STUDY OF ORGN001 IN NEWBORNS, INFANTS AND CHILDREN WITH MOLYBDENUM COFACTOR DEFICIENCY (MOCD) TYPE A

Phase 1

• Conditions: Molydenum CoFactor Deficiency (MoCD) Type A; MedDRA version: 20.1 Level: PT Classification code 10069687 Term: Molybdenum cofactor deficiency System Organ Class: 10010331 - Congenital, familial and genetic disorders; Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]

• Registration Number: EUCTR2013-002702-30-GB
• Lead Sponsor: Origin Biosciences, Inc.

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2015-11-10
• Last Update Posted: 11 March 2020

Recruitment & Eligibility

• Status: Authorised-recruitment may be ongoing or finished
• Sex: Not specified
• Target Recruitment: 10

Inclusion Criteria

-Male or female neonatal patients (1 to 28 days of age [inclusive] at the time of ORGN001 administration, with day 1 of age corresponding to the day of birth) or infant (29 days to <2 years of age) or child (2 to 5 years of age [inclusive]) with MoCD Type A, previously untreated with ORGN001 or treated with ORGN001 through the EAP
-In neonates, diagnosis of MoCD Type A, based on
o Prenatal genetic diagnosis, or
o Onset of clinical and/or laboratory signs and symptoms consistent with MoCD Type A within the first 28 days after birth
- In infants or children, diagnosis of MoCD Type A, based on:
o Confirmed genetic diagnosis (genetic confirmation of the diagnosis of MoCD Type A may be obtained after initiation of ORGN001 therapy in certain cases), biochemical profile, and clinical presentation consistent with MoCD Type A
-Parent or legal guardian must have signed the informed consent form (ICF) prior to any study procedures being performed.
Are the trial subjects under 18? yes
Number of subjects for this age range: 10
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

-Diagnosis other than MoCD Type A (may be determined after the initiation of study drug)
-Condition that is considered by the treating physician to be a contraindication to therapy, including evidence of abnormalities on brain imaging not attributable to MoCD Type A, or that might otherwise interfere with the patient’s participation in the study, pose any additional risk for the patient, or confound patient assessments
-Antenatal and/or postnatal brain imaging prior to initiation of treatment with ORGN001 that indicates cortical or subcortical cystic encephalomalacia, clinically significant intracranial hemorrhage, or other abnormalities on brain imaging determined by the treating physician to be clinically significant
-Modified Glasgow Coma Scale (mGCS) for Infants and Children score of less than 7 for more than 24 hours (does not apply to children less than 1 day of age).

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Main Objective: To evaluate the safety and efficacy of ORGN001 in neonate, infant , and pediatric patients with MoCD Type A who are either treatment-naïve or who have received compassionate use ORGN001;<br> Secondary Objective: - To evaluate the effect of ORGN001 on MoCD-associated urine and blood biomarker concentrations<br> - To evaluate the effect of ORGN001 on growth and development using age-appropriate assessments<br> - To evaluate the effect of ORGN001 on pediatric measures of functional ability and activities of daily living<br> - To characterize the pharmacokinetics (PK) of ORGN001 and the impact on pharmacodynamic (PD) biomarkers<br> ;Primary end point(s): Overall survival (OS);Timepoint(s) of evaluation of this end point: Through study completion.