Back to results

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

Primary Purpose

Lipodystrophy

Status

Completed

Phase

Not Applicable

Locations

United States

Study Type

Interventional

Intervention

metreleptin

About this trial

This is an interventional treatment trial for Lipodystrophy focused on measuring metreleptin, leptin, Amylin, diabetes mellitus, hypertriglyceridemia, lipodystrophy

Eligibility Criteria

5 Years - undefined (Child, Adult, Older Adult)All SexesDoes not accept 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

Sites / Locations

Research Site
Research Site
Research Site
Local Institution
Research Site

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Arm Description

Outcomes

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

Secondary Outcome Measures

To monitor the safety and tolerability of metreleptin in subjects with lipodystrophy that is associated with diabetes mellitus and/or hypertriglyceridemia

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

Full Information

NCT ID

NCT00677313

First Posted

May 12, 2008

Last Updated

April 8, 2015

Sponsor

Bristol-Myers Squibb

1. Study Identification

Unique Protocol Identification Number

NCT00677313

Brief Title

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

Official Title

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

Study Type

Interventional

2. Study Status

Record Verification Date

February 2015

Overall Recruitment Status

Completed

Study Start Date

March 2009 (undefined)

Primary Completion Date

January 2015 (Actual)

Study Completion Date

January 2015 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Sponsor

Name of the Sponsor

Bristol-Myers Squibb

4. Oversight

5. Study Description

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

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Lipodystrophy

Keywords

metreleptin, leptin, Amylin, diabetes mellitus, hypertriglyceridemia, lipodystrophy

7. Study Design

Primary Purpose

Treatment

Study Phase

Not Applicable

Interventional Study Model

Single Group Assignment

Masking

None (Open Label)

Allocation

N/A

Enrollment

41 (Actual)

8. Arms, Groups, and Interventions

Arm Title

Arm Type

Experimental

Intervention Type

Drug

Intervention Name(s)

metreleptin

Intervention Description

metreleptin injection

Primary Outcome Measure Information:

Title

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

Secondary Outcome Measure Information:

Title

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

Title

Time Frame

open ended

10. Eligibility

Sex

All

Minimum Age & Unit of Time

5 Years

Accepts Healthy Volunteers

Eligibility 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): 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. 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

Overall Study Officials:

First Name & Middle Initial & Last Name & Degree

Bristol-Myers Squibb

Organizational Affiliation

Bristol-Myers Squibb

Official's Role

Study Director

Facility Information:

Facility Name

Research Site

City

Santa Barbara

State/Province

California

Country

United States

Facility Name

Research Site

City

Chicago

State/Province

Illinois

Country

United States

Facility Name

Research Site

City

Ann Arbor

State/Province

Michigan

Country

United States

Facility Name

Local Institution

City

Reno

State/Province

Nevada

ZIP/Postal Code

89502

Country

United States

Facility Name

Research Site

City

Greenville

State/Province

North Carolina

Country

United States

12. IPD Sharing Statement

Citations:

PubMed Identifier

34677608

Citation

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.

Results Reference

derived

PubMed Identifier

27642538

Citation

Ajluni 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.

Results Reference

derived

PubMed Identifier

26589105

Citation

Chan 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.

Results Reference

derived

PubMed Identifier

23439261

Citation

Safar 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.

Results Reference

derived

Learn more about this trial

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

We'll reach out to this number within 24 hrs