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Pegzilarginase in Infants and Young Children With Arginase 1 Deficiency
2024-510797-25-00Phase 3Completed
- Sponsor
- Immedica
- Start
- Dec 2024
- Modality
- Replacement Enzymes → Therapeutic Enzymes → Recombinant Proteins and Enzymes
- Countries
- 3
01
Overview
Phase 3, open-label, single-arm trial of pegzilarginase in arginase 1 deficiency (ARG1-D), sponsored by Immedica. Primary endpoint: Change from baseline in plasma arginine after 12 weeks of pegzilarginase treatment. Started Dec 2024; status: completed.
02
Drugs
pegzilarginase
03
Conditions
arginase 1 deficiency (ARG1-D)
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