Trial record 3 of 4 for:    "Crigler-Najjar syndrome"

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

This study is currently recruiting participants. (see Contacts and Locations)
Verified January 2014 by Promethera Biosciences
Sponsor:
Information provided by (Responsible Party):
Promethera Biosciences
ClinicalTrials.gov Identifier:
NCT02051049
First received: January 29, 2014
Last updated: January 30, 2014
Last verified: January 2014
  Purpose

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


Condition
Urea Cycle Disorders,
Crigler Najjar Syndrome

Study Type: Observational
Study Design: Observational Model: Cohort
Time Perspective: Prospective
Official Title: SAF 001: A 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: Up to 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: Up to 4 years ] [ Designated as safety issue: Yes ]

    This assessment will be 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 reported as AESI
    • Metabolic parameters
    • Report on supportive treatment and any adjustment of:

      • Phototherapy
      • Medication (eg phenobarbital treatment)

    Indications II: Urea cycle disorders

    • Frequency and severity of metabolic decompensation reported as AESI
    • Metabolic parameters
    • Report on supportive treatment and any adjustment of:

      • diet (natural protein intake, total protein intake, amino acid supplements)
      • Medication (eg nitrogen scavengers)


Biospecimen Description:

Blood samples


Estimated Enrollment: 20
Study Start Date: March 2013
Estimated Study Completion Date: March 2017
Estimated Primary Completion Date: March 2017 (Final data collection date for primary outcome measure)
Groups/Cohorts
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.

  Eligibility

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 PB.

Criteria

Inclusion Criteria:

  • All patients having received infusions of HepaStem during a former interventional clinical study conducted by PB and who have terminated their participation in that study.
  • For subjects under 18 years of age (16 year in UK) and for adult subjects legally incapable: the subject (if capable of signing) and parents or legal representative have provided a written informed assent/consent before enrolment or For an adult subject legally capable: the subject has provided a written informed consent before enrolment.

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

Contacts
Contact: Béatrice De Vos, MD, PhD 32 10 39 43 07 beatrice.devos@promethera.com
Contact: Joëlle Thonnard, MD 32 10 39 43 16 joelle.thonnard@promethera.com

Locations
Belgium
Promethera Biosciences Recruiting
Mont-Saint-Guibert, Belgium, 1435
Contact: Béatrice De Vos, MD, PhD    +32 10 39 43 07    beatrice.devos@promethera.com   
Contact: Joelle Thonnard, MD    +32 10 39 43 16    joelle.thonnard@promethera.com   
Sponsors and Collaborators
Promethera Biosciences
Investigators
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)
  More Information

No publications provided

Responsible Party: Promethera Biosciences
ClinicalTrials.gov Identifier: NCT02051049     History of Changes
Other Study ID Numbers: SAF001
Study First Received: January 29, 2014
Last Updated: January 30, 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

Keywords provided by Promethera Biosciences:
CN
UCD

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

ClinicalTrials.gov processed this record on July 24, 2014