Study of ORGN001 (Formerly ALXN1101) in Neonates, Infants and Children With Molybdenum Cofactor Deficiency (MOCD) Type A

Study Overview
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Brief Summary
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To evaluate the safety and efficacy of ORGN001(formerly ALXN1101) in neonate patients with MoCD Type A

Official Title
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A Phase 2/3, Multicenter, Multinational, Open Label Study to Evaluate the Efficacy and Safety of ORGN001 (Formerly ALXN1101) in Neonates, Infants and Children With Molybdenum Cofactor Deficiency (MOCD) Type A

Conditions
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Molybdenum Cofactor Deficiency, Type A

Intervention / Treatment
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* Drug: ORGN001 (formerly ALXN1101)


Participation Criteria
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Eligibility Criteria
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Patients must meet all of the following inclusion criteria to be considered for enrollment in this study: Male or female neonatal patient (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/Individual Named Patient access In neonates, diagnosis of MoCD Type A, based on: Prenatal genetic diagnosis, or Onset of clinical and/or laboratory signs and symptoms consistent with MoCD Type A (eg, seizures, exaggerated startle response, high-pitched cry, axial hypotonia, limb hypertonia, feeding difficulties, elevated urinary sulfite and/or SSC, elevated xanthine in urine or blood, or low or absent uric acid in the urine or blood) within the first 28 days after birth In infants or children, diagnosis of MoCD Type A, based on: 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 Patients will be excluded from participating in the study if they meet any of the following 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 in age).

Ages Eligible for Study
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Minimum Age: 1 Day
Maximum Age: 5 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: ORGN001 (formerly ALXN1101)<br> | Drug: ORGN001 (formerly ALXN1101)<br> <br> |

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Overall Survival | Patients with a confirmed diagnosis of MOCD Type A, treated with ORGN001 and still alive at last observation. | Through last observation (average of 24 months) | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Feeding Pattern | Number of patients who can feed orally | At Month 12 visit | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Molybdenum Cofactor Deficiency (MoCD), Molybdenum Cofactor (MoCo) biosynthesis, sulfite oxidase (SO), S sulfocysteine (SSC), xanthine oxidoreductase, aldehyde oxidase