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

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ClinicalTrials.gov Identifier: NCT02047461
Recruitment Status : Enrolling by invitation
First Posted : January 28, 2014
Last Update Posted : August 20, 2019
Sponsor:
Information provided by (Responsible Party):
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.

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.

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 7 participants
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 : April 2014
Estimated Primary Completion Date : December 2020
Estimated Study Completion Date : December 2020


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




Primary Outcome Measures :
  1. Safety of ORGN001 (formerly ALXN1101) [ Time Frame: 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 :
  1. Pharmacokinetics parameters of ORGN001 (formerly ALXN1101) [ Time Frame: 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)

  2. Effect of ORGN001 (formerly ALXN1101) on urine and blood biomarkers [ Time Frame: Until study completion (approx. 72 months) ]
    Change from baseline in urine and blood biomarkers.

  3. Effect of ORGN001 (formerly ALXN1101) on neurologic function including motor examination [ Time Frame: 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.

  4. Long-term safety of ORGN001 (formerly ALXN1101) [ Time Frame: 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

  5. Effect of ORGN001 (formerly ALXN1101) on cognitive functions [ Time Frame: 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



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Ages Eligible for Study:   Child, Adult, Older Adult
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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.


Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02047461


Locations
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United States, Wisconsin
Children's Hospital of Wisconsin
Milwaukee, Wisconsin, United States, 53226
Australia
Monash Medical Centre
Melbourne, Australia
Netherlands
Beatrix Children's Hospital
Groningen, Netherlands
Tunisia
Unité des maladies métaboliques
Tunis, Tunisia
United Kingdom
Royal Hospital for Sick Children
Glasgow, United Kingdom
Sponsors and Collaborators
Origin Biosciences

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Responsible Party: Origin Biosciences
ClinicalTrials.gov Identifier: NCT02047461     History of Changes
Other Study ID Numbers: ALXN1101-MCD-201
2013-002701-56 ( EudraCT Number )
First Posted: January 28, 2014    Key Record Dates
Last Update Posted: August 20, 2019
Last Verified: August 2019
Keywords provided by Origin Biosciences:
Molybdenum cofactor deficiency (MoCD)
molybdenum cofactor (MoCo) biosynthesis
sulfite oxidase (SO)
xanthine dehydrogenase
aldehyde oxidase
S sulfocysteine (SSC)
Additional relevant MeSH terms:
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Metal Metabolism, Inborn Errors
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Metabolic Diseases
Molybdenum
Trace Elements
Micronutrients
Nutrients
Growth Substances
Physiological Effects of Drugs