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

Molybdenum Cofactor Deficiency, Type A Clinical Trial

Official title: 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

Clinical Trial 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.

Clinical Trial 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. ;

Related Conditions & MeSH terms

• Molybdenum Cofactor Deficiency, Type A

• NCT number: NCT02047461
• Study type: Interventional
• Source: Origin Biosciences
• Status: Completed
• Phase: Phase 2
• Start date: April 2014
• Completion date: October 2022