SEQUOIA-HCM
Aficamten for Symptomatic Obstructive Hypertrophic Cardiomyopathy
Design:
- A phase 3, double-blind, randomized trial
Objective:
- To evaluate the efficacy and safety of aficamten in adult patients with symptomatic obstructive hypertrophic cardiomyopathy (HCM).
Patients:
- 282 patients
Inclusion criteria:
- Aged 18-85 years
- BMI <35 kg/m²
- Diagnosed with HCM
- Resting LVOT-G ≥30 mmHg
- Post-Valsalva LVOT-G ≥50 mmHg
- LVEF ≥60%
- NYHA Functional Class II or III
Exclusion criteria:
- Significant valvular heart disease
- Paroxysmal or permanent atrial fibrillation
- Treated with septal reduction therapy
Comparison:
- Aficamten (5-20 mg OD for 24 weeks) (n = 142)
- Versus
- Placebo (n = 140)
Primary Outcome:
- Mean change in the peak oxygen uptake at 24 weeks (ml/kg/min)
- Aficamten: 1.8
- Placebo: 0.0
- Difference: 1.7, 95% CI: 1.0 to 2.4 (P < 0.001)
Secondary Outcomes:
- Improvement of ≥1 NYHA functional class at week 24 (%)
- Aficamten: 58.5%
- Placebo: 24.3%
- Difference: 34.2, 95% CI: 23.4 to 45.0 (P < 0.001)
- Mean change in LVOT-G after Valsalva maneuver at week 24 (mmHg)
- Aficamten: -47.6
- Placebo: -1.8
- Difference: -50, 95% CI: -57 to -44 (P < 0.001)
Conclusion:
- Among patients with symptomatic obstructive HCM, treatment with aficamten resulted in a significantly greater improvement in peak oxygen uptake than placebo.
Reference:
- Maron MS et al. N Engl J Med 2024;doi:10.1056/NEJMoa2401424
This data highlights the key points of the SEQUOIA-HCM trial, comparing the outcomes of aficamten versus placebo in patients with symptomatic obstructive hypertrophic cardiomyopathy.