A Long-term Study for the Outcome of Pompe Disease
Recruiting
- Conditions
- Pompe Disease
- Registration Number
- NCT02399748
- Lead Sponsor
- National Taiwan University Hospital
- Brief Summary
Prospectively follow patients with Pompe disease underwent enzyme replacement therapy.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- RECRUITING
- Sex
- All
- Target Recruitment
- 50
Inclusion Criteria
Confirm diagnosis with Pompe disease, is or is preparing receiving enzyme replacement therapy
Exclusion Criteria
No
Study & Design
- Study Type
- OBSERVATIONAL
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method all cause morbidities 10 years
- 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 are the long-term molecular outcomes of enzyme replacement therapy in Pompe disease patients enrolled in NCT02399748?
How does enzyme replacement therapy in NCT02399748 compare to standard-of-care treatments for Pompe disease in terms of efficacy and safety?
What biomarkers are associated with improved response to enzyme replacement therapy in Pompe disease as observed in NCT02399748?
What adverse events have been reported in long-term enzyme replacement therapy studies for Pompe disease, including NCT02399748?
Are there emerging therapies or combination approaches targeting acid alpha-glucosidase deficiency in Pompe disease beyond enzyme replacement therapy?
Trial Locations
- Locations (1)
National Taiwan University Hospital
🇨🇳Taipei, Taiwan
National Taiwan University Hospital🇨🇳Taipei, TaiwanYin-Hsiu Chien, MD, PhDContact+886223123456chienyh@ntu.edu.tw