An Open-Label Treatment Protocol to Provide Metreleptin for the Treatment of Diabetes Mellitus and/or Hypertriglyceridemia Associated With Lipodystrophy
2 other identifiers
interventional
41
1 country
5
Brief 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
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P25-P50 for not_applicable
Started Mar 2009
Longer than P75 for not_applicable
5 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
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Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
May 12, 2008
CompletedFirst Posted
Study publicly available on registry
May 14, 2008
CompletedStudy Start
First participant enrolled
March 1, 2009
CompletedPrimary Completion
Last participant's last visit for primary outcome
January 1, 2015
CompletedStudy Completion
Last participant's last visit for all outcomes
January 1, 2015
CompletedApril 30, 2015
February 1, 2015
5.8 years
May 12, 2008
April 8, 2015
Conditions
Keywords
Outcome Measures
Primary Outcomes (1)
To provide metreleptin, an investigational medication, under a treatment protocol to subjects with lipodystrophy that is associated with diabetes mellitus and/or hypertriglyceridemia
open ended
Secondary Outcomes (2)
To monitor the safety and tolerability of metreleptin in subjects with lipodystrophy that is associated with diabetes mellitus and/or hypertriglyceridemia
open ended
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
open ended
Study Arms (1)
1
EXPERIMENTALInterventions
Eligibility Criteria
You may qualify if:
- Is male or female ≥5 years old
- If female of childbearing potential (including perimenopausal women who have had a menstrual period within one year):
- Not breastfeeding
- Negative pregnancy test result
- 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:
- Diabetes Mellitus
- 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.
You may not qualify if:
- 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
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (5)
Research Site
Santa Barbara, California, United States
Research Site
Chicago, Illinois, United States
Research Site
Ann Arbor, Michigan, United States
Local Institution
Reno, Nevada, 89502, United States
Research Site
Greenville, North Carolina, United States
Related Publications (4)
Meral R, Malandrino N, Walter M, Neidert AH, Muniyappa R, Oral EA, Brown RJ. Endogenous Leptin Concentrations Poorly Predict Metreleptin Response in Patients With Partial Lipodystrophy. J Clin Endocrinol Metab. 2022 Mar 24;107(4):e1739-e1751. doi: 10.1210/clinem/dgab760.
PMID: 34677608DERIVEDAjluni N, Dar M, Xu J, Neidert AH, Oral EA. Efficacy and Safety of Metreleptin in Patients with Partial Lipodystrophy: Lessons from an Expanded Access Program. J Diabetes Metab. 2016 Mar;7(3):659. doi: 10.4172/2155-6156.1000659. Epub 2016 Mar 23.
PMID: 27642538DERIVEDChan JL, Koda J, Heilig JS, Cochran EK, Gorden P, Oral EA, Brown RJ. Immunogenicity associated with metreleptin treatment in patients with obesity or lipodystrophy. Clin Endocrinol (Oxf). 2016 Jul;85(1):137-49. doi: 10.1111/cen.12980. Epub 2016 Feb 2.
PMID: 26589105DERIVEDSafar Zadeh E, Lungu AO, Cochran EK, Brown RJ, Ghany MG, Heller T, Kleiner DE, Gorden P. The liver diseases of lipodystrophy: the long-term effect of leptin treatment. J Hepatol. 2013 Jul;59(1):131-7. doi: 10.1016/j.jhep.2013.02.007. Epub 2013 Feb 21.
PMID: 23439261DERIVED
Related Links
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Study Officials
- STUDY DIRECTOR
Bristol-Myers Squibb
Bristol-Myers Squibb
Study Design
- Study Type
- interventional
- Phase
- not applicable
- Allocation
- NA
- Masking
- NONE
- Purpose
- TREATMENT
- Intervention Model
- SINGLE GROUP
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
May 12, 2008
First Posted
May 14, 2008
Study Start
March 1, 2009
Primary Completion
January 1, 2015
Study Completion
January 1, 2015
Last Updated
April 30, 2015
Record last verified: 2015-02