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

NCT02051049 | Completed DMC

• 1 other identifier: SAF001
• Study Type: observational
• Target: 9
• Locations: 1 country
• Sites: 1

Brief Summary

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

Conditions

Urea Cycle Disorders; Crigler Najjar Syndrome

Keywords

CN; UCD

Outcome Measures

Primary Outcomes (1)

• 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 Outcomes (1)

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

  4 years

Study Arms (1)

Inborn errors of liver metabolism

Eligibility Criteria

• Age: Up to 23 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)
• 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.

You may qualify if:

• \- Subject having received HepaStem during a former interventional clinical study and who have terminated their participation in that study.

You may not qualify if:

• \- Subject has received mature liver cells, stem cells transplantation other than HepaStem, or organ liver transplant.

Sponsors & Collaborators

• Cellaion SA: lead

Study Sites (1)

Promethera Biosciences

Mont-Saint-Guibert, 1435, Belgium

Location

MeSH Terms

Conditions

Urea Cycle Disorders, Inborn; Crigler-Najjar Syndrome

Condition Hierarchy (Ancestors)

Brain Diseases, Metabolic, Inborn; Brain Diseases, Metabolic; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Amino Acid Metabolism, Inborn Errors; Metabolism, Inborn Errors; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Metabolic Diseases; Nutritional and Metabolic Diseases; Hyperbilirubinemia, Hereditary

Study Officials

• 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

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: January 29, 2014
• First Posted: January 31, 2014
• Study Start: March 1, 2013
• Primary Completion: April 1, 2018
• Study Completion: February 1, 2019
• Last Updated: May 28, 2019

Record last verified: 2019-05

Locations

BE (1)