NCT02047461

Safety & Efficacy Study of ORGN001 (Formerly ALXN1101) in Pediatric Patients With MoCD Type A Currently Treated With rcPMP

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: Phase 2
• Study type: Interventional
• Enrollment: 8 (actual)
• Sponsor: Origin Biosciences · Industry
• Sex: All
• Age: —
• Healthy volunteers: Not accepted

Summary

This study will include a screening period, a 6-month treatment period, followed by long-term extension period expected to last approximately 72 months.

Detailed description

Patients will receive daily IV infusions of ORGN001 (formerly ALXN1101) starting on Day 1. After a prescribed period, dosing will increase monthly based on defined patient safety measures. After Month 6, patients will continue daily dosing at their last tolerated dose.

Conditions

• Molybdenum Cofactor Deficiency, Type A

Interventions

Type	Name	Description
DRUG	ORGN001 (formerly ALXN1101)	IV infusion

Timeline

• Start date: 2014-04-01
• Primary completion: 2022-08-01
• Completion: 2022-10-01
• First posted: 2014-01-28
• Last updated: 2023-10-17
• Results posted: 2023-10-17

Locations

6 sites across 5 countries: United States, Australia, Netherlands, Tunisia, United Kingdom