Trial Outcomes & Findings for Short-term Effects of Leptin in People With Lipodystrophy (NCT NCT01778556)
NCT ID: NCT01778556
Last Updated: 2019-11-13
Results Overview
Total body insulin sensitivity (measured as glucose disposal rate during a hyperinsulinemic, euglycemic clamp)
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
25 participants
Primary outcome timeframe
Intervention 1 (5 days), Intervention 2 (14 days), and Long-term follow-up (6 months)
Results posted on
2019-11-13
Participant Flow
Participant milestones
| Measure |
Leptin Naive
Studied for 5 days without metreleptin, then 14 days while taking metreleptin
Metreleptin: Recombinant analog of the human hormone, leptin
|
On-leptin
Studied for 5 days while taking metreleptin, then 14 days during metreleptin withdrawal
Metreleptin: Recombinant analog of the human hormone, leptin
|
|---|---|---|
|
Intervention 1 (5 Days)
STARTED
|
15
|
10
|
|
Intervention 1 (5 Days)
COMPLETED
|
15
|
8
|
|
Intervention 1 (5 Days)
NOT COMPLETED
|
0
|
2
|
|
Intervention 2 (14 Days)
STARTED
|
15
|
8
|
|
Intervention 2 (14 Days)
COMPLETED
|
14
|
8
|
|
Intervention 2 (14 Days)
NOT COMPLETED
|
1
|
0
|
|
Long-term Follow-up
STARTED
|
15
|
0
|
|
Long-term Follow-up
COMPLETED
|
14
|
0
|
|
Long-term Follow-up
NOT COMPLETED
|
1
|
0
|
Reasons for withdrawal
| Measure |
Leptin Naive
Studied for 5 days without metreleptin, then 14 days while taking metreleptin
Metreleptin: Recombinant analog of the human hormone, leptin
|
On-leptin
Studied for 5 days while taking metreleptin, then 14 days during metreleptin withdrawal
Metreleptin: Recombinant analog of the human hormone, leptin
|
|---|---|---|
|
Intervention 1 (5 Days)
Protocol Violation
|
0
|
1
|
|
Intervention 1 (5 Days)
Withdrawal by Subject
|
0
|
1
|
|
Intervention 2 (14 Days)
Protocol Violation
|
1
|
0
|
|
Long-term Follow-up
noncompliance with metreleptin
|
1
|
0
|
Baseline Characteristics
Short-term Effects of Leptin in People With Lipodystrophy
Baseline characteristics by cohort
| Measure |
Leptin Naive
n=15 Participants
Studied for 5 days without metreleptin, then 14 days while taking metreleptin
Metreleptin: Recombinant analog of the human hormone, leptin
|
On-leptin
n=8 Participants
Studied for 5 days while taking metreleptin, then 14 days during metreleptin withdrawal
Metreleptin: Recombinant analog of the human hormone, leptin
|
Total
n=23 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Continuous
|
32 years
STANDARD_DEVIATION 17 • n=5 Participants
|
25 years
STANDARD_DEVIATION 6 • n=7 Participants
|
28 years
STANDARD_DEVIATION 14 • n=5 Participants
|
|
Sex: Female, Male
Female
|
12 Participants
n=5 Participants
|
5 Participants
n=7 Participants
|
17 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
3 Participants
n=5 Participants
|
3 Participants
n=7 Participants
|
6 Participants
n=5 Participants
|
|
Race/Ethnicity, Customized
White
|
9 Participants
n=5 Participants
|
4 Participants
n=7 Participants
|
13 Participants
n=5 Participants
|
|
Race/Ethnicity, Customized
Hispanic
|
4 Participants
n=5 Participants
|
2 Participants
n=7 Participants
|
6 Participants
n=5 Participants
|
|
Race/Ethnicity, Customized
African American
|
0 Participants
n=5 Participants
|
2 Participants
n=7 Participants
|
2 Participants
n=5 Participants
|
|
Race/Ethnicity, Customized
Asian
|
1 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
|
Race/Ethnicity, Customized
Other
|
1 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
|
Type of lipodystrophy
Generalized
|
3 Participants
n=5 Participants
|
8 Participants
n=7 Participants
|
11 Participants
n=5 Participants
|
|
Type of lipodystrophy
Partial
|
12 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
12 Participants
n=5 Participants
|
|
Subtype of lipodystrophy
Congenital generalized lipodystrophy
|
3 Participants
n=5 Participants
|
7 Participants
n=7 Participants
|
10 Participants
n=5 Participants
|
|
Subtype of lipodystrophy
Familial partial lipodystrophy
|
12 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
12 Participants
n=5 Participants
|
|
Subtype of lipodystrophy
Acquired generalized lipodystrophy
|
0 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
|
Endogenous leptin level
|
9.5 ng/dl
STANDARD_DEVIATION 10.2 • n=5 Participants
|
1.2 ng/dl
STANDARD_DEVIATION 0.5 • n=7 Participants
|
7.4 ng/dl
STANDARD_DEVIATION 10.1 • n=5 Participants
|
PRIMARY outcome
Timeframe: Intervention 1 (5 days), Intervention 2 (14 days), and Long-term follow-up (6 months)Population: One patient at Period 2 (14 days) in Leptin naive arm was withdrew due to protocol violation.
Total body insulin sensitivity (measured as glucose disposal rate during a hyperinsulinemic, euglycemic clamp)
Outcome measures
| Measure |
Leptin Naive
n=15 Participants
Studied for 5 days without metreleptin, then 14 days while taking metreleptin
Metreleptin: Recombinant analog of the human hormone, leptin
|
On-leptin
n=8 Participants
Studied for 5 days while taking metreleptin, then 14 days during metreleptin withdrawal
Metreleptin: Recombinant analog of the human hormone, leptin
|
|---|---|---|
|
Total Body Insulin Sensitivity
Intervention 1 (5 days)
|
4.4 mg/kg fat-free mass/min
Standard Deviation 2.3
|
10.9 mg/kg fat-free mass/min
Standard Deviation 4.1
|
|
Total Body Insulin Sensitivity
Intervention 2 (14 days)
|
5.8 mg/kg fat-free mass/min
Standard Deviation 2.2
|
6.4 mg/kg fat-free mass/min
Standard Deviation 1.8
|
|
Total Body Insulin Sensitivity
Long-term follow-up (6 months)
|
8.0 mg/kg fat-free mass/min
Standard Deviation 4.0
|
—
|
SECONDARY outcome
Timeframe: Intervention 1 (5 days), Intervention 2 (14 days), and Long-term follow-up (6 months)Population: Measurements were not available.
Hepatic insulin sensitivity (measured as suppression of endogenous glucose production during a hyperinsulinemic, euglycemic clamp)
Outcome measures
| Measure |
Leptin Naive
n=14 Participants
Studied for 5 days without metreleptin, then 14 days while taking metreleptin
Metreleptin: Recombinant analog of the human hormone, leptin
|
On-leptin
n=8 Participants
Studied for 5 days while taking metreleptin, then 14 days during metreleptin withdrawal
Metreleptin: Recombinant analog of the human hormone, leptin
|
|---|---|---|
|
Insulin-mediated Suppression of Hepatic Glucose Production
Intervention 1 (5 days)
|
61.2 percentage
Standard Deviation 23.4
|
77.1 percentage
Standard Deviation 42.7
|
|
Insulin-mediated Suppression of Hepatic Glucose Production
Intervention 2 (14 days)
|
75.2 percentage
Standard Deviation 33.1
|
63.8 percentage
Standard Deviation 31.7
|
|
Insulin-mediated Suppression of Hepatic Glucose Production
Long-term follow-up (6 months)
|
85.6 percentage
Standard Deviation 17.9
|
—
|
SECONDARY outcome
Timeframe: Intervention 1 (5 days), Intervention 2 (14 days), and Long-term follow-up (6 months)Population: Measurements were not available.
Endogenous Rate of Appearance of Palmitate is measured in plasma.
Outcome measures
| Measure |
Leptin Naive
n=14 Participants
Studied for 5 days without metreleptin, then 14 days while taking metreleptin
Metreleptin: Recombinant analog of the human hormone, leptin
|
On-leptin
n=8 Participants
Studied for 5 days while taking metreleptin, then 14 days during metreleptin withdrawal
Metreleptin: Recombinant analog of the human hormone, leptin
|
|---|---|---|
|
Endogenous Rate of Appearance of Palmitate
Long-term follow-up (6 months)
|
2.2 μmol/kg fat-free mass/min
Standard Deviation 0.7
|
—
|
|
Endogenous Rate of Appearance of Palmitate
Intervention 1 (5 days)
|
3.2 μmol/kg fat-free mass/min
Standard Deviation 1.3
|
1.4 μmol/kg fat-free mass/min
Standard Deviation 0.7
|
|
Endogenous Rate of Appearance of Palmitate
Intervention 2 (14 days)
|
2.8 μmol/kg fat-free mass/min
Standard Deviation 1.0
|
1.7 μmol/kg fat-free mass/min
Standard Deviation 0.6
|
Adverse Events
Intervention 1 (5 Days) : Leptin Naive
Serious events: 0 serious events
Other events: 0 other events
Deaths: 0 deaths
Intervention 2 (14 Days): Leptin Naive
Serious events: 1 serious events
Other events: 2 other events
Deaths: 0 deaths
Long-term Follow-up (6 Months): Leptin Naive
Serious events: 2 serious events
Other events: 4 other events
Deaths: 0 deaths
Intervention 1 ( 5 Days) : On-Leptin
Serious events: 0 serious events
Other events: 0 other events
Deaths: 0 deaths
Intervention 1 ( 14 Days): On-Leptin
Serious events: 0 serious events
Other events: 0 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Intervention 1 (5 Days) : Leptin Naive
n=15 participants at risk
Studied for 5 days without taking metreleptin.
Metreleptin: Recombinant analog of the human hormone, leptin
|
Intervention 2 (14 Days): Leptin Naive
n=15 participants at risk
Studied for 14 days while taking metreleptin after the intervention 1 (5 days) : Leptin Naive Metreleptin: Recombinant analog of the human hormone, leptin
|
Long-term Follow-up (6 Months): Leptin Naive
n=15 participants at risk
Studied for 6 months while taking metreleptin after intervention 2 (14 days): Leptin Naive
Metreleptin: Recombinant analog of the human hormone, leptin
|
Intervention 1 ( 5 Days) : On-Leptin
n=8 participants at risk
Studied for 5 days while taking metreleptin
Metreleptin: Recombinant analog of the human hormone, leptin
|
Intervention 1 ( 14 Days): On-Leptin
n=8 participants at risk
Studied for 14 days during metreleptin withdrawal after Intervention 1 ( 5 days) : On-Leptin
Metreleptin: Recombinant analog of the human hormone, leptin
|
|---|---|---|---|---|---|
|
Gastrointestinal disorders
abdominal pain of unknown etiology
|
0.00%
0/15 • 6 months
|
0.00%
0/15 • 6 months
|
6.7%
1/15 • Number of events 1 • 6 months
|
0.00%
0/8 • 6 months
|
0.00%
0/8 • 6 months
|
|
Cardiac disorders
angioedema secondary to angiotensin- converting enzyme inhibitor use
|
0.00%
0/15 • 6 months
|
0.00%
0/15 • 6 months
|
6.7%
1/15 • Number of events 1 • 6 months
|
0.00%
0/8 • 6 months
|
0.00%
0/8 • 6 months
|
|
Endocrine disorders
anemia secondary to menorrhagia
|
0.00%
0/15 • 6 months
|
6.7%
1/15 • Number of events 4 • 6 months
|
0.00%
0/15 • 6 months
|
0.00%
0/8 • 6 months
|
0.00%
0/8 • 6 months
|
Other adverse events
| Measure |
Intervention 1 (5 Days) : Leptin Naive
n=15 participants at risk
Studied for 5 days without taking metreleptin.
Metreleptin: Recombinant analog of the human hormone, leptin
|
Intervention 2 (14 Days): Leptin Naive
n=15 participants at risk
Studied for 14 days while taking metreleptin after the intervention 1 (5 days) : Leptin Naive Metreleptin: Recombinant analog of the human hormone, leptin
|
Long-term Follow-up (6 Months): Leptin Naive
n=15 participants at risk
Studied for 6 months while taking metreleptin after intervention 2 (14 days): Leptin Naive
Metreleptin: Recombinant analog of the human hormone, leptin
|
Intervention 1 ( 5 Days) : On-Leptin
n=8 participants at risk
Studied for 5 days while taking metreleptin
Metreleptin: Recombinant analog of the human hormone, leptin
|
Intervention 1 ( 14 Days): On-Leptin
n=8 participants at risk
Studied for 14 days during metreleptin withdrawal after Intervention 1 ( 5 days) : On-Leptin
Metreleptin: Recombinant analog of the human hormone, leptin
|
|---|---|---|---|---|---|
|
Metabolism and nutrition disorders
decreased appetite
|
0.00%
0/15 • 6 months
|
0.00%
0/15 • 6 months
|
6.7%
1/15 • Number of events 1 • 6 months
|
0.00%
0/8 • 6 months
|
0.00%
0/8 • 6 months
|
|
Metabolism and nutrition disorders
weight loss
|
0.00%
0/15 • 6 months
|
0.00%
0/15 • 6 months
|
6.7%
1/15 • Number of events 1 • 6 months
|
0.00%
0/8 • 6 months
|
0.00%
0/8 • 6 months
|
|
Skin and subcutaneous tissue disorders
hair loss
|
0.00%
0/15 • 6 months
|
0.00%
0/15 • 6 months
|
6.7%
1/15 • Number of events 1 • 6 months
|
0.00%
0/8 • 6 months
|
0.00%
0/8 • 6 months
|
|
Metabolism and nutrition disorders
hypoglycemia (in a subject treated with insulin)
|
0.00%
0/15 • 6 months
|
0.00%
0/15 • 6 months
|
6.7%
1/15 • Number of events 1 • 6 months
|
0.00%
0/8 • 6 months
|
0.00%
0/8 • 6 months
|
|
Injury, poisoning and procedural complications
injection site reaction
|
0.00%
0/15 • 6 months
|
6.7%
1/15 • Number of events 1 • 6 months
|
0.00%
0/15 • 6 months
|
0.00%
0/8 • 6 months
|
0.00%
0/8 • 6 months
|
|
Endocrine disorders
menorrhagia
|
0.00%
0/15 • 6 months
|
6.7%
1/15 • Number of events 1 • 6 months
|
0.00%
0/15 • 6 months
|
0.00%
0/8 • 6 months
|
0.00%
0/8 • 6 months
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place