Trial Outcomes & Findings for Leptin to Treat Lipodystrophy (NCT NCT00025883)
NCT ID: NCT00025883
Last Updated: 2016-08-16
Results Overview
Percentage of glycosylated hemoglobin at Baseline, 6 months, and 12 months on treatment with metreleptin
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
103 participants
Primary outcome timeframe
Baseline, 6 months, 12 months
Results posted on
2016-08-16
Participant Flow
Participant milestones
| Measure |
Metreleptin
subcutaneous metreleptin injections in one to two daily doses ranging from 0.06 to 0.24 mg/kg/day.
|
|---|---|
|
Overall Study
STARTED
|
103
|
|
Overall Study
Generalized Lipodystrophy
|
63
|
|
Overall Study
Partial Lipodystrophy
|
40
|
|
Overall Study
COMPLETED
|
86
|
|
Overall Study
NOT COMPLETED
|
17
|
Reasons for withdrawal
| Measure |
Metreleptin
subcutaneous metreleptin injections in one to two daily doses ranging from 0.06 to 0.24 mg/kg/day.
|
|---|---|
|
Overall Study
had atypical progeriod lipodystrophy
|
4
|
|
Overall Study
no baseline data
|
1
|
|
Overall Study
Death
|
1
|
|
Overall Study
Adverse Event
|
1
|
|
Overall Study
didn't reach 6 months of Metreleptin
|
10
|
Baseline Characteristics
Leptin to Treat Lipodystrophy
Baseline characteristics by cohort
| Measure |
Metreleptin With Generalized Lipodystrophy
n=55 Participants
patients with generalized lipodystrophy with subcutaneous metreleptin injection (0.06-0.24 mg/kg/day)
|
Metreleptin With Patial Lipodystrophy
n=31 Participants
patients with partial lipodystrophy with subcutaneous metreleptin injection (0.06-0.24 mg/kg/day)
|
Total
n=86 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Continuous
|
18 years
STANDARD_DEVIATION 12 • n=5 Participants
|
35 years
STANDARD_DEVIATION 14 • n=7 Participants
|
24 years
STANDARD_DEVIATION 15 • n=5 Participants
|
|
Sex: Female, Male
Female
|
42 Participants
n=5 Participants
|
31 Participants
n=7 Participants
|
73 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
13 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
13 Participants
n=5 Participants
|
|
Pediatric patients
aged <20 years
|
42 participants
n=5 Participants
|
7 participants
n=7 Participants
|
49 participants
n=5 Participants
|
|
Pediatric patients
aged >=20 years
|
13 participants
n=5 Participants
|
24 participants
n=7 Participants
|
37 participants
n=5 Participants
|
|
Body mass index-standard deviation score (BMI-SDS)
|
0.26 units on a scale
STANDARD_DEVIATION 0.98 • n=5 Participants
|
0.66 units on a scale
STANDARD_DEVIATION 0.7 • n=7 Participants
|
0.41 units on a scale
STANDARD_DEVIATION 0.9 • n=5 Participants
|
|
Percentage body fat
|
9 percentage of body fat
STANDARD_DEVIATION 2 • n=5 Participants
|
22 percentage of body fat
STANDARD_DEVIATION 4 • n=7 Participants
|
15 percentage of body fat
STANDARD_DEVIATION 7 • n=5 Participants
|
|
Leptin
|
1.13 ng/mL
STANDARD_DEVIATION 0.74 • n=5 Participants
|
6.23 ng/mL
STANDARD_DEVIATION 3.96 • n=7 Participants
|
3.03 ng/mL
STANDARD_DEVIATION 3.44 • n=5 Participants
|
|
Glucose
|
180 mg/dL
STANDARD_DEVIATION 80 • n=5 Participants
|
182 mg/dL
STANDARD_DEVIATION 87 • n=7 Participants
|
181 mg/dL
STANDARD_DEVIATION 83 • n=5 Participants
|
|
HbA1c
|
8.4 percentage of glycated hemoglobin
STANDARD_DEVIATION 2.3 • n=5 Participants
|
8.1 percentage of glycated hemoglobin
STANDARD_DEVIATION 2.2 • n=7 Participants
|
8.3 percentage of glycated hemoglobin
STANDARD_DEVIATION 2.3 • n=5 Participants
|
|
Fasting insulin
|
122 µU/mL
STANDARD_DEVIATION 318 • n=5 Participants
|
82 µU/mL
STANDARD_DEVIATION 157 • n=7 Participants
|
108 µU/mL
STANDARD_DEVIATION 273 • n=5 Participants
|
|
C-peptide
|
5.61 ng/mL
STANDARD_DEVIATION 4.03 • n=5 Participants
|
3.56 ng/mL
STANDARD_DEVIATION 2.27 • n=7 Participants
|
4.82 ng/mL
STANDARD_DEVIATION 3.62 • n=5 Participants
|
|
Antidiabetic medications per patient
|
1.13 antidiabetic medications
STANDARD_DEVIATION 0.7 • n=5 Participants
|
1.79 antidiabetic medications
STANDARD_DEVIATION 0.68 • n=7 Participants
|
1.36 antidiabetic medications
STANDARD_DEVIATION 0.81 • n=5 Participants
|
|
Insulin users
Yes
|
30 participants
n=5 Participants
|
15 participants
n=7 Participants
|
45 participants
n=5 Participants
|
|
Insulin users
No
|
24 participants
n=5 Participants
|
14 participants
n=7 Participants
|
38 participants
n=5 Participants
|
|
Insulin users
not known
|
1 participants
n=5 Participants
|
2 participants
n=7 Participants
|
3 participants
n=5 Participants
|
|
Lipid-lowering medications per patient
|
0.61 lipid-lowering medications
STANDARD_DEVIATION 0.84 • n=5 Participants
|
1.07 lipid-lowering medications
STANDARD_DEVIATION 1.04 • n=7 Participants
|
0.82 lipid-lowering medications
STANDARD_DEVIATION 0.93 • n=5 Participants
|
|
Total cholesterol
|
214 mg/dL
STANDARD_DEVIATION 110 • n=5 Participants
|
235 mg/dL
STANDARD_DEVIATION 147 • n=7 Participants
|
222 mg/dL
STANDARD_DEVIATION 126 • n=5 Participants
|
|
Triglycerides
|
467 mg/dL
n=5 Participants
|
483 mg/dL
n=7 Participants
|
473 mg/dL
n=5 Participants
|
|
HDL-C
|
29 mg/dL
STANDARD_DEVIATION 9 • n=5 Participants
|
31 mg/dL
STANDARD_DEVIATION 9 • n=7 Participants
|
30 mg/dL
STANDARD_DEVIATION 9 • n=5 Participants
|
|
LDL-C
|
104 mg/dL
STANDARD_DEVIATION 50 • n=5 Participants
|
101 mg/dL
STANDARD_DEVIATION 36 • n=7 Participants
|
103 mg/dL
STANDARD_DEVIATION 46 • n=5 Participants
|
|
Vitamin A
|
57 µg/dL
STANDARD_DEVIATION 33 • n=5 Participants
|
73 µg/dL
STANDARD_DEVIATION 20 • n=7 Participants
|
61 µg/dL
STANDARD_DEVIATION 25 • n=5 Participants
|
|
Vitamin E
|
26 mg/dL
STANDARD_DEVIATION 32 • n=5 Participants
|
34 mg/dL
STANDARD_DEVIATION 21 • n=7 Participants
|
31 mg/dL
STANDARD_DEVIATION 28 • n=5 Participants
|
|
25-Hydroxyvitamin D
|
16 ng/mL
STANDARD_DEVIATION 11 • n=5 Participants
|
23 ng/mL
STANDARD_DEVIATION 13 • n=7 Participants
|
19 ng/mL
STANDARD_DEVIATION 12 • n=5 Participants
|
|
PT
|
14.2 sec
STANDARD_DEVIATION 1.2 • n=5 Participants
|
13.2 sec
STANDARD_DEVIATION 0.6 • n=7 Participants
|
13.8 sec
STANDARD_DEVIATION 1.1 • n=5 Participants
|
|
International Normalized Ratio (INR)
|
1.10 ratio
STANDARD_DEVIATION 0.14 • n=5 Participants
|
0.98 ratio
STANDARD_DEVIATION 0.06 • n=7 Participants
|
1.05 ratio
STANDARD_DEVIATION 0.13 • n=5 Participants
|
|
Daily total insulin units per patient
|
625 insulin units
STANDARD_DEVIATION 1099 • n=5 Participants
|
278 insulin units
STANDARD_DEVIATION 214 • n=7 Participants
|
362 insulin units
STANDARD_DEVIATION 620 • n=5 Participants
|
PRIMARY outcome
Timeframe: Baseline, 6 months, 12 monthsPercentage of glycosylated hemoglobin at Baseline, 6 months, and 12 months on treatment with metreleptin
Outcome measures
| Measure |
Generalized Lipodystrophy (GLD)
n=55 Participants
patients with generalized lipodystrophy (GLD) with initiation of subcutaneous metreleptin injection (0.06-0.24 mg/kg/day)
|
Partial Lipodystrophy (PLD)
n=31 Participants
patients with partial lipodystrophy (PLD) with initiation of subcutaneous metreleptin injection (0.06-0.24 mg/kg/day)
|
|---|---|---|
|
Percentage of Glycosylated Hemoglobin at Baseline, 6 Months, and 12 Months on Treatment With Metreleptin
Baseline
|
8.4 percentage of glycated hemoglobin
Standard Deviation 2.3
|
8.1 percentage of glycated hemoglobin
Standard Deviation 2.2
|
|
Percentage of Glycosylated Hemoglobin at Baseline, 6 Months, and 12 Months on Treatment With Metreleptin
6 months
|
6.6 percentage of glycated hemoglobin
Standard Deviation 1.7
|
7.2 percentage of glycated hemoglobin
Standard Deviation 1.2
|
|
Percentage of Glycosylated Hemoglobin at Baseline, 6 Months, and 12 Months on Treatment With Metreleptin
12 months
|
6.4 percentage of glycated hemoglobin
Standard Deviation 1.5
|
7.3 percentage of glycated hemoglobin
Standard Deviation 1.6
|
PRIMARY outcome
Timeframe: Baseline, 6 months, 12 monthsOutcome measures
| Measure |
Generalized Lipodystrophy (GLD)
n=55 Participants
patients with generalized lipodystrophy (GLD) with initiation of subcutaneous metreleptin injection (0.06-0.24 mg/kg/day)
|
Partial Lipodystrophy (PLD)
n=31 Participants
patients with partial lipodystrophy (PLD) with initiation of subcutaneous metreleptin injection (0.06-0.24 mg/kg/day)
|
|---|---|---|
|
Triglycerides at Baseline, 6 Months, and 12 Months on Treatment With Metreleptin
Baseline
|
467 mg/dL
Interval 200.0 to 847.0
|
483 mg/dL
Interval 232.0 to 856.0
|
|
Triglycerides at Baseline, 6 Months, and 12 Months on Treatment With Metreleptin
6 months
|
198 mg/dL
Interval 122.0 to 283.0
|
339 mg/dL
Interval 211.0 to 530.0
|
|
Triglycerides at Baseline, 6 Months, and 12 Months on Treatment With Metreleptin
12 months
|
180 mg/dL
Interval 106.0 to 312.0
|
326 mg/dL
Interval 175.0 to 478.0
|
Adverse Events
Metreleptin
Serious events: 10 serious events
Other events: 64 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Metreleptin
n=103 participants at risk
subcutaneous metreleptin injection (0.06-0.24 mg/kg/day)
|
|---|---|
|
Gastrointestinal disorders
Abdominal pain requiring hospitalization
|
0.97%
1/103 • Number of events 5 • 1 year
|
|
Gastrointestinal disorders
Acute exacerbation of pancreatitis
|
0.97%
1/103 • Number of events 1 • 1 year
|
|
Infections and infestations
Severe acute bronchitis
|
0.97%
1/103 • Number of events 1 • 1 year
|
|
Infections and infestations
Pneumonia
|
1.9%
2/103 • Number of events 2 • 1 year
|
|
Gastrointestinal disorders
Osteomyelitis
|
0.97%
1/103 • Number of events 1 • 1 year
|
|
Infections and infestations
Cellulitis
|
0.97%
1/103 • Number of events 2 • 1 year
|
|
Infections and infestations
Group B streptococcus bacteremia
|
0.97%
1/103 • Number of events 1 • 1 year
|
|
Cardiac disorders
Exacerbations of heart failure
|
1.9%
2/103 • Number of events 5 • 1 year
|
|
Pregnancy, puerperium and perinatal conditions
Miscarriage
|
0.97%
1/103 • Number of events 1 • 1 year
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Ovarian cyst requiring bilateral oophorectomy and total abdominal hysterectomy
|
0.97%
1/103 • Number of events 1 • 1 year
|
Other adverse events
| Measure |
Metreleptin
n=103 participants at risk
subcutaneous metreleptin injection (0.06-0.24 mg/kg/day)
|
|---|---|
|
Gastrointestinal disorders
Abdominal pain
|
7.8%
8/103 • Number of events 9 • 1 year
|
|
Musculoskeletal and connective tissue disorders
Musculoskeletal pain
|
14.6%
15/103 • Number of events 19 • 1 year
|
|
Infections and infestations
Infection
|
6.8%
7/103 • Number of events 12 • 1 year
|
|
General disorders
Anxiety
|
5.8%
6/103 • Number of events 6 • 1 year
|
|
Blood and lymphatic system disorders
Anemia
|
5.8%
6/103 • Number of events 6 • 1 year
|
|
Endocrine disorders
Hypoglycemia
|
9.7%
10/103 • Number of events 12 • 1 year
|
|
Metabolism and nutrition disorders
Weight loss
|
11.7%
12/103 • Number of events 12 • 1 year
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
tumor, benign
|
5.8%
6/103 • Number of events 6 • 1 year
|
|
Endocrine disorders
Fatigue
|
6.8%
7/103 • Number of events 8 • 1 year
|
|
General disorders
Insomnia
|
4.9%
5/103 • Number of events 5 • 1 year
|
|
General disorders
Headache
|
3.9%
4/103 • Number of events 4 • 1 year
|
|
Gastrointestinal disorders
Nausea
|
8.7%
9/103 • Number of events 9 • 1 year
|
|
Gastrointestinal disorders
Constipation
|
4.9%
5/103 • Number of events 5 • 1 year
|
|
Gastrointestinal disorders
Diarrhea
|
4.9%
5/103 • Number of events 5 • 1 year
|
|
Gastrointestinal disorders
Decreased appetite
|
3.9%
4/103 • Number of events 4 • 1 year
|
|
Musculoskeletal and connective tissue disorders
Joins pain
|
3.9%
4/103 • Number of events 5 • 1 year
|
|
General disorders
Depression
|
3.9%
4/103 • Number of events 4 • 1 year
|
|
Blood and lymphatic system disorders
Iron deficiency
|
3.9%
4/103 • Number of events 5 • 1 year
|
Additional Information
Dr. Phillip Gorden
National Institute of DIabetes and Digestive and Kidney Dseases, National Institutes of Health
Phone: 301-402-7340
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place