Safety & Efficacy Study of ALXN1101 in Pediatric Patients With MoCD Type A Currently Treated With rcPMP
| Status: | Active, not recruiting |
|---|---|
| Healthy: | No |
| Age Range: | Any |
| Updated: | 3/21/2019 |
| Start Date: | April 2014 |
| End Date: | June 2020 |
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)
This study will include a screening period, a 6-month treatment period, and a 60-month,
long-term extension period.
long-term extension period.
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 66 at their last tolerated dose.
period, dosing will increase monthly based on defined patient safety measures. After Month 6,
patients will continue daily dosing through Month 66 at their last tolerated dose.
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.
We found this trial at
2
sites
9000 W Wisconsin Ave #270
Milwaukee, Wisconsin 53226
Milwaukee, Wisconsin 53226
(414) 266-2000
Children's Hospital of Wisconsin Nothing matters more than our children. At Children's Hospital of Wisconsin,...
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