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

Completed

• Conditions: Urea Cycle Disorders; Crigler Najjar Syndrome

• Registration Number: NCT02051049
• Lead Sponsor: Cellaion SA

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.

Study Timeline

• Study Start: 2013-03
• Study First Submitted: 2014-01-29
• Study First Submitted QC: 2014-01-30
• Study First Posted: 2014-01-31
• Primary Completion: 2018-04
• Study Completion: 2019-02
• Status Verified: 2019-05
• Last Update Submitted: 2019-05-23
• Last Update Posted: 2019-05-28

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 9

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.

Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Characterisation of the long term safety profile of HepaStem therapy.	4 years	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.

Secondary Outcome Measures

Name	Time	Method
To characterize the disease evolution after having received HepaStem therapy and to report on general safety.	4 years	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)

Trial Locations

Locations (1)

Promethera Biosciences; 🇧🇪 Mont-Saint-Guibert, Belgium