Clinical trial NCT02489292: Study to Evaluate the Efficacy of HepaStem in Urea Cycle Disorders Paediatric Patients (HEP002)
Official title: Prospective, Open Label, Multicenter, Efficacy and Safety Study of Several Infusions of HepaStem in Urea Cycle Disorders Paediatric Patients
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Urea Cycle Disorders
Keywords: Urea Cycle Disorder
UCD
cell therapy
stem cell
Lead sponsor: Cellaion SA
Sponsor class: INDUSTRY
First posted: 2015-07-03
Enrollment: 5
Sex: ALL
Maximum age (years): 12
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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


Eligibility 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.

Primary outcomes:
measure: Efficacy as determined by de novo ureagenesis (C13 tracer method)
timeFrame: at 6m post-first infusion day
Secondary outcomes:
measure: Efficacy as determined by de novo ureagenesis (C13 tracer method)
timeFrame: at 3, 9 and 12 months post-first infusion day
measure: Efficacy as determined by Ammonia (NH3) values
timeFrame: up to 12 months post-first infusion day
measure: Efficacy as determined by amino acids in plasma
timeFrame: up to 12 months post-first infusion day
measure: Efficacy as determined by report of metabolic decompensations
timeFrame: up to 12 months post-first infusion day
measure: Efficacy as determined by report on actual supportive treatment, adjustment of protein restriction and amino acids supplements
timeFrame: up to 12 months post-first infusion day
measure: Efficacy as determined report on behavior, cognitive skills and health-related quality-of-life indicators
timeFrame: up to 12 months post-first infusion day
measure: To evaluate the safety during the year following HepaStem infusions (composite)
timeFrame: up to 12 months post-first infusion day
Other outcomes:
Locations:
city: Brussels
country: Belgium
facility: Cliniques Universitaires Saint-Luc
city: Lille
country: France
facility: Hôpital Jeanne de Flandre, CHRU Lille
city: Warszawa
country: Poland
facility: Instytut - Pomnik Centrum Zdrowia Dziecka
city: Badajoz
country: Spain
facility: Hospital Materno Infatil de Badajoz
city: Barcelona
country: Spain
facility: Hospital Universitari Vall d'Hebron de Barcelona
city: Málaga
country: Spain
facility: Hospital Materno Infantil de Málaga
Study officials:
References: