Clinical Trial: Study of ALXN1101 in Neonates With Molybdenum Cofactor Deficiency (MOCD) Type A
Study Status: Recruiting
Recruit Status: Recruiting
Study Type: Interventional
Official Title: A Phase 2/3, Multicenter, Multinational, Open Label Study to Evaluate the Efficacy and Safety of ALXN1101 in Neonates With Molybdenum Cofactor Deficiency (MOCD) Type A
Brief Summary: To evaluate the safety and efficacy of ALXN1101 in neonate patients with MoCD Type A
Detailed Summary:
Sponsor: Alexion Pharma GmbH
Current Primary Outcome: Response, defined as patients alive and able to sit upright independently for at least 30 seconds [ Time Frame: At 12 months ]
Original Primary Outcome: Ability to sit upright independently for at least 30 seconds [ Time Frame: At 12 months ]
Current Secondary Outcome:
- Bayley Scales of Infant Development® - Third Edition (Bayley - III®) [ Time Frame: First 12 months ]
- Pediatric Evaluation of Disability Inventory (PEDI) [ Time Frame: First 12 months ]
Original Secondary Outcome: Same as current
Information By: Alexion Pharma GmbH
Dates:
Date Received: December 3, 2015
Date Started: May 2016
Date Completion: September 2020
Last Updated: October 31, 2016
Last Verified: October 2016
