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Phase 2/3 study of ALXN1101 in neonates with molybdenum cofactor deficiency type A
2013-002702-30Phase 2Completed
- Sponsor
- Origin Biosciences, Inc.
- Start
- Nov 2015
- Modality
- Metabolically Activated Prodrugs → Prodrugs/Conditional Activator Small Molecules → Small Molecules, Recombinant Proteins and Enzymes
- Countries
- 1
01
Overview
Phase 2, open-label, single-arm trial of fosdenopterin in molybdenum cofactor deficiency (MoCD), sponsored by Origin Biosciences, Inc. Primary endpoint: Response (survival and independent sitting). Started Nov 2015; status: completed.
02
Drugs
fosdenopterin
03
Conditions
molybdenum cofactor deficiency (MoCD)
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