Table 1.
Summary of completed rapamycin clinical trials*
| Population | mTOR inhibitor dosing regimen | Duration | Study design | Primary endpoint | Primary endpoint met? | Reference |
|---|---|---|---|---|---|---|
| 11 men (28 ± 1 years) | Rapamycin, 6 mg single dose | Once | Randomized, double-blinded, placebo controlled, cross-over design | Increase in peripheral insulin sensitivity during insulin clamp plus AA infusion | Yes | Krebs et al. 2007 [15] |
| 15 men (29 ± 2 years) | Rapamycin 12 mg (12 × 1 mg tablets) single dose 2h before resistance exercise (RE) | Once | Acute bout of RE ± rapamycin | Difference in post-RE mixed-muscle protein synthesis rates | Yes | Drummond et al. 2009 [16] |
| 16 men (26 ± 1 years) | Rapamycin 16 mg (16 × 1 mg tablets) single dose 1h before blood flow restricted resistance exercise (RE) | Once | Acute bout of blood flow restriction RE ± rapamycin | Difference in post-RE mixed-muscle protein synthesis rates | Yes | Gunderman 2014 [17] |
| 218 adults age ≥ 65 years |
Everolimus 0.5 mg daily Everolimus 5 mg weekly Everolimus 20 mg weekly Placebo |
6 weeks | Phase 2, randomized, single observer blinded, placebo-controlled | Improvement in influenza vaccination response | Yes for 0.5 mg daily and 5 mg weekly | Mannick et al. 2014 [18] |
| 264 adults age ≥ 65 years |
Everolimus 0.1 mg daily Everolimus 0.5 mg daily BEZ235 10 mg daily BEZ235 10 mg+0.1 mg everolimus daily Placebo |
6 weeks | Phase 2a, randomized, double-blinded, placebo-controlled | Improvement in influenza vaccination response | Yes for BEZ235 10 mg + 0.1 mg everolimus daily | Mannick et al. 2018 [19] |
| 25 healthy adults 70–95 years |
Rapamycin 1 mg Placebo |
8 weeks | Phase 2, Randomized, double-blinded, placebo-controlled | Immunological response | No | Kraig et al., 2018 [20] |
| 36 adults with aging skin > 40 years |
Topical rapamycin (10 μM) Topical placebo |
8 months | Phase 2, randomized, placebo-controlled | Decreased p16-positive senescent cells in skin | Yes | Chung et al. 2019 [21] |
| 1024 adults age ≥ 65 years |
RTB101 10 mg daily Placebo |
16 weeks | Phase 3, randomized, placebo-controlled | Decrease in clinically symptomatic respiratory illness | No | Mannick et al. 2021 [22] |
*Studies are presented in chronological order from the publication date. AA, amino acid