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)
Phase of Trial: Phase II
Latest Information Update: 18 Nov 2019
Price : $35 *
At a glance
- Drugs Cyclic pyranopterin monophosphate (Primary)
- Indications Molybdenum cofactor deficiency
- Focus Adverse reactions
- Sponsors Alexion Pharmaceuticals; Origin Biosciences
- 16 Nov 2019 Planned End Date changed from 1 Jun 2020 to 1 Dec 2020.
- 16 Nov 2019 Planned primary completion date changed from 1 Jun 2020 to 1 Dec 2020.
- 16 Nov 2019 Planned number of patients changed from 7 to 8.