A Phase 2 Open-label Pilot Study Evaluating MYK-461 in Subjects With Symptomatic Hypertrophic Cardiomyopathy and Left Ventricular Outflow Tract Obstruction

Summary

The purpose of this phase 2 open-label pilot study is to evaluate the efficacy, pharmacokinetics (PK), pharmacodynamics (PD), safety, and tolerability of MYK-461 in subjects with symptomatic HCM and LVOT obstruction aged 18-70 years.

Timeline

Start

2016-08

Primary completion

2017-11

Completion

2017-11

Outcome

Met primary endpoint

paper mean change, -89.5 mm Hg [95% CI, -138.3 to -40.7 mm Hg]; P = 0.008 PMID 31035291 ↗

paper mean change, -25.0 mm Hg [CI, -47.1 to -3.0 mm Hg]; P = 0.020 PMID 31035291 ↗

Publications

• Heitner SB, Jacoby D, Lester SJ, Owens A, Wang A, Zhang D, Lambing J, Lee J, Semigran M, Sehnert AJ. Mavacamten Treatment for Obstructive Hypertrophic Cardiomyopathy: A Clinical Trial. Ann Intern Med. 2019 Jun 4;170(11):741-748. doi: 10.7326/M18-3016. Epub 2019 Apr 30.

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	Mavacamten	Small molecule	2 mg	Oral
Subject	Mavacamten	Small molecule	5 mg	Oral
Subject	Mavacamten	Small molecule	10 mg	Oral
Subject	Mavacamten	Small molecule	20 mg	Oral

Indications