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Leptin to Treat Lipodystrophy

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
National Institutes of Health Clinical Center (CC) ( National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) )
ClinicalTrials.gov Identifier:
NCT00025883
First received: October 27, 2001
Last updated: August 12, 2016
Last verified: August 2016
Results First Received: March 1, 2016  
Study Type: Interventional
Study Design: Endpoint Classification: Safety/Efficacy Study;   Intervention Model: Single Group Assignment;   Masking: Open Label;   Primary Purpose: Treatment
Condition: Lipodystrophy
Intervention: Drug: Metreleptin

  Participant Flow
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Recruitment Details
Key information relevant to the recruitment process for the overall study, such as dates of the recruitment period and locations
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Pre-Assignment Details
Significant events and approaches for the overall study following participant enrollment, but prior to group assignment
No text entered.

Reporting Groups
  Description
Metreleptin subcutaneous metreleptin injections in one to two daily doses ranging from 0.06 to 0.24 mg/kg/day.

Participant Flow:   Overall Study
    Metreleptin  
STARTED     103  
Generalized Lipodystrophy     63  
Partial Lipodystrophy     40  
COMPLETED     86  
NOT COMPLETED     17  
had atypical progeriod lipodystrophy                 4  
no baseline data                 1  
Death                 1  
Adverse Event                 1  
didn't reach 6 months of Metreleptin                 10  



  Baseline Characteristics
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Population Description
Explanation of how the number of participants for analysis was determined. Includes whether analysis was per protocol, intention to treat, or another method. Also provides relevant details such as imputation technique, as appropriate.
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Reporting Groups
  Description
Metreleptin With Generalized Lipodystrophy patients with generalized lipodystrophy with subcutaneous metreleptin injection (0.06-0.24 mg/kg/day)
Metreleptin With Patial Lipodystrophy patients with partial lipodystrophy with subcutaneous metreleptin injection (0.06-0.24 mg/kg/day)
Total Total of all reporting groups

Baseline Measures
    Metreleptin With Generalized Lipodystrophy     Metreleptin With Patial Lipodystrophy     Total  
Number of Participants  
[units: participants]
  55     31     86  
Age  
[units: years]
Mean (Standard Deviation)
  18  (12)     35  (14)     24  (15)  
Gender  
[units: participants]
     
Female     42     31     73  
Male     13     0     13  
Pediatric patients  
[units: participants]
     
aged <20 years     42     7     49  
aged >=20 years     13     24     37  
Body mass index-standard deviation score (BMI-SDS) [1]
[units: units on a scale]
Mean (Standard Deviation)
  0.26  (0.98)     0.66  (0.7)     0.41  (0.9)  
Percentage body fat [2]
[units: percentage of body fat]
Mean (Standard Deviation)
  9  (2)     22  (4)     15  (7)  
Leptin  
[units: ng/mL]
Mean (Standard Deviation)
  1.13  (0.74)     6.23  (3.96)     3.03  (3.44)  
Glucose  
[units: mg/dL]
Mean (Standard Deviation)
  180  (80)     182  (87)     181  (83)  
HbA1c  
[units: percentage of glycated hemoglobin]
Mean (Standard Deviation)
  8.4  (2.3)     8.1  (2.2)     8.3  (2.3)  
Fasting insulin  
[units: µU/mL]
Mean (Standard Deviation)
  122  (318)     82  (157)     108  (273)  
C-peptide  
[units: ng/mL]
Mean (Standard Deviation)
  5.61  (4.03)     3.56  (2.27)     4.82  (3.62)  
Antidiabetic medications per patient  
[units: antidiabetic medications]
Mean (Standard Deviation)
  1.13  (0.7)     1.79  (0.68)     1.36  (0.81)  
Insulin users  
[units: participants]
     
Yes     30     15     45  
No     24     14     38  
not known     1     2     3  
Lipid-lowering medications per patient  
[units: lipid-lowering medications]
Mean (Standard Deviation)
  0.61  (0.84)     1.07  (1.04)     0.82  (0.93)  
Total cholesterol  
[units: mg/dL]
Mean (Standard Deviation)
  214  (110)     235  (147)     222  (126)  
Triglycerides  
[units: mg/dL]
Geometric Mean (Inter-Quartile Range)
  467  
  (200 to 847)  
  483  
  (232 to 856)  
  473  
  (200 to 898)  
HDL-C [3]
[units: mg/dL]
Mean (Standard Deviation)
  29  (9)     31  (9)     30  (9)  
LDL-C [4]
[units: mg/dL]
Mean (Standard Deviation)
  104  (50)     101  (36)     103  (46)  
Vitamin A  
[units: µg/dL]
Mean (Standard Deviation)
  57  (33)     73  (20)     61  (25)  
Vitamin E  
[units: mg/dL]
Mean (Standard Deviation)
  26  (32)     34  (21)     31  (28)  
25-Hydroxyvitamin D  
[units: ng/mL]
Mean (Standard Deviation)
  16  (11)     23  (13)     19  (12)  
PT [5]
[units: sec]
Mean (Standard Deviation)
  14.2  (1.2)     13.2  (0.6)     13.8  (1.1)  
International Normalized Ratio (INR)  
[units: ratio]
Mean (Standard Deviation)
  1.10  (0.14)     0.98  (0.06)     1.05  (0.13)  
Daily total insulin units per patient  
[units: insulin units]
Mean (Standard Deviation)
  625  (1099)     278  (214)     362  (620)  
[1] Body mass index -standard deviation score (BMI-SDS) represents a measure of weight, adjusted for height, sex, and age. sample range of BMI-SDS is (-3.23, 1.91). Higher value indicates more body fat.
[2] percentage of body fat
[3] high-density lipoprotein cholesterol
[4] low-density lipoprotein cholesterol
[5] prothrombin time



  Outcome Measures
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1.  Primary:   Percentage of Glycosylated Hemoglobin at Baseline, 6 Months, and 12 Months on Treatment With Metreleptin   [ Time Frame: Baseline, 6 months, 12 months ]

2.  Primary:   Triglycerides at Baseline, 6 Months, and 12 Months on Treatment With Metreleptin   [ Time Frame: Baseline, 6 months, 12 months ]


  Serious Adverse Events


  Other Adverse Events


  Limitations and Caveats
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Limitations of the study, such as early termination leading to small numbers of participants analyzed and technical problems with measurement leading to unreliable or uninterpretable data
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  More Information
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Certain Agreements:  
All Principal Investigators ARE employed by the organization sponsoring the study.


Results Point of Contact:  
Name/Title: Dr. Phillip Gorden
Organization: National Institute of DIabetes and Digestive and Kidney Dseases, National Institutes of Health
phone: 301-402-7340
e-mail: phillipg@mail.nih.gov


Publications:
Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):

Responsible Party: National Institutes of Health Clinical Center (CC) ( National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) )
ClinicalTrials.gov Identifier: NCT00025883     History of Changes
Other Study ID Numbers: 020022
02-DK-0022 ( Other Identifier: National Institutes of Health Clinical Center )
Study First Received: October 27, 2001
Results First Received: March 1, 2016
Last Updated: August 12, 2016
Health Authority: United States: Federal Government
United States: Food and Drug Administration