Betaine vs. Placebo for Serologically Diagnosed Metabolic Dysfunction-associated Steatohepatitis (MASH)

NCT07276204 | Not Yet Recruiting Phase 2 DMC

• 1 other identifier: Betaine for MASH
• Study Type: interventional
• Target: 70
• Locations: 0 countries
• Sites: N/A

Brief Summary

The study will evaluate whether betaine reduces liver injury in people with metabolic dysfunction-associated steatohepatitis (MASH). MASH is a type of liver disease that occurs in some people with fatty liver. Betaine is a normal component in the human body and will be taken as a pill. Seventy (70) participants will be randomized to receive either betaine or placebo for 24 weeks. After stopping treatment, participants will be seen in clinic for another 24 weeks (total participation in the study is approximately 1 year). Procedures performed during the study include blood tests, MRI examinations, questionnaires, and clinic visits. We will measure improvement in liver injury with blood tests and with MRI.

Conditions

MASH - Metabolic Dysfunction-Associated Steatohepatitis

Keywords

MASH; ALT; Betaine

Outcome Measures

Primary Outcomes (1)

• NIS2+

  The primary endpoint is percent of participants with NIS2+™ \<0.6815 (no longer having at-risk MASH) at Week 24 (end of treatment).

  AT of 24 weeks of treatment with Betaine/Placebo

Secondary Outcomes (1)

• MRI-PDFF

  at Week 24 (end of treatment).

Study Arms (2)

Betaine

EXPERIMENTAL

betaine capsule (oral) 1 gram twice a day

Drug: Betaine

Placebo

PLACEBO COMPARATOR

Placebo capsule (oral) twice a day

Drug: Placebo

Interventions

Betaine DRUG

Two capsules of betaine, 500 mg, will be consumed twice a day for 24 weeks.

Betaine

Placebo DRUG

Two placebo capsules will be taken orally twice a day

Placebo

Eligibility Criteria

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

You may not qualify if:

• MRE liver stiffness \>5.0 kPa
• Prior liver biopsy demonstrating cirrhosis
• History of esophageal varices, ascites, or hepatic encephalopathy
• The following laboratory tests during screening (or on blood tests performed within 60 days prior to randomization, if they are not repeated during screening):
• HDL \>46 mg/dL for a male or \>58 mg/L for a woman
• AST or ALT \>5 x ULN for the clinical laboratory at the local study site
• Serum albumin \<3.5 g/dL
• Direct bilirubin \>0.6 mg/dL
• Platelet count less than 125,000 /mm3
• eGFR \< 40 mL/min
• INR \>1.2 (in a participant not receiving anticoagulation medicines)
• 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 the presence of hepatitis C virus (HCV) RNA or prior treatment for hepatitis C
• A diagnosis of autoimmune liver disease as defined by compatible liver histology or receipt of treatment for presumed autoimmune liver disease.

Sponsors & Collaborators

• Southern California Institute for Research and Education: lead

MeSH Terms

Interventions

Betaine

Intervention Hierarchy (Ancestors)

Trimethyl Ammonium Compounds; Quaternary Ammonium Compounds; Amines; Organic Chemicals; Onium Compounds

Central Study Contacts

Timothy R Morgan, MD

CONTACT

949 400 4432; timothy.morgan@va.gov

Aliya A Uddin, MPH

CONTACT

562-826-5212; aliya.uddin@va.gov

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: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Chief, Hepatology

Study Record Dates

• First Submitted: November 28, 2025
• First Posted: December 10, 2025
• Study Start: March 31, 2026
• Primary Completion (Estimated): December 31, 2029
• Study Completion (Estimated): December 31, 2035
• Last Updated: January 8, 2026

Record last verified: 2026-01

Data Sharing

• IPD Sharing: Will not share