LIBRETTO-001

Podcasts, YouTube videos and X posts in our index that name this trial. A report screens them, searches for more and cites what clinicians said when it is built.

Official title

A Phase 1/2 Study of Oral Selpercatinib (LOXO-292) in Patients With Advanced Solid Tumors, Including RET Fusion-Positive Solid Tumors, Medullary Thyroid Cancer, and Other Tumors With RET Activation (LIBRETTO-001)

Phase

Phase 1/Phase 2

Status

Active not recruiting

Conditions

Non-Small Cell Lung Cancer; Medullary Thyroid Cancer; Colon Cancer; Any Solid Tumor

Interventions

LOXO-292

Arms

• Phase 1: 20 mg Selpercatinib QD · Experimental
• Phase 1: 20 mg Selpercatinib BID · Experimental
• Phase 1: 40 mg Selpercatinib BID · Experimental
• Phase 1: 60 mg Selpercatinib BID · Experimental
• Phase 1: 160 mg Selpercatinib QD · Experimental
• Phase 1: 80 mg Selpercatinib BID · Experimental
• Phase 1: 120 mg Selpercatinib BID · Experimental
• Phase 1: 160 mg Selpercatinib BID · Experimental
• Phase 1: 200 mg Selpercatinib BID · Experimental
• Phase 1: 240 mg Selpercatinib BID · Experimental
• Phase 2, Cohort 1: RET Fusion Solid Tumor · Experimental
• Phase 2, Cohort 2: RET Fusion Solid Tumor Without Standard Therapy · Experimental
• Phase 2, Cohort 3: RET Mutant MTC · Experimental
• Phase 2, Cohort 4: RET Mutant MTC Without Standard Therapy · Experimental
• Phase 2, Cohort 5: Advanced RET Altered Solid Tumor · Experimental
• Phase 2, Cohort 6: RET Inhibitor-Discontinued Participants · Experimental

Enrollment

857

Primary outcomes

• Phase 1: Maximum Tolerated Dose (MTD)
• Phase 1: Recommended Phase 2 Dose (RP2D)
• Phase 2: Objective Response Rate (ORR) Based on Independent Review Committee (IRC) Assessment

Sponsor

Eli Lilly and Company with Loxo Oncology, Inc.

Start

May 2, 2017

Primary completion

Feb 14, 2025

Results posted

Yes

Registry record

NCT03157128 · updated Apr 16, 2026

• Gautschi O, Park K, Solomon BJ, Tomasini P, Loong HH, De Braud F, Goto K, Peterson P, Barker S, Liming K, Oxnard GR, Frimodt-Moller B, Drilon A. Selpercatinib in RET Fusion-Positive Non-Small Cell Lung Cancer: Final Safety and Efficacy, Including Overall Survival, From the LIBRETTO-001 Phase I/II Trial. J Clin Oncol. 2025 May 20;43(15):1758-1764. doi: 10.1200/JCO-24-02076. Epub 2025 Feb 21. PubMed 39983053
• Raez LE, Massey AC, Barker SS, Peterson PM, Liming K, Pennell NA. Long-term safety of selpercatinib for Rearranged during transfection (RET)-activated advanced solid tumors in LIBRETTO-001: differing patterns of adverse events over time. Oncologist. 2024 Dec 6;29(12):1068-1078. doi: 10.1093/oncolo/oyae282. PubMed 39471424
• Wirth LJ, Brose MS, Subbiah V, Worden F, Solomon B, Robinson B, Hadoux J, Tomasini P, Weiler D, Deschler-Baier B, Tan DSW, Maeda P, Lin Y, Singh R, Bayt T, Drilon A, Cassier PA. Durability of Response With Selpercatinib in Patients With RET-Activated Thyroid Cancer: Long-Term Safety and Efficacy From LIBRETTO-001. J Clin Oncol. 2024 Sep 20;42(27):3187-3195. doi: 10.1200/JCO.23.02503. Epub 2024 Aug 2. PubMed 39094065
• Deschler-Baier B, Krebs M, Kroiss M, Chatterjee M, Gundel D, Kestler C, Kerscher A, Kunzmann V, Appenzeller S, Maurus K, Rosenwald A, Bargou R, Gerhard-Hartmann E, Venkataramani V. Rapid response to selpercatinib in RET fusion positive pancreatic neuroendocrine carcinoma confirmed by smartwatch. NPJ Precis Oncol. 2024 Jul 31;8(1):167. doi: 10.1038/s41698-024-00659-x. PubMed 39085487
• Deschler-Baier B, Konda B, Massarelli E, Hu MI, Wirth LJ, Xu X, Wright J, Clifton-Bligh RJ. Clinical Activity of Selpercatinib in RET-mutant Pheochromocytoma. J Clin Endocrinol Metab. 2025 Feb 18;110(3):e600-e606. doi: 10.1210/clinem/dgae283. PubMed 38661071
• Subbiah V, Burris HA 3rd, Kurzrock R. Revolutionizing cancer drug development: Harnessing the potential of basket trials. Cancer. 2024 Jan;130(2):186-200. doi: 10.1002/cncr.35085. Epub 2023 Nov 7. PubMed 37934000
• Duke ES, Bradford D, Marcovitz M, Amatya AK, Mishra-Kalyani PS, Nguyen E, Price LSL, Fourie Zirkelbach J, Li Y, Bi Y, Kraft J, Dorff SE, Scepura B, Stephenson M, Ojofeitimi I, Nair A, Han Y, Tezak Z, Lemery SJ, Pazdur R, Larkins E, Singh H. FDA Approval Summary: Selpercatinib for the Treatment of Advanced RET Fusion-Positive Solid Tumors. Clin Cancer Res. 2023 Sep 15;29(18):3573-3578. doi: 10.1158/1078-0432.CCR-23-0459. PubMed 37265412
• Murciano-Goroff YR, Falcon CJ, Lin ST, Chacko C, Grimaldi G, Liu D, Wilhelm C, Iasonos A, Drilon A. Central Nervous System Disease in Patients With RET Fusion-Positive NSCLC Treated With Selpercatinib. J Thorac Oncol. 2023 May;18(5):620-627. doi: 10.1016/j.jtho.2023.01.008. Epub 2023 Jan 16. PubMed 36657661
• Drilon A, Subbiah V, Gautschi O, Tomasini P, de Braud F, Solomon BJ, Shao-Weng Tan D, Alonso G, Wolf J, Park K, Goto K, Soldatenkova V, Szymczak S, Barker SS, Puri T, Bence Lin A, Loong H, Besse B. Selpercatinib in Patients With RET Fusion-Positive Non-Small-Cell Lung Cancer: Updated Safety and Efficacy From the Registrational LIBRETTO-001 Phase I/II Trial. J Clin Oncol. 2023 Jan 10;41(2):385-394. doi: 10.1200/JCO.22.00393. Epub 2022 Sep 19. PubMed 36122315
• Subbiah V, Wolf J, Konda B, Kang H, Spira A, Weiss J, Takeda M, Ohe Y, Khan S, Ohashi K, Soldatenkova V, Szymczak S, Sullivan L, Wright J, Drilon A. Tumour-agnostic efficacy and safety of selpercatinib in patients with RET fusion-positive solid tumours other than lung or thyroid tumours (LIBRETTO-001): a phase 1/2, open-label, basket trial. Lancet Oncol. 2022 Oct;23(10):1261-1273. doi: 10.1016/S1470-2045(22)00541-1. Epub 2022 Sep 12. PubMed 36108661
• Rolfo C, Hess LM, Jen MH, Peterson P, Li X, Liu H, Lai Y, Sugihara T, Kiiskinen U, Vickers A, Summers Y. External control cohorts for the single-arm LIBRETTO-001 trial of selpercatinib in RET+ non-small-cell lung cancer. ESMO Open. 2022 Aug;7(4):100551. doi: 10.1016/j.esmoop.2022.100551. Epub 2022 Aug 2. PubMed 35930972
• Subbiah V, Gainor JF, Oxnard GR, Tan DSW, Owen DH, Cho BC, Loong HH, McCoach CE, Weiss J, Kim YJ, Bazhenova L, Park K, Daga H, Besse B, Gautschi O, Rolfo C, Zhu EY, Kherani JF, Huang X, Kang S, Drilon A. Intracranial Efficacy of Selpercatinib in RET Fusion-Positive Non-Small Cell Lung Cancers on the LIBRETTO-001 Trial. Clin Cancer Res. 2021 Aug 1;27(15):4160-4167. doi: 10.1158/1078-0432.CCR-21-0800. Epub 2021 Jun 4. PubMed 34088726
• Wirth LJ, Sherman E, Robinson B, Solomon B, Kang H, Lorch J, Worden F, Brose M, Patel J, Leboulleux S, Godbert Y, Barlesi F, Morris JC, Owonikoko TK, Tan DSW, Gautschi O, Weiss J, de la Fouchardiere C, Burkard ME, Laskin J, Taylor MH, Kroiss M, Medioni J, Goldman JW, Bauer TM, Levy B, Zhu VW, Lakhani N, Moreno V, Ebata K, Nguyen M, Heirich D, Zhu EY, Huang X, Yang L, Kherani J, Rothenberg SM, Drilon A, Subbiah V, Shah MH, Cabanillas ME. Efficacy of Selpercatinib in RET-Altered Thyroid Cancers. N Engl J Med. 2020 Aug 27;383(9):825-835. doi: 10.1056/NEJMoa2005651. PubMed 32846061
• Drilon A, Oxnard GR, Tan DSW, Loong HHF, Johnson M, Gainor J, McCoach CE, Gautschi O, Besse B, Cho BC, Peled N, Weiss J, Kim YJ, Ohe Y, Nishio M, Park K, Patel J, Seto T, Sakamoto T, Rosen E, Shah MH, Barlesi F, Cassier PA, Bazhenova L, De Braud F, Garralda E, Velcheti V, Satouchi M, Ohashi K, Pennell NA, Reckamp KL, Dy GK, Wolf J, Solomon B, Falchook G, Ebata K, Nguyen M, Nair B, Zhu EY, Yang L, Huang X, Olek E, Rothenberg SM, Goto K, Subbiah V. Efficacy of Selpercatinib in RET Fusion-Positive Non-Small-Cell Lung Cancer. N Engl J Med. 2020 Aug 27;383(9):813-824. doi: 10.1056/NEJMoa2005653. PubMed 32846060