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Phase 3 study of pariglasgene brecaparvovec versus placebo in glycogen storage disease type Ia
NL-OMON58793Phase 3Completed
- Sponsor
- Ultragenyx Pharmaceutical Inc.
- Start
- May 2022
- Modality
- Gene Therapies
- Countries
- 1
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Overview
Phase 3, randomised, double-blind trial in glycogen storage disease type 1a (GSD1a) comparing Pariglasgene brecaparvovec with Placebo (2 arms). Primary endpoint: Percent change in daily cornstarch intake. Started May 2022; status: completed.
02
Drugs
pariglasgene brecaparvovec
03
Conditions
glycogen storage disease type 1a (GSD1a)
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