Compassionate Use of Metreleptin in Previously Treated People With Generalized Lipodystrophy

NCT02262832 | Active Not Recruiting Phase 3 FDA Drug

• 2 other identifiers: 15000315-DK-0003
• Study Type: interventional
• Target: 24
• Locations: 1 country
• Sites: 1

Brief Summary

Background: \- Generalized 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:

• People ages 6 months and older with generalized lipodystrophy who:
• have received metreleptin through NIH studies AND
• cannot get it through approved or compassionate use mechanisms in their home country. Design:
• Participants will come to NIH approximately every 6 months during year one, then every 1 2 years. Financial assistance may be available for travel within the U.S.
• At visits, participants will get a supply of metreleptin to take home for daily injections. They will have:
• plastic catheter placed in an arm vein.
• blood tests, urine collection, and physical exam.
• oral glucose tolerance test, drinking a sweet liquid.
• ultrasound of the heart, liver, uterus, and ovaries. A gel and a probe are placed on the skin and pictures are taken of the organs.
• echocardiogram, which takes pictures of the heart with sound waves.
• Resting Metabolic Rate taken. A plastic hood is worn over the head while the oxygen they breathe is measured.
• Participants will have up to 3 DEXA scan x-rays per year.
• Participants may have:
• annual bone x-rays.
• liver biopsies every few years. A needle will be inserted into the liver to obtain a small piece. Participants will sign a separate consent for this.
• Participants must be seen regularly by their local doctors and have blood tests at least every 3 6 months at home.

Conditions

Lipodystrophy; Diabetes; Hyperlipidemia

Keywords

Diabetes; Leptin; Lipodystrophy; Hypertriglyceridemia

Outcome Measures

Primary Outcomes (2)

• Serum triglycerides

  Improvements in lab value.

  every 6-12 months

• Serum hemoglobin A1C

  Improvements in lab value.

  every 6-12 months

Secondary Outcomes (4)

• Steatohepatosis

  every 12 months

• Pituitary & Reproductive Function

  every 6-12 months

• Ectopic lipid & body composition

  every 12 months

• Bone mineral density & metabolism

  every 12 months

Study Arms (1)

Leptin study drug

OTHER

Administration of study drug SQ BID

Drug: Metreleptin

Interventions

Metreleptin DRUG

administered subcutaneously 1-2 times/day

Leptin study drug

Eligibility Criteria

• Age: 6 Months - 98 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• Age greater than or equal to 6 months.
• Generalized lipodystrophy (either congenital or acquired).
• Those who cannot obtain metreleptin through approved or compassionate use mechanisms in their home country.

You may not qualify if:

• Availability of metreleptin to the patient either as an approved drug, or through local compassionate use or expanded access programs.
• Known HIV infection or HIV-associated lipodystrophy.
• Any medical condition or medication that will increase risk to the subject.
• Current alcohol or substance abuse.
• Subjects who have a known hypersensitivity to E. coli derived proteins (as leptin is derived from such proteins).

Sponsors & Collaborators

• National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK): lead

Study Sites (1)

National Institutes of Health Clinical Center

Bethesda, Maryland, 20892, United States

Location

Related Publications (3)

• Malandrino N, Reynolds JC, Brychta RJ, Chen KY, Auh S, Gharib AM, Startzell M, Cochran EK, Brown RJ. Visceral fat does not contribute to metabolic disease in lipodystrophy. Obes Sci Pract. 2019 Jan 24;5(1):75-82. doi: 10.1002/osp4.319. eCollection 2019 Feb.

  PMID: 30847226 BACKGROUND

• Meral R, Ryan BJ, Malandrino N, Jalal A, Neidert AH, Muniyappa R, Akinci B, Horowitz JF, Brown RJ, Oral EA. "Fat Shadows" From DXA for the Qualitative Assessment of Lipodystrophy: When a Picture Is Worth a Thousand Numbers. Diabetes Care. 2018 Oct;41(10):2255-2258. doi: 10.2337/dc18-0978.

  PMID: 30237235 BACKGROUND

• Brown RJ, Meehan CA, Cochran E, Rother KI, Kleiner DE, Walter M, Gorden P. Effects of Metreleptin in Pediatric Patients With Lipodystrophy. J Clin Endocrinol Metab. 2017 May 1;102(5):1511-1519. doi: 10.1210/jc.2016-3628.

  PMID: 28324110 DERIVED

Related Links

• NIH Clinical Center Detailed Web Page

MeSH Terms

Conditions

Lipodystrophy; Diabetes Mellitus; Hyperlipidemias; Hypertriglyceridemia

Interventions

metreleptin

Condition Hierarchy (Ancestors)

Skin Diseases, Metabolic; Skin Diseases; Skin and Connective Tissue Diseases; Lipid Metabolism Disorders; Metabolic Diseases; Nutritional and Metabolic Diseases; Glucose Metabolism Disorders; Endocrine System Diseases; Dyslipidemias

Study Officials

• Rebecca J Brown, M.D.

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

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: NIH
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: October 10, 2014
• First Posted: October 13, 2014
• Study Start: October 9, 2014
• Primary Completion (Estimated): July 31, 2027
• Study Completion (Estimated): July 31, 2027
• Last Updated: March 24, 2026

Record last verified: 2026-03-09

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)