Hypertrophic Cardiomyopathy (HCM)
Conditions
Keywords
aficamten, mavacamten, cardiac myosin inhibitor, CMI
Brief summary
This is an investigator-initiated two-center study. The goal of this study is to investigate the feasibility, safety and efficacy outcomes of a seamless transition from mavacamten to aficamten in patients with obstructive hypertrophic cardiomyopathy (oHCM).
Interventions
DRUGAficamten
Patients will be switched from mavacamten to aficamten. Mavacamten will be stopped at enrollment, and aficamten started 2 weeks later.
Sponsors
Oregon Health and Science University
Saint Lukes Hospital Mid America Heart Institute
Cytokinetics
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Intervention model description
This is a single group study where patients will transition from mavacamten to aficamten following a standardized protocol
Eligibility
Sex/Gender
ALL
Age
18 Years to 90 Years
Healthy volunteers
No
Inclusion criteria
* Documented history of oHCM with documented resting and/or Valsalva LVOT obstruction ≥ 50 mmHg who are currently receiving mavacamten commercially. * Echo-derived LVEF ≥55% on mavacamten at the time of enrollment. * Patient willing to consent for the study and undergo the study procedures.
Exclusion criteria
* Severe aortic stenosis or sub-aortic obstruction * Known infiltrative or storage disorder causing cardiac hypertrophy that mimics HCM (eg, Noonan syndrome, Fabry disease, amyloidosis). * History of LVEF \<30%. * Paroxysmal atrial fibrillation (AF) with documented episode within 3 months. * Atrial fibrillation (paroxysmal or permanent) not on systemic anticoagulation. * Documented history of current obstructive coronary artery disease (\> 70% stenosis in one or more epicardial coronary arteries) or documented history of myocardial infarction.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Safety Endpoints | Up to 16 weeks | To describe the safety of this proposed protocol. These include: 1. Participant incidence of reported AEs 2. Participant incidence of reported SAEs 3. Participant incidence of LVEF \< 40% |
Secondary
| Measure | Time frame |
|---|---|
| Proportional change from baseline in resting and Valsalva LVOT gradients during each assessment | Up to 16 weeks |
| Proportion of participants with resting LVOT gradient < 30 mmHg and Valsalva LVOT gradient < 50 mmHg | Up to 16 weeks |
| Proportional change from baseline in NT-proBNP and high-sensitivity troponin I | Up to 16 weeks |
| Proportional improvement in NYHA functional classification by 1 functional class | Up to 16 weeks |
Countries
United States
Contacts
CONTACTAhmad Masri
Outcome results
None listed