Clinical trial NCT04229901: Study to Evaluate the Efficacy and Safety of HepaStem in Patients With Acute on Chronic Liver Failure (ACLF)
Official title: Randomized, Placebo-controlled, Double Blind, Multi-centre Phase IIb Study to Evaluate the Efficacy and Safety of HepaStem in Patients With Acute on Chronic Liver Failure (ACLF
Study type: INTERVENTIONAL
Overall status: TERMINATED
Phase: PHASE2
Conditions: Acute on Chronic Liver Failure
Keywords: ACLF
Hepastem
HALPC
Lead sponsor: Cellaion SA
Sponsor class: INDUSTRY
First posted: 2020-01-18
Enrollment: 133
Sex: ALL
Minimum age (years): 18
Maximum age (years): 75
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: This is an interventional, double blind, randomized (2:1), and placebo-controlled study of 2 infusions of a 1 dose regimen of HepaStem in patients recently diagnosed (≤1 week) with ACLF grade 1 or 2 on top of Standard of Care (SoC), and for whom the diagnosis is not resolved on the day of infusion.


Eligibility criteria: Inclusion Criteria:

1. Are adults aged between 18 and 75 years old.
2. Have an initial diagnosis of ACLF at the investigational site.
3. Have ACLF grade 1 or 2 according to the EASL-CLIF Consortium definition.
4. Have a total bilirubin ≥ 5 mg/dL.
5. Are able to read, understand and give written informed consent.

Main Exclusion Criteria:

1. Have a MELD-Na score \> 35.
2. Have underlying cirrhosis due to biliary disease.
3. Have underlying cirrhosis due to autoimmune hepatitis.
4. Have active bleeding at a non-compressible site or at a compressible site that, in the opinion of the investigator, poses an unacceptable risk for the patient's participation in the study.
5. Have received treatment for bleeding complications during the current hospitalization and has a persistent high risk for re-bleeding that, in the opinion of the investigator, poses an unacceptable risk for the patient's participation in the study.
6. Have a complete portal vein thrombosis.
7. Have coagulation disturbances defined as:

   * fibrinogen \< 80 mg/dL
   * platelets \< 50 x 10³/mm³
8. Are requiring chronic dialysis therapy.
9. Have had a cerebrovascular, myocardial, limb arterial thrombotic event, or history for both thrombotic and hemorrhagic cerebrovascular events within 12 months prior to the Screening and not considered stabilized by the investigator.
10. Have a previous history of myocardial infarction and/or cardiac failure, with an ejection fraction rate (EFR) ≤ 40%.

Primary outcomes:
measure: Survival
timeFrame: Day 90
Secondary outcomes:
measure: Liver transplant-free survival
timeFrame: Day 90
measure: Transplant-free survival while free of ACLF
timeFrame: Day 90
measure: Transplant-free survival with MELD-Na score < 15
timeFrame: Day 90
measure: Duration of overall hospitalization and hospitalization in ICU and non-ICU during the index hospitalization
timeFrame: up to Day 90
Other outcomes:
Locations:
city: Vienna
country: Austria
facility: Medical University Of Vienna
city: Antwerp
country: Belgium
facility: UZ Antwerpen
city: Brussels
country: Belgium
facility: CHU Brugmann
city: Brussels
country: Belgium
facility: CHU Erasme
city: Gent
country: Belgium
facility: Universitair Ziekenhuis Gent
city: Leuven
country: Belgium
facility: UZ Leuven
city: Liège
country: Belgium
facility: CHC MontLégia
city: Woluwe-Saint-Lambert
country: Belgium
facility: Cliniques Universitaires Saint-Luc
city: Pleven
country: Bulgaria
facility: University Multiprofile Hospital for Active Treatment "Dr. Georgi Stranski"
city: Ruse
country: Bulgaria
facility: UMHAT Medica
city: Sofia
country: Bulgaria
facility: Military Medical Academy
city: Sofia
country: Bulgaria
facility: UMHAT Sveta Anna
city: Sofia
country: Bulgaria
facility: UMHAT Tzarita Joanna
city: Aarhus
country: Denmark
facility: Aarhus University Hospital
city: Tallinn
country: Estonia
facility: North Estonia Regional Hospital
city: Tallinn
country: Estonia
facility: Lääne-Tallinna Keskhaigla AS
city: Angers
country: France
facility: CHU Angers
city: Clichy
country: France
facility: Hopital Beaujon
city: Créteil
country: France
facility: Assistance Publique Hopitaux De Paris
city: Grenoble
country: France
facility: Centre Hospitalier Universitaire Grenoble Alpes
city: Lyon
country: France
facility: Hospices Civils De Lyon
city: Marseille
country: France
facility: Assistance Publique Hopitaux De Marseille
city: Montpellier
country: France
facility: CHU Montpellier
city: Paris
country: France
facility: Assistance Publique-Hopitaux de Paris - Hopitaux Universitaires Pitie-Salpetriere
city: Strasbourg
country: France
facility: Les Hopitaux Universitaires De Strasbourg
city: Toulouse
country: France
facility: CHU De Toulouse
city: Villejuif
country: France
facility: Hospital Paul Brousse
city: Hanover
country: Germany
facility: Medizinische Hochschule Hannover
city: Leipzig
country: Germany
facility: University Hospital Of Leipzig
city: Ulm
country: Germany
facility: University Hospital Of Ulm AöR
city: Milan
country: Italy
facility: Azienda Ospedaliera Ospedale Niguarda CA' Granda
city: Palermo
country: Italy
facility: Azienda Ospedaliera Universitaria Policlinico
city: Udine
country: Italy
facility: Azienda Sanitaria Universitaria Friuli Centrale
city: Riga
country: Latvia
facility: Pauls Stradins Clinical University Hospital
city: Kaunas
country: Lithuania
facility: Lietuvos Sveikatos Mokslų Universiteto Ligoninė Kauno Klinikos
city: Skopje
country: North Macedonia
facility: PHI General City Hospital "8mi Septemvri"
city: Skopje
country: North Macedonia
facility: PHI University Clinic of Gastroenterohepatology
city: Mysłowice
country: Poland
facility: Id Clinic
city: Rzeszów
country: Poland
facility: Kliniczny Szpital Wojewódzki Nr 2 Im. Św. Jadwigi Królowej W Rzeszowie
city: Banská Bystrica
country: Slovakia
facility: F.D. Roosevelt University General Hospital Of Banská Bystrica
city: Barcelona
country: Spain
facility: Hospital Universitari Vall D'Hebron
city: Córdoba
country: Spain
facility: Hospital Universitario Reina Sofia
city: Madrid
country: Spain
facility: University Hospital Ramón Y Cajal
city: Majadahonda
country: Spain
facility: Hospital Universitario Puerta De Hierro De Majadahonda
city: Sabadell
country: Spain
facility: Sabadell Hospital
city: Zaragoza
country: Spain
facility: Hospital Clinico Universitario Lozano Blesa
city: Zaragoza
country: Spain
facility: Hospital Unviersitario Miguel Servet
Study officials:
References: