NCT07834320Recruiting
Pompe Disease RWE Long-term Monitoring Program
Long Term Follow-up for Patients With Pompe Disease Undergoing Compassionate Use and Transitioning to Medication Listed Under the National Health Insurance.
Source: ClinicalTrials.gov · Last updated 22 Sept 2026
Brief summary (registry, in English)
Evaluate short- and long-term biomarker, motor function, respiratory function and safety with avalglucosidase alfa in IOPD and LOPD patients who switched from other ERT.
- Status (registry)
- Recruiting
- Study type
- Observational
- Enrollment
- 40 (estimated)
- First submitted
- 19 Apr 2024
- Start date
- Oct 2023
- Primary completion date
- Oct 2036
- Lead sponsor
- National Taiwan University Hospital
- Diseases
- Pompe disease
- NCT number
- NCT07834320
What you commit to
- Study type: Observational
- Planned participants: 40
- From Oct 2023 to the primary completion date, Oct 2036
- 1 site in 1 country
- Visits and tests: ask the trial team, the registry does not list them
Built from the registry fields of ClinicalTrials.gov. It describes the study, not your situation.
Who cannot take part
Copied verbatim from ClinicalTrials.gov and grouped by topic. Only the trial team decides who can take part.
Other illness
- Having severe allergic reaction to avalglucosidase alfa.
Age
- Patient/parent/legal guardian is not agreeing to provide signed informed consent, and the patient, if \<18 years of age
Who the study is looking for
- -Confirmed GAA enzyme deficiency (any tissue source) and/or confirmed 2 pathogenic GAA gene variants Pompe patient
Sex: All · Does not accept healthy volunteers
Sites (1)
Sites in registry order. Pick your city on the disease page to sort them by distance.
| Site | City | Country | Site status |
|---|---|---|---|
| National Taiwan University Hospital Coordinator of this site? Confirm its status | Taipei | Taiwan | Recruiting |
This page is information, not medical advice. Discuss any trial with your doctor.