Trial Outcomes & Findings for A Study To Examine The Efficacy And Safety Of Pramlintide+Metreleptin In Obese Subjects (NCT NCT01235741)

NCT ID: NCT01235741

Last Updated: 2015-04-15

Results Overview

Treatment-Emergent Adverse Events are defined as those with an onset date and time on or after the first dose of randomized study medication and on or before the last dose of randomized study medication. Post-treatment Adverse Events are defined as those with an onset date after the date of last dose (imputed if not available) of randomized study medication. Participants experiencing multiple episodes of a given adverse event are counted once.

Recruitment status

TERMINATED

Study phase

PHASE2

Target enrollment

213 participants

Primary outcome timeframe

Day 1 up to Month 6 Follow-Up

Results posted on

2015-04-15

Participant Flow

5 January 2011 Lead-In Period started. 28 September 2011 last participants final visit procedure. Clinics where participants with body mass index \[BMI\] greater than, equal to (≥)35 kilogram per meter squared (kg/m\^2) and less than, equal to (≤)45 kg/m2) could be enrolled.

6 week lead-in period:low-calorie diet (LCD); 16 week randomized treatment period: participants who lost \>=2% body weight at end of LCD were randomized to an arm. Sponsor terminated study during randomization period due to neutralizing activity to metreleptin observed in Study DFA102, NCT00673387. Safety follow-up 2, 4, 6 months post treatment.

Participant milestones

Participant milestones
Measure
Low Calorie Diet Only
6 Week Lead-in Period with low calorie diet (LCD); in the last week of the 6 week LCD, self administered subcutaneous (SC) injections of placebo once a day (QD) was initiated and then followed by randomization to either study drug or placebo in the Randomization period.
Placebo
Self administered subcutaneous (SC) injection of placebo matched to pramlintide (Placebo-P) plus placebo matched to metreleptin (Placebo-M) twice a day (BID) for 16 weeks.
Pramlintide + Metreleptin
Self administered SC injection of pramlintide 360 micrograms (µg) + metreleptin 5.0 milligrams (mg) BID for 16 weeks
Non-Randomized Low Calorie Diet Lead-In
STARTED
213
0
0
Non-Randomized Low Calorie Diet Lead-In
COMPLETED
72
0
0
Non-Randomized Low Calorie Diet Lead-In
NOT COMPLETED
141
0
0
Randomized to Treatment
STARTED
0
36
36
Randomized to Treatment
COMPLETED
0
0
0
Randomized to Treatment
NOT COMPLETED
0
36
36
Follow-Up Post Treatment up to Month 6
STARTED
0
36
36
Follow-Up Post Treatment up to Month 6
COMPLETED
0
27
29
Follow-Up Post Treatment up to Month 6
NOT COMPLETED
0
9
7

Reasons for withdrawal

Reasons for withdrawal
Measure
Low Calorie Diet Only
6 Week Lead-in Period with low calorie diet (LCD); in the last week of the 6 week LCD, self administered subcutaneous (SC) injections of placebo once a day (QD) was initiated and then followed by randomization to either study drug or placebo in the Randomization period.
Placebo
Self administered subcutaneous (SC) injection of placebo matched to pramlintide (Placebo-P) plus placebo matched to metreleptin (Placebo-M) twice a day (BID) for 16 weeks.
Pramlintide + Metreleptin
Self administered SC injection of pramlintide 360 micrograms (µg) + metreleptin 5.0 milligrams (mg) BID for 16 weeks
Non-Randomized Low Calorie Diet Lead-In
Withdrawal by Subject
9
0
0
Non-Randomized Low Calorie Diet Lead-In
Sponsor terminated study
121
0
0
Non-Randomized Low Calorie Diet Lead-In
Physician Decision
1
0
0
Non-Randomized Low Calorie Diet Lead-In
Protocol Violation
1
0
0
Non-Randomized Low Calorie Diet Lead-In
Lost to Follow-up
5
0
0
Non-Randomized Low Calorie Diet Lead-In
Failed to meet weight loss in Period 1
4
0
0
Randomized to Treatment
Withdrawal by Subject
0
0
1
Randomized to Treatment
Adverse Event
0
1
1
Randomized to Treatment
Sponsor terminated study
0
35
34
Follow-Up Post Treatment up to Month 6
Withdrawal by Subject
0
3
6
Follow-Up Post Treatment up to Month 6
Lost to Follow-up
0
5
1
Follow-Up Post Treatment up to Month 6
Other
0
1
0

Baseline Characteristics

A Study To Examine The Efficacy And Safety Of Pramlintide+Metreleptin In Obese Subjects

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure
Non-Randomized Lead-in LCD
n=141 Participants
6 Week Lead-in with low calorie diet (LCD); in the last week of the 6 week LCD, self administered subcutaneous (SC) injections of placebo once a day (QD) was initiated and then followed by randomization to study drug in the Randomization period.
Placebo
n=36 Participants
Self administered subcutaneous (SC ) injection of placebo matched to pramlintide (Placebo-P) plus placebo matched to metreleptin (Placebo-M) twice a day (BID) for 16 weeks.
Pramlintide + Metreleptin
n=36 Participants
Self administered SC injection of pramlintide 360 µg + metreleptin 5.0 mg BID for 16 weeks
Total
n=213 Participants
Total of all reporting groups
Age, Continuous
46.1 years
STANDARD_DEVIATION 11.81 • n=5 Participants
46.2 years
STANDARD_DEVIATION 10.84 • n=7 Participants
46.4 years
STANDARD_DEVIATION 10.45 • n=5 Participants
46.1 years
STANDARD_DEVIATION 11.38 • n=4 Participants
Sex: Female, Male
Female
106 Participants
n=5 Participants
27 Participants
n=7 Participants
29 Participants
n=5 Participants
162 Participants
n=4 Participants
Sex: Female, Male
Male
35 Participants
n=5 Participants
9 Participants
n=7 Participants
7 Participants
n=5 Participants
51 Participants
n=4 Participants
Region of Enrollment
United States
141 participants
n=5 Participants
36 participants
n=7 Participants
36 participants
n=5 Participants
213 participants
n=4 Participants
Body Weight
110.86 kg
STANDARD_DEVIATION 13.210 • n=5 Participants
109.10 kg
STANDARD_DEVIATION 16.850 • n=7 Participants
108.34 kg
STANDARD_DEVIATION 12.032 • n=5 Participants
110.13 kg
STANDARD_DEVIATION 13.678 • n=4 Participants
Body Mass Index
39.51 kg/m^2
STANDARD_DEVIATION 2.875 • n=5 Participants
39.17 kg/m^2
STANDARD_DEVIATION 3.279 • n=7 Participants
39.09 kg/m^2
STANDARD_DEVIATION 2.869 • n=5 Participants
39.38 kg/m^2
STANDARD_DEVIATION 2.937 • n=4 Participants

PRIMARY outcome

Timeframe: Day 1 up to Month 6 Follow-Up

Population: The intent to treat (ITT) population received at least one dose of randomized study treatment.

Treatment-Emergent Adverse Events are defined as those with an onset date and time on or after the first dose of randomized study medication and on or before the last dose of randomized study medication. Post-treatment Adverse Events are defined as those with an onset date after the date of last dose (imputed if not available) of randomized study medication. Participants experiencing multiple episodes of a given adverse event are counted once.

Outcome measures

Outcome measures
Measure
Placebo
n=36 Participants
Self administered SC injection of placebo matched to pramlintide (Placebo-P) plus placebo matched to metreleptin (Placebo-M) BID for 16 weeks.
Pramlintide + Metreleptin
n=36 Participants
Self administered SC injection of pramlintide 360 µg + metreleptin 5.0 mg BID for 16 weeks
Post Treatment Placebo Arm
n=36 Participants
From date after the date of last dose (imputed if not available) of randomized study medication up to 6 Months post treatment.
Post Treatment Pramlintide + Metreleptin Arm
n=36 Participants
From date after the date of last dose (imputed if not available) of randomized study medication up to 6 Months post treatment.
Number of Participants With Treatment Emergent Adverse Events and Number With Post Treatment Adverse Events - Intent to Treat Population
6 participants
13 participants
13 participants
16 participants

PRIMARY outcome

Timeframe: Baseline to Month 6 Follow Up

Population: All participants who received a dose of metreleptin and provided a sample were analyzed. Number of participants analyzed for Week 2, and follow up Months 2, 4, 6 were 6, and 33, 29, 29, respectively.

Participants who received metreleptin were analyzed; no placebo treated participants were analyzed. Baseline was Day 1 measurement or the last measurement taken prior to first dose of randomized study medication, if Day 1 measure was missing. Leptin was measured in nanograms per milliliter (ng/mL).

Outcome measures

Outcome measures
Measure
Placebo
n=36 Participants
Self administered SC injection of placebo matched to pramlintide (Placebo-P) plus placebo matched to metreleptin (Placebo-M) BID for 16 weeks.
Pramlintide + Metreleptin
Self administered SC injection of pramlintide 360 µg + metreleptin 5.0 mg BID for 16 weeks
Post Treatment Placebo Arm
From date after the date of last dose (imputed if not available) of randomized study medication up to 6 Months post treatment.
Post Treatment Pramlintide + Metreleptin Arm
From date after the date of last dose (imputed if not available) of randomized study medication up to 6 Months post treatment.
Change From Baseline to Week 2, and to Follow up Months 2, 4, 6 in Fasting Leptin Concentration - Intent to Treat Population
Change from baseline to Week 2 of treatment
223.38 ng/mL
Standard Deviation 512.088
Change From Baseline to Week 2, and to Follow up Months 2, 4, 6 in Fasting Leptin Concentration - Intent to Treat Population
Change from baseline to Month 2 Follow up
15.46 ng/mL
Standard Deviation 34.885
Change From Baseline to Week 2, and to Follow up Months 2, 4, 6 in Fasting Leptin Concentration - Intent to Treat Population
Change from baseline to Month 4 Follow up
20.08 ng/mL
Standard Deviation 35.732
Change From Baseline to Week 2, and to Follow up Months 2, 4, 6 in Fasting Leptin Concentration - Intent to Treat Population
Month 6 Follow up
10.70 ng/mL
Standard Deviation 18.913

PRIMARY outcome

Timeframe: Week 1 to Month 6 Follow-Up

Population: All participants who received at least one dose of metreleptin and provided a sample to analyze; number analyzed (n) for Week 1, Week 2, early termination, Month 2, Month 4, Month 6 Follow-up were: n=22, 5, 35, 32, 25, 28.

Anti-leptin antibodies measured at Weeks 1 and 2 of drug treatment, early termination visit, and at Months 2, 4, and 6 post treatment follow-up in participants who received metreleptin.

Outcome measures

Outcome measures
Measure
Placebo
n=36 Participants
Self administered SC injection of placebo matched to pramlintide (Placebo-P) plus placebo matched to metreleptin (Placebo-M) BID for 16 weeks.
Pramlintide + Metreleptin
Self administered SC injection of pramlintide 360 µg + metreleptin 5.0 mg BID for 16 weeks
Post Treatment Placebo Arm
From date after the date of last dose (imputed if not available) of randomized study medication up to 6 Months post treatment.
Post Treatment Pramlintide + Metreleptin Arm
From date after the date of last dose (imputed if not available) of randomized study medication up to 6 Months post treatment.
Number of Participants With Anti-leptin Antibodies Who Received Metreleptin - Intent to Treat Population
Month 4 Follow-Up
10 participants
Number of Participants With Anti-leptin Antibodies Who Received Metreleptin - Intent to Treat Population
Month 6 Follow-Up
8 participants
Number of Participants With Anti-leptin Antibodies Who Received Metreleptin - Intent to Treat Population
Week 1
0 participants
Number of Participants With Anti-leptin Antibodies Who Received Metreleptin - Intent to Treat Population
Week 2
1 participants
Number of Participants With Anti-leptin Antibodies Who Received Metreleptin - Intent to Treat Population
Early Termination
9 participants
Number of Participants With Anti-leptin Antibodies Who Received Metreleptin - Intent to Treat Population
Month 2 Follow-Up
15 participants

PRIMARY outcome

Timeframe: Baseline to Month 6 Follow-Up

In vitro assays were conducted to determine if neutralizing activity to metreleptin developed in participants treated with at least one dose of the drug during the study. Baseline is Day 1 of the Randomization Period, prior to administration of metreleptin.

Outcome measures

Outcome measures
Measure
Placebo
n=36 Participants
Self administered SC injection of placebo matched to pramlintide (Placebo-P) plus placebo matched to metreleptin (Placebo-M) BID for 16 weeks.
Pramlintide + Metreleptin
Self administered SC injection of pramlintide 360 µg + metreleptin 5.0 mg BID for 16 weeks
Post Treatment Placebo Arm
From date after the date of last dose (imputed if not available) of randomized study medication up to 6 Months post treatment.
Post Treatment Pramlintide + Metreleptin Arm
From date after the date of last dose (imputed if not available) of randomized study medication up to 6 Months post treatment.
Number of Participants With Neutralizing Activity to Metreleptin at Early Termination or During Post Treatment Follow-Up - Intent to Treat Population Who Received Metreleptin
0 participants

PRIMARY outcome

Timeframe: Screening to 6 Month Follow-Up

Population: The intent to treat (ITT) population received at least one dose of randomized study treatment. Number analyzed (n) in ITT included participants who provided a laboratory measurement. Hemoglobin, hematocrit n=27, 29 in placebo and pramlintide + metreleptin, respectively; urinalysis n=31, 29, in placebo and pramlintide + metreleptin, respectively.

Criteria for laboratory values of potential clinical importance for obese and overweight (BMI \>= 25 kg/m\^2) participants: Platelets high (H) \>500,000/µL; low (L) \<75,000/µL. Hematocrit males \<36%, females \<30%. Hemoglobin males \<12 g/dL, females \<10 g/dL. White blood cell count (WBC) H \>18,000/µL; L \<1,500/µL. Urine protein H \>= 3+ or \>= 500 mg/dL. Urine glucose H \>= 3+ or \>= 500 mg/dL. Urine ketones \>= 3+ or Large.

Outcome measures

Outcome measures
Measure
Placebo
n=36 Participants
Self administered SC injection of placebo matched to pramlintide (Placebo-P) plus placebo matched to metreleptin (Placebo-M) BID for 16 weeks.
Pramlintide + Metreleptin
n=36 Participants
Self administered SC injection of pramlintide 360 µg + metreleptin 5.0 mg BID for 16 weeks
Post Treatment Placebo Arm
From date after the date of last dose (imputed if not available) of randomized study medication up to 6 Months post treatment.
Post Treatment Pramlintide + Metreleptin Arm
From date after the date of last dose (imputed if not available) of randomized study medication up to 6 Months post treatment.
Number of Participants With Hematology and Urinalysis Laboratory Values of Potential Clinical Importance - Intent to Treat Population
Hemoglobin
0 participants
1 participants
Number of Participants With Hematology and Urinalysis Laboratory Values of Potential Clinical Importance - Intent to Treat Population
Hematocrit
0 participants
1 participants
Number of Participants With Hematology and Urinalysis Laboratory Values of Potential Clinical Importance - Intent to Treat Population
Glucose in Urine
0 participants
1 participants

PRIMARY outcome

Timeframe: Screening to Month 6 Follow-Up

Population: The intent to treat (ITT) population received at least one dose of randomized study treatment. The number analyzed in the ITT included those participants who provided a laboratory measurement.

Criteria for laboratory values of potential clinical importance for obese and overweight (BMI \>= 25 kg/m\^2) participants: Total bilirubin High (H) \> 2 mg/dL; Plasma or serum glucose fasting or non-fasting H \> 200 mg/dL, low (L) \< 60 mg/dL; Albumin L \<2.5 g/dL; Creatine kinase H \> 3\*Upper limit of Normal (ULN); Sodium L \<130 milliequivalents per liter (mEq/L), H \> 150 mEq/L; potassium L\<3.0 mEq/L, H\> 5.5 mEq/L; bicarbonate L\<18 mEq/L, H\>35 mEq/L;calcium L \<8mg/dL, H\> 11 mg/dL; triglycerides H\> 500 mg/dL; Cholesterol L \< 100 mg/dL, H \> 350 mg/dL; Alkaline phosphatase H \> 3\*ULN; Gamma-glutamyltransferase H\>3\*ULN; creatinine males \> 1.6 mg/dL, females \> 1.4 mg/dL; alanine aminotransferase H \> 3\*ULN; aspartate aminotransferase H \> 3\*ULN; urea nitrogen H \> 45 mg/dL; uric acid males \> 10.0 mg/dL, females \> 8.0 mg/dL; Phosphorus L \< 1.0 mg/dL H \> 6.0 mg/dL.

Outcome measures

Outcome measures
Measure
Placebo
n=36 Participants
Self administered SC injection of placebo matched to pramlintide (Placebo-P) plus placebo matched to metreleptin (Placebo-M) BID for 16 weeks.
Pramlintide + Metreleptin
n=36 Participants
Self administered SC injection of pramlintide 360 µg + metreleptin 5.0 mg BID for 16 weeks
Post Treatment Placebo Arm
From date after the date of last dose (imputed if not available) of randomized study medication up to 6 Months post treatment.
Post Treatment Pramlintide + Metreleptin Arm
From date after the date of last dose (imputed if not available) of randomized study medication up to 6 Months post treatment.
Number of Participants With Chemistry Laboratory Value of Potential Clinical Importance - Intent to Treat Population
Creatine Kinase
2 participants
3 participants
Number of Participants With Chemistry Laboratory Value of Potential Clinical Importance - Intent to Treat Population
Bilirubin
0 participants
1 participants
Number of Participants With Chemistry Laboratory Value of Potential Clinical Importance - Intent to Treat Population
Urate
0 participants
2 participants

PRIMARY outcome

Timeframe: Baseline to Month 6 Follow-Up

Population: The intent to treat (ITT) population received at least one dose of drug treatment. The number analyzed in the ITT included those participants who provided a vital sign measurement up to 6 Months follow-up.

Baseline was Day 1 measurement or the last measurement taken prior to first dose of randomized study medication, if Day 1 measure was missing. Follow-up was up to 6 months post treatment. Blood pressure included systolic and diastolic pressures measured in millimeters of mercury (mmHg).

Outcome measures

Outcome measures
Measure
Placebo
n=27 Participants
Self administered SC injection of placebo matched to pramlintide (Placebo-P) plus placebo matched to metreleptin (Placebo-M) BID for 16 weeks.
Pramlintide + Metreleptin
n=29 Participants
Self administered SC injection of pramlintide 360 µg + metreleptin 5.0 mg BID for 16 weeks
Post Treatment Placebo Arm
From date after the date of last dose (imputed if not available) of randomized study medication up to 6 Months post treatment.
Post Treatment Pramlintide + Metreleptin Arm
From date after the date of last dose (imputed if not available) of randomized study medication up to 6 Months post treatment.
Mean Change From Baseline to Month 6 Follow-Up in Blood Pressure - Intent to Treat Population
Diastolic Blood Pressure
2.7 mmHg
Standard Deviation 7.40
2.7 mmHg
Standard Deviation 8.07
Mean Change From Baseline to Month 6 Follow-Up in Blood Pressure - Intent to Treat Population
Systolic Blood Pressure
4.3 mmHg
Standard Deviation 9.40
3.1 mmHg
Standard Deviation 8.90

PRIMARY outcome

Timeframe: Baseline up to Month 6 Follow-Up

Population: The intent to treat (ITT) population received at least one dose of randomized study treatment. The number analyzed in the ITT included those participants who provided a vital sign measurement.

Baseline was Day 1 measurement or the last measurement taken prior to first dose of randomized study medication, if Day 1 measure was missing. Heart rate was measured in beats per minute (beats/min).

Outcome measures

Outcome measures
Measure
Placebo
n=27 Participants
Self administered SC injection of placebo matched to pramlintide (Placebo-P) plus placebo matched to metreleptin (Placebo-M) BID for 16 weeks.
Pramlintide + Metreleptin
n=29 Participants
Self administered SC injection of pramlintide 360 µg + metreleptin 5.0 mg BID for 16 weeks
Post Treatment Placebo Arm
From date after the date of last dose (imputed if not available) of randomized study medication up to 6 Months post treatment.
Post Treatment Pramlintide + Metreleptin Arm
From date after the date of last dose (imputed if not available) of randomized study medication up to 6 Months post treatment.
Mean Change From Baseline to Month 6 Follow-Up in Heart Rate - Intent to Treat Population
1.2 beats/min
Standard Deviation 8.85
1.2 beats/min
Standard Deviation 7.98

PRIMARY outcome

Timeframe: Baseline to 6 Month Follow-Up

Population: The intent to treat (ITT) population received at least one dose of randomized study treatment. The number analyzed in the ITT included those participants who provided a laboratory measurement up to Month 6 Follow-Up.

Baseline was Day 1 measurement or the last measurement taken prior to first dose of randomized study medication, if Day 1 measure was missing. Follow up was 6 months post last dose of randomized study medication. Fasting glucose measured in milligrams per deciliter (mg/dL).

Outcome measures

Outcome measures
Measure
Placebo
n=25 Participants
Self administered SC injection of placebo matched to pramlintide (Placebo-P) plus placebo matched to metreleptin (Placebo-M) BID for 16 weeks.
Pramlintide + Metreleptin
n=29 Participants
Self administered SC injection of pramlintide 360 µg + metreleptin 5.0 mg BID for 16 weeks
Post Treatment Placebo Arm
From date after the date of last dose (imputed if not available) of randomized study medication up to 6 Months post treatment.
Post Treatment Pramlintide + Metreleptin Arm
From date after the date of last dose (imputed if not available) of randomized study medication up to 6 Months post treatment.
Mean Change From Baseline to 6 Month Follow-Up in Fasting Plasma Glucose - Intent to Treat Population
6.8 mg/dL
Standard Deviation 8.31
-1.0 mg/dL
Standard Deviation 9.03

PRIMARY outcome

Timeframe: Baseline up to Month 6 Follow-Up

Population: The intent to treat (ITT) population received at least one dose of randomized study treatment. The number analyzed in the ITT included those participants who provided a laboratory measurement.

Baseline was Day 1 measurement or the last measurement taken prior to first dose of randomized study medication, if Day 1 measure was missing. Follow up was 6 months post last dose of randomized study medication. Insulin measured in milliunits per liter (mU/L).

Outcome measures

Outcome measures
Measure
Placebo
n=27 Participants
Self administered SC injection of placebo matched to pramlintide (Placebo-P) plus placebo matched to metreleptin (Placebo-M) BID for 16 weeks.
Pramlintide + Metreleptin
n=29 Participants
Self administered SC injection of pramlintide 360 µg + metreleptin 5.0 mg BID for 16 weeks
Post Treatment Placebo Arm
From date after the date of last dose (imputed if not available) of randomized study medication up to 6 Months post treatment.
Post Treatment Pramlintide + Metreleptin Arm
From date after the date of last dose (imputed if not available) of randomized study medication up to 6 Months post treatment.
Mean Change From Baseline to Month 6 Follow-Up in Insulin - Intent to Treat Population
3.41 mU/L
Standard Deviation 8.990
3.46 mU/L
Standard Deviation 7.617

PRIMARY outcome

Timeframe: Baseline up to Month 6 Follow-Up

Population: The intent to treat (ITT) population received at least one dose of randomized study treatment. The number analyzed in the ITT included those participants who provided a laboratory measurement up to Month 6 Follow-Up.

Baseline was Day 1 measurement or the last measurement taken prior to first dose of randomized study medication, if Day 1 measure was missing. Follow up was 6 months post last dose of randomized study medication. Lipids measured included total cholesterol, high density lipoprotein (HDL) cholesterol, low density lipoprotein (LDL) cholesterol, and triglycerides. Lipids were measured in milligrams per deciliter (mg/dL).

Outcome measures

Outcome measures
Measure
Placebo
n=27 Participants
Self administered SC injection of placebo matched to pramlintide (Placebo-P) plus placebo matched to metreleptin (Placebo-M) BID for 16 weeks.
Pramlintide + Metreleptin
n=29 Participants
Self administered SC injection of pramlintide 360 µg + metreleptin 5.0 mg BID for 16 weeks
Post Treatment Placebo Arm
From date after the date of last dose (imputed if not available) of randomized study medication up to 6 Months post treatment.
Post Treatment Pramlintide + Metreleptin Arm
From date after the date of last dose (imputed if not available) of randomized study medication up to 6 Months post treatment.
Mean Change From Baseline to Month 6 Follow-Up in Lipids - Intent to Treat Population
Total Cholesterol
21.7 mg/dL
Standard Deviation 19.21
14.7 mg/dL
Standard Deviation 25.51
Mean Change From Baseline to Month 6 Follow-Up in Lipids - Intent to Treat Population
HDL cholesterol
7.7 mg/dL
Standard Deviation 7.25
6.6 mg/dL
Standard Deviation 7.60
Mean Change From Baseline to Month 6 Follow-Up in Lipids - Intent to Treat Population
LDL cholesterol
14.0 mg/dL
Standard Deviation 17.47
9.6 mg/dL
Standard Deviation 21.05
Mean Change From Baseline to Month 6 Follow-Up in Lipids - Intent to Treat Population
Triglycerides
10.4 mg/dL
Standard Deviation 35.12
3.0 mg/dL
Standard Deviation 38.30

SECONDARY outcome

Timeframe: Baseline up to Month 6 Follow-Up

Population: The intent to treat (ITT) population received at least one dose of randomized study treatment. The number analyzed in the ITT included those participants who provided a measurement. Number analyzed (n) for Week 1 provided above; 6 Month Follow-Up n=27, 29, in placebo and Pramlintide + Metreleptin, respectively.

Baseline was Day 1 measurement or the last measurement taken prior to first dose of randomized study medication, if Day 1 measure was missing. Follow up was 6 months post last dose of randomized study medication.

Outcome measures

Outcome measures
Measure
Placebo
n=20 Participants
Self administered SC injection of placebo matched to pramlintide (Placebo-P) plus placebo matched to metreleptin (Placebo-M) BID for 16 weeks.
Pramlintide + Metreleptin
n=23 Participants
Self administered SC injection of pramlintide 360 µg + metreleptin 5.0 mg BID for 16 weeks
Post Treatment Placebo Arm
From date after the date of last dose (imputed if not available) of randomized study medication up to 6 Months post treatment.
Post Treatment Pramlintide + Metreleptin Arm
From date after the date of last dose (imputed if not available) of randomized study medication up to 6 Months post treatment.
Percent Change From Baseline to Week 1 and From Baseline to Month 6 Follow-up in Body Weight - Intent to Treat Population
Week 1
-0.34 percentage of change in weight
Standard Deviation 0.924
-0.26 percentage of change in weight
Standard Deviation 0.819
Percent Change From Baseline to Week 1 and From Baseline to Month 6 Follow-up in Body Weight - Intent to Treat Population
6 Month Follow-Up
-0.02 percentage of change in weight
Standard Deviation 6.575
-1.80 percentage of change in weight
Standard Deviation 6.063

Adverse Events

Placebo-P + Placebo-M

Serious events: 0 serious events
Other events: 1 other events
Deaths: 0 deaths

Pramlintide + Metreleptin

Serious events: 1 serious events
Other events: 14 other events
Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure
Placebo-P + Placebo-M
n=36 participants at risk
Self administered subcutaneous injection once a day (QD) of placebo matched to pramlintide (Placebo-P) plus placebo matched to metreleptin (Placebo-M) for 1 week followed by BID dosing for 15 weeks (16 weeks total).
Pramlintide + Metreleptin
n=36 participants at risk
Self administered subcutaneous injection once a day (QD) of pramlintide 360 micrograms (µg) plus metreleptin 5.0 milligrams (mg ) for 1 week followed by twice a week (BID) dosing for 15 weeks (Total of 16 Weeks treatment).
Cardiac disorders
Coronary artery disease
0.00%
0/36 • Treatment emergent adverse events (AEs) and Serious AEs (SAEs): onset date/ time on or after first dose of randomized study medication and on or before the last dose of randomized study medication.
AE=any new unfavorable symptom, sign, or disease or worsening of a preexisting condition. SAE=medical event that results in death, persistent or significant disability/incapacity, or drug dependency/abuse; is life-threatening, an important medical event, or a congenital anomaly/birth defect; or requires or prolongs hospitalization.
2.8%
1/36 • Number of events 1 • Treatment emergent adverse events (AEs) and Serious AEs (SAEs): onset date/ time on or after first dose of randomized study medication and on or before the last dose of randomized study medication.
AE=any new unfavorable symptom, sign, or disease or worsening of a preexisting condition. SAE=medical event that results in death, persistent or significant disability/incapacity, or drug dependency/abuse; is life-threatening, an important medical event, or a congenital anomaly/birth defect; or requires or prolongs hospitalization.

Other adverse events

Other adverse events
Measure
Placebo-P + Placebo-M
n=36 participants at risk
Self administered subcutaneous injection once a day (QD) of placebo matched to pramlintide (Placebo-P) plus placebo matched to metreleptin (Placebo-M) for 1 week followed by BID dosing for 15 weeks (16 weeks total).
Pramlintide + Metreleptin
n=36 participants at risk
Self administered subcutaneous injection once a day (QD) of pramlintide 360 micrograms (µg) plus metreleptin 5.0 milligrams (mg ) for 1 week followed by twice a week (BID) dosing for 15 weeks (Total of 16 Weeks treatment).
Gastrointestinal disorders
Nausea
2.8%
1/36 • Number of events 1 • Treatment emergent adverse events (AEs) and Serious AEs (SAEs): onset date/ time on or after first dose of randomized study medication and on or before the last dose of randomized study medication.
AE=any new unfavorable symptom, sign, or disease or worsening of a preexisting condition. SAE=medical event that results in death, persistent or significant disability/incapacity, or drug dependency/abuse; is life-threatening, an important medical event, or a congenital anomaly/birth defect; or requires or prolongs hospitalization.
19.4%
7/36 • Number of events 9 • Treatment emergent adverse events (AEs) and Serious AEs (SAEs): onset date/ time on or after first dose of randomized study medication and on or before the last dose of randomized study medication.
AE=any new unfavorable symptom, sign, or disease or worsening of a preexisting condition. SAE=medical event that results in death, persistent or significant disability/incapacity, or drug dependency/abuse; is life-threatening, an important medical event, or a congenital anomaly/birth defect; or requires or prolongs hospitalization.
General disorders
injection site hematoma
0.00%
0/36 • Treatment emergent adverse events (AEs) and Serious AEs (SAEs): onset date/ time on or after first dose of randomized study medication and on or before the last dose of randomized study medication.
AE=any new unfavorable symptom, sign, or disease or worsening of a preexisting condition. SAE=medical event that results in death, persistent or significant disability/incapacity, or drug dependency/abuse; is life-threatening, an important medical event, or a congenital anomaly/birth defect; or requires or prolongs hospitalization.
8.3%
3/36 • Number of events 3 • Treatment emergent adverse events (AEs) and Serious AEs (SAEs): onset date/ time on or after first dose of randomized study medication and on or before the last dose of randomized study medication.
AE=any new unfavorable symptom, sign, or disease or worsening of a preexisting condition. SAE=medical event that results in death, persistent or significant disability/incapacity, or drug dependency/abuse; is life-threatening, an important medical event, or a congenital anomaly/birth defect; or requires or prolongs hospitalization.
General disorders
injection site induration
0.00%
0/36 • Treatment emergent adverse events (AEs) and Serious AEs (SAEs): onset date/ time on or after first dose of randomized study medication and on or before the last dose of randomized study medication.
AE=any new unfavorable symptom, sign, or disease or worsening of a preexisting condition. SAE=medical event that results in death, persistent or significant disability/incapacity, or drug dependency/abuse; is life-threatening, an important medical event, or a congenital anomaly/birth defect; or requires or prolongs hospitalization.
5.6%
2/36 • Number of events 2 • Treatment emergent adverse events (AEs) and Serious AEs (SAEs): onset date/ time on or after first dose of randomized study medication and on or before the last dose of randomized study medication.
AE=any new unfavorable symptom, sign, or disease or worsening of a preexisting condition. SAE=medical event that results in death, persistent or significant disability/incapacity, or drug dependency/abuse; is life-threatening, an important medical event, or a congenital anomaly/birth defect; or requires or prolongs hospitalization.
General disorders
injection site pruritus
0.00%
0/36 • Treatment emergent adverse events (AEs) and Serious AEs (SAEs): onset date/ time on or after first dose of randomized study medication and on or before the last dose of randomized study medication.
AE=any new unfavorable symptom, sign, or disease or worsening of a preexisting condition. SAE=medical event that results in death, persistent or significant disability/incapacity, or drug dependency/abuse; is life-threatening, an important medical event, or a congenital anomaly/birth defect; or requires or prolongs hospitalization.
5.6%
2/36 • Number of events 2 • Treatment emergent adverse events (AEs) and Serious AEs (SAEs): onset date/ time on or after first dose of randomized study medication and on or before the last dose of randomized study medication.
AE=any new unfavorable symptom, sign, or disease or worsening of a preexisting condition. SAE=medical event that results in death, persistent or significant disability/incapacity, or drug dependency/abuse; is life-threatening, an important medical event, or a congenital anomaly/birth defect; or requires or prolongs hospitalization.

Additional Information

Peter Ohman, Medical Science Director

AstraZeneca

Results disclosure agreements

  • Principal investigator is a sponsor employee Bristol-Myers Squibb Co. agreements with investigators vary; constant is our right to embargo communications regarding trial results prior to public release for a period ≤60 days from submittal for review. We will not prohibit investigators from publishing, but will prohibit the disclosure of previously undisclosed confidential information other than study results, and request postponement of single-center publications until after disclosure of the clinical trial's primary publication.
  • Publication restrictions are in place

Restriction type: OTHER