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D

Delta Research Partners | Bastrop, LA

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A Study to Evaluate the Efficacy and Safety of Rencofilstat in Subjects With NASH and Advanced Liver Fibrosis (ASCEND)

H

Hepion Pharmaceuticals

Status and phase

Active, not recruiting
Phase 2

Conditions

Fibrosis, Liver
Nonalcoholic Steatohepatitis (NASH)
NAFLD

Treatments

Drug: Rencofilstat
Drug: Placebo

Study type

Interventional

Funder types

Industry

Identifiers

NCT05402371
HEPA-CRV431-207

Details and patient eligibility

About

This is a randomized, double-blind, placebo-controlled, parallel-dosing, multi-center study to evaluate the efficacy and safety of Rencofilstat as evidenced by histopathological improvements in fibrosis in adult NASH subjects with F2 or F3 fibrosis (NASH CRN system). Antifibrotic biomarker activity will be evaluated on an exploratory basis.

Full description

This is a randomized, double-blind, placebo-controlled, parallel-dosing, multi-center study to evaluate the efficacy and safety of Rencofilstat as evidenced by histopathological improvements in fibrosis in adult NASH subjects with F2 or F3 fibrosis (NASH CRN system). Antifibrotic biomarker activity will be evaluated on an exploratory basis.

This study consists of 3 phases: (i) Screening and Randomization; (ii) treatment; and (iii) follow up. During Screening, each subject will provide informed consent prior to starting any study specific procedures. The randomization of the F2/F3 NASH subjects will be performed in a 1:1:1:1 ratio between Rencofilstat 75mg, Rencofilstat 150mg, Rencofilstat 225mg, and matching placebo. Subjects will be stratified by presence or absence of Type 2 diabetes, fibrosis stage and a maximum of 34 F2 subjects in each cohort.

Enrollment

120 patients

Sex

All

Ages

18 to 75 years old

Volunteers

No Healthy Volunteers

Inclusion and exclusion criteria

Inclusion Criteria Subjects must fulfill all the following inclusion criteria to be eligible for participation in the study.

  1. Male or female between 18 and 75 years of age (inclusive).

  2. Capable of giving written informed consent and able to effectively communicate with the investigator and study personnel.

  3. Histologic evidence of NASH based on central readings of the screening biopsy obtained no more than 6 months before Screening defined by presence of all 3 key histological features, Nonalcoholic Fatty Liver Disease Activity Score (NAS) ≥ 4 with at least 1 point each in lobular inflammation and hepatocyte ballooning.

    a. Historical biopsy may be substituted for Screening biopsy to determine eligibility if the following are met: i. Historical biopsy was obtained no more than 180 ± 5 days prior to the first day of Screening.

    ii. Hepatic tissue or slides are available for central histologic evaluation. iii. No new therapeutic intervention for NASH was made 90 days prior to screening (e.g., obeticholic acid, vitamin E ≥ 400 IU/day, pioglitazone, incretins [e.g., liraglutide, semaglutide], sodium-glucose cotransporter-2 [SGLT2] inhibitors).

    iv. Subjects must have been metabolically stable since the biopsy (no significant weight loss ≥ 7% of body weight, no major deterioration of glycemic control, and no introduction of new or investigational drugs for the treatment of Type 2 Diabetes).

  4. Histologic liver fibrosis stage 2 or 3 as defined by the Nonalcoholic Steatohepatitis Clinical Research Network (NASH CRN) scoring of liver fibrosis based on central reading of the Screening biopsy (refer to criteria 4a regarding use of a historical biopsy as a substitute for the Screening biopsy).

  5. Blood pressure up to 160/100 mmHg; potential subjects who meet other eligibility requirements, but who have out of range blood pressure measurements deemed to be not clinically significant by the investigator, may still be considered for study inclusion.

Exclusion Criteria Subjects who meet any of the following criteria prior to the first dose of study drug are not eligible for randomization.

  1. Pregnant or breastfeeding or planning to become pregnant during the study period.

  2. Known allergy to Rencofilstat, cyclosporine, or any of their inactive ingredients.

  3. Positive test for hepatitis B surface antigen (HBsAg), hepatitis C virus antibodies (HCVAb) or human immunodeficiency virus antibodies (HIVAb). If HCVAb test is positive, then an HCV-RNA test will be performed. If this test is negative, the subject is allowed to participate in the study, as long as the subject meets all other inclusion criteria and has never been treated for HCV or was treated >2 years ago and achieved a sustained virologic response at that time.

  4. Subjects with suspected and symptomatic severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2) infection identified prior to first dose.

  5. Subjects with a history of clinically significant acute cardiac events within 30 days prior to Screening such as stroke, transient ischemic attack, or coronary heart disease (angina pectoris requiring therapy, myocardial infarction, revascularization procedures with left ventricular ejection fraction [LVEF] <50% as determined by previous echocardiography or multiple gated acquisition [MUGA] scan).

  6. Subjects with uncontrolled or unstable cardiac arrhythmias:

    1. Severe conduction disturbance (e.g., second-degree or third-degree AV block).
    2. History of congenital long QT syndrome, congenital short QT syndrome, Torsades de Pointes, or Wolff Parkinson White syndrome.
  7. Subjects with transaminases >5 x upper limit of normal (ULN).

  8. Subjects with ALP >2 x ULN.

  9. Subjects with total serum bilirubin >1.5 x ULN.

  10. Subjects with a platelet count <100,000/mm3.

  11. Subjects with an INR ≥ 1.3 in the absence of anticoagulants.

  12. Subjects with albumin <3.5 g/dL.

  13. Model for End-Stage Liver Disease (MELD) score >12, unless due to an alternate etiology such as therapeutic anticoagulation.

  14. Current or previous (within the past 6 months) Child-Pugh (CP) score ≥ 7 for F2 or F3 subjects, unless due to an alternative etiology such as Gilbert's syndrome or therapeutic anticoagulation.

  15. An estimated glomerular filtration rate (eGFR) <60 mL/min/1.73 m2 (calculated by the Chronic Kidney Disease Epidemiology Collaboration [CKD-EPI] method).

  16. Subjects with hemoglobin A1c (HbA1c) >9.5%.

  17. Subjects with any history or presence of decompensated cirrhosis including ascites, hepatic encephalopathy or variceal bleeding.

  18. Other well documented causes of chronic liver disease according to standard diagnostic procedures including, but not restricted to:

    1. Suspicion of drug-induced liver disease.
    2. Alcoholic liver disease.
    3. Autoimmune hepatitis.
    4. Wilson's disease.
    5. Primary biliary cholangitis, primary sclerosing cholangitis.
    6. Genetic hemochromatosis (Homozygosity for C282Y or C282Y/H63D compound heterozygote).
    7. Known or suspected hepatocellular carcinoma (HCC).
    8. History or planned liver transplant.
    9. Clinical evidence of portal hypertension such as esophageal varices, ascites, history of hepatic encephalopathy, or splenomegaly.
  19. History of hepatic decompensating events or subjects who develop manifestations of hepatic decompensation between screening and enrollment should not be randomized, inclusive of new or worsening jaundice and ascites, new esophageal varices, etc.

  20. Subjects with Type 2 diabetes who have recent (< 3 months) changes in medication class or dose of the following antidiabetic medications: Glucagon-like-peptide-1 (GLP-1) receptor agonist, dipeptidyl peptidase-4 (DPP-4) inhibitor, sodium/glucose cotransporter 2 (SGLT2) inhibitor, thiazolidinediones (TZD).

  21. Received an investigational drug or investigational vaccine or used an investigational medical device within 60 days prior to first dose of study drug.

  22. Received any investigation products being evaluated for the treatment of liver fibrosis or NASH in the 6 months prior to the Screening liver biopsy.

  23. Inability to undergo MRE procedure due to unremovable metal or foreign object(s) or contraindication for any other reason including but not limited to pacemaker, shrapnel injury, extensive tattoos, severe claustrophobia, ear (cochlea) implant.

Trial design

Primary purpose

Treatment

Allocation

Randomized

Interventional model

Parallel Assignment

Masking

Quadruple Blind

120 participants in 4 patient groups, including a placebo group

Cohort A: Rencofilstat 75 mg
Experimental group
Description:
Eighty-four (84) biopsy-proven NASH F2/F3 subjects to complete study on Rencofilstat 75 mg daily.
Treatment:
Drug: Rencofilstat
Cohort B: Rencofilstat 150 mg
Experimental group
Description:
Eighty-four (84) biopsy-proven NASH F2/F3 subjects to complete study on Rencofilstat 150 mg daily.
Treatment:
Drug: Rencofilstat
Cohort C: Rencofilstat 225 mg
Experimental group
Description:
Eighty-four (84) biopsy-proven NASH F2/F3 subjects to complete study on Rencofilstat 225 mg daily.
Treatment:
Drug: Rencofilstat
Cohort D: Placebo
Placebo Comparator group
Description:
Eighty-four (84) biopsy-proven NASH F2 / F3 subjects to complete study on matching placebo.
Treatment:
Drug: Placebo

Trial contacts and locations

41

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Central trial contact

Kristen Fetchko

Data sourced from clinicaltrials.gov

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