RecruitingPhase 1

Human Models of Selective Insulin Resistance: Alpelisib, Part I

Who can join

As published by the study on ClinicalTrials.gov. "Not stated" means the study hasn't said — ask the study team.

Age
18 Years to 70 Years
Sex
All sexes
Healthy volunteers
Accepted
Compensation
Mentioned in the study description

Other requirements are listed under Eligibility below. Only the study team can confirm whether you can join.

What to expect

No one can promise a trial will help you personally — that is what the research is trying to find out.

What participation involves

  • Procedures mentioned: Infusions

Only what the study's summary and detailed description on ClinicalTrials.gov say. Time periods may describe the whole study or a part of it — ask the study team what applies to you.

Show the study's own words
  • "Participants will be asked to stay twice overnight in the hospital, take single doses of alpelisib and placebo (one or the other on each of the two hospital stays), and receive intravenous (into the vein) infusions of non-radioactive "tracer" molecules that allow researchers to measure the production of glucose (sugar) and fats by the liver."
  • "During each admission, they will take a dose of either alpelisib or placebo (in randomized order) in the evening and receive infusions of \[13C\] sodium acetate and \[2H\] D-glucose to measure DNL and endogenous glucose production (EGP), respectively, during an overnight fast."
Compensation
Mentioned in the study description
  • Study-related exams and procedures are typically provided at no cost. This is how many trials work in general — confirm the details for this study with the study team.
  • Participants are monitored closely by the research team throughout the study.
  • Some participants may receive access to an investigational treatment, which is still being studied.
  • Your participation helps researchers learn whether this approach could help future patients.

About this study

In plain language

This study tests how alpelisib, a drug that blocks some insulin actions, affects the liver's sugar and fat production. It is for adults ages 18–70 whose bodies respond well to insulin or resist its effects and are at risk for type 2 diabetes. Participants have two overnight hospital stays and receive alpelisib at one stay and a placebo, which has no active drug, at the other.

The goal of this clinical trial is to understand how the blood sugar-lowering hormone insulin works in healthy adults versus those who are at risk for type 2 diabetes. The study will use a drug called alpelisib, which interferes with insulin's actions in the body, to answer the study's main question: does the liver continue to respond to insulin's stimulation of fat production even when it loses the ability to stop making glucose (sugar) in response to insulin. Researchers will compare the impact of single doses of both alpelisib and placebo (inert non-drug) in random order (like flipping a coin) in study participants. Participants will be asked to stay twice overnight in the hospital, take single doses of alpelisib and placebo (one or the other on each of the two hospital stays), and receive intravenous (into the vein) infusions of non-radioactive "tracer" molecules that allow researchers to measure the production of glucose (sugar) and fats by the liver. Measurements will be done both while participants are fasting (not eating or drinking other than water) and while consuming a standardized diet of nutritional beverages. The objective is to evaluate the effect of lowering insulin levels, while maintaining constant mild hyperglycemia, on plasma glucose and lipid levels.

Compensation is read from the study's own description and may be missing — always confirm with the study team.

Eligibility

Who can join

  • Be 18–70 years old.
  • Understand written and spoken English or Spanish.
  • For the group that responds well to insulin: have a BMI of 18–25. BMI is a measure of weight compared with height.
  • For that same group: have fasting insulin of 10 or less, fasting blood sugar below 100, and A1c below 5.7%. A1c measures average blood sugar.
  • For that same group: have an insulin resistance score (HOMA-IR) below 2.5 and a liver scarring risk score (FIB-4) below 1.3.
  • For the group that resists insulin's effects: have a BMI of 30–45 and fasting insulin of 13 or more.
  • For the insulin-resistant group: also have fasting blood sugar of 100–125, A1c of 5.7–6.4%, or a HOMA-IR score of 2.5 or more.

Who can't join

  • Cannot give informed consent in English or Spanish; are pregnant, have a positive pregnancy test, or are breastfeeding.
  • Have diabetes, had diabetes during pregnancy within 5 years, may make too little insulin, or used listed diabetes drugs other than metformin within 90 days.
  • Have unsafe blood pressure, heart rate, or blood tests; serious heart or liver disease; certain mental health conditions; blood clots, bleeding problems, significant anemia, or certain cancers. Risk of fluid buildup or another condition that makes the study unsafe may also rule you out.
  • You or a parent, sibling, or child has certain inherited blood fat disorders; or you recently used certain blood fat medicines or steroid pills or shots above the allowed dose.
  • Had gastric bypass or certain other weight-loss surgery, or had a lap band or stomach sleeve within 6 months.
  • Have concerns about alcohol overuse or illegal drug use other than marijuana; regularly smoke or vape nicotine more than 1 day a week; or had an illness with fever within 30 days.
  • Have allergies to study drugs, foods, or supplies, including soy, cow's milk, or gluten; another important ongoing allergy; or food rules that prevent taking gelatin capsules.
  • Took part in another study of an experimental drug or biological treatment within the required waiting period—at least 6 months, or longer for some drugs. The study doctor may allow prior participation in his own studies.

Simplified for readability. The official criteria below are what the study team uses.

Official eligibility criteria

Inclusion Criteria: 1. Adults aged 18-70 years 2. Able to understand written and spoken English and/or Spanish 3. Body mass index of: * For Group IS: BMI 18-25 kg/m2 * For Group IR: BMI 30-45 kg/m2 4. Evidence of insulin sensitivity or insulin resistance: * Insulin sensitive (for Group IS) defined as all of the following: (1) Fasting serum insulin ≤ 10 µIU/mL, (2) Absence of dysglycemia (fasting plasma glucose \< 100 mg/dL and hemoglobin A1c \< 5.7%), (3) Homeostasis Model Assessment of Insulin Resistance (HOMA-IR) score \< 2.5, and (4) Fibrosis-4 (FIB-4) score \< 1.3 * Insulin resistant (for Group IR) defined as fasting serum insulin ≥ 13 µIU/mL plus at least one of the following: (1) Presence of prediabetic state (fasting plasma glucose 100-125 mg/dL and/or hemoglobin A1c 5.7-6.4%), and/or HOMA-IR ≥ 2.5 Exclusion Criteria: 1. Inability to provide informed consent in English or Spanish 2. Concerns arising at screening visit: * Abnormal vital signs: (1) Systolic blood pressure \< 90 mm Hg or \> 160 mm Hg and/or (2) Diastolic blood pressure \< 55 mm Hg or \> 100 mm Hg and/or (3) Abnormal resting heart rate \< 55 bpm (except at PI's discretion) or ≥ 110 bpm * Abnormal screening serum electrolytes judged by the PI to be potentially clinically significant, including liver function abnormalities (either of the following): (1) Transaminases (AST or ALT) \> 3.0 x the upper limit of normal and/or (2) Total bilirubin \> 1.25 x the upper limit of normal * Laboratory evidence of diabetes mellitus: (1) Hemoglobin A1c ≥ 6.5%, and/or (2) Fasting plasma glucose ≥ 126 mg/dL 3. Reproductive concerns i. Positive qualitative β-hCG (i.e., pregnancy test) in women of childbearing potential ii. Women currently pregnant iii. Women currently breastfeeding 4. Concerns related to glucose metabolism * History of having met any of the American Diabetes Association's definitions of diabetes mellitus (i.e., overt diabetes) * History of gestational diabetes mellitus within the previous 5 years * Use of most antidiabetic medications (other than metformin) within the 90 days prior to screening: thiazolidinediones, sulfonylureas, meglitinides, dipeptidyl peptidase-4 (DPP4) inhibitors, glucagon-like peptide-1 (GLP-1) receptor agonists, sodium-glucose cotransporter-2 (SGLT2) inhibitors, amylin mimetics, acarbose, insulin iv. Clinical concern for absolute insulin deficiency (e.g., type 1 diabetes, pancreatic disease) 5. Concerns related to lipid metabolism * Known diagnoses of familial hypercholesterolemia, familial combined hyperlipidemia, or familial hyperchylomicronemia in the participant or a first-degree relative * Use of certain lipid-lowering drugs within 14 d prior to screening visit: fibrates (e.g., fenofibrate, gemfibrozil), prescription-strength omega-3 fatty acids (e.g., icosapent ethyl), high-dose niacin (\>100 mg daily) 6. Known, documented history, at the time of screening, of any of the following medical conditions: * Significant cardiovascular diseases (N.B. uncomplicated hypertension is not exclusionary) * Severe liver disease, including advanced fibrosis (e.g., fibrosis score F3-F4 by vibration-controlled transient elastography) and cirrhosis * Psychiatric diseases causing functional impairment that: (1) Are or have been decompensated within 1 year of screening, and/or (2) Require use of anti-dopaminergic antipsychotic drugs associated with significant weight gain/metabolic dysfunction (e.g., clozapine, olanzapine) * Venous thromboembolic disease (deep vein thrombosis or pulmonary embolism) or any required use of therapeutic anticoagulation * Bleeding disorders, including due to anticoagulation, or significant anemia (see above) * Active malignancy, or hormonally active benign neoplasm, except allowances for non-melanoma skin cancer and differentiated thyroid cancer (Stage I only) 7. Clinical concern for increased risk of volume overload, including due to medications and/or heart/liver/kidney problems, as listed above 8. Use of oral or parenteral corticosteroids (at greater than prednisone 5 mg daily, or equivalent) for more than 3 days within the previous 30 days; topical and inhaled formulations are permitted 9. History of certain weight-loss (bariatric) surgery, including: * Roux-en-Y gastric bypass * Biliopancreatic diversion * Restrictive procedures (lap band, sleeve gastrectomy) performed within the past 6 months 10. Clinical concern for alcohol overuse based on chart review and/or by recruit's report of more than 14 standard drinks per week for males or more than 7 standard drinks per week for females 11. Regular use of tobacco, either daily or an average of at least 1 cigarette per day, and/or nicotine vaping more than 1 day per week 12. Clinical concern for use of illicit drugs other than marijuana or lawfully prescribed medications based on recruit's report, chart review, and point-of-care urine drug test at screening 13. History of or ongoing febrile illness within 30 days of screening 14. Any other disease or condition or laboratory value that, in the opinion of the investigator, would place the participant at an unacceptable risk and/or interfere with the analysis of study data. 15. Known allergy/hypersensitivity to any component of the medicinal product formulations (including soy, cow dairy, or gluten), other biologics, venipuncture materials, plastics, adhesive or silicone, or ongoing clinically important allergy/hypersensitivity as judged by the investigator. 16. Dietary restrictions (e.g., vegan, kosher, halal) on gelatin present in overencapsulation 17. Concurrent enrollment in another clinical study of any investigational drug/biologic therapy within 6 months prior to screening or within 5 half-lives of an investigational agent or biologic, whichever is longer. * Prior participation in other studies led by Dr. Cook (PI) is excluded from this prohibition according to his medical/scientific judgment.

Study sites (1)

NY