A Phase 2 interventional study of Metreleptin in Severe Insulin Resistance, sponsored by National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK). Active, not recruiting at 1 site in United States. Open to participants aged 5 Years and older. Per ClinicalTrials.gov, last updated 2025-01-30.
Sponsored by National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) · Phase 2, Interventional, and Treatment
Study Description:
Patients with mutations of the insulin receptor have diabetes that is challenging to control with conventional therapies, leading to early morbidity and mortality. We hypothesize that recombinant leptin (metreleptin) in these patients will improve glycemia control.
Objectives:
Primary Objective: To determine if 1 year of metreleptin will improve glycemia control in patients with genetic defects of the insulin receptor. Secondary Objectives: To determine mechanisms by which metreleptin improves glycemia.
Endpoints:
Primary Endpoint: Hemoglobin A1c.
Secondary Endpoints: fasting plasma glucose, fasting insulin/C-peptide, glucose/insulin/C-peptide area under the curve during oral glucose tolerance test.
Study Population:
20 male or female patients with mutations of the insulin receptor, age (Bullet)5 years, at the NIH Clinical Center.
Description of Sites/Facilities Enrolling Participants: Description of Study Intervention:
NIH Clinical Center
Open label study of metreleptin, 0.2 mg/kg/day (max dose 0.24 mg/kg/day).
Study Description:
Patients with mutations of the insulin receptor have diabetes that is challenging to control with conventional therapies, leading to early morbidity and mortality. We hypothesize that recombinant leptin (metreleptin) in these patients will improve glycemia control.
Objectives:
Primary Objective: To determine if 1 year of metreleptin will improve glycemia control in patients with genetic defects of the insulin receptor. Secondary Objectives: To determine mechanisms by which metreleptin improves glycemia.
Endpoints:
Primary Endpoint: Hemoglobin A1c.
Secondary Endpoints: fasting plasma glucose, fasting insulin/C-peptide, glucose/insulin/C-peptide area under the curve during oral glucose tolerance test.
Study Population:
20 male or female patients with mutations of the insulin receptor, age (Bullet)5 years, at the NIH Clinical Center.
Description of Sites/Facilities Enrolling Participants: Description of Study Intervention:
NIH Clinical Center
Open label study of metreleptin, 0.2 mg/kg/day (max dose 0.24 mg/kg/day).
1,960 studies on the registry are indexed under Insulin Resistance; 306 are open to participants now.
This study's enrollment of 11 is below the median of 40 across 1,536 interventional studies indexed under Insulin Resistance.
Browse Insulin Resistance studies →National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) is the lead sponsor of 529 studies on the registry; 54 are open to participants now.
Of its 79 completed or terminated interventional studies of FDA-regulated products, 50 (63%) have results posted.
Counted across the registry records on this site, refreshed daily.
Presence of at least one of the following metabolic abnormalities:
Presence of diabetes as defined by the 2006 American Diabetes Association (ADA) criteria:
EXCLUSION CRITERIA:
300 mg of study drug administered via subcutaneous (SC) injections.
Drug: Metreleptin
Administered SC twice/day to achieve physiological concentrations that will be effective in improving the severe state of insulin resistance seen in patients with genetic defects on their insulin receptor mutation
Change in HbA1C
Change in HbA1C at month 12 from baseline.
Time frame: Change at month 12 from baseline
Change in Fasting Insulin Level
Change in fasting insulin level at month 12 from baseline
Time frame: Change at month 12 from baseline
Change in Fasting Blood Glucose
Change in fasting blood glucose at month 12 from baseline.
Time frame: Change at month 12 from baseline
| Milestone | Leptin Treatment |
|---|---|
| Started | 11 |
| Completed | 5 |
| Not completed | 6 |
Change in HbA1C at month 12 from baseline.
| percent | Leptin Treatment |
|---|---|
| Change in HbA1C | -0.336 ± 1.921 |
Change in fasting insulin level at month 12 from baseline
| mcU/mL | Leptin Treatment |
|---|---|
| Change in Fasting Insulin Level | -58.31 ± 85.21 |
Change in fasting blood glucose at month 12 from baseline.
| mg/dL | Leptin Treatment |
|---|---|
| Change in Fasting Blood Glucose | -6.91 ± 86.1 |
Collected over 1 year. Non-serious events are listed at a 0% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| Leptin Treatment | 0/11 (0%) | 5/11 (45.5%) | 7/11 (63.6%) |
| Event | Leptin Treatment |
|---|---|
| DKAEndocrine disorders | 2/11 |
| Acute kidney injuryRenal and urinary disorders | 1/11 |
| Brain lesionNervous system disorders | 1/11 |
| EdemaGeneral disorders | 1/11 |
| HyperglycemiaEndocrine disorders | 1/11 |
| papillary carcinoma of thyroidNeoplasms benign, malignant and unspecified (incl cysts and polyps) | 1/11 |
| SeizureNervous system disorders | 1/11 |
| Septic shockInfections and infestations | 1/11 |
| Urinary tract infectionInfections and infestations | 1/11 |
| Event | Leptin Treatment |
|---|---|
| Weight lossMetabolism and nutrition disorders | 3/11 |
| Dental abscessInfections and infestations | 2/11 |
| HyperglycemiaMetabolism and nutrition disorders | 2/11 |
| AcrochordonSkin and subcutaneous tissue disorders | 1/11 |
| Alcohol withdrawalPsychiatric disorders | 1/11 |
| AnxietyPsychiatric disorders | 1/11 |
| CandidiasisInfections and infestations | 1/11 |
| CataractEye disorders | 1/11 |
| Cerumen impactionEar and labyrinth disorders | 1/11 |
| CholesteatomaEar and labyrinth disorders | 1/11 |
| Age, Continuous(years) | Leptin Treatment |
|---|---|
| Mean | 13.12 ± 5.47 |
| Sex: Female, Male(Participants) | Leptin Treatment |
|---|---|
| Female | 5 |
| Male | 6 |
| Ethnicity (NIH/OMB)(Participants) | Leptin Treatment |
|---|---|
| Hispanic or Latino | 3 |
| Not Hispanic or Latino | 8 |
| Unknown or Not Reported | 0 |
| Race/Ethnicity, Customized(Participants) | Leptin Treatment |
|---|---|
| Race — African | 1 |
| Race — Asian | 1 |
| Race — Caucasian | 7 |
| Race — Filipino | 2 |
| A1c(percent) | Leptin Treatment |
|---|---|
| Mean | 10.38 ± 1.17 |
| Fasting blood glucose(mg/dL) | Leptin Treatment |
|---|---|
| Mean | 155.27 ± 71.22 |
| Fasting Insulin(mcU/mL) | Leptin Treatment |
|---|---|
| Mean | 597.26 ± 787.15 |
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National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)