An Open-Label Treatment Protocol to Provide Metreleptin for the Treatment of Diabetes Mellitus and/or Hypertriglyceridemia Associated With Lipodystrophy

Bristol-Myers Squibb (BMS)

Status

Completed

Conditions

Lipodystrophy

Treatments

Drug: metreleptin

Study type

Interventional

Funder types

Industry

Identifiers

NCT00677313

MB002-002

FHA101 (Other Identifier)

Details and patient eligibility

About

This is an open-label study to provide metreleptin for the treatment of diabetes mellitus and/or hypertriglyceridemia associated with lipodystrophy. This study intends to provide guidance to investigators with respect to identification of appropriate subjects for metreleptin treatment, guidance on metreleptin dosing, and collection of safety and efficacy data following metreleptin treatment in this population

Enrollment

41 patients

Sex

All

Ages

5+ years old

Volunteers

No Healthy Volunteers

Inclusion criteria

Is male or female ≥5 years old
If female of childbearing potential (including perimenopausal women who have had a menstrual period within one year):
1. Not breastfeeding
2. Negative pregnancy test result
3. Must practice and be willing to continue to practice appropriate birth control (defined as a method which results in a low failure rate, i.e., less than 1% per year, when used consistently and correctly, such as implants, injectables, oral contraceptives, some intrauterine contraceptive devices, sexual abstinence, tubal ligation, or a vasectomized partner) during the entire duration of the study (Double barrier methods including the use of female diaphragm and male condom with spermicide can also be used.)
Has physician-confirmed lipodystrophy as defined by evidence of generalized (whole body) or partial (limbs) loss of body fat outside the range of normal variation
Has been diagnosed with at least one of the following 2 metabolic disorders:
1. Diabetes Mellitus
2. Hypertriglyceridemia as defined by fasting triglyceride concentrations >200 mg/dL
If ≥18 years of age, is able to read, understand, and sign the Informed Consent Form (ICF) and an Authorization to Use and Disclose Protected Health Information form, communicate with the investigator, and understand and comply with protocol requirements
If <18 years of age, has a parent or legal guardian to read and understand the ICF and Child Assent Form, communicate with the investigator, and understand and comply with protocol requirements. Adolescent subjects must also read and understand the Child Assent Form; if the child is too young or unable to read, then the Child Assent Form must be explained to the child.

Exclusion criteria

Has been diagnosed with HIV infection
Has known infectious liver disease
Has known allergies to E. coli-derived proteins or hypersensitivity to any component of study treatment