This trial is active, not recruiting.

Conditions urea cycle disorders,, crigler najjar syndrome
Sponsor Promethera Biosciences
Start date March 2013
End date October 2018
Trial size 9 participants
Trial identifier NCT02051049, SAF001


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

United States No locations recruiting
Other countries No locations recruiting

Study Design

Observational model cohort
Time perspective prospective

Primary Outcomes

Characterisation of the long term safety profile of HepaStem therapy.
time frame: 4 years

Secondary Outcomes

To characterize the disease evolution after having received HepaStem therapy and to report on general safety.
time frame: 4 years

Eligibility Criteria

Male or female participants up to 23 years old.

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.

Additional Information

Official title Long-term Safety Follow-up Study of Patients Having Received Infusions of HepaStem
Principal investigator Françoise Smets, MD
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.
Trial information was received from ClinicalTrials.gov and was last updated in August 2016.
Information provided to ClinicalTrials.gov by Promethera Biosciences.