Compassionate Use of Metreleptin in Previously Treated People With Partial Lipodystrophy

NCT02262806 | Active Not Recruiting Phase 2 FDA Drug

• 2 other identifiers: 15000215-DK-0002
• Study Type: interventional
• Target: 29
• Locations: 1 country
• Sites: 1

Brief Summary

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:

• People age 6 months and older with partial lipodystrophy who
• have received metreleptin through NIH studies and shown improvement AND
• cannot get metreleptin other ways. 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, or it can be shipped to them inside the U.S. 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

Diabetes; Lipodystrophy; Hyperlipidemia

Keywords

Lipodystrophy; Leptin; Hypertriglyceridemia; Diabetes

Outcome Measures

Primary Outcomes (2)

• Serum hemoglobin A1C

  improvement in lab value

  every 6-12 months

• Serum triglycerides

  improvement in lab value

  every 6-12 months

Study Arms (1)

Leptin therapy

OTHER

leptin administered via SC injections BID

Drug: Metreleptin

Interventions

Metreleptin DRUG

A leptin analog indicated as an adjunct to diet as replacement therapy to treat the complications of leptin deficiency in patients with partial lipodystrophy.

Leptin therapy

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
• Partial lipodystrophy (either genetic or acquired)
• Previously or currently treated with metreleptin under NIH study 02-DK-0022 and/or NIH study 13-DK-0057.
• Documented metabolic benefit from prior or current metreleptin treatment, defined as one or more of the following:
• TG reduction greater than or equal to 30% OR
• HbA1c reduction greater than or equal to 1% OR
• Decrease in insulin requirements greater than or equal to 40% OR
• Decrease in episodes of pancreatitis OR
• Improvement in steatohepatitis OR
• Withdrawal of metreleptin led to marked worsening of metabolic parameters

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.
• Psychiatric disorder impeding competence or compliance.
• 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.

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)

• Akinci B, Oral EA, Neidert A, Rus D, Cheng WY, Thompson-Leduc P, Cheung HC, Bradt P, Foss de Freitas MC, Montenegro RM, Fernandes VO, Cochran E, Brown RJ. Comorbidities and Survival in Patients With Lipodystrophy: An International Chart Review Study. J Clin Endocrinol Metab. 2019 Nov 1;104(11):5120-5135. doi: 10.1210/jc.2018-02730.

  PMID: 31314093 BACKGROUND

• Sekizkardes H, Cochran E, Malandrino N, Garg A, Brown RJ. Efficacy of Metreleptin Treatment in Familial Partial Lipodystrophy Due to PPARG vs LMNA Pathogenic Variants. J Clin Endocrinol Metab. 2019 Aug 1;104(8):3068-3076. doi: 10.1210/jc.2018-02787.

  PMID: 31194872 DERIVED

• 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

Diabetes Mellitus; Lipodystrophy; Hyperlipidemias; Hypertriglyceridemia

Interventions

metreleptin

Condition Hierarchy (Ancestors)

Glucose Metabolism Disorders; Metabolic Diseases; Nutritional and Metabolic Diseases; Endocrine System Diseases; Skin Diseases, Metabolic; Skin Diseases; Skin and Connective Tissue Diseases; Lipid Metabolism Disorders; 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 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: NIH
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: October 9, 2014
• First Posted: October 13, 2014
• Study Start: October 14, 2014
• Primary Completion (Estimated): July 31, 2027
• Study Completion (Estimated): July 31, 2027
• Last Updated: April 20, 2026

Record last verified: 2026-03-09

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)