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

• Status: Completed
• Trial end date: 2015-01-01
• Target enrollment: 0
• Participant gender: All

Summary

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

• Phase: N/A
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Bristol-Myers Squibb

Criteria

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