Taking people in TaiwanNo phase
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.
Key facts
- Who runs it
- National Taiwan University Hospital
- Where
- Taipei, TaiwanSee the site
- Trial code
- Registry title
- Pompe Disease RWE Long-term Monitoring Program
- Status
- Recruiting
- People taking part
- 40 people, planned
- Started
- 1 Oct 2023
- Expected to end
- 1 Oct 2036
- Type of study
- Observational
- Record updated
- 22 September 2026
Who can take part
AI summary
From the trial's eligibility criteria. Machine-read from the registry text on this page, so it can be wrong. The registry text below and the trial team decide who can take part.
The trial is looking for people with Pompe disease who are moving to avalglucosidase alfa from another enzyme replacement therapy.
- Sex
- All sexes can join.
- Disease stage
- Pompe disease, infantile-onset (IOPD) or late-onset (LOPD).
- Earlier treatment
- Switched from other enzyme replacement therapy (ERT) to avalglucosidase alfa.
- Biomarker or test result
- Confirmed GAA enzyme deficiency (any tissue source) and/or 2 confirmed pathogenic GAA gene variants.
Main reasons for not taking part
- Severe allergic reaction to avalglucosidase alfa
- No signed informed consent from the patient, parent or legal guardian
- No signed informed consent from the patient, if under 18 years of age
The registry criteria, word for word
Show all 3 criteriaShow less
Who can take part
- -Confirmed GAA enzyme deficiency (any tissue source) and/or confirmed 2 pathogenic GAA gene variants Pompe patient
Who cannot take part
- 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
Where in Taiwan
- National Taiwan University HospitalTaipei, TaiwanContact: Yin-Hsiu Chien, MD, PhDTaking people
About the trial
What the trial wants to find out
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.
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?
Contact the study team: see the registry record → Contacts and Locations
Print a page for my doctorData and sources
- Registry record
- Listed on ClinicalTrials.gov.
- Official title
- Long Term Follow-up for Patients With Pompe Disease Undergoing Compassionate Use and Transitioning to Medication Listed Under the National Health Insurance.
- Plain-language title
- Long-term Follow-up of People With Pompe Disease Switching Enzyme TreatmentGenerated from the registry record. The registry title is the official one.
- AI summary
- From the trial's eligibility criteria. Read by machine on 12 November 2026.
- Record updated
- 22 September 2026