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

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Cellaïon SA

Participant type

Pediatric, Patients

Age range

0-17 years, 18-64 years, 65+ years

Gender

Male and Female

Therapeutic area

Diseases [C] - Nutritional and Metabolic Diseases [C18]

Trial duration

12 Apr 2018 → 23 May 2024

Decision date (initial)

2023-03-02

Transition trial

Yes

Low-intervention

No

Rare-disease indication

Yes

Vulnerable population

Yes

Funding sources

Cellaïon SA

External identifiers

EU CT number

2022-500251-22-00

EudraCT number

2017-003989-27

ClinicalTrials.gov

NCT03343756

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Safety

To assess the long-term safety of HepaStem therapy post administration in patients who have been infused with at least one dose of HepaStem in a previous interventional clinical study conducted by Promethera Therapeutics.

Conditions and MedDRA coding

This study will include all patients having received at least one infusion of the Investigational Medicinal Product (IMP) HepaStem HALPC during a previous interventional clinical study conducted by Promethera Biosciences or Promethera Therapeutics including patients suffering from urea cycle disorders (UCD), Crigler-Najjar (CN) and Fibroinflammatory liver diseases.

Regulatory references

Scientific advice from competent authorities

European Medicines Agency, Medicines And Healthcare Products Regulatory Agency, Paul Ehrlich Institute, Federal Agency For Medicines And Health Products

EMA paediatric investigation plan (PIP)

EMEA-001155-PIP01-11

Plan to share IPD

No

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 2

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 doubleblinded 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 adult 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.

Exclusion criteria 2

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.

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. To document the occurrence of adverse events of special interest (AESIs) as defined below: • Event with fatal outcome (death) • Organ transplantation and outcome • Development of malignancy or unwanted tissue formation in different organs (tumor development malignant or not) • Disease linked to transmission of adventitious agents or reactivation of latent pathogens • Any adverse event (AE) judged to have a plausible causal relationship to HepaStem.

Investigational products

Investigational medicinal products (IMPs), comparators, placebo, auxiliary

Test 1

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Cellaïon SA

Sponsor organisation

Cellaïon SA

Address

Rue Granbonpre 11

City

Mont-Saint-Guibert

Postcode

1435

Country

Belgium

Scientific contact point

Organisation

Cellaïon SA

Contact name

Regulatory Affairs

Public contact point

Organisation

Cellaïon SA

Contact name

Regulatory Affairs

Third parties 1

Locations

5 EU/EEA countries · 29 investigational sites

By country

Investigational sites

Country notifications

Trial-start, recruitment-start, end and early-termination notifications submitted per Member State

Results and documents

Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial

Summary of results Art. 37(4) CTR

Layperson summary Annex V

Documents 2 files

Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.

Application history

7 submissions — initial application plus substantial / non-substantial modifications