The Farnesoid X Receptor (FXR) Ligand Obeticholic Acid in NASH Treatment Trial(FLINT)

NCT ID: NCT01265498

Last Updated: 2018-04-06

Study Results

Results available

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Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE2

Total Enrollment

283 participants

Study Classification

INTERVENTIONAL

Study Start Date

2011-03-31

Study Completion Date

2014-09-30

Brief Summary

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Administration of the farnesoid X receptor (FXR) ligand obeticholic acid (OCA) for 72 weeks to subjects with biopsy evidence of nonalcoholic steatohepatitis (NASH) will result in improvement in their liver disease as measured by changes in the nonalcoholic fatty liver disease (NAFLD) activity score (NAS).

Detailed Description

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To evaluate whether treatment with obeticholic acid, 25 mg daily for 72 weeks compared to treatment with placebo, improves the severity of nonalcoholic fatty liver disease (NAFLD) as determined from hepatic histology.

Conditions

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Nonalcoholic Fatty Liver Disease (NAFLD) Nonalcoholic Steatohepatitis (NASH)

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

TRIPLE

Participants Caregivers Investigators

Study Groups

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Obeticholic acid

obeticholic acid

Group Type ACTIVE_COMPARATOR

obeticholic acid

Intervention Type DRUG

25 mg daily for 72 weeks

Placebo

Placebo

Group Type PLACEBO_COMPARATOR

placebo

Intervention Type DRUG

placebo capsule, 25 mg daily for 72 weeks

Interventions

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obeticholic acid

25 mg daily for 72 weeks

Intervention Type DRUG

placebo

placebo capsule, 25 mg daily for 72 weeks

Intervention Type DRUG

Other Intervention Names

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farnesoid X receptor (FXR) ligand obeticholic acid (OCA) Placebo for obeticholic acid

Eligibility Criteria

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Inclusion Criteria

* 18 years of age or older as of the initial screening interview and provision of consent
* Histologic evidence of definite or probable nonalcoholic steatohepatitis (NASH) based upon a liver biopsy obtained no more than 90 days prior to randomization and a nonalcoholic fatty liver disease activity score (NAS) of 4 or greater with at least 1 in each component of the NAS score (steatosis scored 0-3, ballooning degeneration scored 0-2, and lobular inflammation scored 0-3).

Exclusion Criteria

* Current or history of significant alcohol consumption for a period of more than 3 consecutive months within 1 year prior to screening (significant alcohol consumption is defined as more than 20 grams per day in females and more than 30 grams per day in males, on average)
* Inability to reliably quantify alcohol consumption based upon local study physician judgment
* Use of drugs historically associated with nonalcoholic fatty liver disease (NAFLD) (amiodarone, methotrexate, systemic glucocorticoids, tetracyclines, tamoxifen, estrogens at doses greater than those used for hormone replacement, anabolic steroids, valproic acid, and other known hepatotoxins) for more than 2 weeks in the year prior to randomization
* Prior or planned (during the study period) bariatric surgery (eg, gastroplasty, roux-en-Y gastric bypass)
* Uncontrolled diabetes defined as Hemoglobin A1c 9.5% or higher within 60 days prior to enrollment
* Presence of cirrhosis on liver biopsy
* A platelet count below 100,000/mm3
* Clinical evidence of hepatic decompensation as defined by the presence of any of the following abnormalities:

* Serum albumin less than 3.2 grams/deciliter (g/dL)
* International Normalized Ratio(INR)greater than 1.3
* Direct bilirubin greater than 1.3 milligrams per deciliter (mg/dL)
* History of esophageal varices, ascites or hepatic encephalopathy
* Evidence of other forms of chronic liver disease:

* Hepatitis B as defined by presence of hepatitis B surface antigen (HBsAg)
* Hepatitis C as defined by presence of hepatitis C virus (HCV) ribonucleic acid (RNA) or positive hepatitis C antibody (anti-HCV)
* Evidence of ongoing autoimmune liver disease as defined by compatible liver histology
* Primary biliary cirrhosis as defined by the presence of at least 2 of these criteria (i) Biochemical evidence of cholestasis based mainly on alkaline phosphatase elevation (ii)Presence of anti-mitochondrial antibody (AMA) (iii)Histologic evidence of nonsuppurative destructive cholangitis and destruction of interlobular bile ducts
* Primary sclerosing cholangitis
* Wilson's disease as defined by ceruloplasmin below the limits of normal and compatible liver histology
* Alpha-1-antitrypsin(A1AT) deficiency as defined by diagnostic features in liver histology (confirmed by alpha-1 antitrypsin level less than normal; exclusion at the discretion of the study physician)
* History of hemochromatosis or iron overload as defined by presence of 3+ or 4+ stainable iron on liver biopsy
* Drug-induced liver disease as defined on the basis of typical exposure and history
* Known bile duct obstruction
* Suspected or proven liver cancer
* Any other type of liver disease other than nonalcoholic steatohepatitis (NASH)
* Serum alanine aminotransferase (ALT) greater than 300 units per liter (U/L)
* Serum creatinine of 2.0 mg/dL or greater
* Use of ursodeoxycholic acid (Ursodiol, Urso) within 90 days prior to enrollment
* Inability to safely obtain a liver biopsy
* History of biliary diversion
* Known positivity for Human Immunodeficiency Virus (HIV) infection
* Active, serious medical disease with likely life expectancy less than 5 years
* Active substance abuse including inhaled or injection drugs in the year prior to screening
* Pregnancy, planned pregnancy, potential for pregnancy and unwillingness to use effective birth control during the trial, breast feeding
* Participation in an investigational new drug (IND) trial in the 30 days before randomization
* Any other condition which, in the opinion of the investigator, would impede compliance or hinder completion of the study
* Failure to give informed consent
Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)

NIH

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Edward Doo, MD

Role: STUDY_DIRECTOR

National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)

Locations

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University of California, San Diego

San Diego, California, United States

Site Status

University of California, San Francisco

San Francisco, California, United States

Site Status

Indiana University

Indianapolis, Indiana, United States

Site Status

St. Louis University

St Louis, Missouri, United States

Site Status

Duke University Medical Center

Durham, North Carolina, United States

Site Status

Case Western Reserve University

Cleveland, Ohio, United States

Site Status

Cleveland Clinic Foundation

Cleveland, Ohio, United States

Site Status

Virginia Commonwealth University

Richmond, Virginia, United States

Site Status

Virginia Mason Medical Center

Seattle, Washington, United States

Site Status

Countries

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United States

References

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Shen W, Middleton MS, Cunha GM, Delgado TI, Wolfson T, Gamst A, Fowler KJ, Alazraki A, Trout AT, Ohliger MA, Shah SN, Bashir MR, Kleiner DE, Loomba R, Neuschwander-Tetri BA, Sanyal AJ, Zhou J, Sirlin CB, Lavine JE. Changes in abdominal adipose tissue depots assessed by MRI correlate with hepatic histologic improvement in non-alcoholic steatohepatitis. J Hepatol. 2023 Feb;78(2):238-246. doi: 10.1016/j.jhep.2022.10.027. Epub 2022 Nov 8.

Reference Type DERIVED
PMID: 36368598 (View on PubMed)

Vilar-Gomez E, Gawrieh S, Liang T, McIntyre AD, Hegele RA, Chalasani N. Interrogation of selected genes influencing serum LDL-Cholesterol levels in patients with well characterized NAFLD. J Clin Lipidol. 2021 Mar-Apr;15(2):275-291. doi: 10.1016/j.jacl.2020.12.010. Epub 2020 Dec 27.

Reference Type DERIVED
PMID: 33454241 (View on PubMed)

Loomba R, Neuschwander-Tetri BA, Sanyal A, Chalasani N, Diehl AM, Terrault N, Kowdley K, Dasarathy S, Kleiner D, Behling C, Lavine J, Van Natta M, Middleton M, Tonascia J, Sirlin C; NASH Clinical Research Network. Multicenter Validation of Association Between Decline in MRI-PDFF and Histologic Response in NASH. Hepatology. 2020 Oct;72(4):1219-1229. doi: 10.1002/hep.31121. Epub 2020 Oct 9.

Reference Type DERIVED
PMID: 31965579 (View on PubMed)

Siddiqui MS, Van Natta ML, Connelly MA, Vuppalanchi R, Neuschwander-Tetri BA, Tonascia J, Guy C, Loomba R, Dasarathy S, Wattacheril J, Chalasani N, Sanyal AJ; NASH CRN. Impact of obeticholic acid on the lipoprotein profile in patients with non-alcoholic steatohepatitis. J Hepatol. 2020 Jan;72(1):25-33. doi: 10.1016/j.jhep.2019.10.006. Epub 2019 Oct 18.

Reference Type DERIVED
PMID: 31634532 (View on PubMed)

Chalasani N, Abdelmalek MF, Loomba R, Kowdley KV, McCullough AJ, Dasarathy S, Neuschwander-Tetri BA, Terrault N, Ferguson B, Shringarpure R, Shapiro D, Sanyal AJ. Relationship between three commonly used non-invasive fibrosis biomarkers and improvement in fibrosis stage in patients with non-alcoholic steatohepatitis. Liver Int. 2019 May;39(5):924-932. doi: 10.1111/liv.13974. Epub 2019 Feb 21.

Reference Type DERIVED
PMID: 30253043 (View on PubMed)

Hameed B, Terrault NA, Gill RM, Loomba R, Chalasani N, Hoofnagle JH, Van Natta ML; NASH CRN. Clinical and metabolic effects associated with weight changes and obeticholic acid in non-alcoholic steatohepatitis. Aliment Pharmacol Ther. 2018 Mar;47(5):645-656. doi: 10.1111/apt.14492. Epub 2018 Jan 14.

Reference Type DERIVED
PMID: 29333665 (View on PubMed)

Neuschwander-Tetri BA, Loomba R, Sanyal AJ, Lavine JE, Van Natta ML, Abdelmalek MF, Chalasani N, Dasarathy S, Diehl AM, Hameed B, Kowdley KV, McCullough A, Terrault N, Clark JM, Tonascia J, Brunt EM, Kleiner DE, Doo E; NASH Clinical Research Network. Farnesoid X nuclear receptor ligand obeticholic acid for non-cirrhotic, non-alcoholic steatohepatitis (FLINT): a multicentre, randomised, placebo-controlled trial. Lancet. 2015 Mar 14;385(9972):956-65. doi: 10.1016/S0140-6736(14)61933-4. Epub 2014 Nov 7.

Reference Type DERIVED
PMID: 25468160 (View on PubMed)

Related Links

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https://jhuccs1.us/nash/default.asp

Nonalcoholic Steatohepatitis Clinical Research Network

http://www2.niddk.nih.gov/

The National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)

Other Identifiers

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U01DK061730

Identifier Type: NIH

Identifier Source: secondary_id

View Link

NASH-FLINT (IND)

Identifier Type: -

Identifier Source: org_study_id

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