Ibutamoren for Nonalcoholic Fatty Liver Disease

Overseen By Laura E. Dichtel, MD, MHS

Age: 18 - 65

Sex: Any

Trial Phase: Phase 2

Sponsor: Massachusetts General Hospital

Must not be taking: Diabetes medications, Oral steroids

Disqualifiers: Diabetes, Cancer, Renal disease, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: Unknown

Duration: 6 months

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial explores whether the drug Ibutamoren can reduce liver fat in individuals with nonalcoholic fatty liver disease (NAFLD). NAFLD, often linked to obesity, is a liver condition not caused by alcohol and can lead to more serious liver issues over time. The study invites generally healthy adults with a BMI of 25 or higher who have been diagnosed with NAFLD or NASH (a more severe form of NAFLD) through imaging or biopsy. Participants should not have diabetes or other significant health conditions that could affect the liver. The goal is to determine if Ibutamoren can effectively lower liver fat levels, potentially preventing further liver damage. As a Phase 2 trial, this research measures Ibutamoren's effectiveness in an initial, smaller group, allowing participants to contribute to important findings.

Will I have to stop taking my current medications?

The trial requires that you do not take medications that may interact with LUM-201 (ibutamoren mesylate). If you are on such medications, you may need to stop them to participate.

Is there any evidence suggesting that Ibutamoren is likely to be safe for humans?

Research has shown that Ibutamoren, also known as LUM-201, is generally well tolerated. In past studies, participants received varying doses, and it was found to be mostly safe. The most common side effects were mild, such as increased appetite and slight swelling. It is important to note that the FDA has not yet approved Ibutamoren for any condition. However, its use in clinical trials indicates that researchers are actively studying its safety and effectiveness. So far, results have been promising in terms of safety, with no major problems reported. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising for NAFLD?

Unlike standard treatments for nonalcoholic fatty liver disease (NAFLD), which often focus on lifestyle changes and medications like vitamin E or pioglitazone, Ibutamoren offers a unique approach by mimicking the action of ghrelin, a hormone that stimulates growth hormone release. This mechanism can potentially improve liver health by promoting cell regeneration and reducing inflammation. Researchers are excited about Ibutamoren because it targets the underlying issues in NAFLD differently than existing treatments, possibly offering an innovative option for patients who haven't responded well to current therapies.

What evidence suggests that Ibutamoren might be an effective treatment for nonalcoholic fatty liver disease?

Research has shown that Ibutamoren, also known as LUM-201, might help reduce liver fat in people with nonalcoholic fatty liver disease (NAFLD). It boosts growth hormone levels, which can lower fat buildup in the liver. Early results suggest it may decrease fat inside the liver, an important factor in treating NAFLD. Other studies have found that Ibutamoren might also address liver issues related to obesity by increasing growth hormone levels. While more research is needed, these early findings are promising for those with NAFLD considering this treatment. Participants in this trial will receive an open-label treatment with Ibutamoren to further evaluate its effects. ^{1 2 6 7 8}

Who Is on the Research Team?

Laura E Dichtel, MD

Principal Investigator

Massachusetts General Hospital

Are You a Good Fit for This Trial?

This trial is for adults aged 21-60 with a BMI of at least 25 who have been diagnosed with nonalcoholic fatty liver disease (NAFLD) or nonalcoholic steatohepatitis (NASH), and have lower than normal levels of IGF-1. It's not open to those with diabetes, other liver diseases, pituitary or hypothalamic disorders, recent use of certain drugs that affect the liver, interactions with LUM-201, history of cancer or significant heart/kidney disease, or if pregnant/breastfeeding.

Inclusion Criteria

I have been diagnosed with fatty liver disease or NASH.

BMI ≥ 25 kg/m2

I am between 21 and 60 years old and in good health.

Exclusion Criteria

Pregnancy or breastfeeding

I have a condition affecting my pituitary or hypothalamus that impacts growth hormone.

Contraindications to MRI imaging

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive open-label oral LUM-201 (ibutamoren mesylate) 25mg daily

6 months

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

• Ibutamoren

Trial Overview The trial tests whether Ibutamoren mesylate (LUM-201) can reduce fat in the liver among people without alcohol-related liver issues. The study will measure changes in intrahepatic lipid content using advanced imaging techniques like proton magnetic resonance spectroscopy.

How Is the Trial Designed?

Group I: Open-label Treatment Experimental Treatment 1 Intervention

Open-label study of oral LUM-201 (ibutamoren mesylate) 25mg daily in otherwise healthy adults with BMI ≥25 kg/m2 and histologic or radiologic diagnosis of NAFLD.

Who Is Running the Clinical Trial?

Massachusetts General Hospital

Lead Sponsor

Trials

3,066

Recruited

13,430,000+

Citations

1. clinicaltrials.gov clinicaltrials.gov/study/NCT05364684

The Impact of Ibutamoren on Nonalcoholic Fatty Liver ... It identifies the role of the intervention that participants receive. Types of arms include experimental arm, active comparator arm, placebo comparator arm, ...

2. withpower.com withpower.com/trial/phase-2-non-alcoholic-fatty-liver-disease-5-2022-05ecc

Ibutamoren for Nonalcoholic Fatty Liver Disease Ibutamoren. Trial Overview The trial tests whether Ibutamoren mesylate (LUM-201) can reduce fat in the liver among people without alcohol-related liver issues.

3. bwh.trialstoday.org bwh.trialstoday.org/trial/NCT05364684

The Impact of Ibutamoren on Nonalcoholic Fatty Liver ... The investigators hypothesize that LUM-201 (Ibutamoren mesylate) will decrease intrahepatic lipid accumulation as quantified by proton magnetic ...

4. investors.lumos-pharma.com investors.lumos-pharma.com/news-releases/news-release-details/lumos-pharma-announces-clinical-collaboration-massachusetts

Press Release - Investors - Lumos Pharma This investigator-initiated Phase 2 trial is a single-site, 6-month, open-label pilot study of daily oral LUM-201 in adults with NAFLD.

5. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC9227666/

A GH Secretagogue Receptor Agonist (LUM-201) Elicits ... In addition, the data suggest that LUM-201 may be effective in increasing GH exposure in some pediatric patients within the GHD spectrum, especially those with ...

6. synapse.patsnap.com synapse.patsnap.com/drug/f0832b8b7f544f8a94fd41577786b6b2

Ibutamoren Mesylate - Drug Targets, Indications, Patents Ibutamoren (also known as MK-677) is an active ingredient not approved by FDA, and therefore its safety and efficacy have not been established. Ibutamoren is a ...

7. downloads.regulations.gov downloads.regulations.gov/FDA-2024-N-4188-0004/attachment_2.pdf

Bulk Drug Substance Nomination We (investigators) evaluated the safety and tolerability and the GH-insulin-like growth factor (IGF) responses to two dosages of oral ibutamoren mesylate given ...

8. lumos-pharma.com lumos-pharma.com/our-pipeline/lum-201/

LUM-201 Important Safety Information – *LUM-201 investigational studies continue. LUM-201 has been generally well tolerated to date. The most frequently reported ...