Working…
ClinicalTrials.gov
ClinicalTrials.gov Menu

Study of ORGN001 (Formerly ALXN1101) in Neonates 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.
 
ClinicalTrials.gov Identifier: NCT02629393
Recruitment Status : Recruiting
First Posted : December 14, 2015
Last Update Posted : August 20, 2019
Sponsor:
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

Layout table for study information
Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 5 participants
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 With Molybdenum Cofactor Deficiency (MOCD) Type A
Actual Study Start Date : May 2016
Estimated Primary Completion Date : December 2020
Estimated Study Completion Date : December 2021


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



Primary Outcome Measures :
  1. Response, defined as patients alive and able to sit upright independently for at least 30 seconds [ Time Frame: At 12 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 ]


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.


Layout table for eligibility information
Ages Eligible for Study:   up to 28 Days   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • 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)
  • 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
  • Parent or legal guardian must have signed the informed consent form (ICF) prior to any study procedures being performed

Exclusion Criteria:

  • 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 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
  • 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 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).

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 ClinicalTrials.gov identifier (NCT number): NCT02629393


Contacts
Layout table for location contacts
Contact: Origin Biosciences (Sponsor) 1-617-322-5165 clinicaltrials@origintx.com

Locations
Layout table for location information
United States, Michigan
Children's Hosptial of Michigan Recruiting
Detroit, Michigan, United States, 48201
Contact: Origin Biosciences         
Principal Investigator: Kara Pappas, M.D.         
United States, Ohio
Cincinnati Children's Hospital Medical Center Active, not recruiting
Cincinnati, Ohio, United States, 45229
United States, Wisconsin
Children's Hospital of Wisconsin Active, not recruiting
Milwaukee, Wisconsin, United States, 53226-4874
France
CHU Lyon - Hôpital Femme Mère- Enfant Withdrawn
Lyon, Rhone, France, 69677
Israel
HaEmek Medical Center Recruiting
Afula, Israel, 18341
Malaysia
Hospital Kuala Lumpur Withdrawn
Kuala Lumpur, Malaysia, 50586
Spain
Hospital Sant Joan de Deu Active, not recruiting
Esplugues de Llobregat, Barcelona, Spain, 08950
Turkey
Hacettepe University of Medicine Active, not recruiting
Ankara, Turkey, 06100
Akdeniz University Medical Faculty Active, not recruiting
Antalya, Turkey, 07058
United Kingdom
Willink Biochemical Genetics Unit Active, not recruiting
Manchester, Greater Manchester, United Kingdom, M13 9WL
Sponsors and Collaborators
Origin Biosciences
Investigators
Layout table for investigator information
Study Director: Liza Squires, M.D. Origin Biosciences

Layout table for additonal information
Responsible Party: Origin Biosciences
ClinicalTrials.gov Identifier: NCT02629393     History of Changes
Other Study ID Numbers: ALXN1101-MCD-202
First Posted: December 14, 2015    Key Record Dates
Last Update Posted: August 20, 2019
Last Verified: August 2019
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:
Layout table for MeSH terms
Metal Metabolism, Inborn Errors
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Metabolic Diseases
Molybdenum
Micronutrients
Nutrients
Growth Substances
Physiological Effects of Drugs
Trace Elements