Study of ORGN001 (Formerly ALXN1101) in Neonates, Infants and Children With Molybdenum Cofactor Deficiency (MOCD) Type A

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

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

Study Overview

• Status: Completed
• Conditions: Molybdenum Cofactor Deficiency, Type A
• Intervention / Treatment: Drug: ORGN001 (formerly ALXN1101)

• Study Type: Interventional
• Enrollment (Actual): 5
• Phase: Phase 2; Phase 3

Contacts and Locations

Study Locations

• Israel
  • Afula, Israel, 18341
    • HaEmek Medical Center
• Norway
  • Stavanger, Norway
    • Stavanger Universitetssjukehus
• Spain
  • Barcelona
    • Esplugues de Llobregat, Barcelona, Spain, 08950
      • Hospital Sant Joan de Deu
• Turkey
  • Ankara, Turkey
    • Gazi University
  • Ankara, Turkey, 06100
    • Hacettepe University of Medicine
  • Antalya, Turkey, 07058
    • Akdeniz University Medical Faculty
• United Kingdom
  • London, United Kingdom
    • Great Ormond Street Hosptial
  • Greater Manchester
    • Manchester, Greater Manchester, United Kingdom, M13 9WL
      • Willink Biochemical Genetics Unit
• United States
  • Michigan
    • Detroit, Michigan, United States, 48201
      • Children's Hosptial of Michigan
  • Ohio
    • Cincinnati, Ohio, United States, 45229
      • Cincinnati Children's Hospital Medical Center
  • Wisconsin
    • Milwaukee, Wisconsin, United States, 53226-4874
      • Children's Hospital of Wisconsin

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 1 day to 5 years (Child)
• Accepts Healthy Volunteers: No

Description

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).

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: ORGN001 (formerly ALXN1101)	Drug: ORGN001 (formerly ALXN1101)

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Overall Survival	Patients with a confirmed diagnosis of MOCD Type A, treated with ORGN001 and still alive at last observation.	Through last observation (average of 24 months)

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Feeding Pattern	Number of patients who can feed orally	At Month 12 visit

Collaborators and Investigators

• Sponsor: Origin Biosciences
• Investigators: Study Director: Liza Squires, M.D., Origin Biosciences

Publications and helpful links

Helpful Links

• FDA press release of Nulibry approval
• FDA Drug Approval Package

Study record dates

Study Major Dates

• Study Start (Actual): May 1, 2016
• Primary Completion (Actual): September 1, 2022
• Study Completion (Actual): October 1, 2022

Study Registration Dates

• First Submitted: December 3, 2015
• First Submitted That Met QC Criteria: December 10, 2015
• First Posted (Estimated): December 14, 2015

Study Record Updates

• Last Update Posted (Actual): October 17, 2023
• Last Update Submitted That Met QC Criteria: September 22, 2023
• Last Verified: September 1, 2023

More Information

Terms related to this study

• Keywords: Molybdenum Cofactor Deficiency (MoCD); Molybdenum Cofactor (MoCo) biosynthesis; sulfite oxidase (SO); S sulfocysteine (SSC); xanthine oxidoreductase; aldehyde oxidase
• Additional Relevant MeSH Terms: Metabolic Diseases; Genetic Diseases, Inborn; Metabolism, Inborn Errors; Metal Metabolism, Inborn Errors
• Other Study ID Numbers: ALXN1101-MCD-202