A Randomized, Double-blind Study to Assess the Safety and Efficacy of EDP-305 in Subjects With Liver-biopsy Proven NASH

NCT04378010 | Terminated Phase 2 Results DMC FDA Drug

• 2 other identifiers: EDP 305-1022019-003876-38
• Study Type: interventional
• Target: 98
• Locations: 6 countries
• Sites: 81

Brief Summary

A randomized, double-blind study to assess the safety and efficacy of EDP-305 in subjects with liver-biopsy proven Non-Alcoholic Steatohepatitis (NASH)

Conditions

Non-Alcoholic Steatohepatitis

Keywords

Fatty Liver, non-alcoholic fatty liver disease, NASH

Outcome Measures

Primary Outcomes (1)

• Proportion of Participants Who Achieve ≥1 Stage Improvement in Fibrosis Without Worsening of Steatohepatitis and/or Resolution of Steatohepatitis and no Worsening of Liver Fibrosis as Determined by Liver Biopsy

  Proportion of participants who achieve ≥1 stage improvement in fibrosis without worsening of steatohepatitis and/or resolution of steatohepatitis and no worsening of liver fibrosis as determined by liver biopsy.

  Week 72

Secondary Outcomes (20)

• Change in 5D-itch Scale From Baseline

  Baseline, Week 12

• Proportion of Participants With Improvement of Fibrosis by at Least 1 Stage and/or Resolution of NASH Without Worsening of Either as Determined by Liver Biopsy

  Week 72

• Proportion of Participants With no Worsening of Fibrosis Combined With no Worsening of NASH as Determined by Liver Biopsy

  Week 72

• Proportion of Participants With Resolution of Fibrosis as Determined by Liver Biopsy

  Week 72

• Proportion of Participants With Improvement in Each Histologic Feature of NASH by at Least 1 Point as Determined by Liver Biopsy

  Week 72

Study Arms (3)

EDP-305 1.5 mg

EXPERIMENTAL

Once a day orally for 72 weeks

Drug: EDP-305 1.5 mg

EDP-305 2 mg

EXPERIMENTAL

Once a day orally for 72 weeks

Drug: EDP-305 2 mg

Placebo

PLACEBO COMPARATOR

Once a day orally for 72 weeks

Drug: Placebo

Interventions

EDP-305 1.5 mg DRUG

Tablet

Also known as: EDP-305

EDP-305 1.5 mg

EDP-305 2 mg DRUG

Tablet

Also known as: EDP-305

EDP-305 2 mg

Placebo DRUG

Tablet

Placebo

Eligibility Criteria

• Age: 18 Years - 75 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Informed consent documentation signed and dated by the participant.
• Male and female participants, of all ethnic origins, between the ages of 18 and 75 years, inclusive.
• Participants of all ethnic origins had to have a Body Mass Index (BMI) \> 25 kg/m2 and ≤ 45 except Asian participants who qualified for the study with BMI \> 23 kg/m2.
• Histological evidence of definite NASH based on NASH Clinical Research Network (CRN) criteria obtained from assessment of a liver biopsy by the central histopathologist. The biopsy may be obtained either 1) during the Screening window or 2) within 26 weeks prior to the Screening visit.
• NAFLD Activity Score (NAS) of 4 or greater with a score of at least 1 in each component of the NAS (steatosis scored 0-3, lobular inflammation scored 0-3, ballooning scored 0-2).
• Fibrosis stage 2 or 3 using the NASH CRN Histologic Scoring System.
• Participants had to have Screening laboratory values for Hepatitis B surface antigen (HBsAg), anti-HCV antibodies and HCV RNA, and Human Immunodeficiency Virus (HIV) 1 and 2 antibodies (Ab) as seronegative. \[Note: participants previously infected by chronic hepatitis C and treated with direct acting antivirals (DAAs) with sustained virologic response (SVR) for at least 3 years were allowed.\]
• A woman of childbearing potential who was sexually active with a male had to agree to use two effective methods of contraception from the date of Screening until 30 days after the last dose of study drug.
• A male participant who had not had a vasectomy and was sexually active with a woman of childbearing potential had to agree to use effective contraception from the date of Screening to 90 days after the last dose of study drug.
• Participant had to be willing and able to adhere to the assessments, visit schedules, prohibitions and restrictions, as described in this protocol.

You may not qualify if:

• Laboratory Screening results as indicated below:
• Total white blood cells (WBC) \<3000 cells/mm3
• Absolute neutrophil count (ANC) \<1500 cells/mm3
• Platelet count \<140,000/mm3
• International Normalized Ratio, INR \>1.2 (unless due to use of anticoagulants)
• Estimated glomerular filtration rate (eGFR) \< 60 mL/min according to the Modification of Diet in Renal Disease (MDRD) equation
• AST ≥5× ULN
• ALT ≥5× ULN
• ALP ≥2× ULN
• Total bilirubin \> 1.5 times ULN during Screening. \[Note: Patients with Gilbert's syndrome were allowed following review by the Medical Monitor if they had a known history of Gilbert's syndrome with a normal direct bilirubin value and normal reticulocyte count.\]
• Pregnant or nursing females.
• MELD: Model for End-stage Liver Disease score \>12.
• Clinical or laboratory evidence of known chronic liver disease such as alcoholic liver disease, primary biliary cholangitis (PBC), primary sclerosing cholangitis (PSC), autoimmune hepatitis, Wilson disease, iron overload, alpha-1-antitrypsin deficiency, drug-induced liver injury, known or suspected hepatocellular carcinoma (HCC).
• History of acute liver complications due to gallstones (e.g., acute cholecystitis or acute biliary obstruction) unless the participant had a cholecytectomy (more than 3 months prior to screening).
• History of liver transplant, or current placement on a liver transplant list.

Sponsors & Collaborators

• Enanta Pharmaceuticals, Inc: lead

Study Sites (81)

Arizona Liver Health

Chandler, Arizona, 85224, United States

Location

The Institute of Liver Health

Glendale, Arizona, 85306, United States

Location

Dignity Health DBA St. Joseph's Hospital and Medical Center

Phoenix, Arizona, 85013, United States

Location

Del Sol Research Management LLC

Tucson, Arizona, 85710, United States

Location

Rajeev Krishan, MD, Inc

Bakersfield, California, 93309, United States

Location

eStudy Site

Chula Vista, California, 91911, United States

Location

Southern California Research Center

Coronado, California, 92118, United States

Location

St. Jude Hospital Yorba Linda DBA St. Joseph Heritage Healthcare

Fullerton, California, 92835, United States

Location

National Institute of Clinical Research, Inc

Garden Grove, California, 92844, United States

Location

eStudySite - La Mesa

La Mesa, California, 91942, United States

Location

Om Research LLC

Lancaster, California, 93534, United States

Location

Keck Medical Center Of USC

Los Angeles, California, 90033, United States

Location

Inland Empire Liver Foundation

Rialto, California, 92377, United States

Location

UC Davis Medical Center

Sacramento, California, 95817, United States

Location

Southern California Gastrointestinal and Liver Centers

San Clemente, California, 92673, United States

Location

Precision Research Institute, Llc

San Diego, California, 92114, United States

Location

Paradigm Clinical Research Institute

Torrance, California, 90502, United States

Location

Universal Axon Clinical Research

Doral, Florida, 33166, United States

Location

Fleming Island Center for Clinical Research

Fleming Island, Florida, 32003, United States

Location

Universal Axon- Homestead, LLC

Homestead, Florida, 33030, United States

Location

Nature Coast Clinical Research

Inverness, Florida, 34452, United States

Location

Westside Center for Clinical Research

Jacksonville, Florida, 32205, United States

Location

Jacksonville Center for Endoscopy - Southside ; Borland Groover Clinic

Jacksonville, Florida, 32226, United States

Location

Encore Borland Groover Clinical Research

Jacksonville, Florida, 32256, United States

Location

Meridien Research

Lakeland, Florida, 33803, United States

Location

Meridien Research

Maitland, Florida, 32751, United States

Location

Research Associates of South Florida

Miami, Florida, 33134, United States

Location

University of Miami, Miller School of Medicine-Don Soffer Clinical Research Center

Miami, Florida, 33136, United States

Location

Well Pharma Medical Research, Corp.

Miami, Florida, 33173, United States

Location

Med Research Of Florida, LLC

Miami, Florida, 33186, United States

Location

San Marcus Research Clinic, Inc.

Miami Lakes, Florida, 33014, United States

Location

Ocala GI Research

Ocala, Florida, 34471, United States

Location

IMIC, Inc.

Palmetto Bay, Florida, 33157, United States

Location

Meridien Research

St. Petersburg, Florida, 33709, United States

Location

Guardian Angel Research

Tampa, Florida, 33614, United States

Location

Agile Clinical Research Trials, LLC

Atlanta, Georgia, 30328, United States

Location

Northwestern University

Chicago, Illinois, 60611, United States

Location

Rush University Medical Center - University Cardiovascular Surgeons

Chicago, Illinois, 60612-3852, United States

Location

Digestive Research Alliance of Michiana

South Bend, Indiana, 46635, United States

Location

University Of Iowa Hospital & Clinics

Iowa City, Iowa, 52242, United States

Location

Ochsner Health System

New Orleans, Louisiana, 70115-6969, United States

Location

University of Maryland

Baltimore, Maryland, 21201, United States

Location

Mercy Medical Center

Baltimore, Maryland, 21202, United States

Location

Digestive Disease Associates, PA

Catonsville, Maryland, 21228, United States

Location

Mid-Atlantic GI Research

Greenbelt, Maryland, 20770, United States

Location

Henry Ford Health Hospital

Detroit, Michigan, 48202, United States

Location

Southern Therapy and Advanced Research LLC GI Associates and Endoscopy Center

Jackson, Mississippi, 39216, United States

Location

St. Louis Univ. School Of Medicine

St Louis, Missouri, 63104, United States

Location

AGA Clinical Research Associates, LLC

Egg Harbor, New Jersey, 08234, United States

Location

Intercity Gastroenterology

Fresh Meadows, New York, 11366, United States

Location

New York University Medical Centre

New York, New York, 10016, United States

Location

University of Rochester Medical Center School of Medicine and Dentistry

Rochester, New York, 14642, United States

Location

Northeast GI Research Division

Concord, North Carolina, 28027, United States

Location

Carolinas HealthCare System Digestive - Huntersville

Huntersville, North Carolina, 28078, United States

Location

Lucas Research

Morehead City, North Carolina, 28557, United States

Location

Cleveland Clinic - Taussig Cancer Institute

Cleveland, Ohio, 44195, United States

Location

University of Pittsburgh Medical Center - Center for Liver Diseases

Pittsburgh, Pennsylvania, 15213, United States

Location

Digestive Health Research, LLC

Hermitage, Tennessee, 37076, United States

Location

Digestive Health Research

Lebanon, Tennessee, 37090, United States

Location

Quality Medical Research

Nashville, Tennessee, 37211, United States

Location

Texas Clinical Research Institute

Arlington, Texas, 76012, United States

Location

Texas Diabetes & Endocrinology

Austin, Texas, 78749, United States

Location

Crescent Health Clinical

DeSoto, Texas, 75115, United States

Location

DHAT Research Institute

Garland, Texas, 75044, United States

Location

American Research Corporation at The Texas Liver Institute

San Antonio, Texas, 78215, United States

Location

Clinical Trials of Texas, Inc.

San Antonio, Texas, 78229, United States

Location

Bon Secours St. Mary's Hospital of Richmond, Inc

Newport News, Virginia, 23602, United States

Location

Liver Institute Northwest

Seattle, Washington, 98105, United States

Location

CINME

Buenos Aires, Buenos Aires F.D., C1056AB, Argentina

Location

University of Calgary

Calgary, Alberta, T2N 4Z6, Canada

Location

Klinikum der Johann Wolfgang Goethe-Universitaet Frankfurt

Frankfurt am Main, Hesse, 60590, Germany

Location

Universitaetsmedizin der Johannes Gutenberg Universitaet Mainz KoeR

Mainz, Rhineland-Palatinate, 55131, Germany

Location

Latin Clinical Trial Center

San Juan, 00909, Puerto Rico

Location

MediNova West London Quality Research Site

Wokingham, Berkshire, RG40 1XS, United Kingdom

Location

MeDiNova East London Quality Research Site

Romford, Essex, RM1 3PJ, United Kingdom

Location

King's College Hospital - King's College Hospital NHS Foundation Trust

London, Greater London, SE5 9RS, United Kingdom

Location

MeDiNova South London Quality Research Site

Sidcup, Kent, DA14 6LT, United Kingdom

Location

MeDiNova Northampton Dedicated research site

Corby, Northamptonshire, NN18 9EZ, United Kingdom

Location

MeDiNova Warwickshire Quality Research Site

Kenilworth, Warwickshire, CV81JD, United Kingdom

Location

MeDiNova Yorkshire Quality Research Site

Shipley, Yorkshire, BD18 3SA, United Kingdom

Location

MeDiNova North London Quality Research Site

Middlesex, HA6 2RN, United Kingdom

Location

MeSH Terms

Conditions

Non-alcoholic Fatty Liver Disease; Fatty Liver

Interventions

EDP-305

Condition Hierarchy (Ancestors)

Liver Diseases; Digestive System Diseases

Limitations and Caveats

No limitations reported for the study.

Results Point of Contact

• Title: Alaa Ahmad
• Organization: Enanta Pharmaceuticals, Inc

aahmad@enanta.com

1-617-607-0800

Study Officials

• Enanta Pharmaceuticals, Inc

  Enanta Pharmaceuticals, Inc

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: TRIPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: May 4, 2020
• First Posted: May 7, 2020
• Study Start: January 27, 2020
• Primary Completion: October 4, 2021
• Study Completion: November 30, 2021
• Last Updated: May 19, 2023
• Results First Posted: May 19, 2023

Record last verified: 2023-04

Data Sharing

• IPD Sharing: Will not share

Locations

PR (1); CA (1); DE (2); US (68); GB (8); AR (1)