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DTX401 long-term disease monitoring program in glycogen storage disease type Ia
NL-OMON58788Phase 4Recruiting
- Sponsor
- Ultragenyx Pharmaceutical Inc.
- Start
- Nov 2024
- Modality
- Gene Therapies
- Countries
- 1
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Overview
Phase 4, 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 related to DTX401, Adverse events of special interest for AAV therapies and 3 more. Started Nov 2024; status: recruiting.
02
Drugs
pariglasgene brecaparvovec
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Conditions
glycogen storage disease type 1a (GSD1a)
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