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Phase 3 randomized double-blind placebo-controlled study of DTX401 gene therapy in glycogen storage disease type Ia
2020-004184-12Phase 3Completed
- Sponsor
- Ultragenyx Pharmaceutical Inc.
- Start
- Aug 2021
- Modality
- Gene Therapies
- Countries
- 1
01
Overview
Phase 3, randomised, double-blind trial in glycogen storage disease type 1a (GSD1a) comparing DTX401 gene therapy with Placebo (2 arms). Primary endpoint: Percent change in daily cornstarch intake at Week 48. Started Aug 2021; status: completed.
02
Drugs
pariglasgene brecaparvovec
03
Conditions
glycogen storage disease type 1a (GSD1a)
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