Expanded Access Metreleptin Study (NCT02404896) | Clinical Trial Compass
AVAILABLENot Applicable
Expanded Access Metreleptin Study
United States
Plain-language summary
Metreleptin was approved in the United States as adjunct to diet as replacement therapy to treat the complications of leptin deficiency in patients with congenital or acquired generalized lipodystrophy in February 2014. The approval was based on results obtained in 2 open-label, investigator-sponsored studies (Studies 991265 and 20010769) conducted at the National Institutes of Health (NIH) to evaluate the safety and efficacy of metreleptin treatment in patients with lipodystrophy and 1 treatment IND (FHA101/MB002-002/MB002-002) conducted by Bristol-Myers Squibb on behalf of AstraZeneca (BMS/AZ) in patients with diabetes mellitus and/or hypertriglyceridemia related to lipodystrophy. These studies enrolled patients with lipodystrophy including both generalized and partial lipodystrophy. Although the marketing authorization restricted the indication to patients with generalized lipodystrophy, meaningful clinical benefit was achieved in a subset of patients with partial lipodystrophy, and these patients from FHA101/MB002-002 form the basis of the request for ongoing treatment under expanded access.
Who can participate
Age range18 Years
SexALL
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Inclusion criteria
✓. Signed Written Informed Consent
✓. Target Population
✓. Ability to comply with visits and procedures required by program
✓. Previously enrolled in study FHA101/MB002-002
✓. Has physician-confirmed partial lipodystrophy and had evidence of benefit with metreleptin treatment based on the following metabolic criteria demonstrated within the last year of metreleptin treatment (if on treatment over 1 year) from baseline values:
✓. Age and Reproductive Status
✓. Male or female, over the age of 6 months
✓. Women of childbearing potential (WOCBP) must have a negative serum or urine pregnancy test (minimum sensitivity 25 IU/L or equivalent units of HCG) within 24 hours prior to the restart of study drug.
✕. Has been diagnosed with generalized lipodystrophy
✕. Has been diagnosed with HIV infection
✕. Has a clinically significant medical condition that could potentially affect the risk/benefit ratio for metreleptin treatment and/or the personal well-being of the patient, as judged by the primary treating physician
✕. Has known infectious liver disease
✕. Has known allergies to E. coli-derived proteins or hypersensitivity to any component of metreleptin treatment