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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)

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Trial Profile

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)

Status: Completed
Phase of Trial: Phase II

Latest Information Update: 14 Apr 2025

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At a glance

  • Drugs Fosdenopterin (Primary)
  • Indications Molybdenum cofactor deficiency
  • Focus Adverse reactions
  • Sponsors Alexion AstraZeneca Rare Disease; Origin Biosciences

Most Recent Events

  • 02 Apr 2025 According a Sentynl Therapeutics media release, the company has published NULIBRY's three clinical studies comparing the treatment effect of NULIBRY versus a natural history study in the Journal of Inherited Metabolic Disease.
  • 16 Apr 2024 According a Sentynl Therapeutics media release, based on data from three studies, company today announced Medicines and Healthcare products Regulatory Agency (MHRA) authorization of NULIBRY (fosdenopterin) for Injection as the first therapy for the treatment of patients in Great Britain (GB) with molybdenum cofactor deficiency (MoCD) Type A.
  • 13 Oct 2022 Status changed from active, no longer recruiting to completed.

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