Ignite Creation Date:
2025-12-24 @ 9:12 PM
Ignite Modification Date:
2025-12-25 @ 7:01 PM
Study NCT ID:
NCT07276204
Status:
NOT_YET_RECRUITING
Last Update Posted:
2025-12-10
First Post:
2025-11-28
Is NOT Gene Therapy:
False
Has Adverse Events:
False
Brief Title:
A Phase II RCT Trial of Betaine vs. Placebo for Serologically Diagnosed Metabolic Dysfunction-associated Steatohepatitis (MASH)
Sponsor:
Southern California Institute for Research and Education
Organization:
Study Overview
Official Title:
A Phase II, Double-masked, Placebo-controlled, Randomized, Parallel Treatment Group Trial to Investigate the Safety and Efficacy of Betaine + Standard of Care (SOC) vs. Placebo + SOC Among Participants With Serologically Diagnosed Metabolic Dysfunction-associated Steatohepatitis (MASH) and Elevated Serum Alanine Aminotransferase (ALT) Level
Status:
NOT_YET_RECRUITING
Status Verified Date:
2025-11
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
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.
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.
Detailed Description:
Study Description: This is a randomized, double-masked phase II trial to test the hypothesis that 24 weeks of treatment with oral betaine, 2 g/day, is a safe and effective treatment for participants with serologically diagnosed metabolic dysfunction-associated steatohepatitis (MASH) and elevated serum alanine aminotransferase (ALT) level.
A total of 70 participants will be randomized at VA Long Beach. Primary endpoint is resolution of at-risk MASH determined by NIS2+™ score at the end of treatment (Week 24). Total study duration for each participant is up to 56 weeks (including screening, treatment, and follow-up).
Primary Objective: To evaluate the efficacy of betaine 2 g/day in reducing liver injury as assessed by percent of participants with NIS2+™ \<0.6815 (ie, no longer having at-risk MASH) at the end of treatment (Week 24).
Secondary Objectives:
To evaluate the efficacy of betaine 2 g/day in reducing liver steatosis as assessed by percent of participants with \>30% decrease in MRI proton density fat fraction (PDFF) at Week 24 (end of treatment).
To evaluate the safety and tolerability of betaine 2 g/d
Primary Endpoint: The primary endpoint is percent of participants with NIS2+™ \<0.6815 (no longer having at-risk MASH) at Week 24 (end of treatment).
Secondary Endpoints:
The percentage of participants with \>30% decline in MRI-PDFF at Week 24 (end of treatment).
Safety and tolerability based on adverse events, clinical laboratory values, vital signs, and patient report.
Exploratory Endpoints: There are 5 independent groups of exploratory endpoints: efficacy, cardiovascular risk, laboratory safety, betaine metabolites, and change in behavior and weight.
Study Population: 70 adults of either sex with serologically-diagnosed metabolic dysfunction-associated steatohepatitis and an ALT ≥50 U/L.
Phase: 2 Facilities Enrolling Participants: VA Long Beach Healthcare System Study Intervention: Group 1: betaine (oral, 1 gram BID) + standard of care Group 2: placebo (oral, BID) + standard of care
Study Duration: 5 years
Participant Duration: Participant participation is approximately 13 months:
Treatment: 24 weeks Post-treatment follow-up: 24 weeks
Statistical Assumptions Treatment groups 2 Randomization 1:1 Sample size estimates A two-sided two-sample Z-test with continuity correction and pooled variance Sample size 70 (35 per group) (includes drop outs) Analysis dataset All randomized participants who received at least one does of study medicine
A total of 70 participants will be randomized at VA Long Beach. Primary endpoint is resolution of at-risk MASH determined by NIS2+™ score at the end of treatment (Week 24). Total study duration for each participant is up to 56 weeks (including screening, treatment, and follow-up).
Primary Objective: To evaluate the efficacy of betaine 2 g/day in reducing liver injury as assessed by percent of participants with NIS2+™ \<0.6815 (ie, no longer having at-risk MASH) at the end of treatment (Week 24).
Secondary Objectives:
To evaluate the efficacy of betaine 2 g/day in reducing liver steatosis as assessed by percent of participants with \>30% decrease in MRI proton density fat fraction (PDFF) at Week 24 (end of treatment).
To evaluate the safety and tolerability of betaine 2 g/d
Primary Endpoint: The primary endpoint is percent of participants with NIS2+™ \<0.6815 (no longer having at-risk MASH) at Week 24 (end of treatment).
Secondary Endpoints:
The percentage of participants with \>30% decline in MRI-PDFF at Week 24 (end of treatment).
Safety and tolerability based on adverse events, clinical laboratory values, vital signs, and patient report.
Exploratory Endpoints: There are 5 independent groups of exploratory endpoints: efficacy, cardiovascular risk, laboratory safety, betaine metabolites, and change in behavior and weight.
Study Population: 70 adults of either sex with serologically-diagnosed metabolic dysfunction-associated steatohepatitis and an ALT ≥50 U/L.
Phase: 2 Facilities Enrolling Participants: VA Long Beach Healthcare System Study Intervention: Group 1: betaine (oral, 1 gram BID) + standard of care Group 2: placebo (oral, BID) + standard of care
Study Duration: 5 years
Participant Duration: Participant participation is approximately 13 months:
Treatment: 24 weeks Post-treatment follow-up: 24 weeks
Statistical Assumptions Treatment groups 2 Randomization 1:1 Sample size estimates A two-sided two-sample Z-test with continuity correction and pooled variance Sample size 70 (35 per group) (includes drop outs) Analysis dataset All randomized participants who received at least one does of study medicine
Study Oversight
Has Oversight DMC:
True
Is a FDA Regulated Drug?:
False
Is a FDA Regulated Device?:
False
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
True
Is an FDA AA801 Violation?: