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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
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).
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.
Age
3 - No limit years
Sex
ALL
Healthy Volunteers
No
Investigational Site Number 8400015
Phoenix, Arizona, United States
Investigational Site Number 8400020
Los Angeles, California, United States
Investigational Site Number 8400011
Orange, California, United States
Investigational Site Number 8400017
Stanford, California, United States
Investigational Site Number 8400016
Gainesville, Florida, United States
Investigational Site Number 8400007
Decatur, Georgia, United States
Investigational Site Number 8400023
Chicago, Illinois, United States
Investigational Site Number 8400002
Iowa City, Iowa, United States
Investigational Site Number 8400012
Kansas City, Kansas, United States
Investigational Site Number 8400010
Boston, Massachusetts, United States
Start Date
November 2, 2016
Primary Completion Date
March 19, 2020
Completion Date
May 31, 2023
Last Updated
April 4, 2024
101
ACTUAL participants
Avalglucosidase alfa (GZ402666)
DRUG
Alglucosidase alfa (GZ419829)
DRUG
Lead Sponsor
Genzyme, a Sanofi Company
Data Source & Attribution
This clinical trial information is sourced from ClinicalTrials.gov, a service of the U.S. National Institutes of Health.
Modifications: This data has been reformatted for display purposes. Eligibility criteria have been parsed into inclusion/exclusion sections. Location data has been geocoded to enable distance-based search. For the authoritative and most current information, please visit ClinicalTrials.gov.
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