Trial Outcomes & Findings for Trial of Leptin Replacement Therapy in Patients With Lipodystrophy (NCT NCT00896298)

NCT ID: NCT00896298

Last Updated: 2024-03-27

Results Overview

• Recruitment status: COMPLETED
• Study phase: PHASE2/PHASE3
• Target enrollment: 25 participants
• Primary outcome timeframe: 4 months
• Results posted on: 2024-03-27

Participant Flow

Participant milestones

Measure	Leptin Therapy Randomized to Leptin Therapy for 4 months.	Placebo Randomized to Placebo for 4 months.
Overall Study STARTED	13	12
Overall Study COMPLETED	10	9
Overall Study NOT COMPLETED	3	3

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Trial of Leptin Replacement Therapy in Patients With Lipodystrophy

Baseline characteristics by cohort

Measure	Leptin Therapy n=13 Participants Participants were randomized to receive Leptin .	Placebo n=12 Participants Participants were randomized to receive Placebo .	Total n=25 Participants Total of all reporting groups
Age, Continuous	27.2 years STANDARD_DEVIATION 11.9 • n=5 Participants	24.9 years STANDARD_DEVIATION 13 • n=7 Participants	26.1 years STANDARD_DEVIATION 12.2 • n=5 Participants
Sex: Female, Male Female	10 Participants n=5 Participants	9 Participants n=7 Participants	19 Participants n=5 Participants
Sex: Female, Male Male	3 Participants n=5 Participants	3 Participants n=7 Participants	6 Participants n=5 Participants
Body weight	65.5 kg STANDARD_DEVIATION 17.5 • n=5 Participants	57.7 kg STANDARD_DEVIATION 16.0 • n=7 Participants	61.8 kg STANDARD_DEVIATION 16.9 • n=5 Participants
Fasting serum Triglycerides	309.3 mg/dL n=5 Participants	443.8 mg/dL n=7 Participants	309.3 mg/dL n=5 Participants
Fasting serum Glucose	157.0 mg/dL STANDARD_DEVIATION 74.2 • n=5 Participants	184.0 mg/dL STANDARD_DEVIATION 87.2 • n=7 Participants	170.0 mg/dL STANDARD_DEVIATION 80.2 • n=5 Participants
HbA1c	8.1 % of haemoglobin STANDARD_DEVIATION 2.5 • n=5 Participants	8.0 % of haemoglobin STANDARD_DEVIATION 2.5 • n=7 Participants	8.0 % of haemoglobin STANDARD_DEVIATION 2.5 • n=5 Participants

PRIMARY outcome

Timeframe: 4 months

Outcome measures

Measure	Leptin Therapy n=10 Participants Randomized to Leptin Therapy for 4 months.	Placebo n=9 Participants Randomized to Placebo for 4 months.
Fasting Serum Triglycerides	203 mg/dL Interval 100.0 to 457.0	304 mg/dL Interval 129.0 to 3875.0

PRIMARY outcome

Timeframe: 4 months

Outcome measures

Measure	Leptin Therapy n=10 Participants Randomized to Leptin Therapy for 4 months.	Placebo n=9 Participants Randomized to Placebo for 4 months.
HbA1c	8.1 % of haemoglobin Interval 5.0 to 12.0	7.6 % of haemoglobin Interval 5.1 to 11.5

SECONDARY outcome

Timeframe: 4 months

Outcome measures

Measure	Leptin Therapy n=10 Participants Randomized to Leptin Therapy for 4 months.	Placebo n=9 Participants Randomized to Placebo for 4 months.
Fasting Serum Glucose	162 mg/dL Interval 77.0 to 310.0	165 mg/dL Interval 77.0 to 327.0

SECONDARY outcome

Timeframe: 4 months

Outcome measures

Measure	Leptin Therapy n=10 Participants Randomized to Leptin Therapy for 4 months.	Placebo n=9 Participants Randomized to Placebo for 4 months.
Body Weight	62.5 kg Standard Deviation 17.5	56.3 kg Standard Deviation 17.6

Adverse Events

Leptin Therapy

Serious events: 0 serious events

Other events: 2 other events

Deaths: 0 deaths

Placebo

Serious events: 0 serious events

Other events: 4 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Measure	Leptin Therapy n=13 participants at risk Randomized to Leptin Therapy for 4 months.	Placebo n=12 participants at risk Randomized to Placebo for 4 months.
Infections and infestations viral illness, sinusitis, backache	7.7% 1/13 • Number of events 1 • 4 month	0.00% 0/12 • 4 month
General disorders Headaches	7.7% 1/13 • Number of events 13 • 4 month	0.00% 0/12 • 4 month
Blood and lymphatic system disorders Pneumonia	0.00% 0/13 • 4 month	8.3% 1/12 • Number of events 1 • 4 month
Surgical and medical procedures Tonsillectomy	0.00% 0/13 • 4 month	8.3% 1/12 • Number of events 1 • 4 month
Surgical and medical procedures Right Ankle Pain	0.00% 0/13 • 4 month	8.3% 1/12 • Number of events 1 • 4 month
Eye disorders Blurry Vision and Epigastric pain	0.00% 0/13 • 4 month	8.3% 1/12 • Number of events 1 • 4 month

Additional Information

Dr. Abhimanyu Garg

UT Southwestern Medical Center

Phone: 214-648-2895

Email: abhimanyu.garg@utsouthwestern.edu

Results disclosure agreements

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

Restriction type: GT60