Clinical Trial: Compassionate Use of Metreleptin in Previously Treated People With Partial Lipodystrophy

Study Status: Enrolling by invitation
Recruit Status: Enrolling by invitation
Study Type: Interventional

Official Title: Compassionate Use of Metreleptin in Previously-Treated Patients With Partial Lipodystrophy

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

    Detailed Summary:

    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:

    • To provide access to metreleptin to patients with partial lipodystrophy who have previously received and derived benefit from metreleptin through NIH studies (protocols 02-DK-0022 and 13-DK-0057).
    • To continue to collect data on the long-term efficacy of metreleptin in ameliorating the metabolic complications of partial lipodystrophy.

    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. Patie
    Sponsor: National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)

    Current Primary Outcome:

    • Serum hemoglobin A1C [ Time Frame: Annually ]
    • Serum triglycerides [ Time Frame: Annually ]


    Original Primary Outcome: Same as current

    Current Secondary Outcome:

    • Bone mineral density & metabolism [ Time Frame: Annually ]
    • Ectopic lipid & body composition [ Time Frame: Annually ]
    • Steatohepatosis [ Time Frame: Annually ]
    • Pituitary & Reproductive Function [ Time Frame: Annually ]


    Original Secondary Outcome:

    • Bone mineral density & amp; metabolism [ Time Frame: Annually ]
    • Ectopic lipid & amp; body composition [ Time Frame: Annually ]
    • Steatohepatosis [ Time Frame: Annually ]
    • Pituitary & amp; Reproductive Function [ Time Frame: Annually ]


    Information By: National Institutes of Health Clinical Center (CC)

    Dates:
    Date Received: October 9, 2014
    Date Started: October 6, 2014
    Date Completion: July 31, 2018
    Last Updated: May 12, 2017
    Last Verified: January 12, 2017