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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 : Completed
First Posted : July 3, 2015
Last Update Posted : October 19, 2020
Sponsor:
Information provided by (Responsible Party):
Promethera Therapeutics

Study Description
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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

Study Design
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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 5 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
Actual Study Start Date : October 2014
Actual Primary Completion Date : March 2017
Actual Study Completion Date : March 2017

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Arms and Interventions
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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


Outcome Measures
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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.


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

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.
Contacts and Locations
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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): NCT02489292


Locations
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Belgium
Cliniques Universitaires Saint-Luc
Brussels, Belgium, 1200
France
Hôpital Jeanne de Flandre, CHRU Lille
Lille, France, 59037
Poland
Instytut - Pomnik Centrum Zdrowia Dziecka
Warszawa, Poland
Spain
Hospital Materno Infatil de Badajoz
Badajoz, Spain, 06010
Hospital Universitari Vall d'Hebron de Barcelona
Barcelona, Spain, 08035
Hospital Materno Infantil de Málaga
Málaga, Spain, 29011
Sponsors and Collaborators
Promethera Therapeutics
More Information
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Responsible Party: Promethera Therapeutics
ClinicalTrials.gov Identifier: NCT02489292    
Other Study ID Numbers: HEP002
First Posted: July 3, 2015    Key Record Dates
Last Update Posted: October 19, 2020
Last Verified: October 2020
Keywords provided by Promethera Therapeutics:
Urea Cycle Disorder
UCD
cell therapy
stem cell
Additional relevant MeSH terms:
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Urea Cycle Disorders, Inborn
Disease
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