STUDY OF ALXN1101 IN NEWBORNS WITH MOLYBDENUM COFACTOR DEFICIENCY (MOCD) TYPE A

Phase 1

• Conditions: Molybdenum CoFactor Deficiency (MoCD) Type A; MedDRA version: 19.0Level: PTClassification code 10069687Term: Molybdenum cofactor deficiencySystem Organ Class: 10010331 - Congenital, familial and genetic disorders; Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]

• Registration Number: EUCTR2013-002702-30-FR
• Lead Sponsor: Alexion Pharma GmbH

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2016-05-19
• Last Update Posted: 2 October 2017

Recruitment & Eligibility

• Status: A
• Sex: All
• Target Recruitment: 10

Inclusion Criteria

- Male or female neonatal patients (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:
?Prenatal genetic diagnosis, or
?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 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, 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 Gasgow 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 of age)

Study & Design

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