A Study to Evaluate Mavacamten in Adults With Symptomatic Obstructive HCM Who Are Eligible for Septal Reduction Therapy
Summary
This is a randomized, double-blind, placebo-controlled, multi-center study in the United States (U.S.) that will evaluate the effect of mavacamten treatment on reducing the number of septal reduction therapy (SRT) procedures performed in subjects with symptomatic obstructive hypertrophic cardiomyopathy (oHCM \[also known as HOCM\]) who are eligible for SRT based on ACCF/AHA 2011 and/or ESC 2014 guidelines.
Timeline
- Start
- 2020-07-06
- Primary completion
- 2022-02-07
- Completion
- 2024-05-20
Outcome
Met primary endpoint
registry COUNT_OF_PARTICIPANTS Mavacamten: 10 Participants COUNT_OF_PARTICIPANTS Placebo to Mavacamten: 43 Participants NCT04349072 ↗
registry analysis (superiority test); Mavacamten vs Placebo to Mavacamten; p <0.0001; Proportion Difference 58.93 (95% CI 43.989 to 73.868); Cochran-Mantel-Haenszel NCT04349072 ↗
release “At 16 weeks the primary and all secondary endpoints were met.” bms.com ↗
release “VALOR-HCM met its primary and all secondary endpoints with a high degree of statistical significance, with no new safety signals observed.” bms.com ↗
release “Approval based on two positive Phase 3 trials, EXPLORER-HCM and VALOR-HCM, demonstrating significant benefit in patients treated with CAMZYOS versus placebo” bms.com ↗
release “The trial met all primary and secondary endpoints with statistical significance: Results showed that CAMZYOS significantly reduced the primary composite endpoint” bms.com ↗
Publications
- Desai MY, Wolski K, Owens A, Geske JB, Saberi S, Wang A, Sherrid M, Cremer PC, Lakdawala NK, Tower-Rader A, Fermin D, Naidu SS, Smedira NG, Schaff H, Gong Z, Mudarris L, Lampl K, Sehnert AJ, Nissen SE; VALOR-HCM Investigators. Mavacamten in Patients With Hypertrophic Cardiomyopathy Referred for Septal Reduction: Week 128 Results From VALOR-HCM. Circulation. 2025 May 13;151(19):1378-1390. doi: 10.1161/CIRCULATIONAHA.124.072445. Epub 2024 Nov 18.
- Desai MY, Okushi Y, Wolski K, Geske JB, Owens A, Saberi S, Wang A, Cremer PC, Sherrid M, Lakdawala NK, Tower-Rader A, Fermin D, Naidu SS, Lampl KL, Sehnert AJ, Nissen SE, Popovic ZB; VALOR-HCM Investigators. Mavacamten-Associated Temporal Changes in Left Atrial Function in Obstructive HCM: Insights From the VALOR-HCM Trial. JACC Cardiovasc Imaging. 2025 Mar;18(3):251-262. doi: 10.1016/j.jcmg.2024.08.005. Epub 2024 Sep 2.
- Desai MY, Okushi Y, Gaballa A, Wang Q, Geske JB, Owens AT, Saberi S, Wang A, Cremer PC, Sherrid M, Lakdawala NK, Tower-Rader A, Fermin D, Naidu SS, Lampl KL, Sehnert AJ, Nissen SE, Popovic ZB; VALOR-HCM Investigators. Serial Changes in Ventricular Strain in Symptomatic Obstructive Hypertrophic Cardiomyopathy Treated With Mavacamten: Insights From the VALOR-HCM Trial. Circ Cardiovasc Imaging. 2024 Sep;17(9):e017185. doi: 10.1161/CIRCIMAGING.124.017185. Epub 2024 Sep 2.
- Desai MY, Owens A, Wolski K, Geske JB, Saberi S, Wang A, Sherrid M, Cremer PC, Lakdawala NK, Tower-Rader A, Fermin D, Naidu SS, Smedira NG, Schaff H, McErlean E, Sewell C, Mudarris L, Gong Z, Lampl K, Sehnert AJ, Nissen SE. Mavacamten in Patients With Hypertrophic Cardiomyopathy Referred for Septal Reduction: Week 56 Results From the VALOR-HCM Randomized Clinical Trial. JAMA Cardiol. 2023 Oct 1;8(10):968-977. doi: 10.1001/jamacardio.2023.3342.
- Cremer PC, Geske JB, Owens A, Jaber WA, Harb SC, Saberi S, Wang A, Sherrid M, Naidu SS, Schaff H, Smedira NG, Wang Q, Wolski K, Lampl KL, Sehnert AJ, Nissen SE, Desai MY. Myosin Inhibition and Left Ventricular Diastolic Function in Patients With Obstructive Hypertrophic Cardiomyopathy Referred for Septal Reduction Therapy: Insights From the VALOR-HCM Study. Circ Cardiovasc Imaging. 2022 Dec;15(12):e014986. doi: 10.1161/CIRCIMAGING.122.014986. Epub 2022 Nov 6.
- Desai MY, Owens A, Geske JB, Wolski K, Saberi S, Wang A, Sherrid M, Cremer PC, Naidu SS, Smedira NG, Schaff H, McErlean E, Sewell C, Balasubramanyam A, Lampl K, Sehnert AJ, Nissen SE. Dose-Blinded Myosin Inhibition in Patients With Obstructive Hypertrophic Cardiomyopathy Referred for Septal Reduction Therapy: Outcomes Through 32 Weeks. Circulation. 2023 Mar 14;147(11):850-863. doi: 10.1161/CIRCULATIONAHA.122.062534. Epub 2022 Nov 6.
- Desai MY, Owens A, Geske JB, Wolski K, Naidu SS, Smedira NG, Cremer PC, Schaff H, McErlean E, Sewell C, Li W, Sterling L, Lampl K, Edelberg JM, Sehnert AJ, Nissen SE. Myosin Inhibition in Patients With Obstructive Hypertrophic Cardiomyopathy Referred for Septal Reduction Therapy. J Am Coll Cardiol. 2022 Jul 12;80(2):95-108. doi: 10.1016/j.jacc.2022.04.048.
- Zampieri M, Argiro A, Marchi A, Berteotti M, Targetti M, Fornaro A, Tomberli A, Stefano P, Marchionni N, Olivotto I. Mavacamten, a Novel Therapeutic Strategy for Obstructive Hypertrophic Cardiomyopathy. Curr Cardiol Rep. 2021 Jun 3;23(7):79. doi: 10.1007/s11886-021-01508-0.
- Desai MY, Wolski K, Owens A, Naidu SS, Geske JB, Smedira NG, Schaff H, Lampl K, McErlean E, Sewell C, Zhang D, Edelberg JM, Sehnert AJ, Nissen SE. Study design and rationale of VALOR-HCM: evaluation of mavacamten in adults with symptomatic obstructive hypertrophic cardiomyopathy who are eligible for septal reduction therapy. Am Heart J. 2021 Sep;239:80-89. doi: 10.1016/j.ahj.2021.05.007. Epub 2021 May 24.
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | Mavacamten | Small molecule | 15 mg | Oral |