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Trial of Leptin Replacement Therapy in Patients With Lipodystrophy

Primary Purpose

Hypoleptinemia, Generalized Lipodystrophy, Partial Lipodystrophy

Status
Completed
Phase
Phase 2
Locations
United States
Study Type
Interventional
Intervention
Leptin
Placebo
Sponsored by
University of Texas Southwestern Medical Center
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Hypoleptinemia focused on measuring Lipodystrophy, Insulin secretory response

Eligibility Criteria

6 Years - 70 Years (Child, Adult, Older Adult)All SexesDoes not accept healthy volunteers

Inclusion Criteria:

  • age > 6 years
  • Partial and generalized lipodystrophy
  • Serum leptin levels less than the 7th percentile of normal values reported by the 3rd National Health and Nutrition Examination survey (less than 7.0 ng/mL in females and less than 3/0 ng/mL in males)

  • Presence of at least one of the following metabolic abnormalities:

    1. Type 2 Diabetes Mellitus
    2. Fasting serum insulin >20 uU/mL
    3. Fasting serum triglycerides > 300 mg/dL
    4. Previous participation in leptin trial (Amgen 991265, GCRC 660) - for Part B of the Study.

Exclusion Criteria:

  • Known liver disease due to causes other than non-alcoholic steatohepatitis.
  • Hematocrit of less than 30%.
  • Current alcohol or substance abuse.
  • Use of anorexigenic drugs, anabolic steroids, GH and thiazolidinediones
  • Active tuberculosis
  • Psychiatric disorder impeding competence or compliance
  • Malignancies
  • HIV infection
  • Subjects who have a known hypersensitivity to E. Coli derived proteins
  • Other condition, which in the opinion of the clinical investigators would impede completion of the study.

Sites / Locations

  • UT Southwestern Medical Center

Arms of the Study

Arm 1

Arm 2

Arm Type

Active Comparator

Placebo Comparator

Arm Label

1 Leptin

2 Sugar pill

Arm Description

Active Comparator for 4 months, then for 8 months.

Placebo for 4 months, then active comparator for 8 months.

Outcomes

Primary Outcome Measures

Fasting Serum Triglycerides
HbA1c

Secondary Outcome Measures

Fasting Serum Glucose
Body Weight

Full Information

First Posted
May 8, 2009
Last Updated
March 21, 2019
Sponsor
University of Texas Southwestern Medical Center
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1. Study Identification

Unique Protocol Identification Number
NCT00896298
Brief Title
Trial of Leptin Replacement Therapy in Patients With Lipodystrophy
Official Title
Double-Blind, Placebo-Controlled Trial of Leptin Replacement Therapy in Patients With Lipodystrophy
Study Type
Interventional

2. Study Status

Record Verification Date
March 2019
Overall Recruitment Status
Completed
Study Start Date
April 2006 (undefined)
Primary Completion Date
November 2014 (Actual)
Study Completion Date
November 2014 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Principal Investigator
Name of the Sponsor
University of Texas Southwestern Medical Center

4. Oversight

Data Monitoring Committee
No

5. Study Description

Brief Summary
Lipodystrophies represent a therapeutic challenge with regards to insulin resistance, hypertriglyceridemia and fatty liver which often is coupled with significant adipose tissue loss. The purpose of the study is to examine the safety and efficacy of Leptin on subjects with lipodystrophy.
Detailed Description
The mechanism by which leptin improves glucose and lipid control is not clear. We will examine the possible mechanisms of leptin action by studying the effects of leptin administration on food intake, insulin resistance, insulin secretory response, hepatic and intramuscular triglyceride stores in a large sample of patients with lipodystrophy. Hypothesis: Leptin replacement in patients with generalized and partial lipodystrophy and hypoleptinemia will be safe and efficacious in improving the metabolic abnormalities associated with insulin resistance.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Hypoleptinemia, Generalized Lipodystrophy, Partial Lipodystrophy, Insulin Resistance
Keywords
Lipodystrophy, Insulin secretory response

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2, Phase 3
Interventional Study Model
Parallel Assignment
Masking
ParticipantCare ProviderInvestigator
Allocation
Randomized
Enrollment
25 (Actual)

8. Arms, Groups, and Interventions

Arm Title
1 Leptin
Arm Type
Active Comparator
Arm Description
Active Comparator for 4 months, then for 8 months.
Arm Title
2 Sugar pill
Arm Type
Placebo Comparator
Arm Description
Placebo for 4 months, then active comparator for 8 months.
Intervention Type
Drug
Intervention Name(s)
Leptin
Other Intervention Name(s)
Recombinant-methionyl Human Leptin., r-metHuLeptin
Intervention Description
Leptin injected subcutaneously twice a day, to be administered at 100% of the estimated replacement dose during the first month, which is a dosage of 0.03mg/kg for female children, 0.04mg/kg for adult females, and0.02 mg/kg for all males, and then at 200% subsequently, or placebo.
Intervention Type
Drug
Intervention Name(s)
Placebo
Intervention Description
Sugar pill
Primary Outcome Measure Information:
Title
Fasting Serum Triglycerides
Time Frame
4 months
Title
HbA1c
Time Frame
4 months
Secondary Outcome Measure Information:
Title
Fasting Serum Glucose
Time Frame
4 months
Title
Body Weight
Time Frame
4 months

10. Eligibility

Sex
All
Minimum Age & Unit of Time
6 Years
Maximum Age & Unit of Time
70 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: age > 6 years Partial and generalized lipodystrophy Serum leptin levels less than the 7th percentile of normal values reported by the 3rd National Health and Nutrition Examination survey (less than 7.0 ng/mL in females and less than 3/0 ng/mL in males) Presence of at least one of the following metabolic abnormalities: Type 2 Diabetes Mellitus Fasting serum insulin >20 uU/mL Fasting serum triglycerides > 300 mg/dL Previous participation in leptin trial (Amgen 991265, GCRC 660) - for Part B of the Study. Exclusion Criteria: Known liver disease due to causes other than non-alcoholic steatohepatitis. Hematocrit of less than 30%. Current alcohol or substance abuse. Use of anorexigenic drugs, anabolic steroids, GH and thiazolidinediones Active tuberculosis Psychiatric disorder impeding competence or compliance Malignancies HIV infection Subjects who have a known hypersensitivity to E. Coli derived proteins Other condition, which in the opinion of the clinical investigators would impede completion of the study.
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Abhimanyu Garg, MD
Organizational Affiliation
UT Southwestern Medical Center
Official's Role
Principal Investigator
Facility Information:
Facility Name
UT Southwestern Medical Center
City
Dallas
State/Province
Texas
ZIP/Postal Code
75390
Country
United States

12. IPD Sharing Statement

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Trial of Leptin Replacement Therapy in Patients With Lipodystrophy

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