Expanded Access Metreleptin Study

NCT02404896 | Unknown

• 1 other identifier: HUM00094843
• Study Type: expanded_access
• Target: N/A
• Locations: 1 country
• Sites: 1

Brief 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.

Conditions

Familial Partial Lipodystrophy

Interventions

Metreleptin DRUG

Metreleptin open-label treatment

Also known as: MyaLept

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Signed Written Informed Consent
• a) Before any program procedures are performed, the details of the program will be described to the patient and the patient will be given a written informed consent document to read. If the patient agrees to participate in the program, consent will be indicated by signing and dating of the informed consent document in the presence of program personnel.
• 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:
• TG reduction ≥ 30% OR
• HbA1c reduction ≥ 1% OR
• Decrease in insulin requirements ≥ 40% OR
• Decrease in episodes of pancreatitis OR
• Improvement in steatohepatitis OR
• Withdrawal of metreleptin led to marked worsening of metabolic parameters
• 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.

You may not qualify if:

• Target Disease Exceptions
• a) Has acquired lipodystrophy and clinically significant hematologic abnormalities (such as neutropenia and/or lymphadenopathy)
• Medical History and Concurrent Diseases
• 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
• Prisoners or patients who are involuntarily incarcerated.
• Patients who are compulsorily detained for treatment of either a psychiatric or physical (eg, infectious disease) illness.

Sponsors & Collaborators

• University of Michigan: lead

Study Sites (1)

University of Michigan

Ann Arbor, Michigan, 48105, United States

AVAILABLE

MeSH Terms

Conditions

Lipodystrophy, Familial Partial

Interventions

metreleptin

Condition Hierarchy (Ancestors)

Laminopathies; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Lipodystrophy; Skin Diseases, Metabolic; Skin Diseases; Skin and Connective Tissue Diseases; Lipid Metabolism, Inborn Errors; Lipid Metabolism Disorders; Metabolic Diseases; Nutritional and Metabolic Diseases

Study Officials

• Elif A Oral, MD

  University of Michigan

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Elif A Oral, MD

CONTACT

734-615-7271; eliforal@med.umich.edu

Adam H Neidert, MS

CONTACT

734-615-0539; aneidert@med.umich.edu

Study Design

• Study Type: expanded access
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Professor, Internal Medicine

Study Record Dates

• First Submitted: January 20, 2015
• First Posted: April 1, 2015
• Last Updated: February 2, 2026

Record last verified: 2026-01

Locations

US (1)