Clinical Trials List
Protocol NumberALXN1101-MCD-202
NCT Number(ClinicalTrials.gov Identfier)NCT02629393
2016-03-11 - 2021-04-19
Phase II/III
Terminated1
ICD-10E61.5
Molybdenum deficiency
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
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Trial Applicant
IQVIA RDS Taiwan Ltd.
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Sponsor
Origin Biosciences
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Trial scale
Multi-Regional Multi-Center
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Update
2025/08/20
Investigators and Locations
Co-Principal Investigator
- PI-CHUAN FAN Division of Pediatrics
- Yin-Hsiu Chien Division of General Internal Medicine
- WUH-LIANG HWU Division of General Internal Medicine
The Actual Total Number of Participants Enrolled
0 Stop recruiting
Condition/Disease
Molybdenum Cofactor Deficiency (MoCD) Type A
Objectives
Primary:
x To evaluate the safety and efficacy of ALXN1101 in neonate patients with MoCD Type A
Secondary:
x To evaluate the effect of ALXN1101 on acquisition of developmental milestones
x To evaluate the effect of ALXN1101 on pediatric measures of functional ability and activities of daily living
x To characterize the pharmacokinetics (PK) of ALXN1101
Exploratory:
x Identification of clinical measures that may be useful for characterizing MoCD Type A
x Characterize changes in MoCD-associated urine and blood biomarker concentrations through Month 36
Test Drug
ALXN1101
Active Ingredient
ALXN1101
Dosage Form
Powder for solution for infusion
Dosage
2.5 mg/10 mL
Endpoints
Primary Efficacy Endpoint:
x Response, defined as patients being alive and able to sit upright independently for at least 30 seconds at
Month 12
Secondary Efficacy Endpoints:
x Bayley Scales of Infant Development® – Third Edition (Bayley – III) Cognitive and Motor Scales as
measured through Month 12
x Functional ability and activities of daily living, measured by the Pediatric Evaluation of Disability Inventory
(PEDI) through Month 12
x Response, defined as patients being alive and able to sit upright independently for at least 30 seconds at
Month 12
Secondary Efficacy Endpoints:
x Bayley Scales of Infant Development® – Third Edition (Bayley – III) Cognitive and Motor Scales as
measured through Month 12
x Functional ability and activities of daily living, measured by the Pediatric Evaluation of Disability Inventory
(PEDI) through Month 12
Inclution Criteria
Inclusion criteria
Patients must meet all of the following inclusion criteria to be considered for enrollment in this study:
x 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)
x 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 (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
x Parent or legal guardian must have signed the informed consent form (ICF) prior to any study procedures
being performed.
Patients must meet all of the following inclusion criteria to be considered for enrollment in this study:
x 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)
x 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 (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
x Parent or legal guardian must have signed the informed consent form (ICF) prior to any study procedures
being performed.
Exclusion Criteria
Exclusion criteria:
Patients will be excluded from participating in the study if they meet any of the following criteria:
x Diagnosis other than MoCD Type A (may be determined after the initiation of study drug)
x 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
x 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
x 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.)
Patients will be excluded from participating in the study if they meet any of the following criteria:
x Diagnosis other than MoCD Type A (may be determined after the initiation of study drug)
x 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
x 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
x 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.)
The Estimated Number of Participants
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Taiwan
1 participants
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Global
10 participants
