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: 20.1Level: 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: 20.1Level: 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-ES

Lead Sponsor: Promethera Biosciences

Brief Summary: Not available

Detailed Description: Not available

Recruitment & Eligibility

Status: Authorised-recruitment may be ongoing or finished

Sex: All

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: Longitudinal evaluation of the long-term safety post-HepaStem administration.;Secondary Objective: Not applicable;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>These AESI should be considered as medically important and reported as SAE.;Timepoint(s) of evaluation of this end point: Patients who previously received the IMP HepaStem in the context of an interventional study with HepaStem administration will be followed-up for long-term safety surveillance up to 5 years in this PROLONGSTEM study.

Secondary Outcome Measures