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Liver FibrosisPhase 2FALCON 1NCT03486899

A Study of Experimental Medication BMS-986036 in Adults With Nonalcoholic Steatohepatitis (NASH) and Stage 3 Liver Fibrosis

A Phase 2 study testing BMS-986036 in people with Liver Fibrosis and Nonalcoholic Fatty Liver Disease (NAFLD), run by Bristol-Myers Squibb at 89 sites in 2 countries.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
Search all trials

Trial summary

This is a study of experimental medication BMS-986036 given to adults with Nonalcoholic Steatohepatitis (NASH; the buildup of fat and inflammation in the liver that is not caused by alcohol) and stage 3 liver fibrosis (severe fibrosis). Read more about this study

Who is it for?
Ages 18 to 75 years
What is being tested?
BMS-986036
Type of treatment
Injection or infusion
Could I receive a placebo?
Yes, some participants may receive a placebo
How many people
197 participants
Where
89 sitesin 2 countries
Study dates
19 Jun 2018to 17 Aug 2021
Phase
Phase 2
Are there results?
Yes, posted in the registrySummary in the doctors' view

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 18 to 75 years
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • Liver biopsy performed within 6 months (26 weeks) prior to the screening period. If historical biopsy is not available, a liver biopsy will be performed during the screening period. Biopsy must be consistent with NASH, with: a) a score of at least 1 for each NAS component (steatosis, lobular inflammation, and ballooning), as assessed by the central reader, and b) stage 3 liver fibrosis according to the NASH CRN classification, as assessed by the central reader
  • Participants taking anti-diabetic, anti-obesity, or anti-dyslipidemic medications must have been on stable regimens for at least 3 months (12 weeks) (6 weeks for statins) prior to and during the screening period
  • Participants taking vitamin E at doses greater than or equal to (>=) 800 IU/day must have been on stable doses for at least 6 months (26 weeks) prior to and during the Screening Period. Vitamin E treatment (>=800 IU/day) must not have been initiated after the qualifying liver biopsy was performed.

Who cannot join

  • Other causes of liver disease (e.g., alcoholic liver disease, hepatitis B virus infection, chronic hepatitis C virus [HCV] infection, autoimmune hepatitis, drug-induced hepatotoxicity, Wilson disease, α-1-antitrypsin deficiency, iron overload, and hemochromatosis); participants with HCV sustained viral response (undetectable HCV RNA) for at least 2 years prior to biopsy confirming study eligibility may be eligible
  • Current or past history of hepatocellular carcinoma (HCC)
  • Past or current evidence of hepatic decompensation (e.g., ascites, variceal bleeding, hepatic encephalopathy and/or spontaneous bacterial peritonitis) or liver transplantation
  • Other protocol defined inclusion/exclusion criteria could apply

Trial locations and contacts

89 sites in 2 countries.

89 sites in 2 countries

United States83 sites

  • University of Michigan

    Ann Arbor, Michigan, 48109

  • Texas Clinical Research Institute

    Arlington, Texas, 76012

  • Local Institution - 0066

    Austin, Texas, 78757

  • Local Institution - 0007

    Baltimore, Maryland, 21202

  • Boston Medical Center

    Boston, Massachusetts, 02118

  • Bridgeport Hospital

    Bridgeport, Connecticut, 06610

  • University at Buffalo

    Buffalo, New York, 14203

  • University of Vermont Medical Center

    Burlington, Vermont, 05401

  • Local Institution - 0067

    Butner, North Carolina, 27509-1626

  • Local Institution - 0057

    Catonsville, Maryland, 21228

  • Local Institution - 0005

    Chandler, Arizona, 85224

  • Local Institution - 0064

    Charlotte, North Carolina, 28204

  • University of Virginia Health System

    Charlottesville, Virginia, 22908

  • Local Institution - 0063

    Chesterfield, Missouri, 63005

  • Local Institution - 0096

    Concord, North Carolina, 28027

  • Local Institution - 0079

    Coral Gables, Florida, 33134

  • Kindred Medical Institute for Clinical Trials

    Corona, California, 92879

  • Local Institution - 0092

    Coronado, California, 92118

  • Top Medical Research

    Cutler Bay, Florida, 33189

  • Local Institution - 0053

    Dallas, Texas, 75203

  • Local Institution - 0052

    Dallas, Texas, 75234

  • Texas Digestive Disease Consultants - Dallas

    Dallas, Texas, 75246

  • University of Texas Southwestern Medical Center

    Dallas, Texas, 75390-88520

  • NECCR PrimaCare Research

    Fall River, Massachusetts, 02721

  • Inova Fairfax Hospital

    Falls Church, Virginia, 22042

  • Texas Digestive Disease Consultants - Southlake

    Fort Worth, Texas, 76104

  • Local Institution - 0100

    Gainesville, Florida, 32610

  • Local Institution - 0047

    Germantown, Tennessee, 38138

  • Local Institution - 0041

    Hermitage, Tennessee, 37076

  • Clinical Research of Homestead

    Homestead, Florida, 33030

  • Local Institution - 0004

    Houston, Texas, 77002

  • Local Institution - 0059

    Houston, Texas, 77030

  • Local Institution - 0062

    Houston, Texas, 77030

  • Local Institution - 0082

    Jacksonville, Florida, 32224

  • Saint Lukes Hospital of Kansas City

    Kansas City, Missouri, 64111

  • University of California San Diego

    La Jolla, California, 92037

  • Local Institution - 0003

    Lakewood Rch, Florida, 34211

  • Local Institution - 0017

    Los Angeles, California, 90036

  • Cedars-Sinai Medical Center

    Los Angeles, California, 90048

  • GastroIntestinal Biosciences

    Los Angeles, California, 90067-2015

  • Local Institution - 0087

    Madison, Alabama, 35758

  • Gastroenterology Associates, PC

    Manassas, Virginia, 20110

  • Local Institution - 0078

    Manhasset, New York, 11030

  • Local Institution - 0105

    Marietta, Georgia, 30060

  • Tandem Clinical Research

    Marrero, Louisiana, 70072

  • Local Institution - 0002

    Miami, Florida, 33136

  • A+ Research

    Miami, Florida, 33144

  • IMIC Research

    Miami, Florida, 33157

  • Catalina Research Institute

    Montclair, California, 91763

  • Vanderbilt University Medical Center

    Nashville, Tennessee, 37232-5280

  • Tulane University Health Sciences Center

    New Orleans, Louisiana, 70112

  • Local Institution - 0027

    New Orleans, Louisiana, 70121

  • Local Institution - 0083

    New York, New York, 10003

  • Local Institution - 0038

    New York, New York, 10016

  • Icahn School of Medicine at Mount Sinai

    New York, New York, 10029

  • Local Institution - 0069

    Norfolk, Virginia, 23502

  • Local Institution - 0008

    Oakland, California, 94611

  • Sensible Healthcare

    Ocoee, Florida, 34761

  • Local Institution - 0081

    Orlando, Florida, 32806

  • Local Institution - 0044

    Oxnard, California, 93030

  • Huntington Medical Research Institutes - HMRI Liver Center

    Pasadena, California, 91105

  • Local Institution - 0019

    Pasadena, California, 91105

  • University of Pennsylvania

    Philadelphia, Pennsylvania, 19104

  • Local Institution - 0009

    Philadelphia, Pennsylvania, 19107

  • Local Institution - 0088

    Phoenix, Arizona, 85013

  • Local Institution - 0001

    Phoenix, Arizona, 85054

  • Local Institution - 0006

    Pittsburgh, Pennsylvania, 15213

  • Local Institution - 0074

    Redwood City, California, 94063

  • The Gastroenterology Group

    Reston, Virginia, 20191

  • Local Institution - 0013

    Rialto, California, 92377

  • Bon Secours Liver Institute of Richmond

    Richmond, Virginia, 23226

  • Local Institution - 0077

    Richmond, Virginia, 23249

  • Local Institution - 0049

    Richmond, Virginia, 23298

  • Local Institution - 0029

    San Antonio, Texas, 78215

  • Local Institution - 0012

    San Antonio, Texas, 78229

  • Local Institution - 0101

    San Antonio, Texas, 78229

  • Local Institution - 0089

    San Clemente, California, 92673

  • Medical Associates Research Group

    San Diego, California, 92123

  • Local Institution - 0068

    San Francisco, California, 94115

  • Saint Louis University

    St Louis, Missouri, 63110

  • Tampa General Hospital

    Tampa, Florida, 33606

  • The Institute for Liver Health - Tucson

    Tucson, Arizona, 85712

  • MedStar Georgetown University Hospital

    Washington D.C., District of Columbia, 20007

Other trials in United States

Japan6 sites

  • Fukushima Medical University Hospital

    Fukushima, 960-1295

  • Local Institution - 0072

    Kashihara, Nara, 6348522

  • Kurume University Hospital

    Kurume, Fukuoka, 8300011

  • Toranomon Hospital

    Minato, Tokyo, 105-8470

  • Keio University Hospital

    Shinjuku-ku, Tokyo, 1600016

  • Local Institution - 0056

    Yokohama, Kanagawa, 236-0004

Other trials in Japan

About this study

Official title: A Phase 2B Randomized Double-Blind, Placebo-Controlled Study Evaluating the Safety and Efficacy of BMS-986036 (PEG-FGF21) in Adults With Nonalcoholic Steatohepatitis (NASH) and Stage 3 Liver Fibrosis

This is a study of experimental medication BMS-986036 given to adults with Nonalcoholic Steatohepatitis (NASH; the buildup of fat and inflammation in the liver that is not caused by alcohol) and stage 3 liver fibrosis (severe fibrosis).

Being tested
BMS-986036, Placebo
Medical subject headings
Liver Cirrhosis, Non-alcoholic Fatty Liver Disease

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • BMS-986036Drug

    Also called Pegbelfermin

    Specified dose on specified days.

  • PlaceboOther

    Specified dose on specified days.

Study groups in NCT03486899
Study groupRoleWhat they receive
BMS-986036 Dose Level 1ExperimentalAdministered by subcutaneous injection.
Receives: Drug: BMS-986036
BMS-986036 Dose Level 2ExperimentalAdministered by subcutaneous injection.
Receives: Drug: BMS-986036
BMS-986036 Dose Level 3ExperimentalAdministered by subcutaneous injection.
Receives: Drug: BMS-986036
PlaceboPlacebo comparatorAdministered by subcutaneous injection.
Receives: Other: Placebo

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. The Percentage of Participants With Improvement in Fibrosis or Nonalcoholic Steatohepatitis (NASH) at Week 24

    The percentage of participants who achieved a ≥1-stage improvement in fibrosis without worsening of NASH or NASH improvement with no worsening of fibrosis at week 24 in liver biopsy. Improvement in fibrosis is defined by the NASH Clinical Research Network (CRN) Fibrosis Score. Improvement in NASH is defined by a ≥2-stage decrease in the nonalcoholic fatty liver disease activity score (NAS). Worsening of NASH is defined as an increase of the nonalcoholic fatty liver disease (NAFLD) Activity Score (NAS) by ≥1 point. Worsening of fibrosis is defined as an increase of fibrosis by ≥1 point as determined by the NASH CRN Fibrosis Score. NASH CRN Fibrosis is staged on a 0-4 scale: 0 (none); 1 (perisinusoidal or periportal fibrosis); 2 (perisinusoidal and portal/periportal fibrosis); 3 (bridging fibrosis); 4 (cirrhosis).

    Measured: From first dose to 24 weeks after first dose

Secondary outcomes (9)

  1. The Percentage of Participants Who Achieved an Improvement in Nonalcoholic Steatohepatitis Clinical Research Network (NASH CRN) Fibrosis Score at Week 24

    An improvement in fibrosis is defined as a decrease of ≥ 1-stage in the NASH CRN Fibrosis Score at week 24 in liver biopsy. NASH CRN Fibrosis is staged on a 0-4 scale: 0 (none); 1 (perisinusoidal or periportal fibrosis); 2 (perisinusoidal and portal/periportal fibrosis); 3 (bridging fibrosis); 4 (cirrhosis).

    Measured: From first dose to 24 weeks after first dose

  2. The Percentage of Participants Who Achieve a ≥ 1-Stage Improvement in Ishak Fibrosis Score at Week 24

    An improvement in Ishak fibrosis is defined as a decrease of fibrosis by ≥ 1-stage in the Ishak fibrosis score at week 24 in liver biopsy. ISHAKs uses a 0-6 scale: 1: centrilobular pericellular fibrosis, 2: centrilobular and periportal fibrosis, 3: bridging fibrosis (few bridges), 4: bridging fibrosis (many bridges), 5: early or incomplete cirrhosis, 6: established or advanced cirrhosis.

    Measured: From first dose to 24 weeks after first dose

  3. The Percentage of Participants With Any Improvement in Collagen Proportionate Area (CPA) at Week 24

    An improvement in CPA is defined as any decrease in CPA at week 24 in liver biopsy.

    Measured: From first dose to 24 weeks after first dose

  4. The Percentage of Participants With Nonalcoholic Steatohepatitis (NASH) Resolution Without Worsening of Fibrosis at Week 24

    The percentage of participants with NASH resolution without worsening of fibrosis at week 24 in liver biopsy. NASH resolution defined by the nonalcoholic fatty liver disease activity score (NAS) component of ballooning = 0 and inflammation = 0-1. Worsening of fibrosis is defined as an increase of fibrosis by ≥ 1-stage in the NASH Clinical Research Network (CRN) Fibrosis Score.

    Ballooning = 0 (none) inflammation = 0 (none) - 1 (Grade <2). NASH CRN Fibrosis is staged on a 0-4 scale: 0 (none); 1 (perisinusoidal or periportal fibrosis); 2 (perisinusoidal and portal/periportal fibrosis); 3 (bridging fibrosis); 4 (cirrhosis).

    Measured: From first dose to 24 weeks after first dose

  5. The Percentage of Participants With Nonalcoholic Steatohepatitis (NASH) Resolution at Week 24

    NASH resolution defined by the nonalcoholic fatty liver disease activity score (NAS) component of ballooning = 0 and inflammation = 0-1 at week 24 in liver biopsy. Ballooning = 0 (none) inflammation = 0 (none) - 1 (Grade <2).

    Measured: From first dose to 24 weeks after first dose

  6. The Percentage of Participants With Nonalcoholic Steatohepatitis (NASH) Improvement Without Worsening of Fibrosis at Week 24

    The percentage of participants with NASH improvement without worsening of fibrosis at week 24 in liver biopsy. NASH improvement defined as a reduction of nonalcoholic fatty liver disease activity score (NAS) by ≥2 points with contribution from >1 NAS component. Worsening of fibrosis is defined as an increase of ≥1-point in the NASH Clinical Research Network (CRN) Fibrosis Score. NASH CRN Fibrosis is staged on a 0-4 scale: 0 (none); 1 (perisinusoidal or periportal fibrosis); 2 (perisinusoidal and portal/periportal fibrosis); 3 (bridging fibrosis); 4 (cirrhosis).

    Measured: From first dose to 24 weeks after first dose

  7. The Percentage of Participants Who Achieve a ≥ 1-Stage Improvement in Fibrosis Without Worsening of Nonalcoholic Steatohepatitis (NASH) at Week 24

    An improvement in fibrosis is defined as a decrease of fibrosis by ≥1-stage in the NASH Clinical Research Network (CRN) Fibrosis Score at week 24 in liver biopsy. Worsening of NASH is defined as an increase of the nonalcoholic fatty liver disease activity score (NAS) by ≥ 1-stage. NASH CRN Fibrosis is staged on a 0-4 scale: 0 (none); 1 (perisinusoidal or periportal fibrosis); 2 (perisinusoidal and portal/periportal fibrosis); 3 (bridging fibrosis); 4 (cirrhosis).

    Measured: From first dose to 24 weeks after first dose

  8. The Percentage of Participants With Nonalcoholic Steatohepatitis (NASH) Improvement at Week 24

    The percentage of participants with NASH improvement at week 24 in liver biopsy. NASH improvement is defined as a reduction of nonalcoholic fatty liver disease activity score (NAS) by ≥ 2 points with contribution from > 1 NAS component. The NASH CRN system assesses liver biopsies for degree of steatosis (0-3), lobular inflammation (0-3), hepatocellular ballooning (0-2), and fibrosis (0-4). The 3 categories are added together in an unweighted fashion to determine the NAS, which ranges from 0 to 8.

    Measured: From first dose to 24 weeks after first dose

  9. The Percentage of Participants With Progression to Cirrhosis at Week 24

    Progression to cirrhosis is defined by the nonalcoholic steatohepatitis clinical research network (NASH CRN) Fibrosis Stage 4 at Week 24 in liver biopsy. NASH CRN Fibrosis is staged on a 0-4 scale: 0 (none); 1 (perisinusoidal or periportal fibrosis); 2 (perisinusoidal and portal/periportal fibrosis); 3 (bridging fibrosis); 4 (cirrhosis).

    Measured: From first dose to 24 weeks after first dose

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted3 Apr 2018
  2. Study start19 Jun 2018
  3. Primary completion14 Sep 2020
  4. Study completion17 Aug 2021

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 2
Primary purpose
Treatment
Allocation
Randomised
Intervention model
Parallel groups
Masking
Quadruple blind
Who is masked
Participant, Care provider, Investigator, Outcomes assessor
Enrolment
197 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT03486899
Sponsor's protocol ID
MB130-068
Acronym
FALCON 1
Status last verified
Aug 2022
First submitted
30 Mar 2018
Last updated
9 Sep 2022
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No

Publications

  • Loomba R, Sanyal AJ, Nakajima A, Neuschwander-Tetri BA, Goodman ZD, Harrison SA, Lawitz EJ, Gunn N, Imajo K, Ravendhran N, Akahane T, Boone B, Yamaguchi M, Chatterjee A, Tirucherai GS, Shevell DE, Du S, Charles ED, Abdelmalek MF. Pegbelfermin in Patients With Nonalcoholic Steatohepatitis and Stage 3 Fibrosis (FALCON 1): A Randomized Phase 2b Study. Clin Gastroenterol Hepatol. 2024 Jan;22(1):102-112.e9. doi: 10.1016/j.cgh.2023.04.011. Epub 2023 Apr 23.

    PubMed 37088457

  • Brown EA, Minnich A, Sanyal AJ, Loomba R, Du S, Schwarz J, Ehman RL, Karsdal M, Leeming DJ, Cizza G, Charles ED. Effect of pegbelfermin on NASH and fibrosis-related biomarkers and correlation with histological response in the FALCON 1 trial. JHEP Rep. 2023 Jan 7;5(4):100661. doi: 10.1016/j.jhepr.2022.100661. eCollection 2023 Apr.

    PubMed 36866389

  • Abdelmalek MF, Charles ED, Sanyal AJ, Harrison SA, Neuschwander-Tetri BA, Goodman Z, Ehman RA, Karsdal M, Nakajima A, Du S, Tirucherai GS, Klinger GH, Mora J, Yamaguchi M, Shevell DE, Loomba R. The FALCON program: Two phase 2b randomized, double-blind, placebo-controlled studies to assess the efficacy and safety of pegbelfermin in the treatment of patients with nonalcoholic steatohepatitis and bridging fibrosis or compensated cirrhosis. Contemp Clin Trials. 2021 May;104:106335. doi: 10.1016/j.cct.2021.106335. Epub 2021 Feb 28.

    PubMed 33657443

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing BMS-986036 in people with Liver Fibrosis and Nonalcoholic Fatty Liver Disease (NAFLD), run by Bristol-Myers Squibb at 89 sites in 2 countries.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 3 inclusion and 4 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 9 Sep 2022. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 89 sites in United States, Japan. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Yes. Results are posted in the registry (first posted 9 Sep 2022). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

The Percentage of Participants With Improvement in Fibrosis or Nonalcoholic Steatohepatitis (NASH) at Week 24 (time frame: From first dose to 24 weeks after first dose). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT03486899, which also lists 3 linked publications.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 9 Sep 2022.

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