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Completed

NCT Number: NCT02051049

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

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

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Key information

Conditions

Urea Cycle Disorders Amino Acid Metabolism, Inborn Errors Brain Diseases Brain Diseases, Metabolic Brain Diseases, Metabolic, Inborn Central Nervous System Diseases Congenital, Hereditary, and Neonatal Diseases and Abnormalities Crigler Najjar Syndrome Crigler-Najjar Syndrome Genetic Diseases, Inborn Hyperbilirubinemia, Hereditary Metabolic Diseases Metabolism, Inborn Errors Nervous System Diseases Nutritional and Metabolic Diseases Urea Cycle Disorders, Inborn

Age range

Up to 23 year

Sex eligibility

All sexes

Study type

Observational

Primary location

Promethera Biosciences

Mont-Saint-Guibert, 1435, Belgium

About this study

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.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

Primary outcomes

  1. Characterisation of the long term safety profile of HepaStem therapy.

    Time frame: 4 years

    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 outcomes

  1. To characterize the disease evolution after having received HepaStem therapy and to report on general safety.

    Time frame: 4 years

    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)

Sponsors and collaborators

Lead sponsor

Cellaion SA

Industry

Registry information

Official study title

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

Important dates

Study start
2013
Primary completion
2018
Study completion
2019
First posted
Jan 31, 2014
Registry last updated
May 28, 2019

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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