A Phase 2 interventional study of Metreleptin in Diabetes, Lipodystrophy and Hyperlipidemia, 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 6 Months to 98 Years. Per ClinicalTrials.gov, last updated 2026-11-09.
Sponsored by National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) · Phase 2, Interventional, and Treatment
Background:
- Partial lipodystrophy can cause high blood fat levels and resistance to insulin. This can lead to health problems including diabetes. Researchers have found that the drug metreleptin improves health in people with this disease.
Objective:
- To test the safety and effectiveness of metreleptin.
Eligibility:
Design:
Leptin is an adipocyte-derived hormone that can be thought of as a signal from adipose tissue to the rest of the body conveying information about long-term nutritional status. Patients with lipodystrophy have leptin deficiency secondary to lack of adipose tissue. The combination of leptin deficiency and ectopic lipid deposition in patients with lipodystrophy leads to metabolic complications including severe insulin resistance and diabetes, hypertriglyceridemia, non-alcoholic steatohepatitis, and polycystic ovarian syndrome. Between 2000 and 2014, the NIDDK IRP conducted an open-label clinical trial of the recombinant human leptin analog, metreleptin, in patients with generalized and partial forms of lipodystrophy. This study showed that metreleptin ameliorates metabolic and endocrine abnormalities in lipodystrophy, including reducing food intake, improving insulin resistance and diabetes, reducing ectopic lipid, and normalizing reproduction. Based on these data, metreleptin was approved by the FDA in February, 2014, for patients with generalized, but not partial, lipodystrophy. Our data have shown, however, that a subgroup of patients with partial lipodystrophy do gain medical benefit from metreleptin.
The purpose of this study is twofold:
Metreleptin will be given at doses of less than or equal to 0.24 mg/kg/day, adjusted based on body weight and metabolic control. Patients will be seen approximately once per year at NIH for evaluation, and potentially less frequently for those who are medically stable and have difficulty traveling to NIH. Laboratory evaluation will be obtained more frequently by the patient s home providers as clinically indicated. The primary outcomes of the study are improvements in serum triglycerides and hemoglobin A1c levels. Secondary outcomes include measures of steatohepatitis and ectopic lipid, body composition, bone mineral density and bone mineral metabolism, and pituitary and reproductive function.
Metreleptin is supplied by Chiesi USA, Inc. Neither the NIH nor Chiesi USA, Inc. can guarantee that leptin will be available indefinitely and/or after the study ends.
8,290 studies on the registry are indexed under Diabetes Mellitus; 1,077 are open to participants now.
This study's enrollment of 29 is below the median of 80 across 6,023 interventional studies indexed under Diabetes Mellitus.
Browse Diabetes Mellitus studies →National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) is the lead sponsor of 538 studies on the registry; 55 are open to participants now.
Of its 80 completed or terminated interventional studies of FDA-regulated products, 51 (64%) have results posted.
Counted across the registry records on this site, refreshed daily.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Documented metabolic benefit from prior or current metreleptin treatment, defined as one or more of the following:
EXCLUSION CRITERIA
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
A blood test of average blood sugar over the past two to three months, given as a percentage or in mmol/mol. 6.5% (48 mmol/mol) or more is the usual threshold for diabetes, normally confirmed by a second test.
The human immunodeficiency virus, which weakens the immune system.
Many studies now accept people whose HIV is controlled on treatment, judged by the viral load and the CD4 count. Check the limits this study sets.
An allergy, or another reaction of the immune system, to a substance. “Hypersensitivity to the study drug” means a known allergic reaction to it — not every side effect a medicine can have.
HbA1c
A blood test of average blood sugar over the past two to three months, given as a percentage or in mmol/mol. 6.5% (48 mmol/mol) or more is the usual threshold for diabetes, normally confirmed by a second test.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
HIV
The human immunodeficiency virus, which weakens the immune system.
Many studies now accept people whose HIV is controlled on treatment, judged by the viral load and the CD4 count. Check the limits this study sets.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Hypersensitivity
An allergy, or another reaction of the immune system, to a substance. “Hypersensitivity to the study drug” means a known allergic reaction to it — not every side effect a medicine can have.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
leptin administered via SC injections BID
Drug: Metreleptin
A leptin analog indicated as an adjunct to diet as replacement therapy to treat the complications of leptin deficiency in patients with partial lipodystrophy.
Serum hemoglobin A1C
improvement in lab value
Time frame: every 6-12 months
Serum triglycerides
improvement in lab value
Time frame: every 6-12 months
Plan to share: No — Subject level data will be shared upon request after appropriate collaboration agreements are in place.
Started Oct 2014 (actual). Primary completion is estimated for Jul 2027, 153 months after the start.
Of 82 non-industry phase 2 interventional studies under Diabetes Mellitus, half reached primary completion within 45 months of starting (the median), counting those still running; a quarter within 21 and three quarters within 100. This study's planned 153 months is above that.
Counting every sponsor, half of 147 studies reached it within 27 months; a quarter within 12 and three quarters within 51.
How these figures are worked out, and which studies are left out of them.
From the registry record's own update history. This site started tracking changes on Sep 25, 2026; for anything earlier, see the record history on ClinicalTrials.gov ↗
This study is active, not recruiting, as verified in Oct 2026. You cannot join it, but the record below documents what was studied.
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Studies of the drug tested here: Metreleptin
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) →