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Clinical Trial Details — Status: Completed

Administrative data

NCT number NCT02489292
Other study ID # HEP002
Secondary ID
Status Completed
Phase Phase 2
First received
Last updated
Start date October 2014
Est. completion date March 2017

Study information

Verified date October 2020
Source Promethera Biosciences
Contact n/a
Is FDA regulated No
Health authority
Study type Interventional

Clinical Trial Summary

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


Recruitment information / eligibility

Status Completed
Enrollment 5
Est. completion date March 2017
Est. primary completion date March 2017
Accepts healthy volunteers No
Gender All
Age group N/A to 12 Years
Eligibility 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.

Study Design


Intervention

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

Locations

Country Name City State
Belgium Cliniques Universitaires Saint-Luc Brussels
France Hôpital Jeanne de Flandre, CHRU Lille Lille
Poland Instytut - Pomnik Centrum Zdrowia Dziecka Warszawa
Spain Hospital Materno Infatil de Badajoz Badajoz
Spain Hospital Universitari Vall d'Hebron de Barcelona Barcelona
Spain Hospital Materno Infantil de Málaga Málaga

Sponsors (1)

Lead Sponsor Collaborator
Promethera Biosciences

Countries where clinical trial is conducted

Belgium,  France,  Poland,  Spain, 

Outcome

Type Measure Description Time frame Safety issue
Primary Efficacy as determined by de novo ureagenesis (C13 tracer method) at 6m post-first infusion day
Secondary Efficacy as determined by de novo ureagenesis (C13 tracer method) at 3, 9 and 12 months post-first infusion day
Secondary Efficacy as determined by Ammonia (NH3) values up to 12 months post-first infusion day
Secondary Efficacy as determined by amino acids in plasma up to 12 months post-first infusion day
Secondary Efficacy as determined by report of metabolic decompensations up to 12 months post-first infusion day
Secondary 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
Secondary Efficacy as determined report on behavior, cognitive skills and health-related quality-of-life indicators up to 12 months post-first infusion day
Secondary To evaluate the safety during the year following HepaStem infusions (composite) 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. up to 12 months post-first infusion day
See also
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