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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT02047461
Recruitment Status : Enrolling by invitation
First Posted : January 28, 2014
Last Update Posted : March 18, 2020
Sponsor:
Information provided by (Responsible Party):
Origin Biosciences

Tracking Information
First Submitted Date  ICMJE January 9, 2014
First Posted Date  ICMJE January 28, 2014
Last Update Posted Date March 18, 2020
Actual Study Start Date  ICMJE April 2014
Estimated Primary Completion Date December 2020   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: August 19, 2019)
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
Original Primary Outcome Measures  ICMJE
 (submitted: January 24, 2014)
Safety of 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
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: August 19, 2019)
  • 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)
  • 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.
  • 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.
  • 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
  • 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
Original Secondary Outcome Measures  ICMJE
 (submitted: January 24, 2014)
  • Pharmacokinetics parameters of 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)
  • Effect of ALXN1101 on urine and blood biomarkers [ Time Frame: Up to Month 36 ]
    Change from baseline in urine and blood biomarkers.
  • Effect of ALXN1101 on neurologic function including motor examination [ Time Frame: Up to Month 36 ]
    Change from baseline on repeated Neurologic examinations such as tone, strengthen and reflexes.
  • Long-term safety of ALXN1101 [ Time Frame: Up to Month 36 ]
    • 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
  • Effect of ALXN1101 on cognitive functions [ Time Frame: Up to Month 36 ]
    Change from baseline in cognitive function using age appropriate measures of cognition, eg. Wechsler Preschoool and Primary Scale of intelligence
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Safety & Efficacy Study of ORGN001 (Formerly ALXN1101) in Pediatric Patients With MoCD Type A Currently Treated With rcPMP
Official Title  ICMJE 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)
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 Type  ICMJE Interventional
Study Phase  ICMJE Phase 2
Study Design  ICMJE Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE Molybdenum Cofactor Deficiency, Type A
Intervention  ICMJE Drug: ORGN001 (formerly ALXN1101)
IV infusion
Study Arms  ICMJE Experimental: ORGN001 (formerly ALXN1101)
daily IV infusions
Intervention: Drug: ORGN001 (formerly ALXN1101)
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Enrolling by invitation
Actual Enrollment  ICMJE
 (submitted: January 19, 2015)
7
Original Estimated Enrollment  ICMJE
 (submitted: January 24, 2014)
4
Estimated Study Completion Date  ICMJE December 2020
Estimated Primary Completion Date December 2020   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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.

Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE Child, Adult, Older Adult
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE Australia,   Netherlands,   Tunisia,   United Kingdom,   United States
Removed Location Countries Singapore
 
Administrative Information
NCT Number  ICMJE NCT02047461
Other Study ID Numbers  ICMJE ALXN1101-MCD-201
2013-002701-56 ( EudraCT Number )
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE Not Provided
Responsible Party Origin Biosciences
Study Sponsor  ICMJE Origin Biosciences
Collaborators  ICMJE Not Provided
Investigators  ICMJE Not Provided
PRS Account Origin Biosciences
Verification Date March 2020

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP