Clinical Study to Evaluate Mavacamten (MYK-461) in Adults With Symptomatic Obstructive Hypertrophic Cardiomyopathy

Summary

This is a multicenter, international, double-blind study of the administration of mavacamten in participants with symptomatic obstructive HCM (oHCM). Approximately 220 participants will be randomized to receive placebo or mavacamten.

Timeline

Start

2018-05-29

Primary completion

2020-03-14

Completion

2020-05-06

Publications

• Saberi S, Naidu SS, Olivotto I, Hagege AA, Zhong Y, Lam J, Wyrwich KW, Fine JT, Dolan C, Allen V, Sehnert AJ, Reaney M. Impact of Mavacamten on Disease-Related Symptoms in Patients With Obstructive Hypertrophic Cardiomyopathy: HCMSQ Outcomes in EXPLORER-HCM. J Card Fail. 2026 Jun 6:S1071-9164(26)00257-5. doi: 10.1016/j.cardfail.2026.03.036. Online ahead of print.
• Wang A, Lakdawala NK, Abraham TP, Nilles EK, Wojdyla DM, Owens AT, Bach RG, Saberi S, Sehnert A, Cresci S. Association Between age or Duration of Diagnosis in Obstructive Hypertrophic Cardiomyopathy and Response to Mavacamten Treatment: Exploratory Analysis of the EXPLORER-HCM Trial. J Card Fail. 2025 Jun;31(6):901-911. doi: 10.1016/j.cardfail.2024.10.449. Epub 2024 Dec 7.
• Arnold SV, Gosch KL, Dolan C, Fine JT, Masri A, Saberi S, Wang A, Elliott PM, Hegde SM, Lam J, Sehnert AJ, Cresci S, Bach RG, Spertus JA. Association of Echocardiographic Parameters and Health Status in Patients With Obstructive Hypertrophic Cardiomyopathy: Insights From EXPLORER-HCM. Circulation. 2024 Nov 5;150(19):1560-1562. doi: 10.1161/CIRCULATIONAHA.123.067470. Epub 2024 Nov 4. No abstract available.
• Sauer AJ, Sherrod CF, Gosch KL, Arnold SV, Reaney M, Zhong Y, Lam J, Wyrwich KW, Spertus JA. The Psychometric Performance of the Kansas City Cardiomyopathy Questionnaire-12 in Symptomatic Obstructive Hypertrophic Cardiomyopathy. J Card Fail. 2025 May;31(5):813-820. doi: 10.1016/j.cardfail.2024.09.010. Epub 2024 Sep 28.
• Sherrod CF 4th, Spertus JA, Gosch KL, Wang A, Elliott PM, Lakdawala NK, Reaney M, Zhong Y, Lam J, Wyrwich KW, Sauer AJ. The Kansas City Cardiomyopathy Questionnaire in Relation to New York Heart Association Class. J Card Fail. 2025 Feb;31(2):481-484. doi: 10.1016/j.cardfail.2024.08.061. Epub 2024 Sep 28.
• Garcia-Pavia P, Oreziak A, Masri A, Barriales-Villa R, Abraham TP, Owens AT, Jensen MK, Wojakowski W, Seidler T, Hagege A, Lakdawala NK, Wang A, Wheeler MT, Choudhury L, Balaratnam G, Shah A, Fox S, Hegde SM, Olivotto I. Long-term effect of mavacamten in obstructive hypertrophic cardiomyopathy. Eur Heart J. 2024 Dec 16;45(47):5071-5083. doi: 10.1093/eurheartj/ehae579.
• Merali S, Salinger DH, Palmisano M, Sehnert AJ, Thanneer N, Back H, Seroogy JD, Gretler DD, Roy A, Perera V. Recommendation of mavacamten posology by model-based analyses in adults with obstructive hypertrophic cardiomyopathy. CPT Pharmacometrics Syst Pharmacol. 2024 Sep;13(9):1448-1461. doi: 10.1002/psp4.13138. Epub 2024 May 2.
• Cresci S, Bach RG, Saberi S, Owens AT, Spertus JA, Hegde SM, Lakdawala NK, Nilles EK, Wojdyla DM, Sehnert AJ, Wang A. Effect of Mavacamten in Women Compared With Men With Obstructive Hypertrophic Cardiomyopathy: Insights From EXPLORER-HCM. Circulation. 2024 Feb 13;149(7):498-509. doi: 10.1161/CIRCULATIONAHA.123.065600. Epub 2023 Nov 14.
• Wang A, Spertus JA, Wojdyla DM, Abraham TP, Nilles EK, Owens AT, Saberi S, Cresci S, Sehnert A, Lakdawala NK. Mavacamten for Obstructive Hypertrophic Cardiomyopathy With or Without Hypertension: Post-Hoc Analysis of the EXPLORER-HCM Trial. JACC Heart Fail. 2024 Mar;12(3):567-579. doi: 10.1016/j.jchf.2023.07.030. Epub 2023 Oct 18.
• Wheeler MT, Olivotto I, Elliott PM, Saberi S, Owens AT, Maurer MS, Masri A, Sehnert AJ, Edelberg JM, Chen YM, Florea V, Malhotra R, Wang A, Oreziak A, Myers J. Effects of Mavacamten on Measures of Cardiopulmonary Exercise Testing Beyond Peak Oxygen Consumption: A Secondary Analysis of the EXPLORER-HCM Randomized Trial. JAMA Cardiol. 2023 Mar 1;8(3):240-247. doi: 10.1001/jamacardio.2022.5099.
• Nassif M, Fine JT, Dolan C, Reaney M, Addepalli P, Allen VD, Sehnert AJ, Gosch K, Spertus JA. Validation of the Kansas City Cardiomyopathy Questionnaire in Symptomatic Obstructive Hypertrophic Cardiomyopathy. JACC Heart Fail. 2022 Aug;10(8):531-539. doi: 10.1016/j.jchf.2022.03.002. Epub 2022 May 4.
• Reaney M, Addepalli P, Allen V, Spertus JA, Dolan C, Sehnert AJ, Fine JT. Longitudinal Psychometric Analysis of the Hypertrophic Cardiomyopathy Symptom Questionnaire (HCMSQ) Using Outcomes from the Phase III EXPLORER-HCM Trial. Pharmacoecon Open. 2022 Jul;6(4):575-586. doi: 10.1007/s41669-022-00340-8. Epub 2022 Jun 20.
• Hegde SM, Lester SJ, Solomon SD, Michels M, Elliott PM, Nagueh SF, Choudhury L, Zemanek D, Zwas DR, Jacoby D, Wang A, Ho CY, Li W, Sehnert AJ, Olivotto I, Abraham TP. Effect of Mavacamten on Echocardiographic Features in Symptomatic Patients With Obstructive Hypertrophic Cardiomyopathy. J Am Coll Cardiol. 2021 Dec 21;78(25):2518-2532. doi: 10.1016/j.jacc.2021.09.1381.
• Xie J, Wang Y, Xu Y, Fine JT, Lam J, Garrison LP. Assessing health-related quality-of-life in patients with symptomatic obstructive hypertrophic cardiomyopathy: EQ-5D-based utilities in the EXPLORER-HCM trial. J Med Econ. 2022 Jan-Dec;25(1):51-58. doi: 10.1080/13696998.2021.2011301.
• Burstein Waldman C, Owens A. A plain language summary of the EXPLORER-HCM study: mavacamten for obstructive hypertrophic cardiomyopathy. Future Cardiol. 2021 Oct;17(7):1269-1275. doi: 10.2217/fca-2021-0044. Epub 2021 May 21.
• Spertus JA, Fine JT, Elliott P, Ho CY, Olivotto I, Saberi S, Li W, Dolan C, Reaney M, Sehnert AJ, Jacoby D. Mavacamten for treatment of symptomatic obstructive hypertrophic cardiomyopathy (EXPLORER-HCM): health status analysis of a randomised, double-blind, placebo-controlled, phase 3 trial. Lancet. 2021 Jun 26;397(10293):2467-2475. doi: 10.1016/S0140-6736(21)00763-7. Epub 2021 May 15.
• Olivotto I, Oreziak A, Barriales-Villa R, Abraham TP, Masri A, Garcia-Pavia P, Saberi S, Lakdawala NK, Wheeler MT, Owens A, Kubanek M, Wojakowski W, Jensen MK, Gimeno-Blanes J, Afshar K, Myers J, Hegde SM, Solomon SD, Sehnert AJ, Zhang D, Li W, Bhattacharya M, Edelberg JM, Waldman CB, Lester SJ, Wang A, Ho CY, Jacoby D; EXPLORER-HCM study investigators. Mavacamten for treatment of symptomatic obstructive hypertrophic cardiomyopathy (EXPLORER-HCM): a randomised, double-blind, placebo-controlled, phase 3 trial. Lancet. 2020 Sep 12;396(10253):759-769. doi: 10.1016/S0140-6736(20)31792-X. Epub 2020 Aug 29.
• Ho CY, Olivotto I, Jacoby D, Lester SJ, Roe M, Wang A, Waldman CB, Zhang D, Sehnert AJ, Heitner SB. Study Design and Rationale of EXPLORER-HCM: Evaluation of Mavacamten in Adults With Symptomatic Obstructive Hypertrophic Cardiomyopathy. Circ Heart Fail. 2020 Jun;13(6):e006853. doi: 10.1161/CIRCHEARTFAILURE.120.006853. Epub 2020 Jun 5.

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