An Open-Label, Multicenter, Multinational, Study of the Safety, Efficacy,Pharmacokinetics, and Pharmacodynamics of Recombinant Human Acid alpha-Glucosidase (rhGAA) Treatment in Patients > 6 and <= 36 Months Old with Infantile-OnsetPompe Disease (Glycogen Storage Disease Type II)
Active, not recruiting
- Conditions
- Treatment for Type II GlycogenosisMedDRA version: 6.1Level: PTClassification code 10053185
- Registration Number
- EUCTR2004-002168-59-IT
- Lead Sponsor
- GENZYME
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Not Recruiting
- Sex
- All
- Target Recruitment
- 20
Inclusion Criteria
Are the trial subjects under 18? yes
Number of subjects for this age range:
F.1.2 Adults (18-64 years) no
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range
Exclusion Criteria
Not provided
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Main Objective: ;Secondary Objective: ;Primary end point(s):
- Secondary Outcome Measures
Name Time Method
Related Research Topics
Explore scientific publications, clinical data analysis, treatment approaches, and expert-compiled information related to the mechanisms and outcomes of this trial. Click any topic for comprehensive research insights.
What molecular mechanisms of rhGAA therapy impact lysosomal function in infantile-onset Pompe disease?
How does rhGAA compare to standard enzyme replacement therapies for Glycogen Storage Disease Type II in pediatric populations?
Which biomarkers correlate with treatment response in EUCTR2004-002168-59-IT rhGAA clinical trials for Pompe disease?
What are the most common adverse events associated with rhGAA treatment in infants and toddlers with Pompe disease?
Are there any combination therapies or alternative enzyme replacement strategies being explored for infantile-onset Pompe disease alongside Genzyme's rhGAA?