Human Models of Selective Insulin Resistance: Alpelisib, Part I

Trial statusRecruiting
Trial phasePhase 1
Trial typeInterventional
Biological sexAll
Age18-70
SponsorColumbia University

About this trial

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.

Eligibility criteria

This trial accepts healthy volunteers

Qualifiers

Adults aged 18-70 years

Able to understand written and spoken English and/or Spanish

For Group IS: BMI 18-25 kg/m2

For Group IR: BMI 30-45 kg/m2

Disqualifiers

Inability to provide informed consent in English or Spanish

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

Trial design

Design model

Crossover

Treatments tested in this trial

  • Alpelisib 300 mg

    Drug

    All participants will ingest one dose of alpelisib 300 mg (2 x 150-mg overencapsulated tablets) on one of two study admissions.

  • Placebo

    Drug

    All participants will ingest one dose of placebo (2 overencapuslated doses of microcrystalline cellulose) on one of two study admissions.

  • [1-13C] sodium acetate

    Drug

    All participants will receive continuous infusions of \[1-13C\] sodium acetate for up to 23 hours on both study visits in order to quantify de novo lipogenesis (DNL). (non-experimental)

  • [6,6-2H2] D-glucose

    Drug

    All participants will receive continuous infusions of \[6,6-2H2\] D-glucose for up to 15 hours on both study visits in order to quantify de novo lipogenesis (DNL). (non-experimental)

  • Mixed Meal Tolerance Test

    Diagnostic test

    All participants will undergo two serial standardized meals of Nestlé BOOST Plus on Study Day 2 over 8 hours. (Non-experimental.)

  • Nestlé BOOST Plus

    Dietary supplement

    Participants will consume standardized portions of Nestlé BOOST Plus as meal replacements on Study Day 1 and as two serial mixed meal tolerance tests on Study Day 2. (Non-experimental.)

Treatment groups

32 Participants
are divided into 4 treatment groups
Group A: Placebo then alpelisib (Insulin Sensitive group)Experimental treatment 6 interventions
Group B: Alpelisib then placebo (Insulin Sensitive group)Experimental treatment 6 interventions
Group C: Placebo then alpelisib (Insulin Resistant group)Experimental treatment 6 interventions
Group D: Alpelisib then placebo (Insulin Resistant group)Experimental treatment 6 interventions

Trial outcomes

Primary outcomes

1

Hepatic de novo lipogenesis (DNL) (absolute value)

Percent incorporation of newly synthesized fatty acids into plasma or VLDL TG. During both inpatient (overnight) study visits, starting after investigational agent dose. units: %

Time frame
Up to 24 hours after dosing
2

Hepatic de novo lipogenesis (DNL) (relative value)

Percent incorporation of newly synthesized fatty acids into plasma or VLDL TG. During both inpatient (overnight) study visits, starting after investigational agent dose. unit: fold difference and/or ∆% versus other group

Time frame
Up to 24 hours after dosing
3

Endogenous glucose production (EGP) (absolute value)

Calculated from D2G tracer enrichment by the Steele equations. During both inpatient (overnight) study visits, starting after investigational agent dose. units: mg/kg/min

Time frame
Up to 15 hours after dosing
4

Endogenous glucose production (EGP) (relative value)

Calculated from D2G tracer enrichment by the Steele equations. During both inpatient (overnight) study visits, starting after investigational agent dose. units: fold difference and/or ∆% versus other group

Time frame
Up to 15 hours after dosing

Secondary outcomes

1

Serum insulin level

Serum insulin levels in response to placebo vs alpelisib following an overnight fast and then hourly during refeeding. During Study Day 2 of both study visits, starting after investigational agent dose. units: µIU/mL

Time frame
Approximately 11-19 hours after dosing
2

Plasma glucose level

Plasma glucose levels in response to placebo vs alpelisib following an overnight fast and then hourly during refeeding. During Study Day 2 of both study visits, starting after investigational agent dose. units: mg/dL

Time frame
Approximately 11-19 hours after dosing
3

Triglycerides level

(Serum or plasma) triglyceride levels in response to placebo vs alpelisib following an overnight fast and then hourly during refeeding. During Study Day 2 of both study visits, starting after investigational agent dose. units: mg/dL

Time frame
Approximately 11-19 hours after dosing
4

Free fatty acids level

(Serum or plasma) triglyceride levels in response to placebo vs alpelisib following an overnight fast and then periodically during refeeding. During Study Day 2 of both study visits, starting after investigational agent dose. units: mmol/L

Time frame
Approximately 11-19 hours after dosing

Other outcomes

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

Columbia University

Lead sponsor

University of California, Berkeley

Collaborator

National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)

Collaborator

Stanford University

Collaborator