Safety Study of HepaStem for the Treatment of Urea Cycle Disorders (UCD) and Crigler-Najjar Syndrome (CN)

Inclusion Criteria

• •GENERAL:
• •1. Subject shows patency of the portal vein and branches, with normal flow velocity in the main portal vein as confirmed by Doppler ultrasound and accessibility of the portal vein, or respectively, accessibility of the umbilical vein.
• •2. Subject (if capable of signing) and parents or legal representative have provided a written informed assent/consent.
• •3. Female subjects of childbearing potential need to have a negative pregnancy test and must agree to use an acceptable method of contraception throughout the study.
• •Crigler-Najjar Syndrome specific:
• •Patient presents with Crigler-Najjar syndrome type 1.
• •Patient presents with Crigler-Najjar syndrome type 2, poorly controlled under phenobarbital treatment, or experiencing serious impairment in quality of life.
• •Diagnosis must be confirmed by genetic mutation analysis if not available.
• •Urea Cycle Disorders specific:
• •Diagnosis of one of the urea cycle disorders of which the disease is of such severity to warrant liver transplantation or alternatives despite full conservative therapy,
• •subject experiencing serious impairment in quality of life despite full conservative therapy.