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

Urea Cycle Disorders Clinical Trial

Official title:

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT02051049
• Other study ID #: SAF001
• Status: Completed
• Start date: March 2013
• Est. completion date: February 2019

Study information

• Verified date: May 2019
• Source: Promethera Biosciences
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational

Clinical Trial Summary

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

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 9
• Est. completion date: February 2019
• Est. primary completion date: April 2018
• Accepts healthy volunteers: No
• Gender: All
• Age group: N/A to 23 Years

Eligibility

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.

Related Conditions & MeSH terms

• Crigler Najjar Syndrome
• Crigler-Najjar Syndrome
• Urea Cycle Disorders
• Urea Cycle Disorders, Inborn

Locations

Country	Name	City	State
Belgium	Promethera Biosciences	Mont-Saint-Guibert

Sponsors (1)

Lead Sponsor	Collaborator
Promethera Biosciences

Country where clinical trial is conducted

Belgium, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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	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