Trial record 2 of 85 for:    urea cycle disorders

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

This study is currently recruiting participants. (see Contacts and Locations)
Verified June 2015 by Promethera Biosciences
Information provided by (Responsible Party):
Promethera Biosciences Identifier:
First received: November 6, 2014
Last updated: July 2, 2015
Last verified: June 2015

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

Condition Intervention Phase
Urea Cycle Disorders
Biological: HepaStem
Phase 2

Study Type: Interventional
Study Design: Endpoint Classification: Efficacy Study
Intervention Model: Single Group Assignment
Masking: 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

Resource links provided by NLM:

Further study details as provided by Promethera Biosciences:

Primary Outcome Measures:
  • 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 ] [ Designated as safety issue: No ]

Secondary Outcome Measures:
  • Efficacy as determined by de novo ureagenesis (C13 tracer method) [ Time Frame: up to 12 months post-first infusion day ] [ Designated as safety issue: No ]
  • Efficacy as determined by Ammonia (NH3) values [ Time Frame: up to 12 months post-first infusion day ] [ Designated as safety issue: No ]
  • Efficacy as determined by amino acids in plasma [ Time Frame: up to 12 months post-first infusion day ] [ Designated as safety issue: No ]
  • Efficacy as determined by report of metabolic decompensations [ Time Frame: up to 12 months post-first infusion day ] [ Designated as safety issue: No ]
  • 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 ] [ Designated as safety issue: No ]
  • 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 ] [ Designated as safety issue: No ]
  • To evaluate the safety during the year following HepaStem infusions (composite) [ Time Frame: up to 12 months post-first infusion day ] [ Designated as safety issue: Yes ]
    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.

Estimated Enrollment: 20
Study Start Date: October 2014
Estimated Study Completion Date: March 2017
Estimated Primary Completion Date: March 2017 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
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


Ages Eligible for Study:   up to 12 Years
Genders Eligible for Study:   Both
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.
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its identifier: NCT02489292

Contact: Joëlle Thonnard, MD +32 10 39 43 16
Contact: Pascale Hermant, PhD +32 10 39 43 12

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         
Hôpital de la Timone, CHU de Marseille Not yet recruiting
Marseille, France, 13385
Contact: Brigitte Chabrol, MD    +33 4 91 38 68 06   
Principal Investigator: Brigitte Chabrol, MD         
Hôpital des Enfants, CHU de Toulouse Not yet recruiting
Toulouse, France, 31059
Contact: Pierre Broué, MD    +33 5 34 55 85 66   
Principal Investigator: Pierre Broué, MD         
Centre hospitalier Pellegrin-Enfants Not yet recruiting
Vandoeuvre Les Nancy, France, 54500
Contact: François Feillet, MD, PhD    + 33 383 15 47 96   
Principal Investigator: François Feillet, MD, PhD         
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 Universitario Virgen de la Arrixaca Not yet recruiting
Murcia, Spain, 30008
Contact: David Gil Ortega, MD    +34 968 36 95 00   
Principal Investigator: David Gil Ortega, MD         
Hospital Materno Infantil de Málaga Not yet recruiting
Málaga, Spain, 29011
Contact: Javier Blasco Alonso, MD    +34 951 292 374   
Principal Investigator: Javier Blasco Alonso, MD         
Hospital Clínico Universitario Not yet recruiting
Santiago de Compostela, Spain, 15782
Contact: Maria Luz Couce, MD    +34 981 95 01 68   
Principal Investigator: Maria Luz Couce, MD         
Sponsors and Collaborators
Promethera Biosciences
  More Information

No publications provided

Responsible Party: Promethera Biosciences Identifier: NCT02489292     History of Changes
Other Study ID Numbers: HEP002
Study First Received: November 6, 2014
Last Updated: July 2, 2015
Health Authority: Belgium: Federal Agency for Medicines and Health Products, FAMHP
France: Agence Nationale de Sécurité du Médicament et des produits de santé
Spain: Agencia Española de Medicamentos y Productos Sanitarios

Keywords provided by Promethera Biosciences:
Urea Cycle Disorder
cell therapy
stem cell

Additional relevant MeSH terms:
Urea Cycle Disorders, Inborn
Amino Acid Metabolism, Inborn Errors
Brain Diseases
Brain Diseases, Metabolic
Brain Diseases, Metabolic, Inborn
Central Nervous System Diseases
Genetic Diseases, Inborn
Metabolic Diseases
Metabolism, Inborn Errors
Nervous System Diseases processed this record on October 09, 2015