ATB200/AT2221 Phase 3 Open-label Extension Study
Phase 3
- Conditions
- Pompe disease
- Registration Number
- JPRN-jRCT2011200003
- Lead Sponsor
- Wright Jacquelyn
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ot Recruiting
- Sex
- All
- Target Recruitment
- 110
Inclusion Criteria
Subject must have completed Study ATB200-03
Exclusion Criteria
1. Subject plans to receive gene therapy or participate in another interventional study for Pompe disease.
2. Subject, if female, is pregnant or breastfeeding.
3. Subject, whether male or female, is planning to conceive a child during the study.
Study & Design
- Study Type
- Interventional
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Proportion of participants with Treatment Emergent Adverse Events (TEAE)
- Secondary Outcome Measures
Name Time Method Change from baseline in 6MWD, sitting FVC (% predicted), the manual muscle test score, physical function, fatigue score and et.al