Recruiting NCT02399748
A Long-term Study for the Outcome of Pompe Disease
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- This is an observational study: the protocol does not assign a study treatment.
- Who it may be relevant to
- Registry conditions: Pompe Disease. Basic parameters: No limits · All.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- Taiwan
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
Unsure about the terms? Read our patient guide →
Overview
Prospectively follow patients with Pompe disease underwent enzyme replacement therapy.
Primary outcome measures
- all cause morbidities [Time frame: 10 years]
Eligibility criteria
Inclusion criteria
Confirm diagnosis with Pompe disease, is or is preparing receiving enzyme replacement therapy
Exclusion criteria
No
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: No
Study design
- Observational model
- Case-only
Study locations
Taiwan · 1 center
- National Taiwan University Hospital — Taipei
Publications
- Hsu YK, Chien YH, Shinn-Forng Peng S, Hwu WL, Lee WT, Lee NC, Po-Yu Huang E, Weng WC. Evaluating brain white matter hyperintensity, IQ scores, and plasma neurofilament light chain concentration in early-treated patients with infantile-onset Pompe disease. Genet Med. 2023 Jan;25(1):27-36. doi: 10.1016/j.gim.2022.10.005. Epub 2022 Nov 18. PMID 36399131
Identifiers
NCT: NCT02399748 · 200703045R