Table of contents
- Trial overview
- Who is being studied
- What the trials measure
- Study design and phases
- Key trial summaries
- What patients should know
Trial overview
The clinical trials in this data study Mavacamten in people with hypertrophic cardiomyopathy, which is a condition where the heart muscle is thicker than normal.[1] The trials look at both obstructive and non-obstructive forms of the disease, and they include adolescents and adults.[1] Most of the studies are later-stage trials, including Phase 3 research, and one study is listed as low intervention.[1]
Who is being studied
One Phase 3 study is in adolescent participants with symptomatic obstructive hypertrophic cardiomyopathy, and it plans to enroll 40 people.[1] Another Phase 3 study is in adults with non-obstructive hypertrophic cardiomyopathy and enrolled 478 participants.[2] A separate long-term study included adults who had already completed earlier mavacamten trials, MAVERICK-HCM or EXPLORER-HCM.[4]
These studies are designed for people with different forms of the same heart condition, so the target group changes from trial to trial.[1][2][4] This helps researchers see whether the study treatment works in specific patient groups rather than in one large mixed group.[1][2]
What the trials measure
The adolescent obstructive HCM study measures the change from baseline in Valsalva LVOT gradient at Week 28.[1] This is a test of how much blood flow is blocked as blood leaves the heart, and the Valsalva maneuver is a way to make that blockage easier to measure.[1]
The adult non-obstructive HCM study measures change from baseline in KCCQ CSS and pVO2 at Week 48.[2] KCCQ CSS is a patient questionnaire score that reflects symptoms and physical limits, while pVO2 is a measure of exercise capacity, or how well the body uses oxygen during exercise.[2]
The imaging study looks at whether Mavacamten reduces heart wall thickness and upper chamber size on cardiac magnetic resonance at Week 48.[3] This means researchers are checking heart structure with a detailed scan, not only symptoms.[3]
The long-term extension study focuses on safety, heart events, and changes in heart structure over time.[4] It measures major adverse cardiac events, hospitalizations, heart failure events, abnormal heart rhythms, and treatment-emergent side effects and lab changes.[4]
Study design and phases
Three of the studies are Phase 3 trials, which are later-stage studies that help show whether a treatment works in a larger group of people.[1][2][4] The imaging study is listed as low intervention, meaning it is focused on observation and follow-up with limited added study procedures.[3]
Several studies compare Mavacamten with placebo, which is a look-alike treatment that does not contain the study drug.[1][2][3] This design helps researchers judge whether changes are linked to the study treatment rather than to chance or time alone.[1][2][3]
Key trial summaries
2023-505650-17-00: A Phase 3 study in adolescents with symptomatic obstructive hypertrophic cardiomyopathy. The main goal is to see whether Mavacamten changes the Valsalva LVOT gradient by Week 28.[1]
NCT05582395: A completed Phase 3 randomized, double-blind, placebo-controlled study in adults with non-obstructive hypertrophic cardiomyopathy. It studied health status and exercise capacity over 48 weeks.[2]
2022-502316-36-00: A completed low-intervention study in symptomatic obstructive hypertrophic cardiomyopathy. It examined heart wall thickness and upper chamber size on cardiac magnetic resonance at Week 48.[3]
NCT03723655: A completed Phase 3 long-term extension study in adults with hypertrophic cardiomyopathy who had finished earlier trials. It tracked safety, serious heart events, and changes in heart structure.[4]
What patients should know
These trials are not all looking at the same question, even though they all study Mavacamten.[1][2][3][4] Some focus on symptom relief, some on exercise ability, some on heart imaging, and some on long-term safety.[1][2][3][4]
The trial data show that researchers are trying to learn whether Mavacamten can help different age groups and different forms of hypertrophic cardiomyopathy.[1][2][4] They are also checking whether the treatment changes heart structure and whether it remains safe during longer follow-up.[3][4]
