PROLONGSTEM is a long term follow-up study of all the patients who havereceived at least one infusion of HepaStem

Phase 1

• Conditions: This study will include all patients having received at least one infusionof the Investigational Medicinal Product (IMP) HepaStem, called Human Allogenic Liver-derived Progenitor Cells (HALPC), duringa previous interventional clinical study conducted by Promethera Biosciences or Promethera Therapeutics including patients suffering from urea cycle disorders (UCD), Crigler-Najjar (CN) and Fibro-inflammatory liver diseases; MedDRA version: 20.1Level: LLTClassification code 10021601Term: Inborn error of metabolism NOSSystem Organ Class: 100000004850; MedDRA version: 20.0Level: LLTClassification code 10008954Term: Chronic liver disease and cirrhosisSystem Organ Class: 100000004871; MedDRA version: 20.1Level: LLTClassification code 10049844Term: Acute liver failureSystem Organ Class: 100000004871; MedDRA version: 21.1Level: LLTClassification code 10013373Term: Disorders of urea cycle metabolismSystem Organ Class: 200000003094; Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]

• Registration Number: EUCTR2017-003989-27-BG
• Lead Sponsor: Promethera Therapeutics

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2019-10-23
• Last Update Posted: 23 May 2022

Recruitment & Eligibility

• Status: A
• Sex: All
• Target Recruitment: 48

Inclusion Criteria

1. Patients who have received at least one infusion of HepaStem during a previous interventional clinical study conducted by Promethera Therapeutics
Note: Patients currently participating in ongoing double-blinded controlled interventional studies during which HepaStem is administered will be invited to enter the PROLONGSTEM study. Once this double-blinded controlled interventional study is complete and/or treatment is unblinded, the patients will be informed about the treatment they received. HepaStem-recipients will remain in the PROLONGSTEM study, and the participation of control-recipients in the PROLONGSTEM study will be terminated.
2. Patients who are able to understand and give written informed consent
For children (as defined according to national regulations), an informed consent shall be sought from the parents or legal representative on behalf of the child. If the child is capable of understanding the purpose of the study, he/she should provide an informed assent and sign an assent form. Minor patients at the time of inclusion who become adult (according to national regulations) during the course of the PROLONGSTEM study should provide an informed consent (and sign the adul ICF) as soon as they reach adulthood.
For adult patients who are legally incapable of providing informed consent at the time of inclusion (due to an incapacitating condition such as brain disease or hepatic encephalopathy), a legal representative should provide a signed informed consent before enrollment. After improvement of the incapacitating condition, and as soon as the investigator considers that the patients have become capable to consent by themselves, the patients must sign the ICF.
Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 38
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 2

Exclusion Criteria

1. Patients who received an orthotopic liver transplantation and who completed the 6-month follow-up Organ Transplantation Questionnaire prior to the start of the PROLONGSTEM study.
Note: Patients who received an orthotopic liver transplantation and who did not complete the 6-month follow-up Organ Transplantation Questionnaire prior to the start of the PROLONGSTEM study may be included in the PROLONGSTEM study. These patients will remain in the PROLONGSTEM study until the completion of the 6-month follow-up Organ Transplantation Questionnaire. Data from the last visit of the previous clinical study conducted by the Sponsor (i.e., the start of the PROLONGSTEM study) up to the date of completion of the 6-month follow-up Organ Transplantation Questionnaire will be collected.
2. Patients who received mature liver cells or stem cells other than HepaStem prior to the start of the PROLONGSTEM study.
Note: Patients who received mature liver cells or stem cells other than HepaStem after the last visit of the previous clinical study conducted by the Sponsor (i.e., the start of the PROLONGSTEM study) may be included in the PROLONGSTEM study. Only retrospective data (between the last visit of the previous study and before the administration of such cells) will be collected from those patients.

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified