Safety & Efficacy Study of ORGN001 (Formerly ALXN1101) in Pediatric Patients With MoCD Type A Currently Treated With rcPMP

Sponsor

Origin Biosciences (Industry)

Overall Status

Active, not recruiting

CT.gov ID

NCT02047461

Collaborator

(none)

Enrollment

Locations

Arm

104

Anticipated Duration (Months)

1.2

Patients Per Site

Patients Per Site Per Month

Study Details

Study Description

Brief Summary

This study will include a screening period, a 6-month treatment period, followed by long-term extension period expected to last approximately 72 months.

Condition or Disease	Intervention/Treatment	Phase
Molybdenum Cofactor Deficiency, Type A	Drug: ORGN001 (formerly ALXN1101)	Phase 2

Detailed Description

Patients will receive daily IV infusions of ORGN001 (formerly ALXN1101) starting on Day 1. After a prescribed period, dosing will increase monthly based on defined patient safety measures. After Month 6, patients will continue daily dosing at their last tolerated dose.

Study Design

Study Type:

Interventional

Actual Enrollment :

7 participants

Allocation:

N/A

Intervention Model:

Single Group Assignment

Masking:

None (Open Label)

Primary Purpose:

Treatment

Official Title:

A Phase 2, Multicenter, Multinational, Open-Label, Dose-Escalation Study to Evaluate the Safety and Efficacy of ORGN001 (Formerly ALXN1101) in Pediatric Patients With Molybdenum Cofactor Deficiency (MoCD) Type A Currently Treated With Recombinant Escherichia Coli-derived Cyclic Pyranopterin Monophosphate (rcPMP)

Actual Study Start Date :

Apr 1, 2014

Anticipated Primary Completion Date :

Dec 1, 2022

Anticipated Study Completion Date :

Dec 1, 2022

Arms and Interventions

Arm	Intervention/Treatment
Experimental: ORGN001 (formerly ALXN1101) daily IV infusions	Drug: ORGN001 (formerly ALXN1101) IV infusion

Arm

Intervention/Treatment

Experimental: ORGN001 (formerly ALXN1101)

daily IV infusions

Drug: ORGN001 (formerly ALXN1101)

IV infusion

Outcome Measures

Primary Outcome Measures

Safety of ORGN001 (formerly ALXN1101) [First 6 months of treatment]
The type, number and frequency of adverse events and severity of adverse events (AEs) and serious adverse events (SAEs) Change from baseline in: clinical laboratory assessments, findings on physical examination, vital sign measurements, and EEG results

Secondary Outcome Measures

Pharmacokinetics parameters of ORGN001 (formerly ALXN1101) [First 6 months of treatment]
Plasma concentration (Cmax), time to maximum observed plasma concentration (tmax), area under the plasma concentration-time curve (AUC), and terminal half-life (t½)
Effect of ORGN001 (formerly ALXN1101) on urine and blood biomarkers [Until study completion (approx. 72 months)]
Change from baseline in urine and blood biomarkers.
Effect of ORGN001 (formerly ALXN1101) on neurologic function including motor examination [Until study completion (approx. 72 months)]
Change from baseline on repeated Neurologic examinations such as muscle strength and tone, as well as sensory and reflex exam.
Long-term safety of ORGN001 (formerly ALXN1101) [Until study completion (approx. 72 months)]
The type, number and frequency of adverse events and serious adverse events (SAEs) Change from baseline in: clinical laboratory assessments, findings on physical examination, vital sign measurements, and EEG results
Effect of ORGN001 (formerly ALXN1101) on cognitive functions [Until study completion (approx. 72 months)]
Change from baseline in cognitive function using age appropriate measures of cognition, eg. Wechsler Preschoool and Primary Scale of intelligence

Eligibility Criteria

Criteria

Ages Eligible for Study:

N/A and Older

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

Inclusion Criteria:

Male or female patients with a genetically confirmed diagnosis of MoCD Type A (MOCS1 mutation)
Currently treated with rcPMP infusions

Exclusion Criteria:

Current or planned treatment with another investigational drug or device, with the exception rcPMP treatment through Day -1.

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	Children's Hospital of Wisconsin	Milwaukee	Wisconsin	United States	53226
2	Monash Medical Centre	Melbourne		Australia
3	Beatrix Children's Hospital	Groningen		Netherlands
4	Unité des maladies métaboliques	Tunis		Tunisia
5	Royal Hospital for Sick Children	Glasgow		United Kingdom
6	Manchester University Hospitals NHS Foundation Trust	Manchester		United Kingdom