Phase II Safety Study of 2 Dose Regimens of HepaStem in Patients With ACLF (HEP101)

Multicenter Phase II Safety and Preliminary Efficacy Study of 2 Dose Regimens of HepaStem in Patients With Acute on Chronic Liver Failure

The study will assess the safety of different dose regimens of HepaStem in cirrhotic Patients with ACLF or with acute decompensation at risk of developing ACLF up to Day 28 of the active study period.

Study Overview

• Status: Completed
• Conditions: Acute-On-Chronic Liver Failure
• Intervention / Treatment: Biological: HepaStem

• Study Type: Interventional
• Enrollment (Actual): 24
• Phase: Phase 2

Contacts and Locations

Study Locations

• Belgium
  • Brussels, Belgium, 1070
    • Hôpital Erasme
  • Bruxelles, Belgium, 1020
    • CHU Brugmann
  • Edegem, Belgium, 2650
    • UZ Antwerpen
  • Gent, Belgium, 9000
    • UZ Gent
  • Leuven, Belgium, 3000
    • KU Leuven
  • Liège, Belgium, 4000
    • CHU de Liege
  • Woluwe-Saint Lambert, Belgium, 1200
    • Cliniques St Luc
• France
  • Clichy, France, 92110
    • Hôpital Beaujon
  • Lyon, France, 69004
    • Hôpital de la Croix Rousse
  • Villejuif, France, 94804
    • Hopital Paul Brousse

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years to 70 years (ADULT, OLDER_ADULT)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

Main Inclusion Criteria:

• Diagnosed cirrhosis
• Patient with Acute Decompensation of cirrhosis
• Serum total Bilirubin ≥ 6 mg/dL (≥100 umol/L)
• The INR measurement has to be : 1.2 ≤ INR < 2

Main Exclusion Criteria:

• Absence of portal vein flow
• Known or medical history of thrombotic events
• Gastrointestinal hemorrhage requiring blood transfusion
• Variceal bading or sclerosis within 4 weeks before infusion
• Septic shock or non-controlled bacterial infection
• Clinical evidence of aspergilus infection.
• Circulatory failure
• Respiratory disordered
• Coagulation disorders defined as INR ≥ 2, Fibrinogen < 100 mg/dL or Platelets < 50.000/mm3
• MELD score > 30.
• Major invasive procedure within 4 weeks before infusion
• Previous organ transplantation and/or ongoing immunosuppressive treatments.
• Renal failure due to chronic kidney disease.
• Clinically significant left-right cardiac shunt.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: TREATMENT
• Allocation: NON_RANDOMIZED
• Interventional Model: SINGLE_GROUP
• Masking: NONE

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
OTHER: Low dose cohort; Two dose regimens of HepaStem will be given, which differ in the amount of cells per infusion. The low dose regimen will be given to the first cohort (first 6 patients included in the study).	Biological: HepaStem
OTHER: High dose cohort; The high dose regimen will be given to the second cohort after evaluation of the safety of the 1st cohort (stepwise approach)	Biological: HepaStem

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
Safety assessed by occurence of Adverse Events (AE) up to Day 28 of the active study period	up to 28Day post first infusion day

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Efficacy assessed by clinical parameters	Clinical efficacy parameters evaluated by mortality, liver transplantation and disease scoring.	28Day, 3 month and 1 year post first infusion day
Efficacy assessed by biological parameters	Biological efficacy parameters evaluated by bilirubin, creatinine, INR and albumin values	28Day, 3 month and 1 year post first infusion day
Long term safety Follow up assessed by the occurence of Adverse Event of Special Interest	Adverse Event of Special Interest defined as : SAE (Serious Adverse Event) with fatal	3 month and 1 year post first infusion day

Collaborators and Investigators

• Sponsor: Promethera Therapeutics

Study record dates

Study Major Dates

• Study Start (ACTUAL): December 1, 2016
• Primary Completion (ACTUAL): July 1, 2019
• Study Completion (ACTUAL): July 1, 2020

Study Registration Dates

• First Submitted: October 13, 2016
• First Submitted That Met QC Criteria: October 25, 2016
• First Posted (ESTIMATE): October 27, 2016

Study Record Updates

• Last Update Posted (ACTUAL): October 14, 2020
• Last Update Submitted That Met QC Criteria: October 13, 2020
• Last Verified: October 1, 2020

More Information

Terms related to this study

• Keywords: Acute-On-Chronic Liver Failure; ACLF
• Additional Relevant MeSH Terms: Digestive System Diseases; Liver Diseases; Liver Failure, Acute; End Stage Liver Disease; Liver Failure; Hepatic Insufficiency; Acute-On-Chronic Liver Failure
• Other Study ID Numbers: HEP101