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

Sponsor

Origin Biosciences (Industry)

Overall Status

Recruiting

CT.gov ID

NCT02629393

Collaborator

(none)

Enrollment

Locations

Arm

Anticipated Duration (Months)

0.4

Patients Per Site

Patients Per Site Per Month

Study Details

Study Description

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

Study Design

Study Type:

Interventional

Anticipated 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 1, 2016

Anticipated Primary Completion Date :

Jul 1, 2022

Anticipated Study Completion Date :

Dec 1, 2022

Arms and Interventions

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

Arm

Intervention/Treatment

Experimental: ORGN001 (formerly ALXN1101)

Drug: ORGN001 (formerly ALXN1101)

Outcome Measures

Primary Outcome Measures

Overall survival [36 months]

Secondary Outcome Measures

Bayley Scales of Infant Development® - Third Edition (Bayley - III®) [First 12 months]
Pediatric Evaluation of Disability Inventory (PEDI) [First 12 months]
Gross Motor Function Measure [First 12 months]
GMFM-88
Gross Motor Function Measure [First 12 months]
GMFCS-E&R
Feeding pattern [First 12 months]
Number of patients who can feed orally
Head circumference measurement [First 12 months]
Length measurement [First 12 months]
Weight measurement [First 12 months]
Weight
BMI measurement [First 12 months]

Eligibility Criteria

Criteria

Ages Eligible for Study:

1 Day to 5 Years

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

Patients must meet all of the following inclusion criteria to be considered for enrollment in this study:

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).

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	Children's Hosptial of Michigan	Detroit	Michigan	United States	48201
2	Cincinnati Children's Hospital Medical Center	Cincinnati	Ohio	United States	45229
3	Children's Hospital of Wisconsin	Milwaukee	Wisconsin	United States	53226-4874
4	CHU Lyon - Hôpital Femme Mère- Enfant	Lyon	Rhone	France	69677
5	HaEmek Medical Center	Afula		Israel	18341
6	Hospital Kuala Lumpur	Kuala Lumpur		Malaysia	50586
7	Stavanger Universitetssjukehus	Stavanger		Norway
8	Hospital Sant Joan de Deu	Esplugues de Llobregat	Barcelona	Spain	08950
9	Hacettepe University of Medicine	Ankara		Turkey	06100
10	Gazi University	Ankara		Turkey
11	Akdeniz University Medical Faculty	Antalya		Turkey	07058
12	Willink Biochemical Genetics Unit	Manchester	Greater Manchester	United Kingdom	M13 9WL
13	Great Ormond Street Hosptial	London		United Kingdom