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

NCT ID: NCT02629393

Last Updated: 2023-10-17

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE2/PHASE3
• Total Enrollment: 5 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2016-05-31
• Study Completion Date: 2022-10-31

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

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

ORGN001 (formerly ALXN1101)

Group Type: EXPERIMENTAL

ORGN001 (formerly ALXN1101)

Intervention Type: DRUG

Interventions

ORGN001 (formerly ALXN1101)

Intervention Type: DRUG

Eligibility Criteria

Inclusion Criteria

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).

• Minimum Eligible Age: 1 Day
• Maximum Eligible Age: 5 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Origin Biosciences

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Liza Squires, M.D.

Role: STUDY_DIRECTOR

Origin Biosciences

Locations

Children's Hosptial of Michigan

Detroit, Michigan, United States

Cincinnati Children's Hospital Medical Center

Cincinnati, Ohio, United States

Children's Hospital of Wisconsin

Milwaukee, Wisconsin, United States

HaEmek Medical Center

Afula, , Israel

Stavanger Universitetssjukehus

Stavanger, , Norway

Hospital Sant Joan de Deu

Esplugues de Llobregat, Barcelona, Spain

Hacettepe University of Medicine

Ankara, , Turkey (Türkiye)

Gazi University

Ankara, , Turkey (Türkiye)

Akdeniz University Medical Faculty

Antalya, , Turkey (Türkiye)

Willink Biochemical Genetics Unit

Manchester, Greater Manchester, United Kingdom

Great Ormond Street Hosptial

London, , United Kingdom

Countries

United States; Israel; Norway; Spain; Turkey (Türkiye); United Kingdom

Provided Documents

Document Type: Study Protocol and Statistical Analysis Plan

View Document

Related Links

https://www.accessdata.fda.gov/drugsatfda_docs/nda/2021/214018Orig1s000TOC.cfm

FDA Drug Approval Package

Other Identifiers

ALXN1101-MCD-202

Identifier Type: -

Identifier Source: org_study_id