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Trial record 1 of 2 for:    HEP002
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Study to Evaluate the Efficacy of HepaStem in Urea Cycle Disorders Paediatric Patients (HEP002)

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. 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
Information provided by (Responsible Party):
Promethera Biosciences

Brief Summary:
The aim of the study is to assess the efficacy of HepaStem treatment in paediatric patients suffering from urea cycle disorders.

Condition or disease Intervention/treatment Phase
Urea Cycle Disorders Biological: HepaStem Phase 2

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 20 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Prospective, Open Label, Multicenter, Efficacy and Safety Study of Several Infusions of HepaStem in Urea Cycle Disorders Paediatric Patients
Study Start Date : October 2014
Estimated Primary Completion Date : March 2017
Estimated Study Completion Date : March 2017

Arm Intervention/treatment
Experimental: HepaStem
Target total dose 50x10E6 cells/kg
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

Primary Outcome Measures :
  1. Efficacy as determined by de novo ureagenesis (C13 tracer method) [ Time Frame: at 6m post-first infusion day ]

Secondary Outcome Measures :
  1. Efficacy as determined by de novo ureagenesis (C13 tracer method) [ Time Frame: at 3, 9 and 12 months post-first infusion day ]
  2. Efficacy as determined by Ammonia (NH3) values [ Time Frame: up to 12 months post-first infusion day ]
  3. Efficacy as determined by amino acids in plasma [ Time Frame: up to 12 months post-first infusion day ]
  4. Efficacy as determined by report of metabolic decompensations [ Time Frame: up to 12 months post-first infusion day ]
  5. 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 ]
  6. 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 ]
  7. 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.

Information from the National Library of Medicine

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Ages Eligible for Study:   up to 12 Years   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

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.

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 identifier (NCT number): NCT02489292

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Contact: Joëlle Thonnard, MD +32 10 39 43 16

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Cliniques Universitaires Saint-Luc Recruiting
Brussels, Belgium, 1200
Contact: Françoise Smets, MD, PhD    +32 2 764 19 20      
Principal Investigator: Françoise Smets, MD, PhD         
Hôpital Jeanne de Flandre, CHRU Lille Not yet recruiting
Lille, France, 59037
Contact: Dries Dobbelaere, MD    +33 3 20 44 41 49   
Principal Investigator: Dries Dobbelaere, MD         
Instytut - Pomnik Centrum Zdrowia Dziecka Recruiting
Warszawa, Poland
Contact: Dariusz Rokicki, MD   
Principal Investigator: Dariusz Rokicki, MD         
Hospital Materno Infatil de Badajoz Recruiting
Badajoz, Spain, 06010
Contact: Ana Maria Marquez, MD    +34 636529711   
Principal Investigator: Ana Maria Marquez, MD         
Hospital Universitari Vall d'Hebron de Barcelona Recruiting
Barcelona, Spain, 08035
Contact: Jesús Quintero Bernabeu, MD    +34 675 78 15 47   
Principal Investigator: Jesús Quintero Bernabeu, MD         
Hospital Materno Infantil de Málaga Recruiting
Málaga, Spain, 29011
Contact: Javier Blasco Alonso, MD    +34 951 292 374   
Principal Investigator: Javier Blasco Alonso, MD         
Sponsors and Collaborators
Promethera Biosciences
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Responsible Party: Promethera Biosciences Identifier: NCT02489292    
Other Study ID Numbers: HEP002
First Posted: July 3, 2015    Key Record Dates
Last Update Posted: August 5, 2016
Last Verified: August 2016
Keywords provided by Promethera Biosciences:
Urea Cycle Disorder
cell therapy
stem cell
Additional relevant MeSH terms:
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Urea Cycle Disorders, Inborn
Pathologic Processes
Brain Diseases, Metabolic, Inborn
Brain Diseases, Metabolic
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Amino Acid Metabolism, Inborn Errors
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Metabolic Diseases