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

Study Status: Active, not recruiting
Recruit Status: Active, not recruiting
Study Type: Observational

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

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

Detailed Summary: 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.
Sponsor: Promethera Biosciences

Current Primary Outcome: 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.


Original Primary Outcome: Characterisation of the long term safety profile of HepaStem therapy. [ Time Frame: Up to 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.


Current Secondary Outcome: 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)


Original Secondary Outcome: To characterize the disease evolution after having received HepaStem therapy and to report on general safety. [ Time Frame: Up to 4 years ]

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)

Indication 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)


Information By: Promethera Biosciences

Dates:
Date Received: January 29, 2014
Date Started: March 2013
Date Completion: October 2018
Last Updated: August 4, 2016
Last Verified: August 2016