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Clinical Trials in Taiwan / NCT07834320
Recruiting Observational

Pompe Disease RWE Long-term Monitoring Program

NCT07834320 · tracked via the Priya Life Science Taiwan tracker
Sponsor
National Taiwan University Hospital
Phase
Observational
Started
2023-10-01
Last updated
2026-09-22

Condition(s) studied

Pompe Disease

Study summary

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.

Eligibility

Sex
ALL
Min age
—
Max age
—
Healthy volunteers
No
Inclusion criteria: -Confirmed GAA enzyme deficiency (any tissue source) and/or confirmed 2 pathogenic GAA gene variants Pompe patient Exclusion criteria: * 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

Primary outcome measure(s)

Trial sites (1)

FacilityCityRegionStatus
National Taiwan University Hospital Taipei Taiwan Recruiting

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Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT07834320 on ClinicalTrials.gov ↗ ← All trials in Taiwan