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

September 22, 2023 updated by: Origin Biosciences

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)

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

Study Overview

Status

Completed

Conditions

Intervention / Treatment

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 Type

Interventional

Enrollment (Actual)

8

Phase

  • Phase 2

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Australia
      • Melbourne, Australia
        • Monash Medical Centre
  • Netherlands
      • Groningen, Netherlands
        • Beatrix Children's Hospital
  • Tunisia
      • Tunis, Tunisia
        • Unité des maladies métaboliques
  • United Kingdom
      • Glasgow, United Kingdom
        • Royal Hospital for Sick Children
      • Manchester, United Kingdom
        • Manchester University Hospitals NHS Foundation Trust
  • United States
    • Wisconsin
      • Milwaukee, Wisconsin, United States, 53226
        • Children's Hospital of Wisconsin

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Child
  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Description

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 Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

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

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Safety of ORGN001 (Formerly ALXN1101)
Time Frame: Baseline to Month 24 for all patients plus additional follow-up up to Month 90
Treatment Emergent Serious Adverse Events
Baseline to Month 24 for all patients plus additional follow-up up to Month 90

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Pharmacokinetics (Actual Plasma Concentration) of ORGN001 (Formerly ALXN1101)
Time Frame: First 6 months at each dose level, where available
ORGN001 levels by dose at pre-infusion and end of infusion (EOI) at scheduled timepoints
First 6 months at each dose level, where available
S-sulfocysteine (Umol/L) Normalized to Urine Creatinine (mmol/L) - Change From Baseline Over Time
Time Frame: 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.
Baseline to Month 24 for all patients plus additional follow-up to Month 90
Effect of ORGN001 (Formerly ALXN1101) on Neurologic Function Including Motor Examination
Time Frame: 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.
Baseline to Month 24 for all patients plus additional follow-up until Month 30
Long-term Safety of ORGN001 (Formerly ALXN1101)
Time Frame: Baseline to Month 24 for all patients plus additional follow up until Month 72
Change from baseline in Seizure frequency
Baseline to Month 24 for all patients plus additional follow up until Month 72

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Origin Biosciences

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

Helpful Links

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

April 1, 2014

Primary Completion (Actual)

August 1, 2022

Study Completion (Actual)

October 1, 2022

Study Registration Dates

First Submitted

January 9, 2014

First Submitted That Met QC Criteria

January 24, 2014

First Posted (Estimated)

January 28, 2014

Study Record Updates

Last Update Posted (Actual)

October 17, 2023

Last Update Submitted That Met QC Criteria

September 22, 2023

Last Verified

September 1, 2023

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • ALXN1101-MCD-201
  • 2013-002701-56 (EudraCT Number)

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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