This trial is active, not recruiting.

Condition molybdenum cofactor deficiency, type a
Treatment alxn1101
Phase phase 2
Sponsor Alexion Pharma GmbH
Start date April 2014
End date December 2015
Trial size 7 participants
Trial identifier NCT02047461, 2013-002701-56, ALXN1101-MCD-201


This study will include a screening period, a 6-month treatment period, and a 30-month, long-term extension period.

United States No locations recruiting
Other countries No locations recruiting

Study Design

Endpoint classification safety/efficacy study
Intervention model single group assignment
Masking open label
Primary purpose treatment
daily IV infusions
IV infusion

Primary Outcomes

Safety of ALXN1101
time frame: First 6 months of treatment

Secondary Outcomes

Pharmacokinetics parameters of ALXN1101
time frame: First 6 months of treatment
Effect of ALXN1101 on urine and blood biomarkers
time frame: Up to Month 36
Effect of ALXN1101 on neurologic function including motor examination
time frame: Up to Month 36
Long-term safety of ALXN1101
time frame: Up to Month 36
Effect of ALXN1101 on cognitive functions
time frame: Up to Month 36

Eligibility Criteria

Male or female participants of any age.

Inclusion Criteria: - Male or female patients with a genetically confirmed diagnosis of MoCD Type A (MOCS1 mutation) and who are 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.

Additional Information

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 (MoCD) Type A Currently Treated With Recombinant Escherichia Coli-derived Cyclic Pyranopterin Monophosphate (rcPMP)
Description Patients will receive daily IV infusions of 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 through Month 36 at their last tolerated dose.
Trial information was received from ClinicalTrials.gov and was last updated in February 2016.
Information provided to ClinicalTrials.gov by Alexion Pharma GmbH.