Taking people in TaiwanNo phase
Long-term follow-up of people with Pompe disease receiving enzyme replacement therapy
Follows people with Pompe disease who receive enzyme replacement therapy over time.
Key facts
- Who runs it
- National Taiwan University Hospital
- Where
- Taipei, TaiwanSee the site
- Trial code
- Registry title
- A Long-term Study for the Outcome of Pompe Disease
- Status
- Recruiting
- People taking part
- 50 people, planned
- Started
- Apr 2007
- Expected to end
- Dec 2026
- Type of study
- Observational
- Record updated
- 18 July 2017
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 a confirmed diagnosis of Pompe disease who are on enzyme replacement therapy or getting ready to start it.
- Sex
- All sexes can join.
- Disease stage
- Confirmed Pompe disease.
- Earlier treatment
- Currently receiving enzyme replacement therapy, or preparing to receive it.
The registry criteria, word for word
Show all 2 criteriaShow less
Who can take part
- Confirm diagnosis with Pompe disease, is or is preparing receiving enzyme replacement therapy
Who cannot take part
- No
Where in Taiwan
- National Taiwan University HospitalTaipei, TaiwanContact: Yin-Hsiu Chien, MD, PhDTaking people
About the trial
What the trial wants to find out
Prospectively follow patients with Pompe disease underwent enzyme replacement therapy.
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
- A Long-term Study for the Outcome of Pompe Disease
- Plain-language title
- Long-term follow-up of people with Pompe disease receiving enzyme replacement therapyGenerated 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
- 18 July 2017