Long-term Safety Follow-up Study of Patients Having Received HepaStem (SAF001)

Study Overview
=================
Brief Summary
-----------------
The purpose of this study is to assess the long-term safety follow-up of patients having been treated with HepaStem.

Detailed Description
-----------------
The primary objective of the SAF001 study is the long-term safety surveillance of the patients post infusion with HepaStem. Furthermore, the evolution of both the metabolic condition and the quality of life are followed. As much as possible, the surveillance will mimic the standard follow-up of the respective diseases (standard of care). The surveillance will end when the patient is undergoing an organ transplant or takes part in another research study. This surveillance will last up to a maximum of 48 months.

Official Title
-----------------
Long-term Safety Follow-up Study of Patients Having Received Infusions of HepaStem

Conditions
-----------------
Urea Cycle Disorders, Crigler Najjar Syndrome

Participation Criteria
=================
Eligibility Criteria
-----------------
Main Inclusion Criteria: - Subject having received HepaStem during a former interventional clinical study and who have terminated their participation in that study. Exclusion Criteria: - Subject has received mature liver cells, stem cells transplantation other than HepaStem, or organ liver transplant.

Ages Eligible for Study
-----------------
Maximum Age: 23 Years

Sexes Eligible for Study
-----------------
All

Accepts Healthy Volunteers
-----------------
No

Study Plan
=================
How is the study designed?
-----------------

Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Inborn errors of liver metabolism<br> | |

What is the study measuring?
-----------------
Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Characterisation of the long term safety profile of HepaStem therapy. | Assessment of safety will be achieved by evaluating the following parameters Physical examination Vital signs Laboratory tests Liver tumor markers Autoimmune markers related to liver pathology Anti-HLA antibodies specific for donor cell haplotypes Morphology of liver, bile ducts, and portal system by ultrasound Morphology of the kidneys by ultrasound Non-serious or serious Adverse Events of Special Interest (AESIs) and Serious Adverse Events (SAEs) related to HepaStem therapy. | 4 years | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| To characterize the disease evolution after having received HepaStem therapy and to report on general safety. | This assessment is based on the evaluation of: Report on cognitive skills, behaviour, and health-related quality of life indicators Non-serious or serious Adverse Events of Special Interest (AESIs) and Serious Adverse Events (SAEs) related to concomitant medications or other causes Indication I: Crigler-Najjar syndrome Frequency and severity of metabolic decompensation Metabolic parameters (serum total bilirubin) Report on supportive treatment and any adjustment of phototherapy and medication (eg phenobarbital treatment) Indication II: Urea cycle disorders Frequency and severity of metabolic decompensation Metabolic parameters (NH3 values, amino acids in plasma) Report on supportive treatment and any adjustment of: diet (natural protein intake, total protein intake, amino acid supplements) Medication (eg nitrogen scavengers) | 4 years | 

 Terms related to the study
=================
Keywords Provided by Centre Hospitalier Valida
-----------------
CN, UCD