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GSDIa Disease Monitoring Program
jRCT2053260130Active, not recruiting
- Sponsor
- Ultragenyx Pharmaceutical Inc.
- Start
- Sep 2025
- Modality
- Gene Therapies
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Overview
Open-label trial of pariglasgene brecaparvovec in glycogen storage disease type 1a (GSD1a) (2 arms), sponsored by Ultragenyx Pharmaceutical Inc. Primary endpoints: Incidence and Severity of Serious Adverse Events (SAEs) Related to DTX401, Adverse Events of Special Interest (AESIs) for AAV Therapies and 4 more. Started Sep 2025; status: active, not recruiting.
02
Drugs
pariglasgene brecaparvovec
03
Conditions
glycogen storage disease type 1a (GSD1a)
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