020 events0 affected5 at risk
EG003
Blister
Skin and subcutaneous tissue disorders
MedDRA 22.0
Systematic Assessment
EG0000 events0 affected6 at risk
EG0010 events0 affected5 at risk
EG0020 events0 affected5 at risk
EG003
Dermatitis Atopic
Skin and subcutaneous tissue disorders
MedDRA 22.0
Systematic Assessment
EG0001 events1 affected6 at risk
EG0010 events0 affected5 at risk
EG0020 events0 affected5 at risk
EG003
Dermatitis Contact
Skin and subcutaneous tissue disorders
MedDRA 22.0
Systematic Assessment
EG0000 events0 affected6 at risk
EG0010 events0 affected5 at risk
EG0022 events1 affected5 at risk
EG003
Dermatitis Diaper
Skin and subcutaneous tissue disorders
MedDRA 22.0
Systematic Assessment
EG0000 events0 affected6 at risk
EG0013 events1 affected5 at risk
EG0020 events0 affected5 at risk
EG003
Eczema
Skin and subcutaneous tissue disorders
MedDRA 22.0
Systematic Assessment
EG0002 events1 affected6 at risk
EG0010 events0 affected5 at risk
EG0020 events0 affected5 at risk
EG003
Erythema
Skin and subcutaneous tissue disorders
MedDRA 22.0
Systematic Assessment
EG0000 events0 affected6 at risk
EG0011 events1 affected5 at risk
EG0020 events0 affected5 at risk
EG003
Intertrigo
Skin and subcutaneous tissue disorders
MedDRA 22.0
Systematic Assessment
EG0000 events0 affected6 at risk
EG0010 events0 affected5 at risk
EG0020 events0 affected5 at risk
EG003
Pruritus
Skin and subcutaneous tissue disorders
MedDRA 22.0
Systematic Assessment
EG0000 events0 affected6 at risk
EG0010 events0 affected5 at risk
EG0020 events0 affected5 at risk
EG003
Rash
Skin and subcutaneous tissue disorders
MedDRA 22.0
Systematic Assessment
EG0000 events0 affected6 at risk
EG0011 events1 affected5 at risk
EG0023 events2 affected5 at risk
EG003
Urticaria
Skin and subcutaneous tissue disorders
MedDRA 22.0
Systematic Assessment
EG0000 events0 affected6 at risk
EG0011 events1 affected5 at risk
EG0020 events0 affected5 at risk
EG003
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.
After determination of the highest tolerated avalglucosidase alfa dose in Cohort 1 and Cohort 2 (after at least 5 participants in each Cohort 1 and Cohort 2 had received the 7th dose of avalglucosidase alfa or completed Week 13 with a minimum of 6 infusions), participants received avalglucosidase alfa 40 mg/kg (the highest tolerated dose) IV infusion qow for 25 weeks in PAP.
OG003
PAP: Cohort 3b: Alglucosidase Alfa
After determination of the highest tolerated avalglucosidase alfa dose in Cohort 1 and Cohort 2 (after at least 5 participants in each Cohort 1 and Cohort 2 had received the 7th dose of avalglucosidase alfa or completed Week 13 with a minimum of 6 infusions), participants received alglucosidase alfa at their current stable dose (defined as dose [between 20 mg/kg qow and 40 mg/kg weekly as per physician] administered regularly for a minimum of 6 months immediately prior to entry in this study) IV infusion for 25 weeks in PAP.
Units
Counts
Participants
OG0006
OG0015
OG0025
OG0036
Title
Denominators
Categories
Protocol-defined IARs
Title
Measurements
OG0000
OG0012
OG0021
OG0031
Algorithm-defined IARs
Title
Measurements
OG0000
OG0012
OG0021
OG003
OG003
PAP: Cohort 3b: Alglucosidase Alfa
After determination of the highest tolerated avalglucosidase alfa dose in Cohort 1 and Cohort 2 (after at least 5 participants in each Cohort 1 and Cohort 2 had received the 7th dose of avalglucosidase alfa or completed Week 13 with a minimum of 6 infusions), participants received alglucosidase alfa at their current stable dose (defined as dose [between 20 mg/kg qow and 40 mg/kg weekly as per physician] administered regularly for a minimum of 6 months immediately prior to entry in this study) IV infusion for 25 weeks in PAP.
Units
Counts
Participants
OG0006
OG0015
OG0025
OG0036
Title
Denominators
Categories
Treatment boosted ADA response
Title
Measurements
OG0000
OG0010
OG0021
OG0032
Treatment induced ADA response
Title
Measurements
OG0000
OG0011
OG0023
OG003
Units
Counts
Participants
OG0006
OG0015
OG0025
Title
Denominators
Categories
Week 1
ParticipantsOG0005
ParticipantsOG0014
ParticipantsOG0024
Title
Measurements
OG000189000± 56700
OG001403000± 171000
OG002250000± 45100
Week 25
ParticipantsOG0005
ParticipantsOG0015
ParticipantsOG0025
Title
Measurements
OG000
Units
Counts
Participants
OG0006
OG0015
OG0025
Title
Denominators
Categories
Week 1
ParticipantsOG0005
ParticipantsOG0014
ParticipantsOG0024
Title
Measurements
OG0004.43(3.90 to 5.33)
OG0017.00(6.00 to 7.25)
OG0026.83(6.65 to 7.22)
Week 25
ParticipantsOG0005
ParticipantsOG0015
ParticipantsOG0025
Title
Measurements
OG000
Units
Counts
Participants
OG0006
OG0015
OG0025
Title
Denominators
Categories
Week 1
ParticipantsOG0005
ParticipantsOG0014
ParticipantsOG0024
Title
Measurements
OG000923000± 352000
OG0012630000± 972000
OG0021720000± 255000
Week 25
ParticipantsOG0005
ParticipantsOG0015
ParticipantsOG0025
Title
Measurements
OG000
Units
Counts
Participants
OG0006
OG0015
OG0025
Title
Denominators
Categories
Week 1
ParticipantsOG0005
ParticipantsOG0014
ParticipantsOG0024
Title
Measurements
OG0000.703± 0.291
OG0011.15± 0.523
OG0020.806± 0.248
Week 25
ParticipantsOG0005
ParticipantsOG0015
ParticipantsOG0025
Title
Measurements
OG000
Units
Counts
Participants
OG0006
OG0015
OG0025
Title
Denominators
Categories
Week 1
ParticipantsOG0005
ParticipantsOG0014
ParticipantsOG0024
Title
Measurements
OG000673± 222
OG001562± 152
OG002529± 150
Week 25
ParticipantsOG0005
ParticipantsOG0015
ParticipantsOG0025
Title
Measurements
OG000
Units
Counts
Participants
OG0006
OG0015
OG0025
Title
Denominators
Categories
Week 1
ParticipantsOG0005
ParticipantsOG0014
ParticipantsOG0024
Title
Measurements
OG0003550± 927
OG0014500± 882
OG0024300± 1420
Week 25
ParticipantsOG0005
ParticipantsOG0015
ParticipantsOG0025
Title
Measurements
OG000
OG002
PAP: Cohort 3a: Avalglucosidase Alfa 40 mg/kg
After determination of the highest tolerated avalglucosidase alfa dose in Cohort 1 and Cohort 2 (after at least 5 participants in each Cohort 1 and Cohort 2 had received the 7th dose of avalglucosidase alfa or completed Week 13 with a minimum of 6 infusions), participants received avalglucosidase alfa 40 mg/kg (the highest tolerated dose) IV infusion qow for 25 weeks in PAP.
OG003
PAP: Cohort 3b: Alglucosidase Alfa
After determination of the highest tolerated avalglucosidase alfa dose in Cohort 1 and Cohort 2 (after at least 5 participants in each Cohort 1 and Cohort 2 had received the 7th dose of avalglucosidase alfa or completed Week 13 with a minimum of 6 infusions), participants received alglucosidase alfa at their current stable dose (defined as dose [between 20 mg/kg qow and 40 mg/kg weekly as per physician] administered regularly for a minimum of 6 months immediately prior to entry in this study) IV infusion for 25 weeks in PAP.
Units
Counts
Participants
OG0006
OG0015
OG0024
OG0036
Title
Denominators
Categories
Title
Measurements
OG0006.50± 22.24
OG0019.80± 13.99
OG00211.00± 10.80
OG00317.00± 8.44
OG002
PAP: Cohort 3a: Avalglucosidase Alfa 40 mg/kg
After determination of the highest tolerated avalglucosidase alfa dose in Cohort 1 and Cohort 2 (after at least 5 participants in each Cohort 1 and Cohort 2 had received the 7th dose of avalglucosidase alfa or completed Week 13 with a minimum of 6 infusions), participants received avalglucosidase alfa 40 mg/kg (the highest tolerated dose) IV infusion qow for 25 weeks in PAP.
OG003
PAP: Cohort 3b: Alglucosidase Alfa
After determination of the highest tolerated avalglucosidase alfa dose in Cohort 1 and Cohort 2 (after at least 5 participants in each Cohort 1 and Cohort 2 had received the 7th dose of avalglucosidase alfa or completed Week 13 with a minimum of 6 infusions), participants received alglucosidase alfa at their current stable dose (defined as dose [between 20 mg/kg qow and 40 mg/kg weekly as per physician] administered regularly for a minimum of 6 months immediately prior to entry in this study) IV infusion for 25 weeks in PAP.
Units
Counts
Participants
OG0006
OG0015
OG0025
OG0036
Title
Denominators
Categories
Baseline: Level I
Title
Measurements
OG0001
OG0011
OG0022
OG0033
Baseline: Level II
Title
Measurements
OG0001
OG0012
OG0021
OG003
Baseline: Level III
Title
Measurements
OG0001
OG0010
OG0021
OG003
Baseline: Level IV
Title
Measurements
OG0002
OG0012
OG0020
OG003
Baseline: Level V
Title
Measurements
OG0001
OG0010
OG0021
OG003
Week 25: Level I
Title
Measurements
OG0001
OG0011
OG0022
OG003
Week 25: Level II
Title
Measurements
OG0001
OG0012
OG0021
OG003
Week 25: Level III
Title
Measurements
OG0002
OG0011
OG0020
OG003
Week 25: Level IV
Title
Measurements
OG0001
OG0011
OG0021
OG003
Week 25: Level V
Title
Measurements
OG0001
OG0010
OG0021
OG003
OG002
PAP: Cohort 3a: Avalglucosidase Alfa 40 mg/kg
After determination of the highest tolerated avalglucosidase alfa dose in Cohort 1 and Cohort 2 (after at least 5 participants in each Cohort 1 and Cohort 2 had received the 7th dose of avalglucosidase alfa or completed Week 13 with a minimum of 6 infusions), participants received avalglucosidase alfa 40 mg/kg (the highest tolerated dose) IV infusion qow for 25 weeks in PAP.
OG003
PAP: Cohort 3b: Alglucosidase Alfa
After determination of the highest tolerated avalglucosidase alfa dose in Cohort 1 and Cohort 2 (after at least 5 participants in each Cohort 1 and Cohort 2 had received the 7th dose of avalglucosidase alfa or completed Week 13 with a minimum of 6 infusions), participants received alglucosidase alfa at their current stable dose (defined as dose [between 20 mg/kg qow and 40 mg/kg weekly as per physician] administered regularly for a minimum of 6 months immediately prior to entry in this study) IV infusion for 25 weeks in PAP.
Units
Counts
Participants
OG0006
OG0015
OG0023
OG0036
Title
Denominators
Categories
Title
Measurements
OG0006.19± 10.55
OG0012.12± 4.04
OG0022.60± 1.72
OG0035.20± 5.95
OG003
PAP: Cohort 3b: Alglucosidase Alfa
After determination of the highest tolerated avalglucosidase alfa dose in Cohort 1 and Cohort 2 (after at least 5 participants in each Cohort 1 and Cohort 2 had received the 7th dose of avalglucosidase alfa or completed Week 13 with a minimum of 6 infusions), participants received alglucosidase alfa at their current stable dose (defined as dose [between 20 mg/kg qow and 40 mg/kg weekly as per physician] administered regularly for a minimum of 6 months immediately prior to entry in this study) IV infusion for 25 weeks in PAP.
Units
Counts
Participants
OG0006
OG0015
OG0024
OG0036
Title
Denominators
Categories
Title
Measurements
OG000-0.17± 4.45
OG0013.20± 4.55
OG0024.25± 3.30
OG0035.17± 4.54
OG003
PAP: Cohort 3b: Alglucosidase Alfa
After determination of the highest tolerated avalglucosidase alfa dose in Cohort 1 and Cohort 2 (after at least 5 participants in each Cohort 1 and Cohort 2 had received the 7th dose of avalglucosidase alfa or completed Week 13 with a minimum of 6 infusions), participants received alglucosidase alfa at their current stable dose (defined as dose [between 20 mg/kg qow and 40 mg/kg weekly as per physician] administered regularly for a minimum of 6 months immediately prior to entry in this study) IV infusion for 25 weeks in PAP.
Units
Counts
Participants
OG0006
OG0015
OG0025
OG0036
Title
Denominators
Categories
Baseline
ParticipantsOG0006
ParticipantsOG0013
ParticipantsOG0025
ParticipantsOG0034
Title
Measurements
OG000-1.10± 1.07
OG0010.13± 2.39
OG002-0.78± 0.70
OG003
Week 25
ParticipantsOG0005
ParticipantsOG0014
ParticipantsOG0025
ParticipantsOG0034
Change from Baseline at Week 25
ParticipantsOG0005
ParticipantsOG0012
ParticipantsOG0025
ParticipantsOG0033
OG003
PAP: Cohort 3b: Alglucosidase Alfa
After determination of the highest tolerated avalglucosidase alfa dose in Cohort 1 and Cohort 2 (after at least 5 participants in each Cohort 1 and Cohort 2 had received the 7th dose of avalglucosidase alfa or completed Week 13 with a minimum of 6 infusions), participants received alglucosidase alfa at their current stable dose (defined as dose [between 20 mg/kg qow and 40 mg/kg weekly as per physician] administered regularly for a minimum of 6 months immediately prior to entry in this study) IV infusion for 25 weeks in PAP.
Units
Counts
Participants
OG0006
OG0015
OG0025
OG0036
Title
Denominators
Categories
Left non-Flash
Title
Measurements
OG000-0.67± 1.54
OG0011.30± 1.48
OG0021.30± 0.76
OG003-0.50± 0.77
Right non-Flash
Title
Measurements
OG000-0.92± 1.66
OG0010.70± 1.25
OG0020.90± 1.14
OG003
OG003
PAP: Cohort 3b: Alglucosidase Alfa
After determination of the highest tolerated avalglucosidase alfa dose in Cohort 1 and Cohort 2 (after at least 5 participants in each Cohort 1 and Cohort 2 had received the 7th dose of avalglucosidase alfa or completed Week 13 with a minimum of 6 infusions), participants received alglucosidase alfa at their current stable dose (defined as dose [between 20 mg/kg qow and 40 mg/kg weekly as per physician] administered regularly for a minimum of 6 months immediately prior to entry in this study) IV infusion for 25 weeks in PAP.
Units
Counts
Participants
OG0006
OG0015
OG0025
OG0036
Title
Denominators
Categories
Left MRD
ParticipantsOG0006
ParticipantsOG0014
ParticipantsOG0024
ParticipantsOG0036
Title
Measurements
OG000-0.08± 1.53
OG001-0.38± 0.85
OG0020.88± 1.11
OG003
Right MRD
ParticipantsOG0006
ParticipantsOG0015
ParticipantsOG0024
ParticipantsOG0036
OG003
PAP: Cohort 3b: Alglucosidase Alfa
After determination of the highest tolerated avalglucosidase alfa dose in Cohort 1 and Cohort 2 (after at least 5 participants in each Cohort 1 and Cohort 2 had received the 7th dose of avalglucosidase alfa or completed Week 13 with a minimum of 6 infusions), participants received alglucosidase alfa at their current stable dose (defined as dose [between 20 mg/kg qow and 40 mg/kg weekly as per physician] administered regularly for a minimum of 6 months immediately prior to entry in this study) IV infusion for 25 weeks in PAP.
Units
Counts
Participants
OG0006
OG0015
OG0025
OG0036
Title
Denominators
Categories
MPD Left Non-Flash
ParticipantsOG0006
ParticipantsOG0014
ParticipantsOG0025
ParticipantsOG0036
Title
Measurements
OG000-0.83± 1.44
OG0010.75± 1.19
OG0020.40± 0.42
OG003
MPD Right Non-Flash
ParticipantsOG0006
ParticipantsOG0015
ParticipantsOG0025
ParticipantsOG0036
After determination of the highest tolerated avalglucosidase alfa dose in Cohort 1 and Cohort 2 (after at least 5 participants in each Cohort 1 and Cohort 2 had received the 7th dose of avalglucosidase alfa or completed Week 13 with a minimum of 6 infusions), participants received alglucosidase alfa at their current stable dose (defined as dose [between 20 mg/kg qow and 40 mg/kg weekly as per physician] administered regularly for a minimum of 6 months immediately prior to entry in this study) IV infusion for 25 weeks in PAP.