Study to Evaluate the Efficacy of HepaStem in Urea Cycle Disorders Paediatric Patients (HEP002)
- Conditions
- Urea Cycle Disorders
- Interventions
- Biological: HepaStem
- Registration Number
- NCT02489292
- Lead Sponsor
- Cellaion SA
- 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 available
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- All
- Target Recruitment
- 5
- 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
- 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.
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SINGLE_GROUP
- Arm && Interventions
Group Intervention Description HepaStem HepaStem Target total dose 50x10E6 cells/kg
- Primary Outcome Measures
Name Time Method Efficacy as determined by de novo ureagenesis (C13 tracer method) at 6m post-first infusion day
- Secondary Outcome Measures
Name Time Method Efficacy as determined by de novo ureagenesis (C13 tracer method) at 3, 9 and 12 months post-first infusion day Efficacy as determined by Ammonia (NH3) values up to 12 months post-first infusion day Efficacy as determined by amino acids in plasma up to 12 months post-first infusion day Efficacy as determined by report of metabolic decompensations 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 up to 12 months post-first infusion day Efficacy as determined report on behavior, cognitive skills and health-related quality-of-life indicators up to 12 months post-first infusion day To evaluate the safety during the year following HepaStem infusions (composite) 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.
Trial Locations
- Locations (6)
Hôpital Jeanne de Flandre, CHRU Lille
🇫🇷Lille, France
Hospital Materno Infatil de Badajoz
🇪🇸Badajoz, Spain
Instytut - Pomnik Centrum Zdrowia Dziecka
🇵🇱Warszawa, Poland
Hospital Universitari Vall d'Hebron de Barcelona
🇪🇸Barcelona, Spain
Hospital Materno Infantil de Málaga
🇪🇸Málaga, Spain
Cliniques Universitaires Saint-Luc
🇧🇪Brussels, Belgium