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A Phase 2, Multicenter, Multinational, Open-Label, Dose-Escalation Study to Evaluate the Safety and Efficacy of ORGN001 (Formerly ALXN1101) in Pediatric Patients With Molybdenum Cofactor Deficiency (MoCD) Type A Currently Treated With Recombinant Escherichia Coli-derived Cyclic Pyranopterin Monophosphate (rcPMP)
Conditions
Interventions
ORGN001 (formerly ALXN1101)
Locations
6
United States
Children's Hospital of Wisconsin
Milwaukee, Wisconsin, United States
Monash Medical Centre
Melbourne, Australia
Beatrix Children's Hospital
Groningen, Netherlands
Unité des maladies métaboliques
Tunis, Tunisia
Royal Hospital for Sick Children
Glasgow, United Kingdom
Manchester University Hospitals NHS Foundation Trust
Manchester, United Kingdom
Start Date
April 1, 2014
Primary Completion Date
August 1, 2022
Completion Date
October 1, 2022
Last Updated
October 17, 2023
Lead Sponsor
Origin Biosciences
Data Source & Attribution
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