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Trial record 1 of 1 for:    NCT02629393
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Study of ORGN001 (Formerly ALXN1101) in Neonates, Infants and Children With Molybdenum Cofactor Deficiency (MOCD) Type A

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details. Identifier: NCT02629393
Recruitment Status : Recruiting
First Posted : December 14, 2015
Last Update Posted : April 29, 2022
Information provided by (Responsible Party):
Origin Biosciences

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

Condition or disease Intervention/treatment Phase
Molybdenum Cofactor Deficiency, Type A Drug: ORGN001 (formerly ALXN1101) Phase 2 Phase 3

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 5 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
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
Actual Study Start Date : May 2016
Estimated Primary Completion Date : July 2022
Estimated Study Completion Date : December 2022

Arm Intervention/treatment
Experimental: ORGN001 (formerly ALXN1101) Drug: ORGN001 (formerly ALXN1101)

Primary Outcome Measures :
  1. Overall survival [ Time Frame: 36 months ]

Secondary Outcome Measures :
  1. Bayley Scales of Infant Development® - Third Edition (Bayley - III®) [ Time Frame: First 12 months ]
  2. Pediatric Evaluation of Disability Inventory (PEDI) [ Time Frame: First 12 months ]
  3. Gross Motor Function Measure [ Time Frame: First 12 months ]

  4. Gross Motor Function Measure [ Time Frame: First 12 months ]

  5. Feeding pattern [ Time Frame: First 12 months ]
    Number of patients who can feed orally

  6. Head circumference measurement [ Time Frame: First 12 months ]
  7. Length measurement [ Time Frame: First 12 months ]
  8. Weight measurement [ Time Frame: First 12 months ]

  9. BMI measurement [ Time Frame: First 12 months ]

Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.

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Ages Eligible for Study:   up to 5 Years   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

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).

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT02629393

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Contact: Origin Biosciences (Sponsor) 1-857-350-3841
Contact: Origin Biosciences (Sponsor)

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United States, Michigan
Children's Hosptial of Michigan Completed
Detroit, Michigan, United States, 48201
United States, Ohio
Cincinnati Children's Hospital Medical Center Completed
Cincinnati, Ohio, United States, 45229
United States, Wisconsin
Children's Hospital of Wisconsin Completed
Milwaukee, Wisconsin, United States, 53226-4874
CHU Lyon - Hôpital Femme Mère- Enfant Withdrawn
Lyon, Rhone, France, 69677
HaEmek Medical Center Recruiting
Afula, Israel, 18341
Hospital Kuala Lumpur Withdrawn
Kuala Lumpur, Malaysia, 50586
Stavanger Universitetssjukehus Recruiting
Stavanger, Norway
Contact: Origin Biosciences         
Hospital Sant Joan de Deu Active, not recruiting
Esplugues de Llobregat, Barcelona, Spain, 08950
Hacettepe University of Medicine Completed
Ankara, Turkey, 06100
Gazi University Not yet recruiting
Ankara, Turkey
Contact: Origin Biosciences         
Akdeniz University Medical Faculty Completed
Antalya, Turkey, 07058
United Kingdom
Willink Biochemical Genetics Unit Active, not recruiting
Manchester, Greater Manchester, United Kingdom, M13 9WL
Great Ormond Street Hosptial Recruiting
London, United Kingdom
Sponsors and Collaborators
Origin Biosciences
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Study Director: Liza Squires, M.D. Origin Biosciences
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Responsible Party: Origin Biosciences Identifier: NCT02629393    
Other Study ID Numbers: ALXN1101-MCD-202
First Posted: December 14, 2015    Key Record Dates
Last Update Posted: April 29, 2022
Last Verified: April 2022
Keywords provided by Origin Biosciences:
Molybdenum Cofactor Deficiency (MoCD)
Molybdenum Cofactor (MoCo) biosynthesis
sulfite oxidase (SO)
S sulfocysteine (SSC)
xanthine oxidoreductase
aldehyde oxidase
Additional relevant MeSH terms:
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Metal Metabolism, Inborn Errors
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Metabolic Diseases