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

Phase 1

• Conditions: This study will include all patients having received at least one infusion of the Investigational Medicinal Product (IMP) HepaStem HHALPC during a previous interventional clinical study conducted by Promethera Biosciences including patients suffering from urea cycle disorders (UCD), Crigler-Najjar (CN) and Fibro-inflammatory liver diseases; MedDRA version: 20.1 Level: LLT Classification code 10021601 Term: Inborn error of metabolism NOS System Organ Class: 100000004850; MedDRA version: 20.0 Level: LLT Classification code 10008954 Term: Chronic liver disease and cirrhosis System Organ Class: 100000004871; MedDRA version: 20.1 Level: LLT Classification code 10049844 Term: Acute liver failure System Organ Class: 100000004871; MedDRA version: 20.1 Level: LLT Classification code 10013373 Term: Disorders of urea cycle metabolism System Organ Class: 200000003094; Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]

• Registration Number: EUCTR2017-003989-27-GB
• Lead Sponsor: Promethera Biosciences

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2019-06-24
• Last Update Posted: 30 June 2019

Recruitment & Eligibility

• Status: Authorised-recruitment may be ongoing or finished
• Sex: Not specified
• Target Recruitment: 22

Inclusion Criteria

1- All patients having received at least one infusion of HepaStem during a previous interventional clinical study conducted by Promethera Biosciences.
2- Written Informed Consent.
For a patient who is a minor (according to the national regulations), and for adult patients legally incapable: the parents or legal representative have provided a signed informed consent before enrolment. If the patient is capable of signing, he/she is required to sign an assent in parallel.
For adult patient (legally capable): patient has provided a written informed consent before enrolment.

Are the trial subjects under 18? yes
Number of subjects for this age range: 8
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 12
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 OLT and completed 6-month FU documentation prior to the start of the PROLONGSTEM study.
2- Patients who have received mature liver cells or stem cells other than HepaStem prior to the start of the PROLONGSTEM study.

Study & Design

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

Primary Outcome Measures

Name	Time	Method
Main Objective: The objective of the PROLONGSTEM study in patients having received HepaStem in a previous interventional study (referred as Study A) is to assess the long-term safety of HepaStem therapy post administration.;Secondary Objective: No secondary objectives;<br> Primary end point(s): Occurrence of Adverse Events of Specific Interest (AESI):<br> - Event with fatal outcome (Death)<br> - Orthotopic Liver Transplantation (OLT) and Outcome.<br> - Development of Malignancy or unwanted tissue formation in different organs (tumor malignant or not).<br> - Disease linked to transmission of adventitious agents or reactivation of latent pathogens.<br> -Any AE which in the opinion of the investigator has a plausible causal relationship to HepaStem.<br><br> These AESI should be considered as medically important and reported as SAE.<br>