Tesamorelin clinical trials

• NCT00123253·Phase 3·HIV: abdominal fatHigh evidence

  Phase 3 Study 1

  Phase 3 placebo-controlled trial of tesamorelin (TH9507) in HIV with excess abdominal fat — FDA label "Study 1"

  Enrolled

  412

  Control

  Placebo

  Duration

  52 weeks

  Published

  2007

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  Design. Multicenter, randomized (2:1), double-blind, placebo-controlled, parallel; 26-week main phase followed by a 26-week re-randomized extension

  Primary endpoint. Percent change from baseline in visceral adipose tissue (CT) at week 26

  Population. HIV: abdominal fat (the approved indication's population)

  Status. Completed, 2005–2007

  Results as reported

  Endpoint	Tesamorelin	Control	Difference / p	When
  Visceral adipose tissue, % change%	-15.2%	+5.0%	p <0.001	26 weeksPMID 18057338
  Triglycerides changemg/dL	-50	+9	p <0.001	26 weeksPMID 18057338
  Total cholesterol:HDL ratio changeratio	-0.31	+0.21	p <0.001	26 weeksPMID 18057338
  IGF-I change%	+81.0%	-5.0%	p <0.001	26 weeksPMID 18057338
  Glycemic measures	No significant differences observed	—	—	26 weeksPMID 18057338
  VAT change, T-T group%	-18% (sustained over 52 weeks)	VAT reaccumulated after discontinuation (T-P)	p <0.001 vs baseline	52 weeksPMID 18690162

  Limitations

  • More patients in the tesamorelin group withdrew because of an adverse event (NEJM abstract)
  • VAT reaccumulated after discontinuation (AIDS 2008 abstract)
  • Industry-sponsored (Theratechnologies)

  Evidence grade: High

  • Multicentre, randomized 2:1, double-blind, placebo-controlled, n = 412
  • Industry-sponsored; more withdrawals for adverse events on tesamorelin

  Primary sources

  • ClinicalTrials.gov NCT00123253 (also registered as NCT00435136)
  • Falutz J et al. N Engl J Med. 2007;357:2359-70 PMID 18057338 · doi:10.1056/NEJMoa072375
  • Falutz J et al. AIDS. 2008;22:1719-28 PMID 18690162 · doi:10.1097/QAD.0b013e32830a5058
• NCT00608023·Phase 3·HIV: abdominal fatHigh evidence

  Phase 3 Study 2 (extension)

  Placebo-controlled safety extension of the second phase 3 trial — FDA label "Study 2"

  Enrolled

  263

  Control

  Placebo (re-randomized)

  Duration

  26 weeks

  Published

  2010

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  Design. Multicenter, randomized, quadruple-masked, placebo-controlled extension (T-T, T-P, P-T arms)

  Primary endpoint. Change from baseline in fasting blood glucose and 2-h OGTT glucose at week 52 (registry)

  Population. HIV: abdominal fat (the approved indication's population)

  Status. Completed, 2007–2008

  Results as reported

  Endpoint	Tesamorelin	Control	Difference / p	When
  Fasting blood glucose change (registry)mg/dL	T-T: 0 (SD 16)	T-P: -2 (SD 34)	p >0.05 (ANCOVA)	Week 52ClinicalTrials.gov posted results
  2-h OGTT glucose change (registry)mg/dL	T-T: -2 (SD 38)	T-P: 2 (SD 35)	p >0.05 (ANCOVA)	Week 52ClinicalTrials.gov posted results
  VAT change, 6-month efficacy phase (parent study, 404 patients)% / cm2	-10.9% (-21 cm2)	-0.6% (-1 cm2)	p <0.0001	26 weeksPMID 20101189
  VAT in patients continuing tesamorelin%	approximately -18%	—	p <0.001	12 monthsPMID 20101189

  Limitations

  • Initial VAT improvements were 'rapidly lost' in those switched to placebo (PMID 20101189)
  • Registry primary endpoints are glucose safety measures, not VAT

  Evidence grade: High

  • Parent trial multicentre, randomized, double-blind, placebo-controlled (404 patients)
  • Registry primary endpoints are glucose safety measures, not visceral fat

  Primary sources

  • ClinicalTrials.gov NCT00608023
  • Falutz J et al. J Acquir Immune Defic Syndr. 2010;53:311-22 PMID 20101189 · doi:10.1097/QAI.0b013e3181cbdaff
• NCT01263717·Not applicable·HIV: abdominal fatModerate evidence

  Stanley 2014 (JAMA)

  Tesamorelin and visceral and liver fat in HIV with abdominal fat accumulation

  Enrolled

  54

  Control

  Placebo

  Duration

  26 weeks

  Published

  2014

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  Design. Randomized, quadruple-masked, placebo-controlled, parallel

  Primary endpoint. Change in visceral adipose tissue and liver fat at 6 months

  Population. HIV: abdominal fat (the approved indication's population)

  Status. Completed, 2010–2014

  Results as reported

  Endpoint	Tesamorelin	Control	Difference / p	When
  VAT changecm2	-34 (95% CI -53 to -15)	8 (95% CI -14 to 30)	-42; 95% CI -71 to -14; p = 0.005	6 monthsPMID 25038357
  Liver fat, median change in lipid-to-water %lipid-to-water %	-2.0% (IQR -6.4 to 0.1)	0.9% (IQR -0.6 to 3.7)	net -2.9%; p = 0.003	6 monthsPMID 25038357
  Fasting glucose changemg/dL	9 (95% CI 5-13)	2 (95% CI -3 to 8)	7; 95% CI 1-14; p = 0.03	2 weeksPMID 25038357
  Fasting glucose changemg/dL	4 (95% CI -2 to 10)	2 (95% CI -4 to 7)	2; 95% CI -6 to 10; p = 0.72 (overall across time points)	6 monthsPMID 25038357

  Limitations

  • Authors call it a preliminary study; 50 randomized, 48 treated
  • Single center

  Evidence grade: Moderate

  • Randomized, masked, placebo-controlled
  • Single centre; described by its authors as preliminary; 50 randomized (54 registered)

  Primary sources

  • ClinicalTrials.gov NCT01263717
  • Stanley TL et al. JAMA. 2014;312:380-9 PMID 25038357 · doi:10.1001/jama.2014.8334
• NCT02196831·Not applicable·HIV: liver fatModerate evidence

  Stanley 2019 (Lancet HIV)

  Tesamorelin and liver fat (NAFLD) in HIV: randomized, double-blind, two-centre trial

  Enrolled

  61

  Control

  Placebo

  Duration

  52 weeks

  Published

  2019

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  Design. Randomized (1:1), double-blind (registry: quadruple), placebo-controlled, two sites, followed by a 6-month open-label phase

  Primary endpoint. Change in hepatic fat fraction (1H-MRS) between baseline and 12 months

  Population. HIV: liver fat (outside the approved indication)

  Status. Completed, 2015–2019

  Results as reported

  Endpoint	Tesamorelin	Control	Difference / p	When
  Hepatic fat fraction, absolute effect sizepercentage points HFF	—	—	-4.1%; 95% CI -7.6 to -0.7; p = 0.018	12 monthsPMID 31611038
  Hepatic fat fraction, relative reduction from baseline%	—	—	-37%; 95% CI -67 to -7; p = 0.016	12 monthsPMID 31611038
  Proportion with HFF <5%% of participants	35%	4%	p = 0.0069	12 monthsPMID 31611038
  Fasting glucose and HbA1c	Not different between groups	—	—	12 monthsPMID 31611038
  Integrase-inhibitor subgroup: VAT median changecm2	-25 [IQR -93, -2]	14 [IQR 3, 41]	p = 0.001	12 monthsPMID 38905488 (post hoc subgroup, 38 at baseline; 15 vs 16 completers)
  Integrase-inhibitor subgroup: hepatic fat median change%	-4.2% [IQR -12.3, -2.7]	-0.5% [IQR -3.9, 2.7]	p = 0.01	12 monthsPMID 38905488

  Limitations

  • 61 enrolled (30 vs 30 received study drug)
  • More localized injection-site complaints with tesamorelin (none judged serious)
  • Authors: further studies needed on long-term histology effects
  • Registry results table reports mean HFF change -4.7 (SD 6.6) vs 0.0 (SD 4.1); the publication reports a model-based effect size

  Evidence grade: Moderate

  • Randomized, double-blind, placebo-controlled, two centres
  • Small (61 enrolled); population outside the approved indication

  Primary sources

  • ClinicalTrials.gov NCT02196831
  • Stanley TL et al. Lancet HIV. 2019;6:e821-e830 PMID 31611038 · doi:10.1016/S2352-3018(19)30338-8
  • Russo SC et al. AIDS. 2024;38:1758-1764 PMID 38905488 · doi:10.1097/QAD.0000000000003965
• NCT00675506·Phase 2·Obesity (no HIV)Moderate evidence

  Makimura 2012 (JCEM)

  Tesamorelin in abdominal obesity with reduced GH secretion (no HIV)

  Enrolled

  60

  Control

  Placebo

  Duration

  52 weeks

  Published

  2012

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  Design. Randomized, double-blind (registry: quadruple), placebo-controlled, parallel

  Primary endpoint. Change in visceral adipose tissue (CT) at 6 and 12 months

  Population. Obesity (no HIV) (outside the approved indication)

  Status. Completed, 2008–2012

  Results as reported

  Endpoint	Tesamorelin	Control	Difference / p	When
  VAT changecm2	-16 ± 9	19 ± 9	-35; 95% CI -58, -12; p = 0.003	12 monthsPMID 23015655
  Carotid intima-media thickness changemm	-0.03 ± 0.01	0.01 ± 0.01	-0.04; 95% CI -0.07, -0.01; p = 0.02	12 monthsPMID 23015655
  Triglycerides changemg/dL	-26 ± 16	12 ± 8	-37; 95% CI -67, -7; p = 0.02	12 monthsPMID 23015655
  Abdominal SAT changecm2	-6 ± 6	3 ± 11	-10; 95% CI -32, +13; p = 0.40	12 monthsPMID 23015655
  IGF-I changeμg/L	86 ± 21	-6 ± 8	92; 95% CI +52, +132; p <0.0001	12 monthsPMID 23015655

  Limitations

  • Small (n=60), single center
  • Population outside the approved indication

  Evidence grade: Moderate

  • Randomized, double-blind, placebo-controlled
  • Small (n = 60), single centre; population outside the approved indication

  Primary sources

  • ClinicalTrials.gov NCT00675506
  • Makimura H et al. J Clin Endocrinol Metab. 2012;97:4769-79 PMID 23015655 · doi:10.1210/jc.2012-2794
• NCT03375788·Phase 2·Obesity (no HIV)Low evidence

  NAFLD in obesity (registry results)

  GHRH analog and nonalcoholic fatty liver disease in obesity (no HIV)

  Enrolled

  51

  Control

  Placebo

  Duration

  52 weeks

  Published

  Not published

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  Design. Randomized, quadruple-masked, placebo-controlled, parallel

  Primary endpoint. Liver fat content, change from baseline to 12 months

  Population. Obesity (no HIV) (outside the approved indication)

  Status. Completed, 2019–2025

  Results as reported

  Endpoint	Tesamorelin	Control	Difference / p	When
  Hepatic fat fraction, median percent change (registry; 18 vs 16 analysed)percent change in hepatic fat fraction	-5.3 (IQR -13.0 to -1.4)	3.6 (IQR -6.2 to 7.9)	p = not reported in the registry record	12 monthsClinicalTrials.gov posted results

  Limitations

  • No peer-reviewed publication found in PubMed searches (2024-2026)
  • No statistical analysis posted for the primary outcome
  • Registry group description says a different formulation ('F4') was used

  Evidence grade: Low

  • Randomized, masked, placebo-controlled design
  • No peer-reviewed publication; no statistical analysis posted; 34 analysed; a different formulation ("F4") was used

  Primary sources

  • ClinicalTrials.gov NCT03375788
• NCT02572323·Phase 2·HIV: cognitionLow evidence

  Ellis 2025 (J Infect Dis)

  Tesamorelin and neurocognitive impairment in HIV with abdominal obesity

  Enrolled

  73

  Control

  Standard of care / deferred treatment

  Duration

  24 weeks

  Published

  2025

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  Design. Randomized 3:2. Publication: open-label, tesamorelin vs standard of care. Registry: crossover model, single masking, immediate vs deferred groups

  Primary endpoint. Change in neurocognitive performance (z-score) at 6 months (24 weeks)

  Population. HIV: cognition (outside the approved indication)

  Status. Completed, 2017–2023

  Results as reported

  Endpoint	Tesamorelin	Control	Difference / p	When
  Change in neurocognitive performancez-score	0.146 (95% CI -0.002 to 0.294; P=0.060 within group)	0.103 (95% CI -0.095 to 0.301; P=0.295 within group)	p = 0.673 (between groups)	6 monthsPMID 39813152; same values in registry
  Waist circumferencecm	—	—	median difference -2.7; p = 0.015	6 monthsPMID 39813152

  Limitations

  • Authors note insufficient power and no placebo arm
  • Between-group cognitive difference not significant

  Evidence grade: Low

  • No placebo arm (publication: open-label vs standard of care)
  • Authors note insufficient power; registry and publication describe masking differently

  Primary sources

  • ClinicalTrials.gov NCT02572323
  • Ellis RJ et al. J Infect Dis. 2025;231:1230-1238 PMID 39813152 · doi:10.1093/infdis/jiaf012
• NCT00257712·Phase 2·Older adults / MCIModerate evidence

  Baker 2012 (SMART)

  GHRH and cognition in healthy older adults and mild cognitive impairment

  Enrolled

  151

  Control

  Placebo

  Duration

  20 weeks

  Published

  2012

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  Design. Randomized, double-blind, placebo-controlled, parallel; 10-week washout

  Primary endpoint. Composite cognitive outcomes (executive function, verbal memory, visual memory)

  Population. Older adults / MCI (outside the approved indication)

  Status. Completed, 2006–2011. Publication reports 152 randomized (66 with MCI) and 137 completers

  Results as reported

  Endpoint	Tesamorelin	Control	Difference / p	When
  Cognition, intent-to-treat (ANOVA)composite	—	—	favorable GHRH effect; p = 0.03	20 weeksPMID 22869065
  Cognition, completer analysiscomposite	—	—	p = 0.002	20 weeksPMID 22869065
  Executive functioncomposite	—	—	p = 0.005	20 weeksPMID 22869065
  IGF-1%	+117%	—	p = printed as 'P.001' in the PubMed abstract	20 weeksPMID 22869065
  Mild adverse events reported% of participants	68%	36%	—	20 weeksPMID 22869065

  Limitations

  • Single center
  • Authors call for longer-duration trials
  • Population outside the approved indication

  Evidence grade: Moderate

  • Randomized, double-blind, placebo-controlled, n ≈ 152
  • Single centre, 20 weeks; population outside the approved indication

  Primary sources

  • ClinicalTrials.gov NCT00257712
  • Baker LD et al. Arch Neurol. 2012;69:1420-9 PMID 22869065 · doi:10.1001/archneurol.2012.1970
  • Friedman SD et al. JAMA Neurol. 2013;70:883-90 PMID 23689947 · doi:10.1001/jamaneurol.2013.1425
• NCT02553603·Phase 1·Older adults / MCILow evidence

  Stewart 2026 (pilot)

  GHRH and cognition / brain connectivity, normal cognition to MCI (pilot)

  Enrolled

  22

  Control

  Placebo

  Duration

  10 weeks

  Published

  2026

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  Design. Randomized, triple-masked, placebo-controlled pilot

  Primary endpoint. Cognitive function (SLUMS), brain perfusion (fMRI) and brain morphology (MRI) at 10 weeks

  Population. Older adults / MCI (outside the approved indication)

  Status. Completed, 2017–2019

  Results as reported

  Endpoint	Tesamorelin	Control	Difference / p	When
  All study measures (body composition, fatigue, sleep, physical performance, glucose tolerance, cognition, brain morphometry and connectivity)	—	—	not directly linked with significant changes	10 weeksPMID 42382101 (only exploratory machine-learning findings reported beyond this)

  Limitations

  • Pilot, n=22
  • No significant changes on prespecified measures

  Evidence grade: Low

  • Randomized, triple-masked, placebo-controlled
  • Pilot, n = 22, 10 weeks; no significant changes on prespecified measures

  Primary sources

  • ClinicalTrials.gov NCT02553603
  • Stewart CE et al. eNeurologicalSci. 2026;44:100616 PMID 42382101 · doi:10.1016/j.ensci.2026.100616
• NCT01264497·Phase 2·Type 2 diabetesModerate evidence

  Clemmons 2017 (type 2 diabetes)

  12-week safety and metabolic study of tesamorelin in stable type 2 diabetes

  Enrolled

  55

  Control

  Placebo

  Duration

  12 weeks

  Published

  2017

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  Design. Randomized, double-blind, placebo-controlled, parallel; three arms (placebo and two tesamorelin dose levels)

  Primary endpoint. Change from baseline in relative insulin response (OGTT)

  Population. Type 2 diabetes (outside the approved indication)

  Status. Completed, 2002–2002. Publication reports 53 patients

  Results as reported

  Endpoint	Tesamorelin	Control	Difference / p	When
  Relative insulin response	—	—	No significant differences between groups	12 weeksPMID 28617838
  Fasting glucose and HbA1c	—	—	Not significantly different at Week 12	12 weeksPMID 28617838

  Limitations

  • Short (12 weeks) and small
  • Industry-sponsored; published about 15 years after completion

  Evidence grade: Moderate

  • Randomized, double-blind, placebo-controlled
  • Small (53 published / 55 registered), 12 weeks; published ~15 years after completion

  Primary sources

  • ClinicalTrials.gov NCT01264497
  • Clemmons DR et al. PLoS One. 2017;12:e0179538 PMID 28617838 · doi:10.1371/journal.pone.0179538
• NCT00850564·Not applicable·Healthy volunteersVery low evidence

  Stanley 2011 (healthy men)

  Short-term GHRH analog, GH pulsatility and insulin sensitivity in healthy men

  Enrolled

  15

  Control

  None (baseline comparison)

  Duration

  2 weeks

  Published

  2011

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  Design. Single-group, open-label, before/after with withdrawal

  Primary endpoint. Mean overnight GH after 2 weeks

  Population. Healthy volunteers (outside the approved indication)

  Status. Completed, 2009–2010. Publication reports 13 men

  Results as reported

  Endpoint	Tesamorelin	Control	Difference / p	When
  Mean overnight GH changeμg/L	+0.5 ± 0.1	—	p = 0.004	2 weeksPMID 20943777
  IGF-I changeμg/L	+181 ± 22	—	p <0.0001	2 weeksPMID 20943777
  Insulin-stimulated glucose uptake (clamp)	not significantly affected	—	p = 0.61	2 weeksPMID 20943777

  Limitations

  • Uncontrolled, n=13
  • Mechanistic physiology study

  Evidence grade: Very low

  • Uncontrolled before/after design, 13 analysed
  • Mechanistic physiology study, not an outcome trial

  Primary sources

  • ClinicalTrials.gov NCT00850564
  • Stanley TL et al. J Clin Endocrinol Metab. 2011;96:150-8 PMID 20943777 · doi:10.1210/jc.2010-1587

Trials not listed: registered studies with no published or posted results (several are still recruiting, and some were terminated early), and one registry entry labelled a mock study. See the inclusion criteria.