FULL DETAILS (Read-only)  -> Click Here to Create PDF for Current Dataset of Trial
CTRI Number  CTRI/2024/11/076383 [Registered on: 08/11/2024] Trial Registered Prospectively
Last Modified On: 29/08/2025
Post Graduate Thesis  No 
Type of Trial  Interventional 
Type of Study   Drug 
Study Design  Randomized, Parallel Group, Placebo Controlled Trial 
Public Title of Study   This Study will be conducted to Evaluate the Safety and Efficacy of Investigational medicine (Efruxifermin) in patients with Compensated Liver Cirrhosis Due to Nonalcoholic Steatohepatitis (NASH)/Metabolic Dysfunction-Associated Steatohepatitis (MASH).  
Scientific Title of Study   A Phase 3, Randomized, Double-Blind, Placebo-Controlled Study Evaluating the Safety and Efficacy of Efruxifermin in Subjects with Compensated Cirrhosis Due to Nonalcoholic Steatohepatitis (NASH)/Metabolic Dysfunction-Associated Steatohepatitis (MASH).  
Trial Acronym   
Secondary IDs if Any  
Secondary ID  Identifier 
AK-US-001-0106, Original Version, Protocol Dated 19 Mar 2024  DCGI 
 
Details of Principal Investigator or overall Trial Coordinator (multi-center study)  
Name  Dr Sawan Bopanna 
Designation  Medical Monitor 
Affiliation  KlinEra Global Services 
Address  Clinical Operations,801, Neelkanth Corporate Park, Near Vidyavihar Station, Vidyavihar West, Mumbai 400 086.

Mumbai (Suburban)
MAHARASHTRA
400086
India 
Phone  912249781252  
Fax    
Email  medicalmonitor@klinera.com  
 
Details of Contact Person
Scientific Query
 
Name  Dr Sawan Bopanna 
Designation  Medical Monitor 
Affiliation  KlinEra Global Services 
Address  Clinical Operations,801, Neelkanth Corporate Park, Near Vidyavihar Station, Vidyavihar West, Mumbai 400 086.


MAHARASHTRA
400086
India 
Phone  912249781252  
Fax    
Email  medicalmonitor@klinera.com  
 
Details of Contact Person
Public Query
 
Name  Rajeev Singh 
Designation  Senior Project Manager-Clinical Operations 
Affiliation  KlinEra Global Services 
Address  Clinical Operations, 801, Neelkanth Corporate Park, Near Vidyavihar Station, Vidyavihar West, Mumbai 400 086.

Mumbai (Suburban)
MAHARASHTRA
400 086
India 
Phone  912249781252  
Fax    
Email  rsingh@klinera.com  
 
Source of Monetary or Material Support  
Akero Therapeutics, Inc. 601 Gateway Blvd., Suite 350, South San Francisco, CA 94080 
 
Primary Sponsor  
Name  Akero Therapeutics, Inc.  
Address  601 Gateway Blvd., Suite 350, South San Francisco, CA 94080, USA 
Type of Sponsor  Pharmaceutical industry-Global 
 
Details of Secondary Sponsor  
Name  Address 
Klinera Global Services  801, Neelkanth Corporate Park, Near Vidyavihar Station, Vidyavihar West, Mumbai 400 086, India. 
 
Countries of Recruitment     Argentina
Australia
Brazil
Canada
Chile
France
India
Israel
Italy
Mexico
Poland
Spain
Switzerland
Turkey
United Kingdom
United States of America
Germany  
Sites of Study
Modification(s)  
No of Sites = 25  
Name of Principal Investigator  Name of Site  Site Address  Phone/Fax/Email 
Dr Mithun Sharma  AIG Hospitals   AIG Hospitals, No 136 Plot No 2-3-4-5, Survey Mindspace Road, Gachibowli, Hyderabad-500032, Telangana, India
Hyderabad
TELANGANA 
8790622655

drmithunsharma@gmail.com 
Dr Rohit Gupta  All India Institute of Medical Science  Department of Gastroenterology, Rishikesh - 249203, Uttarakhand, India
Dehradun
UTTARANCHAL 
7579067715

docgupta1976@gmail.com 
Dr Shalimar  All India Institution of Medical Sciences (AIIMS)  Room no. 127, First floor, Old OT Block, AIIMS Ansari nagar, Delhi - 110029
New Delhi
DELHI 
9868397211

dr.shalimar@gmail.com 
Dr Parshottam Koradia  BAPS Pramukh Swami Hospital  Shri Pramukh Swami Maharaj Marg, Adajan, Surat - 395009 Gujarat, India
Surat
GUJARAT 
9825312027

purushottam_koradia@yahoo.co.in 
Dr Nitin Behl  Deep Hospital  481, Model Town, Ludhiana-141002, Punjab, India
Ludhiana
PUNJAB 
8427000080

drnitinbehl@gmail.com 
Dr Chandan Kumar  Gleneagles Hospital  6-1-1070/1 to 4, Lakdi-ka-pul, Hyderabad - 500 004, Telangana, India
Hyderabad
TELANGANA 
9986003257

drchandan@gmail.com 
Dr Krishnadas Devadas  Government Medical college  Department of Medical Gastroenterology, Superspeciality block, Thiruvanathpuram-695011, Kerala, India
Thiruvananthapuram
KERALA 
9847111824

kdas40@gmail.com 
Dr Abhijit Chowdhury  Indian Institute of Liver and Digestive Sciences(IILDS)  JCMLRI,Indian Institute of Liver and Digestive Sciences campus, Sitala east, Sonarpur, Kolkata - 700150, West Bengal, India
Kolkata
WEST BENGAL 
9433045435

achowdhury2002@yahoo.co.in 
Dr Shiv Kumar Sarin  Institute of Liver and Biliary Sciences  D-1, Vasant Kunj, New Delhi, Delhi 110070, Delhi, India
New Delhi
DELHI 
011-46300000

shivsarin@gmail.com 
Dr Dawesh Prakash Yadav  Institute of Medical Sciences  Department of gastroenterology, Banaras Hindu University, Varanasi-221005, Uttar Pradesh, India
Varanasi
UTTAR PRADESH 
8130856563

devesh.thedoc@gmail.com 
Dr Kausik Das  IPGME&R and SSKM Hospital  244, A.J.C Bose Road, Kolkata - 700020, West Bengal, India
Kolkata
WEST BENGAL 
9433162770

kausikdasmail@gmail.com 
Dr Dharmendra BL  K R Hospital Princess Krishnajammanni Super Speciality Hospital  K R Hospital Princess Krishnajammanni Super Speciality Hospital, 1st Cross Rd, Brindavan Extension 1st stage, Opp to ESI Hospital, Mysore, Karnataka- 570001
Mysore
KARNATAKA 
9844400382

Drdharmu21@gmail.com 
Dr Saubhik Ghosh  Medical College and Hospital  88 College Street, Kolkata - 700073, West Bengal, India
Kolkata
WEST BENGAL 
8017585988

souvikpgi@gmail.com 
Dr Shrikant Mukewar  Midas Hospital  392, Behind Empress Palace, Opp Singh Saab Dhaba, Wardha Road, Parsodi, Nagpur – 441108, Maharashtra, India.
Nagpur
MAHARASHTRA 
7720033280

shrikant_mukewar@yahoo.com 
Dr Dharmendra BL  Mysore Medical College and Research Institute  Princess Krishnajammanni Super Speciality Hospital, 1st Cross Rd, Brindavan Extension 1st stage, Opp to ESI Hospital, Mysore, Karnataka- 570015
Mysore
KARNATAKA 
9844400382

Drdharmu21@gmail.com 
Dr Pravin Rathi  Nair Hospital  OPD Building, 7th floor, Room No.714/715, Dr. A L Nair Road, Mumbai Central 400008, Maharashtra
Mumbai
MAHARASHTRA 
9322406438

rathipmpp@gmail.com 
Dr Ajay Kumar Duseja  Post Graduate Institute of Medical Education & Research  Nehru Hospital Extension Block (NHEB), Ground floor, Room no-36, Sector 12, PGIMER, Chandigarh - 160012, India
Chandigarh
CHANDIGARH 
172-2754791

ajayduseja@yahoo.co.in 
Dr Hemant Kumar Gupta  Samvedna Hospital  B-27/88-G, New Colony, Ravindrapuri, Varanasi - 221005, Uttar Pradesh, India
Varanasi
UTTAR PRADESH 
8573888800

hemantkrg26@gmail.com 
Dr Arun Vaidya  Seth G.S. Medical College and KEM Hospital  Department of Gastroenterology, Ward 32A,9th Floor, M.S. Building, Mumbai, Maharashtra-400012
Mumbai
MAHARASHTRA 
9699044016

arunvaidyagastro@gmail.com 
Dr Mayank Kabrawala  SIDS Hospital & Research centre  A unit of SIDS health care private limited, Off ring road, Near shell petrol pump, Ring road-sosyo circle lane, Surat-395002, Gujarat, India
Surat
GUJARAT 
9825130363

mayankkabrawala@hotmail.com 
Dr Shivam Khare  Sir Ganga Ram Hospital  Institute of Liver, Gastroenterology & Pancreatico-Biliary Sciences Rajinder Nagar, New Delhi - 110060, India
New Delhi
DELHI 
9754478909

drshivamkhare01@gmail.com 
Dr Sudhir Maharshi  SMS Super Speciality Hospital  Department of Gastroenterology Vivekanand Marg, C-Scheme, Jaipur – 302004, Rajasthan, India
Jaipur
RAJASTHAN 
8130369247

sudhir.maharshi@gmail.com 
Dr Mukesh Kalla  SR Kalla Memorial Gastro and General Hospital  78-79, Dhuleshwar Garden, behind HSBC bank, Sardar Patel Marg, C-Scheme, Jaipur - 302001, Rajasthan, India
Jaipur
RAJASTHAN 
9829050622

drmkalla@rediffmail.com 
Dr V G Mohan Prasad  VGM Hospital  VGM Hospital, 2100, Trichy Rd, Busstop, Singanallur, Coimbatore, Tamil Nadu 641005
Coimbatore
TAMIL NADU 
9842204995

drvgm@hotmail.com 
Dr Kiran Shinde  Vishwaraj Hospital  Near Loni Railway station Loni Kalbhor, Solapur - Pune Hwy, Pune - 412201, Maharashtra, India
Pune
MAHARASHTRA 
9986003257

drkiranshinde@gmail.com 
 
Details of Ethics Committee
Modification(s)  
No of Ethics Committees= 25  
Name of Committee  Approval Status 
Institutional Ethics Committee Institute of Liver and Biliary Sciences  Submittted/Under Review 
BAPS Hospital Institutional Ethics Committee BAPS Pramukh Swami Hospital  Approved 
Ethics Committee relating to Clinical Trial, All India Institute Of Medical Sciences, Rishikesh  Approved 
Ethics Committee Sir Ganga Ram Hospital,  Approved 
Ethics Committee, SMS Medical College and attached Hospitals  Approved 
Human Ethics Committee Government Medical College  Approved 
Human Research Ethics Committee, Indian Institute of Liver and Digestive Science   Approved 
IEC-MMC and RI and Associated Hospital  Approved 
Institutional Ethics Committee Asian Institute of Gastroenterology  Approved 
Institutional Ethics Committee Deep Hospital  Approved 
Institutional Ethics Committee for Human Research Medical College And Hospital  Submittted/Under Review 
Institutional ethics Committee Gleneagles Hospital  Approved 
Institutional Ethics Committee TNMC NAIR HOSPITAL  Approved 
Institutional Ethics Committee(IEC), Seth G.S Medical College and KEM Hospital  Approved 
Institutional Ethics Committee, Post Graduate Institute of Medical Education and Research  Submittted/Under Review 
Institutional Ethics Committee, All India Institute of Medical Sciences  Submittted/Under Review 
Institutional Ethics Committee, Institute of Medical Sciences, Banaras Hindu University  Approved 
Institutional Ethics Committee, MAEER’s Vishwaraj Hospital  Approved 
Institutional Ethics Committee, Midas Multispeciality Hospital Pvt. Ltd  Submittted/Under Review 
Institutional Ethics Committee, Mysore Medical College, Mysore  Approved 
Institutional Ethics Committee, VGM Hospital VGM Hospital Institute Of Gastroenterology   Approved 
IPGME&R Research Oversight Committee  Approved 
Samvedna Hospital Ethics Committee  Approved 
SR Kalla Memorial Ethical Committee for Human Research, SR Kalla Memorial Gastro and General Hospital  Approved 
Surat Institute of Digestive Sciences Ethics Committee, SIDS Hospital & Research centre  Submittted/Under Review 
 
Regulatory Clearance Status from DCGI  
Status 
Approved/Obtained 
 
Health Condition / Problems Studied  
Health Type  Condition 
Patients  (1) ICD-10 Condition: K758||Other specified inflammatory liverdiseases,  
 
Intervention / Comparator Agent  
Type  Name  Details 
Intervention  Efruxifermin (EFX) 50 mg   Efruxifermin is a fusion protein, comprised of human IgG1Fc linked to modified, human Fibroblast Growth Factor 21 (FGF21). EFX will be provided as a lyophilized powder in one chamber of a dual chamber syringe (LyoJect 3S DCS, Vetter) for once weekly subcutaneous (SC) administration immediately after reconstitution with diluent contained in the other chamber of the device. dose of study drug Efruxifermin (50mg) will be administered at the Baseline/Day 1 to week 260 visit as a SC injection into the abdomen. 
Comparator Agent  Placebo to match Efruxifermin(EFX)  Placebo to match Efruxifermin 50mg is composed of lyophilized Placebo formulation which closely matches the visual appearance and solution properties of the active drug product. Placebo to match Efruxifermin will be administered subcutaneously once a week, dose will be administered at the Baseline/Day 1 to week 260 visit as a SC injection into the abdomen 
 
Inclusion Criteria  
Age From  18.00 Year(s)
Age To  80.00 Year(s)
Gender  Both 
Details  1.Males and non-pregnant, non-lactating females between 18–80 years of age, inclusive, on the day of signing informed consent.2.Previous history or presence of type 2 diabetes (T2D) (as determined by medical history or based on screening lab values if previously undiagnosed [i.e., HbA1c greater than or equal to 6.5%]) or 2 out of 4 components of metabolic syndrome (obesity, dyslipidemia, elevated blood pressure, elevated fasting glucose).3. Body mass index (BMI) greater than or equal to 25.0 kg/m².4.Cohort 1 Subjects only: Subjects who do not have a historical liver biopsy specimen (meeting Inclusion Criteria 5 below) must meet either inclusion criterion 4a OR 4b: a. FibroScan liver stiffness measurement (LSM) greater than or equal to 15 kilopascals (kPa) Note: All subjects must complete a FibroScan examination during the screening period, unless historical values for a FibroScan assessment performed within 12 weeks prior to baseline/Day 1 (or up to 14 weeks prior to baseline/Day 1 with an approved screening window extension) are available.b. ELF score greater than or equal to 9.8.5.Cohort 1 Subjects only: Biopsy-proven compensated cirrhosis (fibrosis stage 4) due to NASH/MASH based on a centrally read biopsy. Must have had a liver biopsy obtained less than or equal to 365 days prior to screening with fibrosis stage 4 and a NAFLD activity score (NAS) of greater than or equal to 3 with at least 1 point in each of the following components: a.Steatosis (scored 0 to 3) b.Ballooning degeneration (scored 0 to 2), and c. Lobular inflammation (scored 0 to 3) 6.Cohort 2 Subjects only: Subjects must meet either Inclusion Criterion 6a, 6b, OR 6c below:a.Biopsy-proven compensated cirrhosis (fibrosis stage 4) with no competing etiology for liver disease. Must have had a liver biopsy specimen collected during screening or within 365 days prior to screening with confirmation of fibrosis stage 4 from a pathologist (centrally or locally read).Note: A subject cannot be considered for Cohort 2 based on Inclusion Criterion 6b or 6c if a biopsy previously submitted for central read failed to meet Inclusion Criterion 6a. If there is reason to suspect progression to cirrhosis, a new biopsy may be obtained and submitted for evaluation (centrally or locally) OR b.FibroScan LSM greater than or equal to 20.0 kPa. Note: All subjects must complete a FibroScan examination during the screening period, unless historical values for a FibroScan assessment performed within 12 weeks prior to baseline/Day 1 (or up to 14 weeks prior to baseline/Day 1 with an approved screening window extension) are available OR c.ELF score greater than or equal to 10.5.7.Central laboratory tests that meet all of the following criteria at screening: a.Albumin greater than or equal to 3.5 g/dL.b.Estimated glomerular filtration rate (eGFR) greater than or equal to 15 mL/min, as calculated by the Chronic Kidney Disease Epidemiology Collaboration (CKD-EPI).c.Hemoglobin A1c (HbA1c) less than or equal to 9.5%.d.International Normalized Ratio (INR) less than or equal to 1.3, unless due to therapeutic anticoagulation.e.Direct bilirubin less than or equal to 0.5 mg/dL.f.Creatine kinase (CK) less than 3 × upper limit of normal (ULN). g.Triglyceride (TG) level less than or equal to 500 mg/dL.h.25-Hydroxy Vitamin D greater than or equal to 13 ng/mL.Note: Laboratory tests for eligibility assessment may be repeated one time at the Investigator’s discretion. A subject who fails to meet Inclusion Criterion 7h will be allowed to retest 25-Hydroxy Vitamin D during the same screening period OR rescreen provided that they agree to take supplementation with Vitamin D.8.Central laboratory tests that meet all of the following criteria at screening and pre-baseline:a.Total bilirubin less than 1.3 mg/dL. For subjects with Gilbert’s syndrome or hemolytic anemia, total bilirubin may be elevated if direct bilirubin less than or equal to ULN.b.Platelet count greater than or equal to 75,000/µL.c.Alanine aminotransferase (ALT) less than or equal to 5 × ULN.d.Aspartate aminotransferase (AST) less than or equal to 5 x ULN.e.Alkaline phosphatase (ALP) less than 1.5 × ULN.Note: Laboratory tests for eligibility assessment may be repeated one time at the Investigator’s discretion 9.Documented stability of ALT and AST levels, as evidenced by no significant worsening of ALT and AST values at pre-baseline relative to screening values and the following parameters: a.If the screening and pre-baseline ALT and AST values are both less than or equal to 1.5 × ULN, there is no limit to the difference between the values b. If at least 1 of the screening or pre-baseline ALT or AST values is greater than 1.5 × ULN and shows worsening at pre-baseline, the percent increase must be less than or equal to 50%.Note: Subjects must have ALT and AST repeated during the screening period (Pre-baseline visit), with a minimum of 28 days between blood draws to confirm either criterion 9a or 9b above.Laboratory tests for eligibility assessment may be repeated one time at the Investigator’s discretion.10.Use of any conditionally allowed medications must follow the stable dose and adjustment criteria as outlined in protocol.11.Willing and able to give written informed consent prior to any study specific procedures being performed.12.Female subjects of childbearing potential (see definition in protocol) must have a negative serum pregnancy test at screening and a negative urine pregnancy test at baseline/Day 1. 13.Male and female subjects of childbearing potential who engage in heterosexual intercourse must agree to use protocol specified method(s) of contraception as described in protocol. 
 
ExclusionCriteria 
Details  1.Weight loss at screening defined as: a. Cohort 1: Weight loss greater than 10% within 90 days prior to the collection date of the liver biopsy specimen used to assess subject eligibility through randomization.b.Cohort 2: Weight loss greater than 10 % within 90 days prior to screening through randomization.2. Type 1 diabetes.3.Unstable T2D defined as: a.Insulin dose adjustment greater than 35% within 30 days prior to screening through randomization. b. Any prior history of diabetic ketoacidosis and/or hyperosmolar hyperglycemic state.4.Hypoglycemia unawareness, hospitalization due to hypoglycemia, or history of severe hypoglycemia (hypoglycemia requiring outside assistance to regain normal neurologic status) within 90 days prior to screening.5.Subjects with osteoporosis, defined as a T-score of less than or equal to minus 2.5 at the femoral neck, total hip, or lumbar spine based on a centrally read dual-energy X-ray absorptiometry (DXA) scan performed during screening.Note: A historical DXA scan performed within 90 days prior to screening may be accepted as the screening DXA scan. The historical scan must have been performed on a scanner previously qualified by the central imaging vendor that is available for use at post-baseline visits.6.Poorly controlled hypertension (systolic blood pressure greater than 160 mm Hg, or diastolic blood pressure greater than 100 mm Hg) at the Screening visit or Pre-baseline visit. Note: Vital signs for eligibility assessment may be repeated one time at the Investigator’s discretion.7.Presence of varices (Cohort 1) OR presence of high-risk varices (Cohort 2), based on a centrally read upper gastrointestinal (GI) EGD exam conducted at screening.8.Any current or prior history of decompensated liver disease including ascites, hepatic encephalopathy (HE), or variceal bleeding.9.Model for End-Stage Liver Disease (MELD) score greater than 12, unless due to hemolytic anemia, therapeutic anticoagulation, or Gilbert’s syndrome.10.Child-Pugh score greater than 6 (Class B or C), unless due to hemolytic anemia, therapeutic anticoagulation, or Gilbert’s syndrome.11.History of significant pancreatic disorders (acute pancreatitis, chronic pancreatitis, hereditary pancreatitis, and pancreatic cancer). 12.Chronic hepatitis B virus (HBV) infection (hepatitis B surface antigen [HBsAg] positive) or acute hepatitis A infection (hepatitis A immunoglobulin M [IgM] antibody positive). For subjects with positive hepatitis B core antibody (HBcAb), HBV DNA by quantitative polymerase chain reaction (PCR) will be required.13.Chronic hepatitis C virus (HCV) infection (HCV antibody [Ab] and HCV RNA positive). Subjects cured of HCV infection less than 2 years prior to the Screening visit (based on date of RNA PCR negative confirmation following conclusion of treatment) are not eligible.Note: Subjects who were previously diagnosed with chronic HCV infection who achieved sustained viral response (SVR) following treatment, or subjects with spontaneous clearance of HCV infection (positive serology for HCV infection, negative for
HCV RNA at screening, and no documented history of acute HCV infection within 2 years prior to screening) may be enrolled.14. Prior (less than 2 years prior to screening) or planned (during the study period) bariatric surgery (e.g., gastroplasty, Roux-en-Y gastric bypass) or reversal or removal of intragastric balloon. Surgery failure less than 2 years prior to screening is also exclusionary
15.Other causes of liver disease based on medical history and/or centralized review of liver histology and/or central laboratory results, including but not limited to: alcoholic liver disease, autoimmune disorders (e.g., primary biliary cholangitis [PBC], primary sclerosing cholangitis [PSC], autoimmune hepatitis), drug induced hepatotoxicity, Wilson disease, clinically significant iron overload, or alpha-1-antitryspin deficiency.16.History of liver transplantation.17. Current or prior history of hepatocellular carcinoma (HCC).18.Current diagnosis of Cushing’s syndrome.19. History of significant alcohol consumption for a period of more than 3 consecutive months within 1 year prior to screening Note: Significant alcohol consumption is defined as an average exceeding 1 ethanol containing drink/day in female subjects and
2 ethanol-containing drinks/day in male subjects.20.Human immunodeficiency virus (HIV) infection.21. Uncontrolled cardiac arrhythmia or confirmed QT interval corrected using Fridericia’s formula (QTcF) greater than 450 msec for males and greater than 470 msec for females at the screening ECG assessment. Subjects with cardiac pacemakers and elevated QTcF (greater than 450 msec for males and greater than 470 msec for females) may be allowed to participate if, in the Investigator’s opinion, the subject’s cardiac function is stable
Note: ECG for eligibility assessment may be repeated one time at the Investigator’s discretion.22.Myocardial infarction, unstable angina, percutaneous coronary intervention, coronary artery bypass graft, or stroke within 90 days prior to screening and through randomization.23.Life expectancy of less than 2 years.24.Use of any investigational medication within 30 days or 5 halflives, whichever is longer, prior to screening or concurrent participation in another therapeutic clinical study.Note: Without written approval from the Sponsor, a historical biopsy cannot be used for screening eligibility if the biopsy was collected prior to or during treatment with an investigational drug.25.Use of any prohibited medication(s) as outlined in protocol, including any prior exposure to EFX Positive urine drug screen for amphetamines, cocaine, or opiates (e.g., heroin, morphine) at screening. Subjects with a 26.positive urine drug screen due to prescription medication (e.g., opiates, methylphenidate) are eligible if the prescription and diagnosis are reviewed and approved by the Investigator. Subjects on stable methadone or buprenorphine maintenance treatment for at least 180 days prior to screening may be included in the study.27. Cohort 1 only: Unable to safely undergo a liver biopsy.28.Presence of any laboratory abnormality or significant systemic or major illnesses (other than liver disease) that, in the opinion of the Investigator, compromises the subject’s ability to safely participate in and complete the study including, but not limited to:
a. Pulmonary disease, heart failure, renal failure, organ transplantation, serious psychiatric disease, malignancy, history of substance abuse and/or a psychiatric condition requiring hospitalization and/or emergency room visit within 180 days of screening.29. Unavailable for follow-up assessment or concern for subject’s compliance with the protocol procedures.30.Known hypersensitivity to the study drug, its metabolites, or formulation excipients.
 
 
Method of Generating Random Sequence   Computer generated randomization 
Method of Concealment   Case Record Numbers 
Blinding/Masking   Participant and Investigator Blinded 
Primary Outcome  
Outcome  TimePoints 
Proportion of subjects who achieve more than or equal to 1 stage improvement in fibrosis (based on NASH CRN fibrosis score) and no worsening of steatohepatitis (defined as no increase in NAS for ballooning, inflammation, or steatosis) at Week 96 (Cohort 1 only).  Week 96 
 
Secondary Outcome  
Outcome  TimePoints 
Proportion of subjects who achieve more than or equal to 1 stage improvement in fibrosis (based on NASH CRN fibrosis score) & no worsening of steatohepatitis (defined as no increase in NAS for ballooning, inflammation, or steatosis) at End of Study(Cohort 1)
Proportion of subjects who achieve more than or equal to 1 stage improvement in fibrosis (based on NASH CRN fibrosis score) at Week 96 & End of Study(Cohort 1)
Proportion of subjects who achieve NASH/MASH resolution (defined as a NAS of 0—1 for inflammation & 0 for ballooning) as determined by the NASH CRN criteria at Week 96 & End of Study.(Cohort 1)
Proportion of subjects who achieve NASH/MASH resolution (defined as a NAS of 0—1 for inflammation & 0 for ballooning) AND more than or equal to 1 stage improvement in fibrosis (based on NASH CRN fibrosis score) at Week 96 & End of Study(Cohort 1) 
Week 96 & End of Study  
Change from baseline in ELF score & components (TIMP-1, HA, PIIINP), Pro-C3, liver
stiffness assessed by FibroScan, & FAST score
Change from baseline in ALT & AST
Change from baseline in total cholesterol, TG, HDL-C, non-HDL-C, & LDL-C
Change from baseline in HbA1c, C-peptide,
adiponectin, insulin, & HOMA-IR
Change from baseline in body weight
Safety & tolerability will be assessed through the
reporting of extent of exposure,AEs,and clinical assessments 
NA 
 
Target Sample Size   Total Sample Size="1150"
Sample Size from India="115" 
Final Enrollment numbers achieved (Total)= "Applicable only for Completed/Terminated trials"
Final Enrollment numbers achieved (India)="Applicable only for Completed/Terminated trials" 
Phase of Trial   Phase 3 
Date of First Enrollment (India)   18/11/2024 
Date of Study Completion (India) Applicable only for Completed/Terminated trials 
Date of First Enrollment (Global)  01/06/2024 
Date of Study Completion (Global) Applicable only for Completed/Terminated trials 
Estimated Duration of Trial   Years="5"
Months="4"
Days="0" 
Recruitment Status of Trial (Global)   Open to Recruitment 
Recruitment Status of Trial (India)  Open to Recruitment 
Publication Details   N/A 
Individual Participant Data (IPD) Sharing Statement

Will individual participant data (IPD) be shared publicly (including data dictionaries)?  

Response - NO
Brief Summary  

Cohort 1: Biopsy-proven Cirrhosis due to NASH/MASH

This is a Phase 3, randomized, double-blind, placebo-controlled study evaluating the clinical benefit and safety of EFX in subjects with compensated cirrhosis due to NASH/MASH. The study will enroll in 2 cohorts. Cohort 1 will enroll approximately 750 subjects with biopsy-proven compensated cirrhosis due to NASH/MASH. Cohort 2 will enroll approximately 400 subjects with a clinical diagnosis of compensated cirrhosis due to NASH/MASH. 

Evaluation of the clinical outcomes primary endpoint will occur when a pre-specified number of adjudicated events has been accrued, which is anticipated to be approximately 156 weeks. This time frame may be extended until the prespecified number of events has accrued. 

A histology primary endpoint analysis will occur after all subjects in Cohort 1 have completed the Histology Primary Endpoint Treatment Duration (i.e., completed 96 weeks of treatment or permanently discontinued from the study prior to Week 96). For evaluation of longer-term safety and efficacy, all subjects will continue to receive their assigned treatment until the pre-specified number of adjudicated events has been accrued.

Subjects meeting the study’s eligibility criteria will be randomly assigned in a 1:1 ratio into 1 of 2 treatment groups as shown in the figures below.  

Cohort 2: Clinical Diagnosis of Cirrhosis due to NASH/MASH  

Subjects will be enrolled into Cohort 2 only after enrollment for Cohort 1 is complete. With Sponsor approval, a subject may be considered for Cohort 2 enrollment prior to completion of Cohort 1 enrollment in scenarios such as those outlined below:

Biopsy confirms fibrosis stage 4 but minimum scores for steatosis, ballooning, and/or lobular inflammation are not met. Esophagogastroduodenoscopy (EGD) confirms either no varices or presence of low-risk varices

Biopsy confirms fibrosis stage 4 and minimum scores for steatosis, ballooning, and lobular inflammation are met. EGD confirms presence of low-risk varices 

 
Close