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

Phase 2

Completed

• Conditions: Molybdenum Cofactor Deficiency, Type A
• Interventions: Drug: ORGN001 (formerly ALXN1101)

• Registration Number: NCT02047461
• Lead Sponsor: Origin Biosciences

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.

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 Timeline

• Study First Submitted: 2014-01-09
• Study First Submitted QC: 2014-01-24
• Study First Posted: 2014-01-28
• Study Start: 2014-04
• Primary Completion: 2022-08
• Study Completion: 2022-10
• Results First Submitted: 2023-08-10
• Status Verified: 2023-09
• Results First Submitted QC: 2023-09-22
• Last Update Submitted: 2023-09-22
• Results First Posted: 2023-10-17
• Last Update Posted: 2023-10-17

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 8

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.

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
ORGN001 (formerly ALXN1101)	ORGN001 (formerly ALXN1101)	daily IV infusions

Primary Outcome Measures

Name	Time	Method
Safety of ORGN001 (Formerly ALXN1101)	Baseline to Month 24 for all patients plus additional follow-up up to Month 90	Treatment Emergent Serious Adverse Events

Secondary Outcome Measures

Name	Time	Method
Long-term Safety of ORGN001 (Formerly ALXN1101)	Baseline to Month 24 for all patients plus additional follow up until Month 72	Change from baseline in Seizure frequency
Pharmacokinetics (Actual Plasma Concentration) of ORGN001 (Formerly ALXN1101)	First 6 months at each dose level, where available	ORGN001 levels by dose at pre-infusion and end of infusion (EOI) at scheduled timepoints
S-sulfocysteine (Umol/L) Normalized to Urine Creatinine (mmol/L) - Change From Baseline Over Time	Baseline to Month 24 for all patients plus additional follow-up to Month 90	Analyses were performed on urine SSC, a biomarker of the MoCD pathway. Levels of SSC measured in urine were normalized to urine creatinine levels. The observed value, change, and percent change in urine and blood SSC levels from baseline were summarized by visit over time.
Effect of ORGN001 (Formerly ALXN1101) on Neurologic Function Including Motor Examination	Baseline to Month 24 for all patients plus additional follow-up until Month 30	Change from baseline on repeated Neurologic examinations such as muscle strength and tone, as well as sensory and reflex exam.

Trial Locations

Locations (6)

Beatrix Children's Hospital; 🇳🇱 Groningen, Netherlands

Monash Medical Centre; 🇦🇺 Melbourne, Australia

Manchester University Hospitals NHS Foundation Trust; 🇬🇧 Manchester, United Kingdom

Unité des maladies métaboliques; 🇹🇳 Tunis, Tunisia

Children's Hospital of Wisconsin; 🇺🇸 Milwaukee, Wisconsin, United States

Royal Hospital for Sick Children; 🇬🇧 Glasgow, United Kingdom