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

This study is ongoing, but not recruiting participants.
Sponsor:
Information provided by (Responsible Party):
Bristol-Myers Squibb
ClinicalTrials.gov Identifier:
NCT00677313
First received: May 12, 2008
Last updated: July 18, 2014
Last verified: July 2014
  Purpose

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


Condition Intervention
Lipodystrophy
Drug: metreleptin

Study Type: Interventional
Study Design: Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: An Open-Label Treatment Protocol to Provide Metreleptin for the Treatment of Diabetes Mellitus and/or Hypertriglyceridemia Associated With Lipodystrophy

Resource links provided by NLM:


Further study details as provided by Bristol-Myers Squibb:

Primary Outcome Measures:
  • To provide metreleptin, an investigational medication, under a treatment protocol to subjects with lipodystrophy that is associated with diabetes mellitus and/or hypertriglyceridemia [ Time Frame: open ended ] [ Designated as safety issue: No ]

Secondary Outcome Measures:
  • To monitor the safety and tolerability of metreleptin in subjects with lipodystrophy that is associated with diabetes mellitus and/or hypertriglyceridemia [ Time Frame: open ended ] [ Designated as safety issue: No ]
  • Information on the efficacy of metreleptin as assessed by its effects on fasting triglyceride concentrations, HbA1c, and fasting glucose concentrations in subjects with lipodystrophy that is associated with diabetes mellitus and/or hypertriglyceridemia [ Time Frame: open ended ] [ Designated as safety issue: No ]

Estimated Enrollment: 41
Study Start Date: November 2008
Estimated Study Completion Date: October 2014
Estimated Primary Completion Date: October 2014 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: 1 Drug: metreleptin
metreleptin injection

  Eligibility

Ages Eligible for Study:   5 Years and older
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
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
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT00677313

Locations
United States, California
Research Site
Santa Barbara, California, United States
United States, Illinois
Research Site
Chicago, Illinois, United States
United States, Michigan
Research Site
Ann Arbor, Michigan, United States
United States, Nevada
Local Institution
Reno, Nevada, United States, 89502
United States, North Carolina
Research Site
Greenville, North Carolina, United States
Sponsors and Collaborators
Bristol-Myers Squibb
Investigators
Study Director: Bristol-Myers Squibb Bristol-Myers Squibb
  More Information

Additional Information:
No publications provided by Bristol-Myers Squibb

Additional publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
Responsible Party: Bristol-Myers Squibb
ClinicalTrials.gov Identifier: NCT00677313     History of Changes
Other Study ID Numbers: MB002-002, FHA101
Study First Received: May 12, 2008
Last Updated: July 18, 2014
Health Authority: United States: Food and Drug Administration

Keywords provided by Bristol-Myers Squibb:
metreleptin
leptin
Amylin
diabetes mellitus
hypertriglyceridemia
lipodystrophy

Additional relevant MeSH terms:
Diabetes Mellitus
Hypertriglyceridemia
Lipodystrophy
Glucose Metabolism Disorders
Metabolic Diseases
Endocrine System Diseases
Hyperlipidemias
Dyslipidemias
Lipid Metabolism Disorders
Skin Diseases, Metabolic
Skin Diseases

ClinicalTrials.gov processed this record on July 26, 2014