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Study to Compare the Efficacy and Safety of Enzyme Replacement Therapies Avalglucosidase Alfa and Alglucosidase Alfa Administered Every Other Week in Patients With Late-onset Pompe Disease Who Have Not Been Previously Treated for Pompe Disease (COMET)

Primary Purpose

Glycogen Storage Disease Type II;Pompe's Disease

Status
Completed
Phase
Phase 3
Locations
International
Study Type
Interventional
Intervention
Avalglucosidase alfa (GZ402666)
Alglucosidase alfa (GZ419829)
Sponsored by
Genzyme, a Sanofi Company
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Glycogen Storage Disease Type II;Pompe's Disease

Eligibility Criteria

3 Years - undefined (Child, Adult, Older Adult)All SexesDoes not accept healthy volunteers

Inclusion criteria :

  • The participant has confirmed acid alpha-glucosidase (GAA) enzyme deficiency from any tissue source and/or 2 confirmed GAA gene mutations.
  • The participant must provide signed, informed consent prior to performing any study related procedures. Consent of a legally authorized guardian(s) is (are) required for legally minor participant as defined by local regulation. If the participant is legally minor, signed written consent shall be obtained from parent(s)/legal guardian and assent obtained from participants, if applicable.

Exclusion criteria:

  • The participant is <3 years of age.
  • The participant has known Pompe specific cardiac hypertrophy.
  • The participant is wheelchair dependent.
  • The participant is not able to ambulate 40 meters (approximately 130 feet) without stopping and without an assistive device.
  • The participant requires invasive-ventilation (non-invasive ventilation is allowed).
  • The participant is not able to successfully perform repeated forced vital capacity (FVC) measurements in upright position of greater than or equal to 30% predicted and less than or equal to 85% predicted.
  • The participant (and participant's legal guardian if participant is legally minor as defined by local regulation) is (are) not able to comply with the clinical protocol.
  • The participant has had previous treatment with alglucosidase alfa or any investigational therapy for Pompe disease.
  • The participant has prior or current use of immune tolerance induction therapy.
  • The participant, if female and of childbearing potential, has a positive pregnancy test (beta-human chorionic gonadotropin) at baseline.

The above information is not intended to contain all considerations relevant to a participant's potential participation in a clinical trial.

Sites / Locations

  • Investigational Site Number 8400015
  • Investigational Site Number 8400020
  • Investigational Site Number 8400011
  • Investigational Site Number 8400017
  • Investigational Site Number 8400016
  • Investigational Site Number 8400007
  • Investigational Site Number 8400023
  • Investigational Site Number 8400002
  • Investigational Site Number 8400012
  • Investigational Site Number 8400010
  • Investigational Site Number 8400001
  • Investigational Site Number 8400019
  • Investigational Site Number 8400026
  • Investigational Site Number 8400008
  • Investigational Site Number 8400006
  • Investigational Site Number 8400009
  • Investigational Site Number 8400014
  • Investigational Site Number 8400025
  • Investigational Site Number 8400018
  • Investigational Site Number 8400005
  • Investigational Site Number 8400024
  • Investigational Site Number 0320001
  • Investigational Site Number 0360001
  • Investigational Site Number 0400001
  • Investigational Site Number 0560003
  • Investigational Site Number 0560001
  • Investigational Site Number 0760004
  • Investigational Site Number 0760001
  • Investigational Site Number 1240003
  • Investigational Site Number 1240002
  • Investigational Site Number 2030001
  • Investigational Site Number 2080003
  • Investigational Site Number 2500008
  • Investigational Site Number 2500007
  • Investigational Site Number 2500011
  • Investigational Site Number 2500004
  • Investigational Site Number 2500010
  • Investigational Site Number 2500005
  • Investigational Site Number 2500006
  • Investigational Site Number 2500001
  • Investigational Site Number 2760006
  • Investigational Site Number 2760001
  • Investigational Site Number 2760003
  • Investigational Site Number 2760002
  • Investigational Site Number 3480001
  • Investigational Site Number 3800006
  • Investigational Site Number 3800001
  • Investigational Site Number 3800002
  • Investigational Site Number 3800007
  • Investigational Site Number 3800003
  • Investigational Site Number 3920002
  • Investigational Site Number 4100001
  • Investigational Site Number 4100002
  • Investigational Site Number 4840001
  • Investigational Site Number 5280001
  • Investigational Site Number 6160001
  • Investigational Site Number 6200001
  • Investigational Site Number 6430001
  • Investigational Site Number 7240002
  • Investigational Site Number 7240003
  • Investigational Site Number 7560002
  • Investigational Site Number 1580001
  • Investigational Site Number 7920001
  • Investigational Site Number 7920002
  • Investigational Site Number 8260005
  • Investigational Site Number 8260002
  • Investigational Site Number 8260001
  • Investigational Site Number 8260004
  • Investigational Site Number 8260003

Arms of the Study

Arm 1

Arm 2

Arm Type

Experimental

Active Comparator

Arm Label

avalglucosidase alfa (GZ402666)

alglucosidase alfa (GZ419829)

Arm Description

Administered intravenously every 2 weeks

Administered intravenously every 2 weeks

Outcomes

Primary Outcome Measures

PAP: Change From Baseline in Percent Predicted Forced Vital Capacity in Upright Position at Week 49
FVC is a standard pulmonary function test used to quantify respiratory muscle weakness. FVC is the volume of air (in liters) that can be forcibly blown out after full inspiration in the upright position. Least square (LS) mean and standard error (SE) were derived from mixed model for repeated measure (MMRM) model with baseline FVC (% predicted, as continuous), sex, age (in years at baseline), treatment group, visit, interaction term between treatment group and visit as fixed effects. Percent of predicted FVC = (actual FVC measurement)/(predicted value of FVC) * 100. After non-inferiority (NI) testing, a test for superiority of avalglucosidase alfa versus alglucosidase alfa was performed with an overall 2-sided 5% level of significance.

Secondary Outcome Measures

PAP: Change From Baseline in Total Distance Walked During Six-minute Walk Test (6MWT) at Week 49
6MWT was a standardized test that measured the distance (in meters) covered by the participant by walking on a flat, hard surface in a period of a 6-minute walk. Mean distance walked gives an indication of functional endurance. The greater the distance (that a participant could walk in 6 minutes), the greater the endurance. LS mean and SE were derived from MMRM model with baseline FVC (% predicted) and baseline 6MWT (distance walked in meter), age (in years, at baseline), gender, treatment group, visit, and treatment-by-visit interaction as fixed effects.
PAP: Change From Baseline in Percent Predicted Maximal Inspiratory Pressure (MIP) in Upright Position at Week 49
MIP is a quick and non-invasive test to measure strength of inspiratory muscles, primarily diaphragm, and allows for assessment of ventilatory failure, restrictive lung disease and respiratory muscle strength. MIP refers to how much air pressure force an individual creates by inhaling through the mouth as hard as possible. LS mean and SE were derived from MMRM model for MIP % predicted adjusted for MIP % predicted at baseline, age (in years, at baseline), gender, treatment group, visit, and treatment-by-visit interaction as fixed effects.
PAP: Change From Baseline in Percent Predicted Maximal Expiratory Pressure (MEP) in Upright Position at Week 49
MEP is a quick and non-invasive test to measure strength of expiratory muscles, primarily diaphragm, and allows for assessment of ventilatory failure, restrictive lung disease and respiratory muscle strength. MEP is the greater pressure generated during maximal expiration. LS mean and SE were derived from MMRM model for MEP % predicted adjusted for MEP % predicted at baseline, age (in years, at baseline), gender, treatment group, visit, and treatment-by-visit interaction as fixed effects.
PAP: Change From Baseline in Lower Extremity Muscle Strength at Week 49 as Assessed by Hand-held Dynamometry (HHD)
HHD: a portable method for strength quantitation. To complete a make test, participant exerted maximal force against dynamometer with gradual increase in force and completed isometric hold for 4-5 seconds. Muscle strengths were collected in Newton. Every muscle group (hip: flexion, extension, abduction; knee: flexion, extension and ankle dorsiflexion) were measured 2 times and highest value was reported. Summary score was sum of 12 measurements (2 measurements per muscle group) from 6 muscle groups on each side (left and right). An increase from Baseline was reflective of increased muscle strength, whereas a decrease from Baseline was reflective of decreased muscle strength. LS mean and SE were derived from MMRM model for HHD lower extremity muscle strength composite score adjusted for summary HHD lower extremity score at baseline, baseline FVC (% predicted), age (in years, at baseline), gender, treatment group, visit, and treatment-by-visit interaction as fixed effects.
PAP: Change From Baseline in Quick Motor Function Test (QMFT) Total Scores at Week 49
The QMFT was an observer administered test to evaluate changes in motor function. QMFT comprised of 16 items specifically difficult for participants with Pompe disease. Each item was scored separately on a 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), higher score represented better outcome. LS mean and SE were derived from MMRM models adjusted for total QMFT score at baseline, baseline FVC (% predicted), age (in years, at baseline), gender, treatment group, visit, and treatment-by-visit interaction as fixed effects.
PAP: Change From Baseline in 12-item Short-form Health Survey (SF-12): Physical Component Summary (PCS) and Mental Component Summary (MCS) Scores at Week 49
SF-12, a 12 item-questionnaire, used to assess health-related quality of life in participants aged >=18 years at screening/baseline. SF-12 consisted of 12 items, which were categorized into eight domains (subscales) of functioning and well-being: physical functioning, role-physical, role emotional, mental health, bodily pain, general health, vitality and social functioning, with each domain score ranged from 0 (poor health) to 100 (better health), higher scores indicated good health condition. These eight domains were further summarized into 2 summary scores, PCS and MCS. The score range for each of these 2 summary scores was from 0 (poor health) to 100 (better health), higher scores indicated a better health-related quality of life. LS mean and SE were derived from MMRM models adjusted for baseline score (PCS or MCS), baseline FVC (% predicted), age (in years, at baseline), gender, treatment group, visit, and treatment-by-visit interaction as fixed effects.
PAP: Number of Participants With Treatment-emergent Adverse Events (TEAEs) and Infusion-associated Reactions (IARs)
AE: any untoward medical occurrence in participant who took study drug and not necessarily have to had causal relationship with treatment. TEAEs: AEs that developed/worsened in grade/became serious during TEAE period in PAP (from time of 1st treatment date to last treatment date+4 weeks for participants who didn't receive any treatment in open-label or to time just prior to 1st treatment in open-label for participants who received treatment in open-label). Protocol-defined IARs: AE of special interest (AESIs) that occurred during either infusion/observation period following infusion which were deemed to be related/possibly related to study drug. Algorithm-defined IARs: any TEAE meeting either 1 of 2 criteria: 1) event occurred from start to end of infusion + 24 hours, considered related to study drug, 2) If AE time component missed, compare AE start date with infusion start and end date. If AE start date was between infusion start and end date + 1 day and it was related to study drug.
Open-label Period: Number of Participants With Treatment-emergent Adverse Events and Infusion-associated Reactions
AE: any untoward medical occurrence in a participant who received study drug and did not necessarily have to had a causal relationship with treatment. TEAEs in open-label: AEs that developed/worsened in grade/became serious during TEAE period in open-label (from time of 1st open-label treatment to last treatment date + 4 weeks). Protocol-defined IARs: defined as AESIs that occurred during either infusion/observation period following infusion which were deemed to be related/possibly related to study drug. Algorithm-defined IARs: any TEAE meeting either 1 of 2 criteria: 1) event occurred from start to end of infusion plus 24 hours, considered related to study drug, 2) If AE time component missed, compare AE start date with infusion start and end date. If AE start date was between infusion start and end date plus 1 day and it was related to study drug.
PAP: Percentage of Participants With Treatment-emergent Antidrug Antibodies (ADA) Response
ADA response categories: 1) Treatment-induced: ADAs developed following administration of the study drug. If the baseline ADA sample was missing or non-reportable and at least one reportable on-treatment ADA sample was available, the baseline sample was considered as "negative". 2) Treatment-boosted: Pre-existing ADAs that were boosted at least two titer steps from baseline (i.e., 4 fold increase in titers) following administration of the study drug (any time after the first drug administration). 3) Treatment emergent: combination of treatment induced and treatment boosted.

Full Information

First Posted
May 23, 2016
Last Updated
June 1, 2023
Sponsor
Genzyme, a Sanofi Company
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1. Study Identification

Unique Protocol Identification Number
NCT02782741
Brief Title
Study to Compare the Efficacy and Safety of Enzyme Replacement Therapies Avalglucosidase Alfa and Alglucosidase Alfa Administered Every Other Week in Patients With Late-onset Pompe Disease Who Have Not Been Previously Treated for Pompe Disease
Acronym
COMET
Official Title
A Phase 3 Randomized, Multicenter, Multinational, Double-blinded Study Comparing the Efficacy and Safety of Repeated Biweekly Infusions of Avalglucosidase Alfa (neoGAA, GZ402666) and Alglucosidase Alfa in Treatment naïve Patients With Late-onset Pompe Disease
Study Type
Interventional

2. Study Status

Record Verification Date
June 2023
Overall Recruitment Status
Completed
Study Start Date
November 2, 2016 (Actual)
Primary Completion Date
March 19, 2020 (Actual)
Study Completion Date
May 31, 2023 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Genzyme, a Sanofi Company

4. Oversight

Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
Yes

5. Study Description

Brief Summary
Primary Objective: To determine the effect of avalglucosidase alfa treatment on respiratory muscle strength measured by percent (%) predicted forced vital capacity (FVC) in the upright position, as compared to alglucosidase alfa. Secondary Objective: To determine the safety and effect of avalglucosidase alfa treatment on functional endurance (6-minute walk test, inspiratory muscle strength (maximum inspiratory pressure), expiratory muscle strength (maximum expiratory pressure), lower extremity muscle strength (hand-held dynamometry), motor function (Quick Motor Function Test), and health-related quality of life (Short Form-12).
Detailed Description
The duration of the study per participant will be up to approximately 6 years that will consist of a 14-day screening period (may be extended up to 8 weeks in pre-specified situations), a 49-week blinded treatment period (except for the subgroup of pediatric patients aged 3 to less than (<) 18 years enrolling directly in the open-label long-term follow-up phase), a 240-week open-label treatment period, and a 4-week post-treatment observation period.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Glycogen Storage Disease Type II;Pompe's Disease

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 3
Interventional Study Model
Parallel Assignment
Masking
ParticipantCare ProviderInvestigatorOutcomes Assessor
Allocation
Randomized
Enrollment
100 (Actual)

8. Arms, Groups, and Interventions

Arm Title
avalglucosidase alfa (GZ402666)
Arm Type
Experimental
Arm Description
Administered intravenously every 2 weeks
Arm Title
alglucosidase alfa (GZ419829)
Arm Type
Active Comparator
Arm Description
Administered intravenously every 2 weeks
Intervention Type
Drug
Intervention Name(s)
Avalglucosidase alfa (GZ402666)
Intervention Description
Pharmaceutical form: powder for concentrate for solution for infusion Route of administration: intravenous
Intervention Type
Drug
Intervention Name(s)
Alglucosidase alfa (GZ419829)
Other Intervention Name(s)
Myozyme, Lumizyme
Intervention Description
Pharmaceutical form: powder for concentrate for solution for infusion Route of administration: intravenous
Primary Outcome Measure Information:
Title
PAP: Change From Baseline in Percent Predicted Forced Vital Capacity in Upright Position at Week 49
Description
FVC is a standard pulmonary function test used to quantify respiratory muscle weakness. FVC is the volume of air (in liters) that can be forcibly blown out after full inspiration in the upright position. Least square (LS) mean and standard error (SE) were derived from mixed model for repeated measure (MMRM) model with baseline FVC (% predicted, as continuous), sex, age (in years at baseline), treatment group, visit, interaction term between treatment group and visit as fixed effects. Percent of predicted FVC = (actual FVC measurement)/(predicted value of FVC) * 100. After non-inferiority (NI) testing, a test for superiority of avalglucosidase alfa versus alglucosidase alfa was performed with an overall 2-sided 5% level of significance.
Time Frame
Baseline, Week 49
Secondary Outcome Measure Information:
Title
PAP: Change From Baseline in Total Distance Walked During Six-minute Walk Test (6MWT) at Week 49
Description
6MWT was a standardized test that measured the distance (in meters) covered by the participant by walking on a flat, hard surface in a period of a 6-minute walk. Mean distance walked gives an indication of functional endurance. The greater the distance (that a participant could walk in 6 minutes), the greater the endurance. LS mean and SE were derived from MMRM model with baseline FVC (% predicted) and baseline 6MWT (distance walked in meter), age (in years, at baseline), gender, treatment group, visit, and treatment-by-visit interaction as fixed effects.
Time Frame
Baseline, Week 49
Title
PAP: Change From Baseline in Percent Predicted Maximal Inspiratory Pressure (MIP) in Upright Position at Week 49
Description
MIP is a quick and non-invasive test to measure strength of inspiratory muscles, primarily diaphragm, and allows for assessment of ventilatory failure, restrictive lung disease and respiratory muscle strength. MIP refers to how much air pressure force an individual creates by inhaling through the mouth as hard as possible. LS mean and SE were derived from MMRM model for MIP % predicted adjusted for MIP % predicted at baseline, age (in years, at baseline), gender, treatment group, visit, and treatment-by-visit interaction as fixed effects.
Time Frame
Baseline, Week 49
Title
PAP: Change From Baseline in Percent Predicted Maximal Expiratory Pressure (MEP) in Upright Position at Week 49
Description
MEP is a quick and non-invasive test to measure strength of expiratory muscles, primarily diaphragm, and allows for assessment of ventilatory failure, restrictive lung disease and respiratory muscle strength. MEP is the greater pressure generated during maximal expiration. LS mean and SE were derived from MMRM model for MEP % predicted adjusted for MEP % predicted at baseline, age (in years, at baseline), gender, treatment group, visit, and treatment-by-visit interaction as fixed effects.
Time Frame
Baseline, Week 49
Title
PAP: Change From Baseline in Lower Extremity Muscle Strength at Week 49 as Assessed by Hand-held Dynamometry (HHD)
Description
HHD: a portable method for strength quantitation. To complete a make test, participant exerted maximal force against dynamometer with gradual increase in force and completed isometric hold for 4-5 seconds. Muscle strengths were collected in Newton. Every muscle group (hip: flexion, extension, abduction; knee: flexion, extension and ankle dorsiflexion) were measured 2 times and highest value was reported. Summary score was sum of 12 measurements (2 measurements per muscle group) from 6 muscle groups on each side (left and right). An increase from Baseline was reflective of increased muscle strength, whereas a decrease from Baseline was reflective of decreased muscle strength. LS mean and SE were derived from MMRM model for HHD lower extremity muscle strength composite score adjusted for summary HHD lower extremity score at baseline, baseline FVC (% predicted), age (in years, at baseline), gender, treatment group, visit, and treatment-by-visit interaction as fixed effects.
Time Frame
Baseline, Week 49
Title
PAP: Change From Baseline in Quick Motor Function Test (QMFT) Total Scores at Week 49
Description
The QMFT was an observer administered test to evaluate changes in motor function. QMFT comprised of 16 items specifically difficult for participants with Pompe disease. Each item was scored separately on a 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), higher score represented better outcome. LS mean and SE were derived from MMRM models adjusted for total QMFT score at baseline, baseline FVC (% predicted), age (in years, at baseline), gender, treatment group, visit, and treatment-by-visit interaction as fixed effects.
Time Frame
Baseline, Week 49
Title
PAP: Change From Baseline in 12-item Short-form Health Survey (SF-12): Physical Component Summary (PCS) and Mental Component Summary (MCS) Scores at Week 49
Description
SF-12, a 12 item-questionnaire, used to assess health-related quality of life in participants aged >=18 years at screening/baseline. SF-12 consisted of 12 items, which were categorized into eight domains (subscales) of functioning and well-being: physical functioning, role-physical, role emotional, mental health, bodily pain, general health, vitality and social functioning, with each domain score ranged from 0 (poor health) to 100 (better health), higher scores indicated good health condition. These eight domains were further summarized into 2 summary scores, PCS and MCS. The score range for each of these 2 summary scores was from 0 (poor health) to 100 (better health), higher scores indicated a better health-related quality of life. LS mean and SE were derived from MMRM models adjusted for baseline score (PCS or MCS), baseline FVC (% predicted), age (in years, at baseline), gender, treatment group, visit, and treatment-by-visit interaction as fixed effects.
Time Frame
Baseline, Week 49
Title
PAP: Number of Participants With Treatment-emergent Adverse Events (TEAEs) and Infusion-associated Reactions (IARs)
Description
AE: any untoward medical occurrence in participant who took study drug and not necessarily have to had causal relationship with treatment. TEAEs: AEs that developed/worsened in grade/became serious during TEAE period in PAP (from time of 1st treatment date to last treatment date+4 weeks for participants who didn't receive any treatment in open-label or to time just prior to 1st treatment in open-label for participants who received treatment in open-label). Protocol-defined IARs: AE of special interest (AESIs) that occurred during either infusion/observation period following infusion which were deemed to be related/possibly related to study drug. Algorithm-defined IARs: any TEAE meeting either 1 of 2 criteria: 1) event occurred from start to end of infusion + 24 hours, considered related to study drug, 2) If AE time component missed, compare AE start date with infusion start and end date. If AE start date was between infusion start and end date + 1 day and it was related to study drug.
Time Frame
From Baseline up to Week 49
Title
Open-label Period: Number of Participants With Treatment-emergent Adverse Events and Infusion-associated Reactions
Description
AE: any untoward medical occurrence in a participant who received study drug and did not necessarily have to had a causal relationship with treatment. TEAEs in open-label: AEs that developed/worsened in grade/became serious during TEAE period in open-label (from time of 1st open-label treatment to last treatment date + 4 weeks). Protocol-defined IARs: defined as AESIs that occurred during either infusion/observation period following infusion which were deemed to be related/possibly related to study drug. Algorithm-defined IARs: any TEAE meeting either 1 of 2 criteria: 1) event occurred from start to end of infusion plus 24 hours, considered related to study drug, 2) If AE time component missed, compare AE start date with infusion start and end date. If AE start date was between infusion start and end date plus 1 day and it was related to study drug.
Time Frame
Week 50 to 145 in open-label long-term period
Title
PAP: Percentage of Participants With Treatment-emergent Antidrug Antibodies (ADA) Response
Description
ADA response categories: 1) Treatment-induced: ADAs developed following administration of the study drug. If the baseline ADA sample was missing or non-reportable and at least one reportable on-treatment ADA sample was available, the baseline sample was considered as "negative". 2) Treatment-boosted: Pre-existing ADAs that were boosted at least two titer steps from baseline (i.e., 4 fold increase in titers) following administration of the study drug (any time after the first drug administration). 3) Treatment emergent: combination of treatment induced and treatment boosted.
Time Frame
From Baseline up to Week 49

10. Eligibility

Sex
All
Minimum Age & Unit of Time
3 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion criteria : The participant has confirmed acid alpha-glucosidase (GAA) enzyme deficiency from any tissue source and/or 2 confirmed GAA gene mutations. The participant must provide signed, informed consent prior to performing any study related procedures. Consent of a legally authorized guardian(s) is (are) required for legally minor participant as defined by local regulation. If the participant is legally minor, signed written consent shall be obtained from parent(s)/legal guardian and assent obtained from participants, if applicable. Exclusion criteria: The participant is <3 years of age. The participant has known Pompe specific cardiac hypertrophy. The participant is wheelchair dependent. The participant is not able to ambulate 40 meters (approximately 130 feet) without stopping and without an assistive device. The participant requires invasive-ventilation (non-invasive ventilation is allowed). The participant is not able to successfully perform repeated forced vital capacity (FVC) measurements in upright position of greater than or equal to 30% predicted and less than or equal to 85% predicted. The participant (and participant's legal guardian if participant is legally minor as defined by local regulation) is (are) not able to comply with the clinical protocol. The participant has had previous treatment with alglucosidase alfa or any investigational therapy for Pompe disease. The participant has prior or current use of immune tolerance induction therapy. The participant, if female and of childbearing potential, has a positive pregnancy test (beta-human chorionic gonadotropin) at baseline. The above information is not intended to contain all considerations relevant to a participant's potential participation in a clinical trial.
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Clinical Sciences & Operations
Organizational Affiliation
Sanofi
Official's Role
Study Director
Facility Information:
Facility Name
Investigational Site Number 8400015
City
Phoenix
State/Province
Arizona
ZIP/Postal Code
85013
Country
United States
Facility Name
Investigational Site Number 8400020
City
Los Angeles
State/Province
California
ZIP/Postal Code
90095
Country
United States
Facility Name
Investigational Site Number 8400011
City
Orange
State/Province
California
ZIP/Postal Code
92868
Country
United States
Facility Name
Investigational Site Number 8400017
City
Stanford
State/Province
California
ZIP/Postal Code
94305
Country
United States
Facility Name
Investigational Site Number 8400016
City
Gainesville
State/Province
Florida
ZIP/Postal Code
32610
Country
United States
Facility Name
Investigational Site Number 8400007
City
Decatur
State/Province
Georgia
ZIP/Postal Code
30033
Country
United States
Facility Name
Investigational Site Number 8400023
City
Chicago
State/Province
Illinois
ZIP/Postal Code
60611
Country
United States
Facility Name
Investigational Site Number 8400002
City
Iowa City
State/Province
Iowa
ZIP/Postal Code
52242
Country
United States
Facility Name
Investigational Site Number 8400012
City
Kansas City
State/Province
Kansas
ZIP/Postal Code
66160-7321
Country
United States
Facility Name
Investigational Site Number 8400010
City
Boston
State/Province
Massachusetts
ZIP/Postal Code
02114
Country
United States
Facility Name
Investigational Site Number 8400001
City
Detroit
State/Province
Michigan
ZIP/Postal Code
48201
Country
United States
Facility Name
Investigational Site Number 8400019
City
Minneapolis
State/Province
Minnesota
ZIP/Postal Code
55455
Country
United States
Facility Name
Investigational Site Number 8400026
City
Great Neck
State/Province
New York
ZIP/Postal Code
11020
Country
United States
Facility Name
Investigational Site Number 8400008
City
Valhalla
State/Province
New York
ZIP/Postal Code
10595
Country
United States
Facility Name
Investigational Site Number 8400006
City
Durham
State/Province
North Carolina
ZIP/Postal Code
27710
Country
United States
Facility Name
Investigational Site Number 8400009
City
Cincinnati
State/Province
Ohio
ZIP/Postal Code
45267-0542
Country
United States
Facility Name
Investigational Site Number 8400014
City
Portland
State/Province
Oregon
ZIP/Postal Code
97239
Country
United States
Facility Name
Investigational Site Number 8400025
City
Pittsburgh
State/Province
Pennsylvania
ZIP/Postal Code
15213
Country
United States
Facility Name
Investigational Site Number 8400018
City
Salt Lake City
State/Province
Utah
ZIP/Postal Code
84132
Country
United States
Facility Name
Investigational Site Number 8400005
City
Fairfax
State/Province
Virginia
ZIP/Postal Code
22030
Country
United States
Facility Name
Investigational Site Number 8400024
City
Morgantown
State/Province
West Virginia
ZIP/Postal Code
26506
Country
United States
Facility Name
Investigational Site Number 0320001
City
Caba
ZIP/Postal Code
C1181ACH
Country
Argentina
Facility Name
Investigational Site Number 0360001
City
Auchenflower
ZIP/Postal Code
4066
Country
Australia
Facility Name
Investigational Site Number 0400001
City
Wien
ZIP/Postal Code
1090
Country
Austria
Facility Name
Investigational Site Number 0560003
City
Bruxelles
ZIP/Postal Code
1070
Country
Belgium
Facility Name
Investigational Site Number 0560001
City
Leuven
ZIP/Postal Code
3000
Country
Belgium
Facility Name
Investigational Site Number 0760004
City
Brasilia
ZIP/Postal Code
71625-009
Country
Brazil
Facility Name
Investigational Site Number 0760001
City
Sao Paulo
ZIP/Postal Code
04037-002
Country
Brazil
Facility Name
Investigational Site Number 1240003
City
Hamilton
ZIP/Postal Code
L8N 3Z5
Country
Canada
Facility Name
Investigational Site Number 1240002
City
Montreal
ZIP/Postal Code
H3A 2B4
Country
Canada
Facility Name
Investigational Site Number 2030001
City
Praha 2
ZIP/Postal Code
12808
Country
Czechia
Facility Name
Investigational Site Number 2080003
City
København Ø
ZIP/Postal Code
2100
Country
Denmark
Facility Name
Investigational Site Number 2500008
City
Angers
ZIP/Postal Code
49933
Country
France
Facility Name
Investigational Site Number 2500007
City
Bordeaux
Country
France
Facility Name
Investigational Site Number 2500011
City
Brest Cedex 2
ZIP/Postal Code
29609
Country
France
Facility Name
Investigational Site Number 2500004
City
Bron
ZIP/Postal Code
69677
Country
France
Facility Name
Investigational Site Number 2500010
City
Clermont Ferrand
ZIP/Postal Code
63003
Country
France
Facility Name
Investigational Site Number 2500005
City
Lille
ZIP/Postal Code
59037
Country
France
Facility Name
Investigational Site Number 2500006
City
Marseille Cedex 5
ZIP/Postal Code
13385
Country
France
Facility Name
Investigational Site Number 2500001
City
Paris
ZIP/Postal Code
75013
Country
France
Facility Name
Investigational Site Number 2760006
City
Bochum
ZIP/Postal Code
44789
Country
Germany
Facility Name
Investigational Site Number 2760001
City
Mainz
ZIP/Postal Code
55131
Country
Germany
Facility Name
Investigational Site Number 2760003
City
München
ZIP/Postal Code
80336
Country
Germany
Facility Name
Investigational Site Number 2760002
City
Münster
ZIP/Postal Code
48149
Country
Germany
Facility Name
Investigational Site Number 3480001
City
Budapest
ZIP/Postal Code
1083
Country
Hungary
Facility Name
Investigational Site Number 3800006
City
Brescia
ZIP/Postal Code
25123
Country
Italy
Facility Name
Investigational Site Number 3800001
City
Messina
ZIP/Postal Code
98125
Country
Italy
Facility Name
Investigational Site Number 3800002
City
Milano
ZIP/Postal Code
20122
Country
Italy
Facility Name
Investigational Site Number 3800007
City
Napoli
ZIP/Postal Code
80131
Country
Italy
Facility Name
Investigational Site Number 3800003
City
Torino
ZIP/Postal Code
10126
Country
Italy
Facility Name
Investigational Site Number 3920002
City
Kodaira-Shi
Country
Japan
Facility Name
Investigational Site Number 4100001
City
Seoul
ZIP/Postal Code
03080
Country
Korea, Republic of
Facility Name
Investigational Site Number 4100002
City
Seoul
ZIP/Postal Code
06273
Country
Korea, Republic of
Facility Name
Investigational Site Number 4840001
City
Mexico
Country
Mexico
Facility Name
Investigational Site Number 5280001
City
Rotterdam
ZIP/Postal Code
3015 GE
Country
Netherlands
Facility Name
Investigational Site Number 6160001
City
Warszawa
ZIP/Postal Code
02-097
Country
Poland
Facility Name
Investigational Site Number 6200001
City
Braga
ZIP/Postal Code
4710-243
Country
Portugal
Facility Name
Investigational Site Number 6430001
City
Moscow
ZIP/Postal Code
125367
Country
Russian Federation
Facility Name
Investigational Site Number 7240002
City
Barcelona
ZIP/Postal Code
08025
Country
Spain
Facility Name
Investigational Site Number 7240003
City
Barcelona
ZIP/Postal Code
08950
Country
Spain
Facility Name
Investigational Site Number 7560002
City
Zürich
ZIP/Postal Code
8091
Country
Switzerland
Facility Name
Investigational Site Number 1580001
City
Taipei
ZIP/Postal Code
10043
Country
Taiwan
Facility Name
Investigational Site Number 7920001
City
Ankara
ZIP/Postal Code
06100
Country
Turkey
Facility Name
Investigational Site Number 7920002
City
Istanbul
ZIP/Postal Code
34390
Country
Turkey
Facility Name
Investigational Site Number 8260005
City
Birmingham
ZIP/Postal Code
B15 2GW
Country
United Kingdom
Facility Name
Investigational Site Number 8260002
City
Cambridge
ZIP/Postal Code
CB2 OQQ
Country
United Kingdom
Facility Name
Investigational Site Number 8260001
City
London
ZIP/Postal Code
NW3 2QG
Country
United Kingdom
Facility Name
Investigational Site Number 8260004
City
Newcastle Upon Tyne
ZIP/Postal Code
NE1 4LP
Country
United Kingdom
Facility Name
Investigational Site Number 8260003
City
Salford
ZIP/Postal Code
M6 8HD
Country
United Kingdom

12. IPD Sharing Statement

Plan to Share IPD
Yes
IPD Sharing Plan Description
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
Citations:
PubMed Identifier
34800399
Citation
Diaz-Manera J, Kishnani PS, Kushlaf H, Ladha S, Mozaffar T, Straub V, Toscano A, van der Ploeg AT, Berger KI, Clemens PR, Chien YH, Day JW, Illarioshkin S, Roberts M, Attarian S, Borges JL, Bouhour F, Choi YC, Erdem-Ozdamar S, Goker-Alpan O, Kostera-Pruszczyk A, Haack KA, Hug C, Huynh-Ba O, Johnson J, Thibault N, Zhou T, Dimachkie MM, Schoser B; COMET Investigator Group. Safety and efficacy of avalglucosidase alfa versus alglucosidase alfa in patients with late-onset Pompe disease (COMET): a phase 3, randomised, multicentre trial. Lancet Neurol. 2021 Dec;20(12):1012-1026. doi: 10.1016/S1474-4422(21)00241-6. Erratum In: Lancet Neurol. 2022 Apr;21(4):e4.
Results Reference
derived

Learn more about this trial

Study to Compare the Efficacy and Safety of Enzyme Replacement Therapies Avalglucosidase Alfa and Alglucosidase Alfa Administered Every Other Week in Patients With Late-onset Pompe Disease Who Have Not Been Previously Treated for Pompe Disease

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