Study Status: Active, not recruiting
Recruit Status: Active, not recruiting
Study Type: Interventional
Official Title: A Phase 2, Multicenter, Multinational, Open-Label, Dose-Escalation Study to Evaluate the Safety and Efficacy of ALXN1101 in Pediatric Patients With Molybdenum Cofactor Deficiency
Brief Summary: This study will include a screening period, a 6-month treatment period, and a 60-month, long-term extension period.