A Phase 1 interventional study of Placebo and Diazoxide Oral Suspension, 2 mg per kg per dose in Hyperinsulinemia, Insulin Resistance and Non-Alcoholic Fatty Liver Disease, sponsored by Columbia University. Recruiting at 1 site in United States. Open to participants aged 18 Years to 65 Years. Per ClinicalTrials.gov, last updated 2026-07-06.
Sponsored by Columbia University · Phase 1, Interventional, and Basic science
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:
Researchers will compare blood tests at the beginning and end of each 1-week study period in participants randomized (like the flip of a coin) to receive either placebo followed by diazoxide or placebo followed by placebo, to see how the drug treatment affects de novo lipogenesis, serum insulin, plasma glucose, and other serum lipid parameters (triglycerides, free fatty acids), among others.
Metabolic dysfunction-associated steatotic liver disease (MASLD) is an under-appreciated complication of lipid dysmetabolism in type 2 diabetes (T2DM). Although it appears that insulin resistance (IR) is a mechanism common to both, the pathophysiology of its connection to unhealthy fat accumulation in the liver remains unclear. The investigators propose that the hyperinsulinemia that accompanies IR drives the excess hepatic de novo lipogenesis (DNL) that characterizes IR-associated MASLD. In other words, hepatic IR may be "selective," such that DNL is more sensitive to stimulation by insulin than is suppression of endogenous glucose production. As such, despite its potential impact on glucose metabolism, lowering insulin levels might attenuate the pro-steatotic drive in patients with IR. The investigators' objective, therefore, is to blunt endogenous insulin secretion using the insulin anti-secretagogue diazoxide in order to assess the impact on DNL.
This is a single-center, randomized, double-blinded, placebo-controlled, crossover clinical trial to determine the lipogenic impact of hyperinsulinemia reduction with diazoxide oral suspension in participants with obesity and insulin resistance (prediabetic state or elevated Homeostasis Model Assessment of Insulin Resistance (HOMA-IR) score, + fasting hyperinsulinemia) who are diagnosed with MASLD. Participants will be randomized to one of two groups. Both groups will receive 14 doses of placebo over 7 days. Then, 4-12 weeks later, one group will cross over to receive 14 doses of diazoxide 2 mg per kg of body weight for 7 days, while the other group will receive a second 1-week course of placebo. Participants will consume heavy (deuterated) water for a total of 18 doses of 50 ml over each 1-week study period to measure de novo lipogenesis. They will present for outpatient blood draws and saliva collections after an overnight fast at the start and conclusion of each study period (Study Days 1, 8, 9 and 16), and will undergo formal assessment of insulin resistance by the insulin suppression test (IST) at the end of each study period (Days 8 and 16).
Presence of uncomplicated metabolic dysfunction-associated steatotic liver disease (MASLD) by vibration-controlled transient elastography (VCTE)
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:
Exclusion Criteria:
Concerns arising at screening visit (any of the following):
Abnormal blood pressure (including on treatment, if prescribed)
Laboratory evidence of diabetes mellitus:
Abnormal screening blood counts (any of the following):
Reproductive concerns
Concerns related to glucose metabolism
History of having met any of the American Diabetes Association's definitions of diabetes mellitus (i.e., overt diabetes):
Concerns related to lipid metabolism
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:
Cardiovascular disease (N.B. uncomplicated hypertension is not exclusionary)
Chronic liver disease other than uncomplicated MASLD, including but not limited to:
Use of certain medications currently or within 90 d prior to screening:
Prescribed medications used for any of the indications in the preceding list of excluded conditions, or their use within 90 d prior to screening, except allowances for:
History of certain weight-loss (bariatric) surgeries, including:
During the first 1-week study period, participants will ingest a placebo solution at 14 doses over 7 days. During the second 1-week study period, 4-12 weeks later, participants will ingest diazoxide oral suspension at 2 mg per kg body weight per dose (14 doses over 7 days). Blinding will occur by completely covering single-dose oral syringes with labels. 80% of participants will be randomized to this arm.
Drug: Placebo · Drug: Diazoxide Oral Suspension, 2 mg per kg per dose · Drug: Deuterated water (2H2O/D2O), 70% · Diagnostic Test: Insulin Suppression Test (IST)
During the first 1-week study period, participants will ingest a placebo solution at 14 doses over 7 days. During the second 1-week study period, 4-12 weeks later, participants will again ingest placebo solution (14 doses over 7 days). Blinding will occur by completely covering single-dose oral syringes with labels. 20% of participants will be randomized to this arm.
Drug: Placebo · Drug: Deuterated water (2H2O/D2O), 70% · Diagnostic Test: Insulin Suppression Test (IST)
Flavor-approximate placebo consisting of peppermint extract in diet tonic water, thickened with xanthan gum, provided in label-obscured single-use oral syringes at 40 µL per kg per dose. 80% of participants will receive placebo (14 doses over 7 days) during the first 1-week study period, while 20% of participants will receive an additional 14 doses of placebo over 7 days during the second study period, 4-12 weeks later.
Also known as: Placebo solution
Eighty percent of participants will ingest diazoxide oral suspension at 2 mg per kg body weight per dose (14 doses over 7 days) during the study's second 1-week treatment period. Blinding will occur by completely covering single-dose oral syringes with labels.
Also known as: Proglycem
All participants will consume 18 aliquots of deuterated water (2H2O/D2O) 50 mL over 7 days during both study periods to assess hepatic de novo lipogenesis. Tracer enrichment will be determined in blood and saliva.
Also known as: Heavy water
Participants receive intravenous infusions of regular insulin (32 milliunits \[mU\] per square meter \[m2\] per minute \[min\]), octreotide acetate (25 µg bolus + 0.27 µg/m2/min continuous infusion), and dextrose 20% in water (267 mg/m2/min continuous infusion) for 3 hours. Insulin resistance is reflected as the steady-state plasma glucose (SSPG) during the final 30 minutes of the procedure. IST is performed at the end of both study periods to determine the impact of placebo versus diazoxide on insulin sensitivity.
Hepatic de novo lipogenesis (absolute values)
Percent incorporation of newly synthesized fatty acids into serum or very low-density lipoprotein (VLDL) triglyceride (TG) (units: %)
Time frame: Study Days 8 and 16
Hepatic de novo lipogenesis (relative/change)
Percent incorporation of newly synthesized fatty acids into serum or VLDL TG (units: fold difference and/or ∆%)
Time frame: Study Days 8 and 16
Fasting plasma/serum insulin (absolute values)
Measurement of fasting endogenous insulin levels during treatment with diazoxide 2 mpk vs. placebo (units: micro-international units \[µIU\] per mL).
Time frame: Study Days 8 and 16
Fasting plasma/serum insulin (relative/change)
Measurement of fasting endogenous insulin levels during treatment with diazoxide 2 mpk vs. placebo (units: fold difference and/or change in µIU/mL).
Time frame: Study Days 8 and 16
Fasting plasma glucose
Measurement of fasting plasma glucose levels during treatment with diazoxide 2 mpk vs. placebo (units: mg/dL).
Time frame: Study Days 8 and 16
Fasting serum or plasma triglycerides
Measurement of fasting plasma or serum triglyceride levels during treatment with diazoxide 2 mpk vs. placebo (units: mg/dL).
Time frame: Study Days 8 and 16
Fasting plasma free fatty acids
Measurement of fasting plasma free fatty acid levels during treatment with diazoxide 2 mpk vs. placebo (units: mmol/L).
Time frame: Study Days 8 and 16
Steady state plasma glucose (SSPG) during IST
Measurement of plasma glucose at 10-minute intervals during 30-minute steady-state period at the end of each IST (units: mg/dL)
Time frame: At time points of 150, 160, 170, and 180 minutes during each 3-hour IST protocol
Skin de novo lipogenesis
Percent incorporation of newly synthesized fatty acids into sebum from healthy skin and, if present, skin from inflammatory skin lesions (units: %)
Time frame: 3 hours
Deuterium tracer enrichment in body water (measured in blood)
Enrichment of total body water with deuterated water (2H2O/D2O) (units: %)
Time frame: Study Days 8 and 16
Deuterium tracer enrichment in body water (measured in saliva)
Enrichment of total body water with deuterated water (2H2O/D2O) (units: %)
Time frame: Study Days 8 and 16
Deuterium tracer enrichment in sebum
Enrichment of sebum with deuterated water (2H2O/D2O) (units: %)
Time frame: 3 hours
Plan to share: Yes
Supporting information: Study protocol
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