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

Molybdenum Cofactor Deficiency, Type A Clinical Trial

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)

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT02047461
• Other study ID #: ALXN1101-MCD-201
• Secondary ID: 2013-002701-56
• Status: Completed
• Phase: Phase 2
• Start date: April 2014
• Est. completion date: October 2022

Study information

• Verified date: September 2023
• Source: Origin Biosciences
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 8
• Est. completion date: October 2022
• Est. primary completion date: August 2022
• Accepts healthy volunteers: No
• Gender: All
• Age group: N/A and older

Eligibility

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.

Related Conditions & MeSH terms

• Molybdenum Cofactor Deficiency, Type A

Intervention

Drug:
• ORGN001 (formerly ALXN1101)
IV infusion

Locations

Country	Name	City	State
Australia	Monash Medical Centre	Melbourne
Netherlands	Beatrix Children's Hospital	Groningen
Tunisia	Unité des maladies métaboliques	Tunis
United Kingdom	Royal Hospital for Sick Children	Glasgow
United Kingdom	Manchester University Hospitals NHS Foundation Trust	Manchester
United States	Children's Hospital of Wisconsin	Milwaukee	Wisconsin

Sponsors (1)

Lead Sponsor	Collaborator
Origin Biosciences

Countries where clinical trial is conducted

United States,  Australia,  Netherlands,  Tunisia,  United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Safety of ORGN001 (Formerly ALXN1101)	Treatment Emergent Serious Adverse Events	Baseline to Month 24 for all patients plus additional follow-up up to Month 90
Secondary	Pharmacokinetics (Actual Plasma Concentration) of ORGN001 (Formerly ALXN1101)	ORGN001 levels by dose at pre-infusion and end of infusion (EOI) at scheduled timepoints	First 6 months at each dose level, where available
Secondary	S-sulfocysteine (Umol/L) Normalized to Urine Creatinine (mmol/L) - Change From Baseline Over Time	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.	Baseline to Month 24 for all patients plus additional follow-up to Month 90
Secondary	Effect of ORGN001 (Formerly ALXN1101) on Neurologic Function Including Motor Examination	Change from baseline on repeated Neurologic examinations such as muscle strength and tone, as well as sensory and reflex exam.	Baseline to Month 24 for all patients plus additional follow-up until Month 30
Secondary	Long-term Safety of ORGN001 (Formerly ALXN1101)	Change from baseline in Seizure frequency	Baseline to Month 24 for all patients plus additional follow up until Month 72

External Links

•   FDA Drug Approval Package
•   FDA press release of Nulibry approval