Trial Outcomes & Findings for Effect of Metreleptin Therapy in the Treatment of Severe Insulin Resistance (NCT NCT00085982)
NCT ID: NCT00085982
Last Updated: 2025-01-30
Results Overview
Change in HbA1C at month 12 from baseline.
Recruitment status
ACTIVE_NOT_RECRUITING
Study phase
PHASE2
Target enrollment
11 participants
Primary outcome timeframe
Change at month 12 from baseline
Results posted on
2025-01-30
Participant Flow
Participant milestones
| Measure |
Leptin Treatment
300 mg of study drug administered via subcutaneous (SC) injections.
Metreleptin: Administered SC twice/day to achieve physiological concentrations that will be effective in improving the severe state of insulin resistance seen in patients with genetic defects on their insulin receptor mutation
|
|---|---|
|
Overall Study
STARTED
|
11
|
|
Overall Study
COMPLETED
|
5
|
|
Overall Study
NOT COMPLETED
|
6
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Effect of Metreleptin Therapy in the Treatment of Severe Insulin Resistance
Baseline characteristics by cohort
| Measure |
Leptin Treatment
n=11 Participants
300 mg of study drug administered via SC injections.
Metreleptin: Administered SC twice/day to achieve physiological concentrations that will be effective in improving the severe state of insulin resistance seen in patients with genetic defects on their insulin receptor mutation
|
|---|---|
|
Age, Continuous
|
13.12 years
STANDARD_DEVIATION 5.47 • n=5 Participants
|
|
Sex: Female, Male
Female
|
5 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
6 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
3 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
8 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
|
Race/Ethnicity, Customized
Race · African
|
1 Participants
n=5 Participants
|
|
Race/Ethnicity, Customized
Race · Asian
|
1 Participants
n=5 Participants
|
|
Race/Ethnicity, Customized
Race · Caucasian
|
7 Participants
n=5 Participants
|
|
Race/Ethnicity, Customized
Race · Filipino
|
2 Participants
n=5 Participants
|
|
A1c
|
10.38 percent
STANDARD_DEVIATION 1.17 • n=5 Participants
|
|
Fasting blood glucose
|
155.27 mg/dL
STANDARD_DEVIATION 71.22 • n=5 Participants
|
|
Fasting Insulin
|
597.26 mcU/mL
STANDARD_DEVIATION 787.15 • n=5 Participants
|
PRIMARY outcome
Timeframe: Change at month 12 from baselineChange in HbA1C at month 12 from baseline.
Outcome measures
| Measure |
Leptin Treatment
n=11 Participants
300 mg of study drug administered via SC injections.
Metreleptin: Administered SC twice/day to achieve physiological concentrations that will be effective in improving the severe state of insulin resistance seen in patients with genetic defects on their insulin receptor mutation
|
|---|---|
|
Change in HbA1C
|
-0.336 percent
Standard Deviation 1.921
|
SECONDARY outcome
Timeframe: Change at month 12 from baselineChange in fasting insulin level at month 12 from baseline
Outcome measures
| Measure |
Leptin Treatment
n=11 Participants
300 mg of study drug administered via SC injections.
Metreleptin: Administered SC twice/day to achieve physiological concentrations that will be effective in improving the severe state of insulin resistance seen in patients with genetic defects on their insulin receptor mutation
|
|---|---|
|
Change in Fasting Insulin Level
|
-58.31 mcU/mL
Standard Deviation 85.21
|
SECONDARY outcome
Timeframe: Change at month 12 from baselineChange in fasting blood glucose at month 12 from baseline.
Outcome measures
| Measure |
Leptin Treatment
n=11 Participants
300 mg of study drug administered via SC injections.
Metreleptin: Administered SC twice/day to achieve physiological concentrations that will be effective in improving the severe state of insulin resistance seen in patients with genetic defects on their insulin receptor mutation
|
|---|---|
|
Change in Fasting Blood Glucose
|
-6.91 mg/dL
Standard Deviation 86.1
|
Adverse Events
Leptin Treatment
Serious events: 5 serious events
Other events: 7 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Leptin Treatment
n=11 participants at risk
300 mg of study drug administered via SC injections.
Metreleptin: Administered SC twice/day to achieve physiological concentrations that will be effective in improving the severe state of insulin resistance seen in patients with genetic defects on their insulin receptor mutation
|
|---|---|
|
Renal and urinary disorders
Acute kidney injury
|
9.1%
1/11 • Number of events 1 • 1 year
|
|
Nervous system disorders
Brain lesion
|
9.1%
1/11 • Number of events 1 • 1 year
|
|
Endocrine disorders
DKA
|
18.2%
2/11 • Number of events 3 • 1 year
|
|
General disorders
Edema
|
9.1%
1/11 • Number of events 1 • 1 year
|
|
Endocrine disorders
Hyperglycemia
|
9.1%
1/11 • Number of events 1 • 1 year
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
papillary carcinoma of thyroid
|
9.1%
1/11 • Number of events 1 • 1 year
|
|
Nervous system disorders
Seizure
|
9.1%
1/11 • Number of events 1 • 1 year
|
|
Infections and infestations
Septic shock
|
9.1%
1/11 • Number of events 1 • 1 year
|
|
Infections and infestations
Urinary tract infection
|
9.1%
1/11 • Number of events 1 • 1 year
|
Other adverse events
| Measure |
Leptin Treatment
n=11 participants at risk
300 mg of study drug administered via SC injections.
Metreleptin: Administered SC twice/day to achieve physiological concentrations that will be effective in improving the severe state of insulin resistance seen in patients with genetic defects on their insulin receptor mutation
|
|---|---|
|
Skin and subcutaneous tissue disorders
Acrochordon
|
9.1%
1/11 • Number of events 1 • 1 year
|
|
Psychiatric disorders
Alcohol withdrawal
|
9.1%
1/11 • Number of events 1 • 1 year
|
|
Psychiatric disorders
Anxiety
|
9.1%
1/11 • Number of events 1 • 1 year
|
|
Infections and infestations
Candidiasis
|
9.1%
1/11 • Number of events 1 • 1 year
|
|
Eye disorders
Cataract
|
9.1%
1/11 • Number of events 1 • 1 year
|
|
Ear and labyrinth disorders
Cerumen impaction
|
9.1%
1/11 • Number of events 1 • 1 year
|
|
Ear and labyrinth disorders
Cholesteatoma
|
9.1%
1/11 • Number of events 1 • 1 year
|
|
Gastrointestinal disorders
Constipation
|
9.1%
1/11 • Number of events 1 • 1 year
|
|
Infections and infestations
COVID-19
|
9.1%
1/11 • Number of events 1 • 1 year
|
|
Musculoskeletal and connective tissue disorders
Cracked tooth
|
9.1%
1/11 • Number of events 1 • 1 year
|
|
Infections and infestations
Dental abscess
|
18.2%
2/11 • Number of events 2 • 1 year
|
|
Psychiatric disorders
Depression
|
9.1%
1/11 • Number of events 2 • 1 year
|
|
General disorders
Edema
|
9.1%
1/11 • Number of events 1 • 1 year
|
|
Metabolism and nutrition disorders
Elevated lactate
|
9.1%
1/11 • Number of events 1 • 1 year
|
|
Gastrointestinal disorders
GERD
|
9.1%
1/11 • Number of events 1 • 1 year
|
|
Eye disorders
Glaucoma
|
9.1%
1/11 • Number of events 1 • 1 year
|
|
Nervous system disorders
Headache
|
9.1%
1/11 • Number of events 1 • 1 year
|
|
Metabolism and nutrition disorders
Hyperglycemia
|
18.2%
2/11 • Number of events 2 • 1 year
|
|
Metabolism and nutrition disorders
Hyperkalemia
|
9.1%
1/11 • Number of events 1 • 1 year
|
|
Metabolism and nutrition disorders
Hypoglycemia
|
9.1%
1/11 • Number of events 1 • 1 year
|
|
Metabolism and nutrition disorders
Hypokalemia
|
9.1%
1/11 • Number of events 1 • 1 year
|
|
Gastrointestinal disorders
Indigestion
|
9.1%
1/11 • Number of events 1 • 1 year
|
|
Metabolism and nutrition disorders
Ketosis
|
9.1%
1/11 • Number of events 1 • 1 year
|
|
Renal and urinary disorders
Kidney stone
|
9.1%
1/11 • Number of events 2 • 1 year
|
|
Surgical and medical procedures
Mastoidectomy & tympanostomy tube placement
|
9.1%
1/11 • Number of events 1 • 1 year
|
|
Cardiac disorders
Mitral & tricupsid valve regurgitation
|
9.1%
1/11 • Number of events 1 • 1 year
|
|
General disorders
Pain
|
9.1%
1/11 • Number of events 1 • 1 year
|
|
Nervous system disorders
Seizure
|
9.1%
1/11 • Number of events 4 • 1 year
|
|
Respiratory, thoracic and mediastinal disorders
Shortness of breath
|
9.1%
1/11 • Number of events 1 • 1 year
|
|
Nervous system disorders
Syncope
|
9.1%
1/11 • Number of events 1 • 1 year
|
|
Endocrine disorders
Throid nodule
|
9.1%
1/11 • Number of events 1 • 1 year
|
|
Gastrointestinal disorders
Vomiting
|
9.1%
1/11 • Number of events 1 • 1 year
|
|
Metabolism and nutrition disorders
Weight loss
|
27.3%
3/11 • Number of events 3 • 1 year
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place