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.|
|ClinicalTrials.gov Identifier: NCT02489292|
Recruitment Status : Completed
First Posted : July 3, 2015
Last Update Posted : October 19, 2020
- Study Details
- Tabular View
- No Results Posted
- How to Read a Study Record
|Condition or disease||Intervention/treatment||Phase|
|Urea Cycle Disorders||Biological: HepaStem||Phase 2|
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||5 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|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|
Target total dose 50x10E6 cells/kg
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
- Efficacy as determined by de novo ureagenesis (C13 tracer method) [ Time Frame: at 6m post-first infusion day ]
- 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.
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, Learn About Clinical Studies.
|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.
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
|Cliniques Universitaires Saint-Luc|
|Brussels, Belgium, 1200|
|Hôpital Jeanne de Flandre, CHRU Lille|
|Lille, France, 59037|
|Instytut - Pomnik Centrum Zdrowia Dziecka|
|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|
|Responsible Party:||Promethera Therapeutics|
|Other Study ID Numbers:||
|First Posted:||July 3, 2015 Key Record Dates|
|Last Update Posted:||October 19, 2020|
|Last Verified:||October 2020|
Urea Cycle Disorder
Urea Cycle Disorders, Inborn
Brain Diseases, Metabolic, Inborn
Brain Diseases, Metabolic
Central Nervous System Diseases
Nervous System Diseases
Amino Acid Metabolism, Inborn Errors
Metabolism, Inborn Errors
Genetic Diseases, Inborn