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Study to Evaluate the Efficacy of HepaStem in Urea Cycle Disorders Paediatric Patients (HEP002)

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ClinicalTrials.gov Identifier: NCT02489292
Recruitment Status : Unknown
Verified August 2016 by Promethera Biosciences.
Recruitment status was:  Recruiting
First Posted : July 3, 2015
Last Update Posted : August 5, 2016
Sponsor:
Information provided by (Responsible Party):
Promethera Biosciences

Tracking Information
First Submitted Date  ICMJE November 6, 2014
First Posted Date  ICMJE July 3, 2015
Last Update Posted Date August 5, 2016
Study Start Date  ICMJE October 2014
Estimated Primary Completion Date March 2017   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: August 4, 2016)
Efficacy as determined by de novo ureagenesis (C13 tracer method) [ Time Frame: at 6m post-first infusion day ]
Original Primary Outcome Measures  ICMJE
 (submitted: July 2, 2015)
Change of ammonia up to 12 months post-first infusion day as compared to prior medical condition. [ Time Frame: at baseline, 3m, 6m, 9m, and 12m follow-up ]
Change History Complete list of historical versions of study NCT02489292 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures  ICMJE
 (submitted: August 4, 2016)
  • Efficacy as determined by de novo ureagenesis (C13 tracer method) [ Time Frame: at 3, 9 and 12 months post-first infusion day ]
  • Efficacy as determined by Ammonia (NH3) values [ Time Frame: up to 12 months post-first infusion day ]
  • Efficacy as determined by amino acids in plasma [ Time Frame: up to 12 months post-first infusion day ]
  • Efficacy as determined by report of metabolic decompensations [ Time Frame: up to 12 months post-first infusion day ]
  • Efficacy as determined by report on actual supportive treatment, adjustment of protein restriction and amino acids supplements [ Time Frame: up to 12 months post-first infusion day ]
  • Efficacy as determined report on behavior, cognitive skills and health-related quality-of-life indicators [ Time Frame: up to 12 months post-first infusion day ]
  • To evaluate the safety during the year following HepaStem infusions (composite) [ Time Frame: up to 12 months post-first infusion day ]
    Safety evaluation in terms of (1) clinical status, (2) portal vein hemodynamics, (3) morphology of the liver, bile ducts and portal system, (4) laboratory tests, (5) De novo detection of donor-specific circulating anti-human leukocyte antigen (HLA) antibodies, and/or other immune-related markers, (6) serious adverse events and clinically significant adverse events related to HepaStem, technical intervention, and concomitant treatments.
Original Secondary Outcome Measures  ICMJE
 (submitted: July 2, 2015)
  • Efficacy as determined by de novo ureagenesis (C13 tracer method) [ Time Frame: up to 12 months post-first infusion day ]
  • Efficacy as determined by Ammonia (NH3) values [ Time Frame: up to 12 months post-first infusion day ]
  • Efficacy as determined by amino acids in plasma [ Time Frame: up to 12 months post-first infusion day ]
  • Efficacy as determined by report of metabolic decompensations [ Time Frame: up to 12 months post-first infusion day ]
  • Efficacy as determined by report on actual supportive treatment, adjustment of protein restriction and amino acids supplements [ Time Frame: up to 12 months post-first infusion day ]
  • Efficacy as determined report on behavior, cognitive skills and health-related quality-of-life indicators [ Time Frame: up to 12 months post-first infusion day ]
  • To evaluate the safety during the year following HepaStem infusions (composite) [ Time Frame: up to 12 months post-first infusion day ]
    Safety evaluation in terms of (1) clinical status, (2) portal vein hemodynamics, (3) morphology of the liver, bile ducts and portal system, (4) laboratory tests, (5) De novo detection of donor-specific circulating anti-human leukocyte antigen (HLA) antibodies, and/or other immune-related markers, (6) serious adverse events and clinically significant adverse events related to HepaStem, technical intervention, and concomitant treatments.
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Study to Evaluate the Efficacy of HepaStem in Urea Cycle Disorders Paediatric Patients (HEP002)
Official Title  ICMJE Prospective, Open Label, Multicenter, Efficacy and Safety Study of Several Infusions of HepaStem in Urea Cycle Disorders Paediatric Patients
Brief Summary The aim of the study is to assess the efficacy of HepaStem treatment in paediatric patients suffering from urea cycle disorders.
Detailed Description Not Provided
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 Urea Cycle Disorders
Intervention  ICMJE Biological: HepaStem
HepaStem will be administered in maximum 4 infusion days, spread over an 8-week period with an interval of 2 to 3 weeks between infusion days. The target total dose of cells will be 50x10E6 cells/kg body weight
Study Arms  ICMJE Experimental: HepaStem
Target total dose 50x10E6 cells/kg
Intervention: Biological: HepaStem
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 Unknown status
Estimated Enrollment  ICMJE
 (submitted: July 2, 2015)
20
Original Estimated Enrollment  ICMJE Same as current
Estimated Study Completion Date  ICMJE March 2017
Estimated Primary Completion Date March 2017   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

Main Inclusion Criteria:

  • Paediatric patients < 12 years prior to infusion
  • Patient presents with UCD
  • Patient shows patency of the portal vein and branches, with normal flow velocity as confirmed by Doppler US and accessibility of the portal vein and /or affluants.

Main Exclusion Criteria:

  • Patient has mild disease severity, easily controlled under standard of care therapy, with no recurrent metabolic crises.
  • Patient is registered on a liver transplant waiting list or is scheduled for living donor liver transplantation before the end of the study.
  • Patient presents acute liver failure.
  • Patient presents clinical or radiological evidence of liver cirrhosis.
  • Patient presents or has a history of hepatic or extrahepatic malignancy.
  • Patient has a known clinically significant cardiac malformation.
  • Patient has a personal history of venous thrombosis, or has a clinically significant abnormal value for protein S, protein C, anti-thrombin III, and /or activated Protein C Resistance (aPCR) at screening. In case of known family history, a complete coagulation work-up should be performed. In all above described cases, results need to be discussed with PB before enrolling the patient in the study.
  • Patient had or has a renal insufficiency treated by dialysis.
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE up to 12 Years   (Child)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE Belgium,   France,   Poland,   Spain
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT02489292
Other Study ID Numbers  ICMJE HEP002
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE Not Provided
Responsible Party Promethera Biosciences
Study Sponsor  ICMJE Promethera Biosciences
Collaborators  ICMJE Not Provided
Investigators  ICMJE Not Provided
PRS Account Promethera Biosciences
Verification Date August 2016

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