Diazoxide for Non-alcoholic Fatty Liver Disease
What You Need to Know Before You Apply
What is the purpose of this trial?
This trial explores whether the drug diazoxide (also known as Proglycem or Hyperstat) can aid individuals with non-alcoholic fatty liver disease (NAFLD) by lowering high insulin levels. Participants will receive either a low or high dose of diazoxide or a placebo over two weeks. The study will assess how these treatments affect the liver's processing of fat and sugar. Individuals with NAFLD and insulin resistance who are at high risk of the disease are suitable candidates. As a Phase 1 trial, the research focuses on understanding how the treatment works in people, offering participants an opportunity to contribute to early-stage medical research.
Will I have to stop taking my current medications?
The trial requires that you stop taking most antidiabetic medications (except metformin) and certain lipid-lowering drugs within 90 days before screening. If you're on these medications, you may need to stop them to participate.
Is there any evidence suggesting that diazoxide is likely to be safe for humans?
Research has shown that diazoxide is usually well-tolerated in humans. It has been studied for its ability to lower insulin levels, which might help reduce liver fat in people with insulin resistance (IR) and non-alcoholic fatty liver disease (NAFLD). Other studies found that diazoxide does not significantly increase blood sugar levels, indicating that its effect on glucose is manageable.
As this is an early-phase trial, the main goal is to assess the safety and tolerability of diazoxide at different doses. Although complete safety data from this study is not yet available, diazoxide's use in other conditions provides some reassurance about its safety. Participants might experience some side effects, but these are usually mild. The trial will examine these safety aspects more closely.12345Why do researchers think this study treatment might be promising for non-alcoholic fatty liver disease?
Unlike other treatments for non-alcoholic fatty liver disease, which often focus on diet and exercise or medications like vitamin E and pioglitazone, diazoxide acts on a different pathway. This drug is unique because it targets mitochondrial function and insulin sensitivity, which are crucial in reducing liver fat. Researchers are excited about diazoxide because it could offer a more direct way to tackle the underlying metabolic issues associated with this condition, potentially leading to faster and more effective results. Additionally, the study explores two different dosages, which might help in fine-tuning the treatment's efficacy and safety.
What evidence suggests that diazoxide might be an effective treatment for non-alcoholic fatty liver disease?
This trial will evaluate the effects of diazoxide on non-alcoholic fatty liver disease (NAFLD). Research suggests that diazoxide might help with NAFLD by reducing liver damage and improving energy processing in the body. Studies have shown that diazoxide can lower signs of liver injury, potentially protecting the liver. Additionally, diazoxide reduces insulin release, which might help manage insulin resistance—a major issue in NAFLD. While early results are promising, more research is needed to confirm these effects in people with NAFLD. Participants in this trial will receive either a placebo or varying doses of diazoxide to assess its effectiveness.23456
Who Is on the Research Team?
Joshua R Cook, MD, PhD
Principal Investigator
Columbia University
Are You a Good Fit for This Trial?
Adults aged 18-70 with overweight/obesity and insulin resistance, at high risk for or diagnosed with NAFLD. Must have prediabetes indicators like fasting plasma glucose of 100-125 mg/dL or HbA1c of 5.7-6.4%. Excludes those with diabetes, abnormal heart rates, recent significant weight loss, certain medical conditions including active COVID-19 within screening period, psychiatric diseases decompensated within a year, known allergies to trial drugs, and women not using effective contraception.Inclusion Criteria
Exclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Treatment
Participants receive diazoxide or placebo for 14 days to assess the impact on glucose and lipid metabolism
Follow-up
Participants are monitored for safety and effectiveness after treatment
What Are the Treatments Tested in This Trial?
Interventions
- Diazoxide
Diazoxide is already approved in United States for the following indications:
- Hypoglycemia
Find a Clinic Near You
Who Is Running the Clinical Trial?
Columbia University
Lead Sponsor
University of California, Berkeley
Collaborator
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
Collaborator