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

NCT02629393 | Completed Phase 2 Results DMC

• 1 other identifier: ALXN1101-MCD-202
• Study Type: interventional
• Target: 5
• Locations: 6 countries
• Sites: 11

Brief Summary

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

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

Outcome Measures

Primary Outcomes (1)

• 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 Outcomes (1)

• Feeding Pattern

  At Month 12 visit

Study Arms (1)

ORGN001 (formerly ALXN1101)

EXPERIMENTAL

Drug: ORGN001 (formerly ALXN1101)

Interventions

ORGN001 (formerly ALXN1101) DRUG

ORGN001 (formerly ALXN1101)

Eligibility Criteria

• Age: 1 Day - 5 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• 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
• 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
• 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
• 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:
• 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 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
• 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).

Sponsors & Collaborators

• Origin Biosciences: lead

Study Sites (11)

Children's Hosptial of Michigan

Detroit, Michigan, 48201, United States

Location

Cincinnati Children's Hospital Medical Center

Cincinnati, Ohio, 45229, United States

Location

Children's Hospital of Wisconsin

Milwaukee, Wisconsin, 53226-4874, United States

Location

HaEmek Medical Center

Afula, 18341, Israel

Location

Stavanger Universitetssjukehus

Stavanger, Norway

Location

Hospital Sant Joan de Deu

Esplugues de Llobregat, Barcelona, 08950, Spain

Location

Hacettepe University of Medicine

Ankara, 06100, Turkey (Türkiye)

Location

Gazi University

Ankara, Turkey (Türkiye)

Location

Akdeniz University Medical Faculty

Antalya, 07058, Turkey (Türkiye)

Location

Willink Biochemical Genetics Unit

Manchester, Greater Manchester, M13 9WL, United Kingdom

Location

Great Ormond Street Hosptial

London, United Kingdom

Location

Related Links

• FDA Drug Approval Package

MeSH Terms

Conditions

Molybdenum Cofactor Deficiency, Complementation Group A; Molybdenum cofactor deficiency

Limitations and Caveats

Due to the small number of patients and no control, there was no SAP for this study. Results were reported by individual patient and were only descriptive. Results in tabular format are unavailable except for binary measures, such as survival and feeding ability. For more information and summary data, please refer to the referenced FDA approval package.

Results Point of Contact

• Title: Business Development and Operations
• Organization: Origin Biosciences (affiliate of BridgeBio)

info@bridgebio.com

650-391-9740

Study Officials

• Liza Squires, M.D.

  Origin Biosciences

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: December 3, 2015
• First Posted: December 14, 2015
• Study Start: May 1, 2016
• Primary Completion: September 1, 2022
• Study Completion: October 1, 2022
• Last Updated: October 17, 2023
• Results First Posted: October 17, 2023

Record last verified: 2023-09

Locations

NO (1); GB (2); US (3); IL (1); ES (1); TU (3)