Trial Outcomes & Findings for Effects of Metreleptin in Type 1 Diabetes Mellitus (NCT NCT01268644)

NCT ID: NCT01268644

Last Updated: 2019-08-28

Results Overview

Change in Hba1c after 12 weeks on Leptin Therapy compared to Baseline value

• Recruitment status: TERMINATED
• Study phase: PHASE1
• Target enrollment: 8 participants
• Primary outcome timeframe: Baseline and 12 weeks
• Results posted on: 2019-08-28

Participant Flow

Patients with T1DM between the 18 and 50 years of age and with a BMI less than 27 kg/m2 and an HbA1c of 7.0-10.0% (53-86 mmol/mol) were eligible for the study.

Participant milestones

Measure	Leptin Therapy in T1DM Patients After a 4-week lead-in period, baseline evaluation was conducted at week 0.
Baseline Period (Week 0) STARTED	8
Baseline Period (Week 0) COMPLETED	8
Baseline Period (Week 0) NOT COMPLETED	0
Phase A (Weeks 0-12) Leptin Therapy STARTED	8
Phase A (Weeks 0-12) Leptin Therapy COMPLETED	8
Phase A (Weeks 0-12) Leptin Therapy NOT COMPLETED	0
Phase B (Weeks 12-20) Leptin Therapy STARTED	7
Phase B (Weeks 12-20) Leptin Therapy COMPLETED	7
Phase B (Weeks 12-20) Leptin Therapy NOT COMPLETED	0
Phase C (Weeks 20-24) Off Leptin Therapy STARTED	7
Phase C (Weeks 20-24) Off Leptin Therapy COMPLETED	7
Phase C (Weeks 20-24) Off Leptin Therapy NOT COMPLETED	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Effects of Metreleptin in Type 1 Diabetes Mellitus

Baseline characteristics by cohort

Measure	Week 0 (Baseline) n=8 Participants After a 4-week lead-in period, baseline evaluation was conducted at week 0.
Age, Categorical <=18 years	0 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	8 Participants n=5 Participants
Age, Categorical >=65 years	0 Participants n=5 Participants
Age, Continuous	33 years STANDARD_DEVIATION 10 • n=5 Participants
Sex: Female, Male Female	5 Participants n=5 Participants
Sex: Female, Male Male	3 Participants n=5 Participants
Region of Enrollment United States	8 participants n=5 Participants
Weight	70.7 kg STANDARD_DEVIATION 5.9 • n=5 Participants
Leptin	23 ng/mL n=5 Participants
Insulin dose	45.9 Units/day STANDARD_DEVIATION 11.5 • n=5 Participants
HbA1c	7.6 % of Hemoglobin STANDARD_DEVIATION 0.4 • n=5 Participants

PRIMARY outcome

Timeframe: Baseline and 12 weeks

Change in Hba1c after 12 weeks on Leptin Therapy compared to Baseline value

Outcome measures

Measure	Leptin Therapy Week 12 n=8 Participants Change in HbA1c after 12 weeks on Leptin Therapy from Baseline.
HbA1c	-0.19 % of hemoglobin Interval -0.44 to 0.07

SECONDARY outcome

Timeframe: Baseline to 12 weeks

Population: One subject withdrew after 12 weeks.

Change in Body Weight after 12 weeks on Leptin Therapy compared to Baseline value

Outcome measures

Measure	Leptin Therapy Week 12 n=8 Participants Change in HbA1c after 12 weeks on Leptin Therapy from Baseline.
Weight	-2.60 kg Interval -4.6 to -0.6

SECONDARY outcome

Timeframe: Baseline to 12 weeks

Population: One subject withdrew after 12 weeks.

Change in Insulin dose after 12 weeks on Leptin Therapy compared to Baseline value

Outcome measures

Measure	Leptin Therapy Week 12 n=8 Participants Change in HbA1c after 12 weeks on Leptin Therapy from Baseline.
Insulin Dose	-5.84 units/day Interval -10.1 to -1.6

SECONDARY outcome

Timeframe: Baseline to Week 20 (On leptin)

Change in Hba1c after 20 weeks on Leptin Therapy compared to Baseline value. With ongoing metreleptin therapy, the concomitant basal insulin dose was actively reduced by 50% after week 12.

Outcome measures

Measure	Leptin Therapy Week 12 n=7 Participants Change in HbA1c after 12 weeks on Leptin Therapy from Baseline.
Change in HbA1c From Baseline to Week 20 on Leptin Therapy	-0.04 % of Hemoglobin Interval -0.31 to 0.22

Adverse Events

Overall Study

Serious events: 0 serious events

Other events: 1 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Measure	Overall Study n=8 participants at risk After a 4-week lead-in period, baseline evaluation was conducted at week 0.
Infections and infestations Injection site reaction	12.5% 1/8 • Number of events 1 • Adverse event data was collected for 24 weeks.

Additional Information

Dr. Abhimanyu Garg

UT Southwestern Medical Center

Phone: 2146482895

Email: abhimanyu.garg@utsouthwestern.edu

Results disclosure agreements

• Principal investigator is a sponsor employee
• Publication restrictions are in place