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

• Status: Active, not recruiting
• Trial end date: 2021-12-01
• Target enrollment: 0
• Participant gender: All

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.

• Phase: Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Alexion Pharma GmbH; Origin Biosciences
• Treatments: Molybdenum

Criteria

Inclusion Criteria:

• Male or female patients with a genetically confirmed diagnosis of MoCD Type A (MOCS1 mutation)

• Currently treated with rcPMP infusions

Exclusion Criteria:

• Current or planned treatment with another investigational drug or device, with the exception rcPMP treatment through Day -1.