avalglucosidase alfa
avalglucosidase alfa: 5 clinical trials tracked.
avalglucosidase alfa evidence translation
What we can confirm
5 tracked trials
What it means
What to watch
Prepared research question
What is verified about avalglucosidase alfa, and what remains unknown?
Traceable citations · Unknowns marked
See linked entities and source evidence1 indications · 1 study sponsors
Related indications
Study sponsors
Clinical Trial Registry Records (5)
A Single-arm, Open-label, Phase 4 Study to Evaluate the Safety and Efficacy of Avalglucosidase Alfa in Chinese Participants With Infantile-onset Pompe Disease (IOPD)
Primary endpoint: Incidence of AEs, SAEs, AESIs including IARs during the TE period
An Open Label, Single Center, Exploratory Study of the Safety and Efficacy of Avalglucosidase Alfa Patients With Non-classic Pompe Disease Aged ≥ 5 Years
Primary endpoint: Incidence/occurrence of adverse events
A French Multicenter Open Label Phase 4 Extension Study of Long-term Safety and Efficacy in Patients With Pompe Disease Who Previously Participated in Avalglucosidase Alfa Development Studies in France
Primary endpoint: Number of participants with adverse events (AE), treatment-emergent adverse events (TEAE), including infusion associated reactions (IAR) and death
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)
Primary endpoint: Proportion of participants who are alive and free of invasive ventilation at Week 52
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
Primary endpoint: PAP: Number of Participants With Treatment-emergent Adverse Events (TEAEs), Serious Treatment-emergent Adverse Events, and Adverse Event of Special Interest (AESI)