Safety and efficacy of ORGN001 in pediatric patients with Molybdenum Cofactor Deficiency Type A
Phase 1
- Conditions
- Molybdenum Cofactor Deficiency (MoCD) Type AMedDRA version: 20.1 Level: PT Classification code 10069687 Term: Molybdenum cofactor deficiency System Organ Class: 10010331 - Congenital, familial and genetic disordersTherapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]
- Registration Number
- EUCTR2013-002701-56-GB
- Lead Sponsor
- Origin Biosciences, Inc.
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Authorised-recruitment may be ongoing or finished
- Sex
- Not specified
- Target Recruitment
- 8
Inclusion Criteria
- Male or female patients with a genetically confirmed diagnosis of MoCD Type A (MOCS1 mutation)
- Currently treated with rcPMP infusions
Are the trial subjects under 18? yes
Number of subjects for this age range: 8
F.1.2 Adults (18-64 years) no
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range
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 clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Main Objective: safety;Secondary Objective: PK, long-term safety and efficacy;Primary end point(s): safety;Timepoint(s) of evaluation of this end point: Trial duration
- Secondary Outcome Measures
Name Time Method <br> Secondary end point(s): - PK parameters <br> - Change from baseline in urine and blood SSC levels<br> - Change from baseline in clinical findings from neurologic examination, cognitive and motor assessment, seizure frequency, neuroimaging, growth parameters and feeding patterns<br> ;Timepoint(s) of evaluation of this end point: Trial duration