Study characteristics and intervention details.
| Study ID | Trial | Location | Study design | Blinding | Population | Number of patients (intervention/placebo) | Study duration | Intervention | Dosage |
|---|---|---|---|---|---|---|---|---|---|
| Mavacamten | |||||||||
| Olivotto et al. 2020 [10] | EXPLORER-HCM | Multi-national | Phase 3 RCT | Double-blind | Obstructive HCM | 251 (123/128) | 30 weeks | Mavacamten oral | Doses starting from 5 mg up to 15 mg |
| Ho et al. 2020 [6] | MAVERICK-HCM | Multi-center, United States | Phase 2 RCT | Double-blind | Non-obstructive HCM | 59 (40/19) | 16 weeks | Mavacamten | Target serum concentrations of either 200 ng/mL or 500 ng/mL |
| Desai et al. 2022 [8] | VALOR-HCM | Multi-center, United States | Phase 3 RCT | Double-blind | Obstructive HCM | 112 (56/56) | 16 weeks | Mavacamten oral | 5 mg daily titrated up to 15 mg |
| Tian et al. 2023 [9] | EXPLORER-CN | Multi-center, China | Phase 3 RCT | Double-blind | Obstructive HCM | 81 (54/27) | 30 weeks | Mavacamten oral | Once daily with starting dose 2.5 mg up to 15 mg |
| Aficamten | |||||||||
| Maron et al. 2023 [11] | REDWOOD-HCM | Multi-national | Phase 2 RCT | Double-blind | Obstructive HCM | 41 (28/13) | 10 weeks | Aficamten | Ranging from 5 mg up to 15 mg in cohort 1 and from 10 to 30 mg in cohort 2 |
| Maron et al. 2024 [7] | SEQUOIA-HCM | Multi-national | Phase 3 RCT | Double-blind | Obstructive HCM | 282 (142/140) | 24 weeks | Aficamten | Once daily starting from 5 mg up to 20 mg |
HCM: hypertrophic cardiomyopathy; RCT: randomized controlled trial.
The supplementary tables and figures for this article are available at: https://www.explorationpub.com/uploads/Article/file/101289_sup_1.pdf.
Pre-print: the pre-print is posted on SSRN: http://dx.doi.org/10.2139/ssrn.5183557. Conference: the abstract of this manuscript was presented to the conference at the APPNA Spring Meeting 2025 and is published in the conference proceedings in Pakistan Heart Journal under the doi: https://pakheartjournal.com/index.php/pk/article/view/3065.
Ayesha A: Writing—original draft, Supervision, Software, Methodology, Formal analysis, Data curation, Conceptualization. BA: Data curation, Methodology, Formal analysis, Writing—original draft. Arfa A: Data curation, Visualization, Formal analysis, Software, Writing—original draft. MM: Methodology, Software, Writing—original draft. ET: Visualization, Software, Methodology, Writing—original draft. AH: Formal analysis, Software, Writing—review & editing. MZB: Writing—original draft, Methodology, Writing—review & editing. Aleena A: Writing—review & editing, Formal analysis, Visualization. SA: Writing—original draft, Software, Visualization. MWZK: Writing—review & editing, Writing—original draft. All authors have read and approved the submitted version of this manuscript.
The authors declare that they have no conflicts of interest.
Not applicable.
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All the data utilized in this study are available in the relevant figures and Tables S3–5.
This research did not receive any specific grant or funding from funding agencies in the public, commercial, or not-for-profit sectors.
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