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. Author manuscript; available in PMC: 2022 Feb 16.
Published in final edited form as: J Am Coll Cardiol. 2021 Feb 16;77(6):745–760. doi: 10.1016/j.jacc.2020.11.069

TABLE 2.

Clinical Trials Investigating the Effect of Leptin Administration in Common Obesity

First Author (Year) (Ref. #) Study Design Subjects N Type of Leptin Duration Dose Leptin Weight Loss Other Effects
Heymsfield et al. (1999) (116) RCT, double blind placebo controlled Overweight/obese BMI 27.6–36 kg/m2 12
6
8
13
8
r-met hu leptin 24 weeks Placebo
0.01 mg/kg/day
0.03 mg/kg/day
0.10 mg/kg/day
0.30 mg/kg/day
+hypocaloric diet (−500 kcal)
−1.3 kg/m2 (4.9 kg)
−0.7 kg/m2 (5.4 kg)
−1.4 kg/m2 (4.1 kg)
−2.4 kg/m2 (5.5 kg)
−7.1 kg/m2 (8.5 kg)
• No significant differences in weight loss between treatment groups reported
• Linear regression models: reduction of weight and fat mass but not fat free mass with increasing dose
Moon et al. (2011) (36) RCT, double blind placebo controlled Obese with T2D BMI 33.2 ± 3.8 kg/m2 71 r-met hu leptin 16 weeks Placebo
10 mg twice daily
−0.5 ± 0.2 kg/m2
−0.7 ± 0.1 kg/m2
=> No significant effect of leptin on weight loss
• HbA1c slightly reduced (p = 0.03) Placebo: 8.01 ± 0.93%; leptin: 7.96 ± 1.12%
• No changes in inflammatory markers
• All metreleptin signaling pathways saturable at 50 ng/ml of free leptin
Mittendorfer et al. (2011) (119) RCT, placebo controlled Obese with newly diagnosed T2D 18 r-met hu leptin 14 days Placebo
15 mg twice daily
40 mg twice daily
=> No changes in BMI in all groups • No effect on total body mass, fat mass, fat free mass, % of body fat
• No effects on insulin sensitivity on hyperinsulinemic-euglycemic clamp
Lejeune et al. (2003) (123) RCT, double blind placebo controlled Overweight/obese BMI 25–32 kg/m2 24 PEG-OB 6 weeks of treatment with VLCD followed by 8 weeks of follow-up VLCD + placebo 80 mg/week for 6 weeks 0–6 weeks of treatment:
−3.6 kg/m2
−4.3 kg/m2
=> Reduction (p = 0.049) of body weight with leptin
Follow-up:
+0.6 kg/m2
+1.1 kg/m2
=> Increase of body weight in subjects previously treated with leptin during weight maintenance
• 0–6 weeks of treatment: no effect of leptin on fat mass, % of body fat, fat-free mass, REE, RQ, hunger
• Follow up: no effect of leptin on total body mass, fat mass, fat-free mass, % of fat mass, hunger
Hukshorn et al. (2000) (118) RCT, double blind placebo controllec Overweight/obese BMI ≥27 kg/m2 30 PEG-OB 12 weeks Placebo 20 mg/week +hypocaloric diet (−500 kcal) −6.4 kg
−4.3 kg
=> No changes in weight
• No effect on body fat %, 24-h EE, SMR, RQ, appetite (ad libitum evening meal)
• No effect on glucose, insulin, glucose disappearance rate, FFAs, glycerol, TGs, TC, LDL-C, HDL-C
Hukshorn et al. (2002) (120) RCT, double blind placebo controlled Overweight/obese BMI ≥27 kg/m2 + loss of ≥1.75 kg after a 4-week lead-in diet period 28 PEG-OB 4 weeks of diet followed by 8 weeks of treatment Placebo 60 mg/week −3.8 kg
−4.8 kg
=> No changes in body weight
• No effects on glucose, insulin, HOMA-IR, FFAs, glycerol, TGs, TC
• No effects on inflammatory markers (sTNF, CRP)
Zelissen et al. (2005) (121) RCT, double blind placebo controlled Overweight/obese BMI 27.5–37.5 kg/m2 + loss of 0–5 kg and BMI >25 kg/m2 after a 3 week lead-in diet period 284 r-met hu leptin 3 weeks of diet followed by 12 weeks of treatment Placebo
10 mg every morning
10 mg every night
10 mg twice daily
−2.6 kg
−2.8 kg
−2.7 kg
−3.4 kg
=> No changes in body weight
• No change in SBP, DBP, heart rate, TGs, TCs, LDL-C, HDL-C, fasting glucose, fasting insulin
Rosenbaum et al. (2005) (125) Single-arm open label stud 3 nonobese, 7 obese 10 r-met hu leptin Diet until 10% weight loss achieved followed by 5 weeks treatment Dose calculated to maintain blood leptin concentrations at levels prior to weight loss −2.1 kg compared with treatment start • 1.0 kg fat mass loss and 1.1 kg free fat mass loss compared with treatment start
• Returned of nonresting EE, SNS tone to pre-weight loss levels
Rosenbaum et al. (2008) (133)
Kissileff etal (2012) (124)
Single-blind placebo-controlled crossover study design Obese subjects 10 r-met hu Leptin Diet until 10% weight loss achieved followed by 5 weeks treatment Placebo Leptin dose calculated to maintain blood leptin concentrations at levels prior to weight loss No significant changes in weight between leptin and placebo (−1.8-kg difference). • EE and VAS for satiation were lower in the placebo group than in the leptin group
• Leptin reverses changes in neural activity in response to visual food stimuli due to weight loss
Korner et al. (2013) (122) RCT double-blind placebo-controlled crossover study design Post-RYGB, weight-stable, relative hypoleptinemic subjects 27 r-met hu Leptin 16 weeks Placebo
0.05 mg/kg twice daily
No differences in body weight • No effect of leptin on fat mass, REE, thyroid hormones, cortisol levels
Chrysafi et al. (2020) (34) Crossover
study
design
5 obese men, 5 lean men, 5 lean women 15 r-met
hu Leptin
(a) 24 h (fed state)
(b) 72 h (fasting) Leptin
administrated at 8 AM/day [1 dose in (a), 4 doses in (b)]
0.01 mg/kg
0.1 mg/kg
0.3 mg/kg (total 9 visits for each subject)
No differences in body weight with escalating leptin doses • No effect of leptin on energy expenditure, SNS activity, adrenal hormones, lipid utilization.

CRP = C-reactive protein; DBP = diastolic blood pressure; EE = energy expenditure; FFA = free fatty acid; HbA1c, glycosylated hemoglobin; HDL-C = high-density lipoprotein cholesterol; HOMA-IR, Homeostatic Model Assessment of Insulin Resistance; LDL-C = low-density lipoprotein cholesterol; PEG-OB = •••; RCT = randomized clinical trial; REE = resting energy expenditure; RQ = respiratory quotient; RYGB = Roux-en-Y gastric bypass; SBP = systolic blood pressure; SMR = sleeping metabolic rate; sTNF, soluble tumor necrosis factor; T2D, type 2 diabetes mellitus; TC = total cholesterol; TG = triglyceride; VAS = •••; VLCD = very low-caloric diet (provided 2.1 MJ/day).