Long-term Follow-up of People With Pompe Disease Switching Enzyme Treatment
Monitors people with Pompe disease who switched to avalglucosidase alfa from other enzyme treatment.
Plain-language title generated from the registry record
Registry title: Pompe Disease RWE Long-term Monitoring Program
Key facts
- Where
- Taipei, TaiwanSee the site
Where in Taiwan
- National Taiwan University HospitalTaipei, TaiwanTaking people
Questions to ask the study team
- How often do I need to come in, and for how long each time?
- Are travel costs paid?
- Can I keep my own doctor?
- What happens if I want to stop?
Trial details
- Who runs it
- National Taiwan University Hospital
- Trial code
- NCT07834320
- Record updated
- 22 September 2026
- Original description
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.Read full original descriptionShow less
Long Term Follow-up for Patients With Pompe Disease Undergoing Compassionate Use and Transitioning to Medication Listed Under the National Health Insurance.
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.
Inclusion: -Confirmed GAA enzyme deficiency (any tissue source) and/or confirmed 2 pathogenic GAA gene variants Pompe patient.
Exclusion: Having severe allergic reaction to avalglucosidase alfa; Patient/parent/legal guardian is not agreeing to provide signed informed consent, and the patient, if <18 years of age.