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

NCT ID: NCT02051049

Last Updated: 2019-05-28

Basic Information

• Recruitment Status: COMPLETED
• Total Enrollment: 9 participants
• Study Classification: OBSERVATIONAL
• Study Start Date: 2013-03-31
• Study Completion Date: 2019-02-28

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.

Conditions

Urea Cycle Disorders; Crigler Najjar Syndrome

Study Design

• Observational Model Type: COHORT
• Study Time Perspective: PROSPECTIVE

Study Groups

Inborn errors of liver metabolism

No interventions assigned to this group

Eligibility Criteria

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.

• Maximum Eligible Age: 23 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Cellaion SA

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Françoise Smets, MD

Role: PRINCIPAL_INVESTIGATOR

Cliniques universitaires Saint-Luc (Belgium)

Dries Dobbelaere, MD/Prof

Role: PRINCIPAL_INVESTIGATOR

CHRU de Lille - Hopital Jeanne de Flandre (France)

Isabel Gonçalves, MD/Prof

Role: PRINCIPAL_INVESTIGATOR

Hospital Pediátrico de Coimbra (Portugal)

Stephanie Grunewald, MD

Role: PRINCIPAL_INVESTIGATOR

Great Ormond Street Children Hospital

Giuliano Torre, MD

Role: PRINCIPAL_INVESTIGATOR

IRCCS OSPEDALE PEDIATRICO DEL BAMBINO GESÃ (Roma)

Hanna Mandel, MD

Role: PRINCIPAL_INVESTIGATOR

Rambam Health Corporation

Locations

Promethera Biosciences

Mont-Saint-Guibert, , Belgium

Countries

Belgium

Other Identifiers

SAF001

Identifier Type: -

Identifier Source: org_study_id