Pompe Disease RWE Long-term Monitoring Program

Sponsor
National Taiwan University Hospital
Study ID
NCT07834320
Status
Recruiting
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Conditions

Eligibility Criteria

Sex
ALL
Age
N/A - N/A
Healthy Volunteers
Not accepted

Study Details

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.

Key Dates

First listed
Sep 22, 2026
Start date
Oct 1, 2023
Status verified
Jan 2026
Primary completion
Oct 1, 2036
Completion
Oct 1, 2036

Study Design

Enrollment
40 participants (estimated)

Primary Outcome Measure

Urinary Glc4/Hex4 [ Time Frame: 3years ]

Central Contacts

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