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出境医 / 临床实验 / Safety and Tolerability of HepaStem in Patients With Cirrhotic and Pre-cirrhotic NASH Patients (PANASH)

Safety and Tolerability of HepaStem in Patients With Cirrhotic and Pre-cirrhotic NASH Patients (PANASH)

Study Description
Brief Summary:
Multicenter, open-label, safety and tolerability study of ascending doses of HepaStem in patients with cirrhotic and pre-cirrhotic non-alcoholic steato-hepatitis (NASH) to determine the safety and tolerability of ascending single and repeated doses of HepaStem administered to patients with cirrhotic and pre-cirrhotic non-alcoholic steato-hepatitis (NASH)

Condition or disease Intervention/treatment Phase
NASH - Nonalcoholic Steatohepatitis Drug: HepaStem Phase 1 Phase 2

Study Design
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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 23 participants
Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Multicenter, Open-label, Safety and Tolerability Study of Ascending Doses of HepaStem in Patients With Cirrhotic and Pre-cirrhotic Non-alcoholic Steato-hepatitis (NASH)
Actual Study Start Date : April 9, 2019
Actual Primary Completion Date : May 28, 2020
Actual Study Completion Date : August 31, 2020
Arms and Interventions
Arm Intervention/treatment
Experimental: F4 patient population
A total of 2 doses are planned to be administered as single or repeated infusions in an ascending manner:
 Drug: HepaStem
Heterologous human adult liver-derived progenitor cells
Experimental: F3 patient population
A total of 2 doses are planned to be administered as single or repeated infusions in an ascending manner:
 Drug: HepaStem
Heterologous human adult liver-derived progenitor cells
Outcome Measures
Primary Outcome Measures :
  1. Incidence of Adverse Event [ Time Frame: up to Day 28 ]
    Safety and Tolerability


Eligibility Criteria
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Ages Eligible for Study:   18 Years to 70 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Main Inclusion Criteria:

  • Able and willing to provide written informed consent and comply with the requirements of this study protocol
  • Age 18 to 70-years old, inclusive
  • Proven diagnosis of NASH based on histological evidence from biopsy performed within 6 months for F3 patients and within 2 years for F4 patients prior to Screening If no biopsy is available within these time windows, a biopsy should be performed at Screening NB: For F4 patients for whom the biopsy cannot confirm the diagnosis of NASH, any other causes of underlying liver diseases should be excluded

Main Exclusion Criteria:

  • Alcoholic liver disease or alcohol consumption exceeding the daily intake of 140g/w (two doses) for women and of 210g/w (three doses) for men
  • Other causes of liver disease including, but not limited to, alcoholic liver disease, active hepatitis B (HbsAg+), hepatitis C (PCR positive), autoimmune disorders, drug-induced hepatotoxicity, Wilson disease, hemochromatosis, and alpha-1-antitryspin deficiency based on medical history and/ or clinical and biological assessment
  • Recent recurrent or ongoing thrombotic or bleeding events within 3 months prior the screening
  • Patients considered at persistent risk of thrombosis or bleeding at the time of screening
  • Patients with high risk of Gastro intestinal bleeding at time of the screening.
  • Cerebrovascular, myocardial, or limb arterial thrombotic event within 12 months prior to the screening and/or not considered stabilized by the investigator
  • Bariatric surgery within 1 year prior to the screening
  • Coagulation disturbances defined as (Drolz et al. 2016, Nadim et al. 2016, Stravitz et al. 2018, Green et al. 2018): fibrinogen at < 80 mg/dL and/or platelets at < 40 x 10³/mm3
  • Severe hepatic encephalopathy (defined by West Haven grade > 2)
  • Acute Decompensation of cirrhosis with Chronic Liver Failure Consortium Acute Decompensation (CLIF-C AD) score > 60
  • Acute on Chronic liver failure (ACLF) grade 1, 2 ,3
  • MELD score > 20
  • Child Pugh score ≥ C
Contacts and Locations

Locations
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Belgium
Cliniques Universitaires St Luc
Brussels, Belgium, 1200
CUB Erasme
Brussel, Belgium, 1070
University Hospital Antwerp (UZA)
Edegem, Belgium, 2650
UZ Gent
Gent, Belgium, 9000
Bulgaria
University Multiprofile Hospital for Active Treatment "Tsaritsa Yoana - ISUL"
Sofia, Bulgaria, 1527
Multiprofile hospital for active treatment (MHAT) Sofia Military Medical Academy
Sofia, Bulgaria, 1606
Trakia Park Hospital
Stara Zagora, Bulgaria, 6004
France
CHU Bordeaux
Bordeaux, France, 33604
Paul Brousse Hospital
Villejuif, France, 94804
Spain
Vall d'Hebron
Barcelona, Spain, 08035
Hospital de la Santa Creu i Sant Pau
Barcelona, Spain, 08041
Hospital General Universitario Gregorio Marañon
Madrid, Spain, 28007
Hospital Universitario Ramón y Cajal
Madrid, Spain, 28034
Sponsors and Collaborators
Promethera Biosciences
Investigators
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Study Chair: Etienne SOKAL, MD, PhD CSMO
Tracking Information
First Submitted Date  ICMJE May 23, 2019
First Posted Date  ICMJE May 28, 2019
Last Update Posted Date October 14, 2020
Actual Study Start Date  ICMJE April 9, 2019
Actual Primary Completion Date May 28, 2020   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: May 24, 2019) 		Incidence of Adverse Event [ Time Frame: up to Day 28 ]
Safety and Tolerability
Original Primary Outcome Measures  ICMJE Same as current
Change History
Current Secondary Outcome Measures  ICMJE Not Provided
Original Secondary Outcome Measures  ICMJE Not Provided
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Safety and Tolerability of HepaStem in Patients With Cirrhotic and Pre-cirrhotic NASH Patients
Official Title  ICMJE Multicenter, Open-label, Safety and Tolerability Study of Ascending Doses of HepaStem in Patients With Cirrhotic and Pre-cirrhotic Non-alcoholic Steato-hepatitis (NASH)
Brief Summary Multicenter, open-label, safety and tolerability study of ascending doses of HepaStem in patients with cirrhotic and pre-cirrhotic non-alcoholic steato-hepatitis (NASH) to determine the safety and tolerability of ascending single and repeated doses of HepaStem administered to patients with cirrhotic and pre-cirrhotic non-alcoholic steato-hepatitis (NASH)
Detailed Description Not Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 1
Phase 2
Study Design  ICMJE Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE NASH - Nonalcoholic Steatohepatitis
Intervention  ICMJE Drug: HepaStem
Heterologous human adult liver-derived progenitor cells
Study Arms  ICMJE
  • Experimental: F4 patient population
    A total of 2 doses are planned to be administered as single or repeated infusions in an ascending manner:
    Intervention: Drug: HepaStem
  • Experimental: F3 patient population
    A total of 2 doses are planned to be administered as single or repeated infusions in an ascending manner:
    Intervention: Drug: HepaStem
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Completed
Actual Enrollment  ICMJE
 (submitted: October 13, 2020) 		23
Original Estimated Enrollment  ICMJE
 (submitted: May 24, 2019) 		24
Actual Study Completion Date  ICMJE August 31, 2020
Actual Primary Completion Date May 28, 2020   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

Main Inclusion Criteria:

  • Able and willing to provide written informed consent and comply with the requirements of this study protocol
  • Age 18 to 70-years old, inclusive
  • Proven diagnosis of NASH based on histological evidence from biopsy performed within 6 months for F3 patients and within 2 years for F4 patients prior to Screening If no biopsy is available within these time windows, a biopsy should be performed at Screening NB: For F4 patients for whom the biopsy cannot confirm the diagnosis of NASH, any other causes of underlying liver diseases should be excluded

Main Exclusion Criteria:

  • Alcoholic liver disease or alcohol consumption exceeding the daily intake of 140g/w (two doses) for women and of 210g/w (three doses) for men
  • Other causes of liver disease including, but not limited to, alcoholic liver disease, active hepatitis B (HbsAg+), hepatitis C (PCR positive), autoimmune disorders, drug-induced hepatotoxicity, Wilson disease, hemochromatosis, and alpha-1-antitryspin deficiency based on medical history and/ or clinical and biological assessment
  • Recent recurrent or ongoing thrombotic or bleeding events within 3 months prior the screening
  • Patients considered at persistent risk of thrombosis or bleeding at the time of screening
  • Patients with high risk of Gastro intestinal bleeding at time of the screening.
  • Cerebrovascular, myocardial, or limb arterial thrombotic event within 12 months prior to the screening and/or not considered stabilized by the investigator
  • Bariatric surgery within 1 year prior to the screening
  • Coagulation disturbances defined as (Drolz et al. 2016, Nadim et al. 2016, Stravitz et al. 2018, Green et al. 2018): fibrinogen at < 80 mg/dL and/or platelets at < 40 x 10³/mm3
  • Severe hepatic encephalopathy (defined by West Haven grade > 2)
  • Acute Decompensation of cirrhosis with Chronic Liver Failure Consortium Acute Decompensation (CLIF-C AD) score > 60
  • Acute on Chronic liver failure (ACLF) grade 1, 2 ,3
  • MELD score > 20
  • Child Pugh score ≥ C
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 18 Years to 70 Years   (Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE Belgium,   Bulgaria,   France,   Spain
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT03963921
Other Study ID Numbers  ICMJE HEP201
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE
Plan to Share IPD: No
Responsible Party Promethera Biosciences
Study Sponsor  ICMJE Promethera Biosciences
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Study Chair: Etienne SOKAL, MD, PhD CSMO
PRS Account Promethera Biosciences
Verification Date October 2020

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP

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