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OpenTrials
Completed

NCT Number: NCT02047461

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

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Key information

Conditions

Molybdenum Cofactor Deficiency, Type A Molybdenum Cofactor Deficiency, Complementation Group A Molybdenum cofactor deficiency

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2

Primary location

Monash Medical Centre, Melbourne, Australia

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About this study

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.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

ORGN001 (formerly ALXN1101)

Drug

IV infusion

Primary outcomes

  1. 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

Secondary outcomes

  1. 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

  2. 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.

  3. 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.

  4. 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

Sponsors and collaborators

Lead sponsor

Origin Biosciences

Industry

Registry information

Official study 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)

Important dates

Study start
2014
Primary completion
2022
Study completion
2022
First posted
Jan 28, 2014
Registry last updated
Oct 17, 2023

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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