Clinical trial NCT02629393: Study of ORGN001 (Formerly ALXN1101) in Neonates, Infants and Children With Molybdenum Cofactor Deficiency (MOCD) Type A
Official title: A Phase 2/3, Multicenter, Multinational, Open Label Study to Evaluate the Efficacy and Safety of ORGN001 (Formerly ALXN1101) in Neonates, Infants and Children With Molybdenum Cofactor Deficiency (MOCD) Type A
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
PHASE3
Conditions: Molybdenum Cofactor Deficiency, Type A
Keywords: Molybdenum Cofactor Deficiency (MoCD)
Molybdenum Cofactor (MoCo) biosynthesis
sulfite oxidase (SO)
S sulfocysteine (SSC)
xanthine oxidoreductase
aldehyde oxidase
Lead sponsor: Origin Biosciences
Sponsor class: INDUSTRY
First posted: 2015-12-14
Enrollment: 5
Sex: ALL
Minimum age (years): 1
Maximum age (years): 5
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: To evaluate the safety and efficacy of ORGN001(formerly ALXN1101) in neonate patients with MoCD Type A


Eligibility criteria: Patients must meet all of the following inclusion criteria to be considered for enrollment in this study:

1. Male or female neonatal patient (1 to 28 days of age \[inclusive\] at the time of ORGN001 administration, with day 1 of age corresponding to the day of birth) or infant (29 days to \<2 years of age) or child (2 to 5 years of age \[inclusive\]) with MoCD Type A, previously untreated with ORGN001 or treated with ORGN001 through Compassionate Use/Individual Named Patient access
2. In neonates, 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 (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
3. In infants or children, diagnosis of MoCD Type A, based on:

   Confirmed genetic diagnosis (genetic confirmation of the diagnosis of MoCD Type A may be obtained after initiation of ORGN001 therapy in certain cases), biochemical profile, and clinical presentation consistent with MoCD Type A
4. Parent or legal guardian must have signed the informed consent form (ICF) prior to any study procedures being performed

Patients will be excluded from participating in the study if they meet any of the following criteria:

1. Diagnosis other than MoCD Type A (may be determined after the initiation of study drug)
2. 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
3. Antenatal and/or postnatal brain imaging prior to initiation of treatment with ORGN001 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
4. Modified Glasgow Coma Scale (mGCS) for Infants and Children score of less than 7 for more than 24 hours (does not apply to children less than 1 day in age).

Primary outcomes:
measure: Overall Survival
timeFrame: Through last observation (average of 24 months)
Secondary outcomes:
measure: Feeding Pattern
timeFrame: At Month 12 visit
Other outcomes:
Locations:
city: Detroit
country: United States
facility: Children's Hosptial of Michigan
state: Michigan
city: Cincinnati
country: United States
facility: Cincinnati Children's Hospital Medical Center
state: Ohio
city: Milwaukee
country: United States
facility: Children's Hospital of Wisconsin
state: Wisconsin
city: Afula
country: Israel
facility: HaEmek Medical Center
city: Stavanger
country: Norway
facility: Stavanger Universitetssjukehus
city: Esplugues de Llobregat
country: Spain
facility: Hospital Sant Joan de Deu
state: Barcelona
city: Ankara
country: Turkey
facility: Hacettepe University of Medicine
city: Ankara
country: Turkey
facility: Gazi University
city: Antalya
country: Turkey
facility: Akdeniz University Medical Faculty
city: Manchester
country: United Kingdom
facility: Willink Biochemical Genetics Unit
state: Greater Manchester
city: London
country: United Kingdom
facility: Great Ormond Street Hosptial
Study officials:
affiliation: Origin Biosciences
name: Liza Squires, M.D.
role: STUDY_DIRECTOR
References: