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

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

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

Study Overview

• Status: Completed
• Conditions: Urea Cycle Disorders; Crigler Najjar Syndrome

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.

• Study Type: Observational
• Enrollment (Actual): 9

Contacts and Locations

Study Locations

• Belgium
  • Mont-Saint-Guibert, Belgium, 1435
    • Promethera Biosciences

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: No older than 23 years (Child, Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

• 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.

Description

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.

Study Plan

How is the study designed?

Design Details

• Observational Models: Cohort
• Time Perspectives: Prospective

Number of groups / cohorts

1

Cohorts and Interventions

Group / Cohort
Inborn errors of liver metabolism

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Characterisation of the long term safety profile of HepaStem therapy.	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.	4 years

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
To characterize the disease evolution after having received HepaStem therapy and to report on general safety.	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)	4 years

Collaborators and Investigators

• Sponsor: Promethera Therapeutics
• 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); 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

Study record dates

Study Major Dates

• Study Start: March 1, 2013
• Primary Completion (Actual): April 1, 2018
• Study Completion (Actual): February 1, 2019

Study Registration Dates

• First Submitted: January 29, 2014
• First Submitted That Met QC Criteria: January 30, 2014
• First Posted (Estimate): January 31, 2014

Study Record Updates

• Last Update Posted (Actual): May 28, 2019
• Last Update Submitted That Met QC Criteria: May 23, 2019
• Last Verified: May 1, 2019

More Information

Terms related to this study

• Keywords: UCD; CN
• Additional Relevant MeSH Terms: Metabolic Diseases; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Genetic Diseases, Inborn; Metabolism, Inborn Errors; Brain Diseases, Metabolic; Brain Diseases, Metabolic, Inborn; Amino Acid Metabolism, Inborn Errors; Hyperbilirubinemia, Hereditary; Urea Cycle Disorders, Inborn; Crigler-Najjar Syndrome
• Other Study ID Numbers: SAF001