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
Conditions
Sponsors
Origin Biosciences, Inc.
Eligibility
Sex/Gender
All
Inclusion criteria
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 =65 years) no F.1.3.1 Number of subjects for this age range
Exclusion criteria
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).
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| 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; Secondary Objective: - To evaluate the effect of ORGN001 on MoCD-associated urine and blood biomarker concentrations - To evaluate the effect of ORGN001 on growth and development using age-appropriate assessments - To evaluate the effect of ORGN001 on pediatric measures of functional ability and activities of daily living - To characterize the pharmacokinetics (PK) of ORGN001 and the impact on pharmacodynamic (PD) biomarkers ;Primary end point(s): Overall survival (OS);Timepoint(s) of evaluation of this end point: Through study completion. | — |
Secondary
| Measure | Time frame |
|---|---|
| Timepoint(s) of evaluation of this end point: through Month 12; Secondary end point(s): - Changes in MoCD-related biomarkers (SSC, xanthine, uric acid in plasma and urine) - Changes in growth parameters (height, weight, body mass index [BMI], head circumference) - Feeding pattern assessments - Gross Motor Function Classification System Expanded and Revised (GMFCS-E&R) results - Assessments of the Bayley Scales of Infant Development® – Third Edition (Bayley – III®) Cognitive and Motor Scales as measured o For children aged 3 and above, for whom the Bayley-III is no longer appropriate, the Wechsler Preschool and Primary Scale of Intelligence – Fourth Edition (WPPSI-IV) will be administered. o For patients with severe developmental delay, the WPPSI may not be an appropriate assessment, and therefore, the Bayley-III may be administered instead. - functionnal ability and activities of daily living, measured by the Pediatric Evaluation of Disability Inventory (PEDI) - Gross Motor Function Measure (GMFM)-88 results | — |
Countries
Argentina, France, Germany, Israel, Italy, Saudi Arabia, Spain, Turkey, United Kingdom, United States
Contacts
Public ContactHead of Clinical Operations
Origin Biosciences, Inc.
Outcome results
None listed