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

Prospective, Open Label, Multicenter, Efficacy and Safety Study of Several Infusions of HepaStem in Urea Cycle Disorders Paediatric Patients

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

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.