A Phase 2, Multicenter, Multinational, Open-Label, Dose-Escalation Study to Evaluate the Safety and Efficacy of ORGN001 (formerly ALXN1101) in Pediatric Patients with Molybdenum Cofactor Deficiency (MoCD) Type A Currently Treated with Recombinant Escherichia coli-derived Cyclic Pyranopterin Monophosphate (rcPMP)
Phase 2
Recruiting
- Conditions
- and aldehyde oxidaseCombined deficiency of sulfite oxidasexanthine dehydrogenase10027664
- Registration Number
- NL-OMON55625
- Lead Sponsor
- Origin Biosciences, Inc.
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Recruiting
- Sex
- Not specified
- Target Recruitment
- 2
Inclusion Criteria
1) Male of female patients with genetically confirmed diagnosis of MoCD Type A
(MOCS1 mutation) and who are currently treated with rcPMP infusions.
2) Parent or legal guardian must have signed the informed consent form (ICF)
prior to any study procedures.
Exclusion Criteria
Current or planned treatment with another investigational drug or device, with
the exception of rcPMP treatment through Day -1
Study & Design
- Study Type
- Interventional
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method <p>Safety over the first 6 months of treatment</p><br>
- Secondary Outcome Measures
Name Time Method