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

This study is ongoing, but not recruiting participants.
Information provided by (Responsible Party):
Promethera Biosciences
ClinicalTrials.gov Identifier:
First received: January 29, 2014
Last updated: October 2, 2014
Last verified: October 2014
The purpose of this study is to assess the long-term safety follow-up of patients having been treated with HepaStem.

Urea Cycle Disorders,
Crigler Najjar Syndrome

Study Type: Observational
Study Design: Observational Model: Cohort
Time Perspective: Prospective
Official Title: Long-term Safety Follow-up Study of Patients Having Received Infusions of HepaStem

Resource links provided by NLM:

Further study details as provided by Promethera Biosciences:

Primary Outcome Measures:
  • Characterisation of the long term safety profile of HepaStem therapy. [ Time Frame: 4 years ] [ Designated as safety issue: Yes ]

    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:
  • To characterize the disease evolution after having received HepaStem therapy and to report on general safety. [ Time Frame: 4 years ] [ Designated as safety issue: Yes ]

    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)

Enrollment: 9
Study Start Date: March 2013
Estimated Study Completion Date: October 2018
Estimated Primary Completion Date: October 2018 (Final data collection date for primary outcome measure)
Inborn errors of liver metabolism

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.

Ages Eligible for Study:   up to 23 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population
The SAF001 study will include all patients having received infusions of HepaStem in any former interventional study conducted by Promethera Biosciences.

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.

  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT02051049

Promethera Biosciences
Mont-Saint-Guibert, Belgium, 1435
Sponsors and Collaborators
Promethera Biosciences
Principal Investigator: Françoise Smets, MD Cliniques universitaires Saint-Luc (Belgium)
Principal Investigator: Dries Dobbelaere, MD/Prof CHRU de Lille - Hopital Jeanne de Flandre (France)
Principal Investigator: Isabel Gonçalves, MD/Prof Hospital Pediátrico de Coimbra (Portugal)
Principal Investigator: Stephanie Grunewald, MD Great Ormond Street Children Hospital
Principal Investigator: Giuliano Torre, MD IRCCS OSPEDALE PEDIATRICO DEL BAMBINO GESÃ (Roma)
Principal Investigator: Hanna Mandel, MD Rambam Health Corporation
  More Information

Responsible Party: Promethera Biosciences
ClinicalTrials.gov Identifier: NCT02051049     History of Changes
Other Study ID Numbers: SAF001 
Study First Received: January 29, 2014
Last Updated: October 2, 2014
Health Authority: Belgium: Federal Agency for Medicines and Health Products, FAMHP
France: Agence Nationale de Sécurité du Médicament et des produits de santé
Portugal: Autoridade Nacional do Medicamento e Produtos de Saude I.P (Infarmed)
Israel: Ministry of Health
Italy: Istituto Superiore di Sanita (ISS)
United Kingdom: Medicines and Healthcare Products Regulatory Agency

Keywords provided by Promethera Biosciences:

Additional relevant MeSH terms:
Crigler-Najjar Syndrome
Urea Cycle Disorders, Inborn
Amino Acid Metabolism, Inborn Errors
Brain Diseases
Brain Diseases, Metabolic
Brain Diseases, Metabolic, Inborn
Central Nervous System Diseases
Genetic Diseases, Inborn
Hyperbilirubinemia, Hereditary
Metabolic Diseases
Metabolism, Inborn Errors
Nervous System Diseases

ClinicalTrials.gov processed this record on May 22, 2016