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Trial record 12 of 31 for:    Metreleptin

Leptin to Treat Lipodystrophy

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ClinicalTrials.gov Identifier: NCT00025883
Recruitment Status : Completed
First Posted : October 29, 2001
Results First Posted : August 16, 2016
Last Update Posted : August 16, 2016
Sponsor:
Information provided by (Responsible Party):
National Institutes of Health Clinical Center (CC) ( National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) )

Study Type Interventional
Study Design Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Condition Lipodystrophy
Intervention Drug: Metreleptin
Enrollment 103
Recruitment Details  
Pre-assignment Details  
Arm/Group Title Metreleptin
Hide Arm/Group Description subcutaneous metreleptin injections in one to two daily doses ranging from 0.06 to 0.24 mg/kg/day.
Period Title: Overall Study
Started 103
Generalized Lipodystrophy 63
Partial Lipodystrophy 40
Completed 86
Not Completed 17
Reason Not Completed
had atypical progeriod lipodystrophy             4
no baseline data             1
Death             1
Adverse Event             1
didn't reach 6 months of Metreleptin             10
Arm/Group Title Metreleptin With Generalized Lipodystrophy Metreleptin With Patial Lipodystrophy Total
Hide Arm/Group Description patients with generalized lipodystrophy with subcutaneous metreleptin injection (0.06-0.24 mg/kg/day) patients with partial lipodystrophy with subcutaneous metreleptin injection (0.06-0.24 mg/kg/day) Total of all reporting groups
Overall Number of Baseline Participants 55 31 86
Hide Baseline Analysis Population Description
[Not Specified]
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 55 participants 31 participants 86 participants
18  (12) 35  (14) 24  (15)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 55 participants 31 participants 86 participants
Female
42
  76.4%
31
 100.0%
73
  84.9%
Male
13
  23.6%
0
   0.0%
13
  15.1%
Pediatric patients  
Measure Type: Number
Unit of measure:  Participants
Number Analyzed 55 participants 31 participants 86 participants
aged <20 years 42 7 49
aged >=20 years 13 24 37
Body mass index-standard deviation score (BMI-SDS)   [1] 
Mean (Standard Deviation)
Unit of measure:  Units on a scale
Number Analyzed 55 participants 31 participants 86 participants
0.26  (0.98) 0.66  (0.7) 0.41  (0.9)
[1]
Measure Description: 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] 
Mean (Standard Deviation)
Unit of measure:  Percentage of body fat
Number Analyzed 55 participants 31 participants 86 participants
9  (2) 22  (4) 15  (7)
[1]
Measure Description: percentage of body fat
Leptin  
Mean (Standard Deviation)
Unit of measure:  ng/mL
Number Analyzed 55 participants 31 participants 86 participants
1.13  (0.74) 6.23  (3.96) 3.03  (3.44)
Glucose  
Mean (Standard Deviation)
Unit of measure:  mg/dL
Number Analyzed 55 participants 31 participants 86 participants
180  (80) 182  (87) 181  (83)
HbA1c  
Mean (Standard Deviation)
Unit of measure:  Percentage of glycated hemoglobin
Number Analyzed 55 participants 31 participants 86 participants
8.4  (2.3) 8.1  (2.2) 8.3  (2.3)
Fasting insulin  
Mean (Standard Deviation)
Unit of measure:  µU/mL
Number Analyzed 55 participants 31 participants 86 participants
122  (318) 82  (157) 108  (273)
C-peptide  
Mean (Standard Deviation)
Unit of measure:  ng/mL
Number Analyzed 55 participants 31 participants 86 participants
5.61  (4.03) 3.56  (2.27) 4.82  (3.62)
Antidiabetic medications per patient  
Mean (Standard Deviation)
Unit of measure:  Antidiabetic medications
Number Analyzed 55 participants 31 participants 86 participants
1.13  (0.7) 1.79  (0.68) 1.36  (0.81)
Insulin users  
Measure Type: Number
Unit of measure:  Participants
Number Analyzed 55 participants 31 participants 86 participants
Yes 30 15 45
No 24 14 38
not known 1 2 3
Lipid-lowering medications per patient  
Mean (Standard Deviation)
Unit of measure:  Lipid-lowering medications
Number Analyzed 55 participants 31 participants 86 participants
0.61  (0.84) 1.07  (1.04) 0.82  (0.93)
Total cholesterol  
Mean (Standard Deviation)
Unit of measure:  mg/dL
Number Analyzed 55 participants 31 participants 86 participants
214  (110) 235  (147) 222  (126)
Triglycerides  
Geometric Mean (Inter-Quartile Range)
Unit of measure:  mg/dL
Number Analyzed 55 participants 31 participants 86 participants
467
(200 to 847)
483
(232 to 856)
473
(200 to 898)
HDL-C   [1] 
Mean (Standard Deviation)
Unit of measure:  mg/dL
Number Analyzed 55 participants 31 participants 86 participants
29  (9) 31  (9) 30  (9)
[1]
Measure Description: high-density lipoprotein cholesterol
LDL-C   [1] 
Mean (Standard Deviation)
Unit of measure:  mg/dL
Number Analyzed 55 participants 31 participants 86 participants
104  (50) 101  (36) 103  (46)
[1]
Measure Description: low-density lipoprotein cholesterol
Vitamin A  
Mean (Standard Deviation)
Unit of measure:  µg/dL
Number Analyzed 55 participants 31 participants 86 participants
57  (33) 73  (20) 61  (25)
Vitamin E  
Mean (Standard Deviation)
Unit of measure:  mg/dL
Number Analyzed 55 participants 31 participants 86 participants
26  (32) 34  (21) 31  (28)
25-Hydroxyvitamin D  
Mean (Standard Deviation)
Unit of measure:  ng/mL
Number Analyzed 55 participants 31 participants 86 participants
16  (11) 23  (13) 19  (12)
PT   [1] 
Mean (Standard Deviation)
Unit of measure:  Sec
Number Analyzed 55 participants 31 participants 86 participants
14.2  (1.2) 13.2  (0.6) 13.8  (1.1)
[1]
Measure Description: prothrombin time
International Normalized Ratio (INR)  
Mean (Standard Deviation)
Unit of measure:  Ratio
Number Analyzed 55 participants 31 participants 86 participants
1.10  (0.14) 0.98  (0.06) 1.05  (0.13)
Daily total insulin units per patient  
Mean (Standard Deviation)
Unit of measure:  Insulin units
Number Analyzed 55 participants 31 participants 86 participants
625  (1099) 278  (214) 362  (620)
1.Primary Outcome
Title Percentage of Glycosylated Hemoglobin at Baseline, 6 Months, and 12 Months on Treatment With Metreleptin
Hide Description Percentage of glycosylated hemoglobin at Baseline, 6 months, and 12 months on treatment with metreleptin
Time Frame Baseline, 6 months, 12 months
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Generalized Lipodystrophy (GLD) Partial Lipodystrophy (PLD)
Hide Arm/Group Description:
patients with generalized lipodystrophy (GLD) with initiation of subcutaneous metreleptin injection (0.06-0.24 mg/kg/day)
patients with partial lipodystrophy (PLD) with initiation of subcutaneous metreleptin injection (0.06-0.24 mg/kg/day)
Overall Number of Participants Analyzed 55 31
Mean (Standard Deviation)
Unit of Measure: percentage of glycated hemoglobin
Baseline 8.4  (2.3) 8.1  (2.2)
6 months 6.6  (1.7) 7.2  (1.2)
12 months 6.4  (1.5) 7.3  (1.6)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Generalized Lipodystrophy (GLD)
Comments A null hypothesis of interest is there is no significant change over three time points (baseline, 6 months, 12 months) in response to metreleptin within GLD group.
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value <0.001
Comments [Not Specified]
Method Mixed Models Analysis
Comments [Not Specified]
Show Statistical Analysis 2 Hide Statistical Analysis 2
Statistical Analysis Overview Comparison Group Selection Partial Lipodystrophy (PLD)
Comments A null hypothesis of interest is there is no significant change over three time points (baseline, 6 months, 12 months) in response to metreleptin within PLD group.
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.004
Comments [Not Specified]
Method Mixed Models Analysis
Comments [Not Specified]
2.Primary Outcome
Title Triglycerides at Baseline, 6 Months, and 12 Months on Treatment With Metreleptin
Hide Description [Not Specified]
Time Frame Baseline, 6 months, 12 months
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Generalized Lipodystrophy (GLD) Partial Lipodystrophy (PLD)
Hide Arm/Group Description:
patients with generalized lipodystrophy (GLD) with initiation of subcutaneous metreleptin injection (0.06-0.24 mg/kg/day)
patients with partial lipodystrophy (PLD) with initiation of subcutaneous metreleptin injection (0.06-0.24 mg/kg/day)
Overall Number of Participants Analyzed 55 31
Geometric Mean (Inter-Quartile Range)
Unit of Measure: mg/dL
Baseline
467
(200 to 847)
483
(232 to 856)
6 months
198
(122 to 283)
339
(211 to 530)
12 months
180
(106 to 312)
326
(175 to 478)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Generalized Lipodystrophy (GLD)
Comments Triglycerides were log transformed for analysis due to non-normal distribution. Changes in triglycedies in response to metreleptin over three time points (baseline, 6 months, 12 months) are tested within GLD group.
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.05
Comments [Not Specified]
Method Mixed Models Analysis
Comments [Not Specified]
Show Statistical Analysis 2 Hide Statistical Analysis 2
Statistical Analysis Overview Comparison Group Selection Partial Lipodystrophy (PLD)
Comments Triglycerides were log transformed for analysis due to non-normal distribution. Changes in triglycedies in response to metreleptin over three time points (baseline, 6 months, 12 months) are tested within PLD group.
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.02
Comments [Not Specified]
Method Mixed Models Analysis
Comments [Not Specified]
Time Frame 1 year
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title Metreleptin
Hide Arm/Group Description subcutaneous metreleptin injection (0.06-0.24 mg/kg/day)
All-Cause Mortality
Metreleptin
Affected / at Risk (%)
Total   --/--    
Show Serious Adverse Events Hide Serious Adverse Events
Metreleptin
Affected / at Risk (%) # Events
Total   10/103 (9.71%)    
Cardiac disorders   
Exacerbations of heart failure *  2/103 (1.94%)  5
Gastrointestinal disorders   
Abdominal pain requiring hospitalization *  1/103 (0.97%)  5
Acute exacerbation of pancreatitis *  1/103 (0.97%)  1
Osteomyelitis *  1/103 (0.97%)  1
Infections and infestations   
Severe acute bronchitis *  1/103 (0.97%)  1
Pneumonia *  2/103 (1.94%)  2
Cellulitis *  1/103 (0.97%)  2
Group B streptococcus bacteremia *  1/103 (0.97%)  1
Neoplasms benign, malignant and unspecified (incl cysts and polyps)   
Ovarian cyst requiring bilateral oophorectomy and total abdominal hysterectomy *  1/103 (0.97%)  1
Pregnancy, puerperium and perinatal conditions   
Miscarriage *  1/103 (0.97%)  1
*
Indicates events were collected by non-systematic assessment
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 4%
Metreleptin
Affected / at Risk (%) # Events
Total   64/103 (62.14%)    
Blood and lymphatic system disorders   
Anemia *  6/103 (5.83%)  6
Iron deficiency *  4/103 (3.88%)  5
Endocrine disorders   
Hypoglycemia *  10/103 (9.71%)  12
Fatigue *  7/103 (6.80%)  8
Gastrointestinal disorders   
Abdominal pain *  8/103 (7.77%)  9
Nausea *  9/103 (8.74%)  9
Constipation *  5/103 (4.85%)  5
Diarrhea *  5/103 (4.85%)  5
Decreased appetite *  4/103 (3.88%)  4
General disorders   
Anxiety *  6/103 (5.83%)  6
Insomnia *  5/103 (4.85%)  5
Headache *  4/103 (3.88%)  4
Depression *  4/103 (3.88%)  4
Infections and infestations   
Infection *  7/103 (6.80%)  12
Metabolism and nutrition disorders   
Weight loss *  12/103 (11.65%)  12
Musculoskeletal and connective tissue disorders   
Musculoskeletal pain *  15/103 (14.56%)  19
Joins pain *  4/103 (3.88%)  5
Neoplasms benign, malignant and unspecified (incl cysts and polyps)   
tumor, benign *  6/103 (5.83%)  6
*
Indicates events were collected by non-systematic assessment
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
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