An Exploratory Study of the Safety and Efficacy of Prophylactic Immunomodulatory Treatment in Myozyme-naive Cross-Reacting Immunologic Material (CRIM[-]) Patients With Infantile-Onset Pompe Disease
PHASE4
The purpose of this study was to evaluate the efficacy, clinical benefits a...
A Study to Assess the Safety of Myozyme® and of Aldurazyme® in Male and Female Participants of Any Age Group With Pompe Disease or With Mucopolysaccharidosis Type I (MPS I) in a Home-care Setting
N/A
Primary objective:
To obtain data pertaining to the safety and tolerabilit...
Status
Completed
Location
Investigational Site Italy
Start Date
2021-10-14
Trial ID
NCT05073783
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
PHASE2
This is a multi-stage, phase 2, open-label, multicenter, multinational, asc...
Status
Active Not Recruiting
Location
Regional Medical Genetics Center of New York Site Number : 8400002