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Leptin to Treat Lipodystrophy

This study has been completed.
Sponsor:
ClinicalTrials.gov Identifier:
NCT00025883
First Posted: October 29, 2001
Last Update Posted: August 16, 2016
The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
Information provided by (Responsible Party):
National Institutes of Health Clinical Center (CC) ( National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) )
Results First Submitted: March 1, 2016  
Study Type: Interventional
Study Design: Intervention Model: Single Group Assignment;   Masking: None (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
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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.
No text entered.

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 
Overall Participants Analyzed 
[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) 
[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.
Percentage body fat [1] 
[Units: Percentage of body fat]
Mean (Standard Deviation)
 9  (2)   22  (4)   15  (7) 
[1] percentage of body fat
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 [1] 
[Units: mg/dL]
Mean (Standard Deviation)
 29  (9)   31  (9)   30  (9) 
[1] high-density lipoprotein cholesterol
LDL-C [1] 
[Units: mg/dL]
Mean (Standard Deviation)
 104  (50)   101  (36)   103  (46) 
[1] low-density lipoprotein cholesterol
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 [1] 
[Units: Sec]
Mean (Standard Deviation)
 14.2  (1.2)   13.2  (0.6)   13.8  (1.1) 
[1] prothrombin time
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) 


  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 )
First Submitted: October 27, 2001
First Posted: October 29, 2001
Results First Submitted: March 1, 2016
Results First Posted: August 16, 2016
Last Update Posted: August 16, 2016