💬In plain words
What the trial is, and whether it could suit you
The goal of this clinical trial is to compare a one-week course of diazoxide (2 mg/kg per dose x 14 doses) and placebo in people with obesity and insulin resistance (IR) with metabolic dysfunction-associated steatotic liver disease (MASLD). The main question it aims to answer are how mitigation of compensatory hyperinsulinemia with diazoxide affects hepatic de novo lipogenesis, a major contributor to MASLD pathophysiology.
Participants will:
* Take 14 doses of placebo over 7 days, followed 4-12 weeks later by either 14 doses of diazoxide (at 2 mg per kg of body weight per dose \[mpk\]) or another 14 doses of placebo, over 7 days
* Take 18 doses of heavy (deuterated) water (50 mL each) over 7 days, twice
* Have blood drawn and saliva collected after an overnight fast on four mornings over the course of the study
* Undergo insulin suppression tests (IST) to assess the degree of insulin r
Sign in to get the eligibility rules as simple questions to take to your doctor.
📋 Eligibility rules (as published)
Inclusion Criteria:
* Adults aged 18-65 years
* Body mass index of 30-45 kg/m2
* Able to understand written and spoken English and/or Spanish
* Able to have pre-randomization screening labs drawn and study protocol initiated within 60 days of eligibility determination
* Presence of uncomplicated metabolic dysfunction-associated steatotic liver disease (MASLD) by vibration-controlled transient elastography (VCTE)
* Steatosis score of S1-S3
* Fibrosis score of F0-F2 (Note that if VCTE result is available from within past 6 months, then do not have to repeat VCTE for study purposes)
* Evidence of insulin resistance, represented by any or all of the following criteria:
* Meeting either of the American Diabetes Association's definitions for prediabetes or impaired fasting glucose (IFG) on screening labs:
* Prediabetes: Hemoglobin A1c 5.7-6.4%
* IFG: plasma glucose of 100-125 mg dL-1 after ≥ 8-h fast
* Homeostasis Model of Insulin Resistance (HOMA-IR) score ≥ 2.73
* Fasting hyperinsulinemia (fasting insulin level ≥ 13 μU/mL) on screening labs
* Written informed consent (in English or Spanish) and any locally required authorization (e.g., Health Insurance Portability and Accountability Act) obtained from the participant prior to performing any protocol-related procedures, including screening evaluations.
Exclusion Criteria:
* Unable to provide informed consent in English or Spanish
* Concerns arising at screening visit (any of the following):
* Documented weight loss of ≥ 5.0% of baseline within the previous 3 months
* Abnormal blood pressure (including on treatment, if prescribed)
* Systolic blood pressure (SBP) \< 90 mm Hg or \> 160 mm Hg, and/or
* Diastolic blood pressure (DBP) \< 60 mm Hg or \> 100 mm Hg
* Resting heart rate \< 55 bpm or ≥ 110 bpm
* Abnormal screening electrocardiogram (or if on file, performed within previous 90 days)
* Laboratory evidence of diabetes mellitus:
* Hemoglobin A1c ≥ 6.5%, and/or
* Fasting plasma glucose ≥ 126 mg/dL
* Positive qualitative serum β-human chorionic gonadotropin (β-hCG, i.e., pregnancy test) in women of childbearing potential
* Liver function abnormalities: transaminases (aspartate aminotransferase or alanine aminotransferase) \> 3.0 x the upper limit of normal, and/or total bilirubin \> 1.25 x the upper limit of normal
* Abnormal screening fasting triglycerides \> 500 mg/dL
* Abnormal screening serum electrolytes that are considered clinically significant according to the clinical judgment of the PI
* Creatinine equating to estimated glomerular filtration rate \< 60 mL/min/1.73 m2
* Abnormal screening blood counts (any of the following):
* Hemoglobin \< 10 g/dL
* White blood cell count below the lower limit of normal for sex
* Platelet count below the lower limit of normal for sex
* Uric acid level above the upper limit of normal
* Reproductive concerns
* Women currently pregnant (tested by serum and/or urine β-hCG)
* Women currently breastfeeding
* Concerns related to glucose metabolism
* History of having met any of the American Diabetes Association's definitions of diabetes mellitus (i.e., overt diabetes):
* Hemoglobin A1c ≥ 6.5%
* Plasma glucose ≥ 126 mg/dL after 8-h fast
* Plasma glucose of ≥ 200 mg/dL at 2 h after ingestion of a 75-g glucose load
* Random plasma glucose ≥ 200 mg/dL associated with typical hyperglycemic symptoms, diabetic ketoacidosis, or hyperglycemic-hyperosmolar state
* History of gestational diabetes mellitus within the previous 5 years
* Use of antidiabetic medications except metformin within the 90 days prior to screening
* Clinical concern for absolute insulin deficiency (e.g., type 1 diabetes, pancreatic disease)
* Concerns related to lipid metabolism
* Known diagnoses of familial hypercholesterolemia, familial combined hyperlipidemia, or familial hyperchylomicronemia
* Use of fibrates, prescription-strength omega-3 fatty acids, or high-dose niacin within the 90 days prior to screening:
* Known, documented history (i.e., not to be newly screened/tested for study purposes), at the time of screening, of any of the following medical conditions:
* Pancreatic pathology, including but not limited to neoplasia, pancreatitis, pancreatectomy
* Cardiovascular disease (N.B. uncomplicated hypertension is not exclusionary)
* Atherosclerotic cardiovascular disease: stable or unstable angina, myocardial infarction, ischaemic or hemorrhagic stroke, or transient ischaemic attack, peripheral arterial disease (claudication), use of dual antiplatelet therapy (aspirin + P2Y12 inhibitor), history of percutaneous coronary intervention
* Heart rhythm abnormalities
* Congestive heart failure of any New York Heart Association class
* Symptomatic valvular heart disease (e.g., aortic stenosis)
* Pulmonary hypertension
* Chronic kidney disease, Stage 3 or higher (estimated glomerular filtration rate \< 60 mL/min/ 1.73 m2), of any cause
* Chronic liver disease other than uncomplicated MASLD, including but not limited to:
* Advanced liver fibrosis, as determined by non-invasive testing, including fibrosis scores of F3-F4 on VCTE
* Cirrhosis of any etiology
* Autoimmune hepatitis or other rheumatologic disorder affecting the liver
* Biliopathy (e.g., progressive sclerosing cholangitis, primary biliary cholangitis)
* Chronic liver infection (e.g., viral hepatitis, parasitic infestation)
* Hepatocellular carcinoma
* Infiltrative disorders (e.g., sarcoidosis, hemochromatosis, Wilson disease)
* Gout
* Chronic infection with hepatitis B virus (HBV), hepatitis C virus (HCV), or human immunodeficiency virus (HIV)
* Malabsorptive conditions
* Active seizure disorder (including controlled with antiepileptic drugs)
* Psychiatric diseases that are or have been decompensated within 1 year of screening, and/or require use of antipsychotic drugs associated with significant weight ga