STUDY OF ORGN001 IN NEWBORNS, INFANTS, CHILDREN AND ADOLESCENTS WITH MOLYBDENUM COFACTOR DEFICIENCY (MOCD) TYPE A
- Conditions
- Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]Molydenum CoFactor Deficiency (MoCD) Type AMedDRA version: 20.1Level: PTClassification code 10069687Term: Molybdenum cofactor deficiencySystem Organ Class: 10010331 - Congenital, familial and genetic disorders
- Registration Number
- EUCTR2013-002702-30-NO
- Lead Sponsor
- Origin Biosciences, Inc.
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ot Recruiting
- Sex
- All
- Target Recruitment
- 8
Inclusion criteria for Primary Study :
- 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 compassionate use
- 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.
Inclusion criteria for Exploratory Study:
- Male or female pediatric patient (6 to 17 years of age [inclusive] at the time of ORGN001 administration) with MoCD Type A, previously untreated with ORGN001 or treated with ORGN001 through compassionate use.
- Parent or legal guardian must have signed the informed consent form (ICF) prior to any study procedures being performed.
- Female participants of childbearing potential and male participants of reproductive potential must agree to remain abstinent for the duration of the study and approximately 30 days after last dose of study drug.
- Female participants of childbearing potential must have a negative urine pregnancy test at Screening/Baseline.
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 for Primary Study :
- 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).
Exclusion criteria for Exploratory Study :
- Diagnosis other than MoCD Type A
- Condition that is considered by the treating physician to be a contraindication to therapy, including evidence of abnormalities on brain imaging not attibutable to MoCD Type A, or that might otherwise interfere with the patient's participation in the study, pse any additional risk for the patient, or confound patient assessments
- 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.
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;Secondary Objective: - To evaluate the effect of ORGN001 on MoCD Type A-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;Primary end point(s): Overall survival (OS);Timepoint(s) of evaluation of this end point: Through study completion.
- Secondary Outcome Measures
Name Time Method