ALXN1101 in Neonates with Molybdenum Cofactors Deficiency Type A


Molybdenum Cofactor Deficiency Type A

Full Study Name

A phase 2/3, multicenter, multinational, open-label study to evaluate the efficacy and safety of ALXN1101 in neonates with Molybdenum Cofactor Deficiency (MOCD) type A

Study ID



Alexion Pharmaceuticals Inc.

Local Principal Investigator

Bernd Schwahn

Study Status


Main Aims

To evaluate the safety and efficacy of ALXN1101 in neonate patients with MoCD Type A

Inclusion Criteria
  • Male or female neonatal patient (1 to 28 days of age, 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 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
  • Parent or legal guardian must have signed
Exclusion Criteria
  • 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 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
  • Brain imaging prior to initiation of treatment that indicates cortical or subcortical cystic encephalomalacia or intracranial hemorrhage
  • Modified Glasgow Coma Scale (mGCS) for Infants and Children score of less than 7 for more than 24 hours
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