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

Alexion Pharma GmbH0 个研究点目标入组 10 人开始时间: 2016年5月19日最近更新: 2017年10月2日

试验速览

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•- 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

•- 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)

发起方