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Phase 3 N=25 Randomized Triple-blind Treatment

Trial of Leptin Replacement Therapy in Patients With Lipodystrophy

Hypoleptinemia · Generalized Lipodystrophy · Partial Lipodystrophy · Insulin Resistance

Enrolled (actual)
25
Serious AEs
0.0%
Results posted
Mar 2019
Primary outcome: Primary: Fasting Serum Triglycerides — 203; 304 mg/dL — p=0.2572

Study Design & Population

Study type
Interventional
Phase
Phase 3
Interventions
Leptin (Drug); Placebo (Drug)
Age
Pediatric, Adult, Older Adult · 6+ yrs
Sex
All
Sponsor
University of Texas Southwestern Medical Center
Primary completion
Nov 2014

Outcome Measures

OutcomeResultp-value
PRIMARY
Fasting Serum Triglycerides
203; 304 0.2572
PRIMARY
HbA1c
8.1; 7.6 0.71
SECONDARY
Fasting Serum Glucose
162; 165 0.68
SECONDARY
Body Weight
62.5; 56.3 0.0256 sig

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.

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.
View full record on ClinicalTrials.gov →

Data sourced from ClinicalTrials.gov (NCT00896298). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.

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