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avalglucosidase alfa

DRUG5 trials

Sponsors

Sanofi-Aventis Recherche & Developpement, Genzyme, a Sanofi Company, Sanofi, Iris Plug

Conditions

Glycogen Storage Disease Type IIGlycogen Storage Disease Type II Late OnsetGlycogen Storage Disease Type II Pompe DiseaseGlycogen storage disease type IIPompe DiseasePompe Disease (Late-onset)Pompe Disease Late-OnsetPompe's Disease

Phase 2

Avalglucosidase Alfa Extension Study
CompletedNCT02032524
Genzyme, a Sanofi CompanyGlycogen Storage Disease Type II Pompe Disease
Start: 2014-02-27End: 2022-12-12Updated: 2024-03-01

Phase 3

Clinical Study for Treatment-naïve IOPD Babies to Evaluate Efficacy and Safety of ERT With Avalglucosidase Alfa
Active, not recruitingNCT04910776
SanofiGlycogen Storage Disease Type II
Start: 2021-09-01End: 2027-08-10Updated: 2026-04-08
An Open-label, Multinational, Multicenter, Intravenous Infusion Study of the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of Avalglucosidase Alfa in Treatment-naïve Pediatric Participants with Infantile-Onset Pompe Disease (IOPD)
Active, not recruitingCTIS2024-513859-33-00
Sanofi-Aventis Recherche & DeveloppementGlycogen storage disease type II
Start: 2022-08-26Target: 8Updated: 2025-04-08

Phase 4

China Post-approval Commitment (PAC) Study of Avalglucosidase Alfa in Participants With IOPD
RecruitingNCT06666413
Genzyme, a Sanofi CompanyGlycogen Storage Disease Type II, Pompe's Disease
Start: 2025-05-07End: 2028-05-02Target: 13Updated: 2026-03-23
Safety and Efficacy of Avalglucosidase Alfa in Patients With Non-classic Pompe Disease Aged ≥ 5 Years
Active, not recruitingNCT07652814
Iris PlugGlycogen Storage Disease Type II Late Onset, Pompe Disease, Pompe Disease (Late-onset) +2
Start: 2022-10-13End: 2028-03-09Target: 6Updated: 2026-06-17

Related Papers

An Indirect Treatment Comparison of Avalglucosidase Alfa versus Cipaglucosidase Alfa Plus Miglustat in Patients with Late-Onset Pompe Disease.
2025-09-08
Efficacy of avalglucosidase alfa on forced vital capacity percent predicted in treatment-naïve patients with late-onset Pompe disease: A pooled analysis of clinical trials.
2024-06-264 citations
Population pharmacokinetic modeling and dosing simulation of avalglucosidase alfa for selecting alternative dosing regimen in pediatric patients with late-onset pompe disease
Journal of Pharmacokinetics and Pharmacodynamics2023-08-034 citations
Long-term Safety and Efficacy of Avalglucosidase Alfa in Patients With Late-Onset Pompe Disease
Neurology2022-06-0242 citations
NEO1 and NEO-EXT Studies: Long-Term Safety and Exploratory Efficacy of Repeat Avalglucosidase Alfa Dosing for 5.5 Years in Late-Onset Pompe Disease Patients (695)
Neurology2020-04-143 citations