STUDY OF ALXN1101 IN NEWBORNS 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-DE
• Lead Sponsor: Origin Biosciences, Inc.

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2015-11-06
• Last Update Posted: 28 February 2019

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: Not specified
• Target Recruitment: 10

Inclusion Criteria

- Male or female neonatal patient (1 to 28 days of age [inclusive] at the time of ALXN1101 administration, with day 1 of age corresponding to the day of birth)
- 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
- 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 ALXN1101 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 (This criterion does not apply to children less than 1 day in 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 ALXN1101 in neonate patients with MoCD Type A;Primary end point(s): Response, defined as patients being alive and able to sit upright independently for at least 30 seconds;Timepoint(s) of evaluation of this end point: Month 12;<br> Secondary Objective: - To evaluate the effect of ALXN1101 on acquisition of developmental milestones<br> - To evaluate the effect of ALXN1101 on pediatric measures of functional ability and activities of daily living<br> - To characterize the pharmacokinetics (PK) of ALXN1101<br>

Secondary Outcome Measures

Name	Time	Method
<br> Secondary end point(s): - Bayley Scales of Infant Development® – Third Edition (Bayley – III) Cognitive and Motor Scales as measured<br> - Functional ability and activities of daily living, measured by the Pediatric Evaluation of Disability Inventory (PEDI)<br> - PK parameters<br> - Safety<br> ;Timepoint(s) of evaluation of this end point: through Month 12