Clinical Trials

Numerous clinical trials spanning Phase 1 through Phase 4 evaluate the therapeutic profile of mavacamten across cardiovascular conditions, primarily focusing on obstructive and general hypertrophic cardiomyopathy, heart failure with preserved ejection fraction, healthy volunteers, and adolescent populations. Sponsored by industry entities such as Bristol-Myers Squibb and LianBio LLC alongside academic centers like Oregon Health and Science University, these studies range in recruitment status from not yet recruiting to active and completed.

NCT Number Recruitment Conditions Sponsor/Collaborators Start Date Phases
NCT07120776 RECRUITING
Hypertrophic Obstructive Cardiomyopathy \(HOCM\); Left Ventricular Outflow Tract Obstruction; Myocardial Ischaemia
University of Manchester
 2026-03-26
NCT07004972 ACTIVE_NOT_RECRUITING
Symptomatic Obstructive Hypertrophic Cardiomyopathy
Bristol-Myers Squibb
 2025-09-04 PHASE4
NCT07600177 RECRUITING
Hypertrophic Cardiomyopathy (HCM)
Oregon Health and Science University
 2026-05-05 PHASE4
NCT07077005 RECRUITING
Cardiomyopathy, Hypertrophic
Technical University of Munich
 2025-11-01
NCT06112743 ACTIVE_NOT_RECRUITING
Cardiomyopathy, Hypertrophic
Bristol-Myers Squibb
 2024-01-24 PHASE4
NCT07103655 NOT_YET_RECRUITING
Hypertrophic Cardiomyopathy (HCM)
Second Affiliated Hospital, Zhejiang University, School of Medicine
 2026-01-01 PHASE4
NCT06947590 ACTIVE_NOT_RECRUITING
HCM - Hypertrophic Cardiomyopathy; Mavacamten
Xu Liu
 2025-04-20
NCT03723655 COMPLETED
Hypertrophic Cardiomyopathy; Obstructive Hypertrophic Cardiomyopathy; Non-obstructive Hypertrophic Cardiomyopathy
Bristol-Myers Squibb
 2018-09-27 PHASE2; PHASE3
NCT06856265 NOT_YET_RECRUITING
Obstructive Hypertrophic Cardiomyopathy; Mavacamten; Radiofrequency Ablation
Shanghai Chest Hospital
 2025-02-26
NCT06253221 ACTIVE_NOT_RECRUITING
Cardiomyopathy, Hypertrophic
Bristol-Myers Squibb
 2024-04-17 PHASE3
NCT05582395 COMPLETED
Cardiomyopathy, Hypertrophic
Bristol-Myers Squibb
 2022-12-14 PHASE3
NCT06211179 COMPLETED
Healthy Volunteers
Bristol-Myers Squibb
 2024-01-10 PHASE1
NCT06338202 Not yet recruiting
Obstructive Hypertrophic Cardiomyopathy (oHCM)
Bristol-Myers Squibb
 2024-04-01 --
NCT06253221 Recruiting
Cardiomyopathy Hypertrophic
Bristol-Myers Squibb
 2024-04-17 Phase 3
NCT04766892 COMPLETED
Heart Failure With Preserved Ejection Fraction
Bristol-Myers Squibb
 2021-03-30 PHASE2
NCT05414175 COMPLETED
Cardiomyopathy, Hypertrophic Obstructive
Bristol-Myers Squibb
 2022-08-19 PHASE3
NCT03496168 COMPLETED
Hypertrophic Cardiomyopathy
Bristol-Myers Squibb
 2018-04-26 PHASE2
NCT06023186 Not yet recruiting
Obstructive Hypertrophic Cardiomyopathy
Michael Ayers|Bristol-Myers Squibb|University of Virginia
 2023-09-15 --
NCT05719805 COMPLETED
Healthy Participants
Bristol-Myers Squibb
 2023-02-20 PHASE1
NCT05658146 COMPLETED
Healthy Participants
Bristol-Myers Squibb
 2023-01-06 PHASE1
NCT05174416 COMPLETED
Obstructive Hypertrophic Cardiomyopathy
LianBio LLC
 2022-01-04 PHASE3
NCT05320094 COMPLETED
Healthy Participants
Bristol-Myers Squibb
 2022-04-15 PHASE1
NCT05362045 COMPLETED
Healthy Participants
Bristol-Myers Squibb
 2022-05-16 PHASE1
NCT05489705 Recruiting
Obstructive Hypertrophic Cardiomyopathy
Bristol-Myers Squibb
 2022-08-16 --
NCT04349072 COMPLETED
HOCM, Hypertrophic Obstructive Cardiomyopathy
Bristol-Myers Squibb
 2020-07-06 PHASE3
NCT05135871 COMPLETED
Hypertrophic Cardiomyopathy
LianBio LLC
 2021-10-31 PHASE1
NCT04766892 Active not recruiting
Heart Failure With Preserved Ejection Fraction
Bristol-Myers Squibb
 2021-03-30 Phase 2
NCT03470545 COMPLETED
Obstructive Hypertrophic Cardiomyopathy
MyoKardia, Inc.
 2018-05-29 PHASE3
NCT03442764 COMPLETED
Non-obstructive Hypertrophic Cardiomyopathy
MyoKardia, Inc.
 2018-03-30 PHASE2
NCT02842242 COMPLETED
Cardiomyopathy, Hypertrophic Obstructive; Left Ventricular Outflow Tract Obstruction
MyoKardia, Inc.
 2016-08 PHASE2
NCT02480296 COMPLETED
Healthy
MyoKardia, Inc.
 2015-08 PHASE1
NCT02329184 COMPLETED
Hypertrophic Cardiomyopathy
MyoKardia, Inc.
 2014-12 PHASE1
NCT02356289 COMPLETED
Healthy
MyoKardia, Inc.
 2015-01 PHASE1

(data from https://clinicaltrials.gov, updated on 2026-05-22)

Check the MYK-461 (Mavacamten) product page for in-depth specifications, including solubility, stock solutions, MOA, and working concentrations.

Compliance for Clinical Use

Mechanism and Biochemical Profile

Mavacamten is a selective small-molecule modulator of cardiac myosin that binds to cardiac myosin ATPase, reducing the formation of active myosin-actin cross-bridges and dampening excessive myocardial force generation. By suppressing sarcomeric hypercontractility and improving diastolic relaxation, this mechanism alleviates dynamic left ventricular outflow tract obstruction, directly addressing the pathophysiological features of obstructive hypertrophic cardiomyopathy and heart failure with preserved ejection fraction studied in clinical trials.

Appendix RUO and cGMP Quality Standards

Quality Dimension RUO (Research Use Only) cGMP (Current Good Manufacturing Practice)
Clinical Applicability Prohibited in human clinical trials or medical diagnostics. Mandatory for human clinical trials (Phase I–III) and therapies.
Regulatory Status Non-regulated grade; exempt from drug manufacturing laws. Legally enforced by health authorities (e.g., FDA, EMA, NMPA).
Facility Environment Unclassified analytical or research laboratories. Validated Cleanrooms (ISO Class 5–8) with continuous monitoring.
Quality Control Basic purity and activity testing. Rigorous safety release testing (Sterility, Endotoxin, Mycoplasma).
Process Validation Basic equipment calibration; no process validation required. Full qualification (IQ/OQ/PQ) and complete batch records.
Quality Assurance Vendor self-declared without required formal QMS. Mandatory QA/QC unit, Change Control, CAPA, and vendor audits.
Regulatory Impact High risk of IND rejection if used as a critical raw material. Required for IND/NDA filings, supported by Drug Master Files (DMF).

Footnotes

Regulatory Note: Governed by FDA (21 CFR Parts 210/211/312), EMA (EudraLex Vol 4), ICH Guidelines (E6/Q7/Q9/Q10), and compendial standards (USP <71>/<85>/<1043>).

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Note: Technical data last updated: Sep 1, 2026.