Ensayo de fase 3, multicéntrico, aleatorizado, doble ciego, para evaluar la eficacia y seguridad de aficamten en comparación con placebo en adultos con miocardiopatía hipertrófica no obstructiva sintomática

Activo, no reclutaFase 3ClinicalTrials.gov

ID: NCT06081894Tipo: INTERVENTIONALInicio: 30 de ago de 2023Fin estimado: 1 de sept de 2026

Resumen

Este ensayo clínico estudiará los efectos de aficamten (versus placebo) sobre la calidad de vida, la capacidad de ejercicio y los resultados clínicos de pacientes con miocardiopatía hipertrófica no obstructiva.

Elegibilidad

Edad mínima: 18 YearsEdad máxima: 85 YearsSexo: ALL

Criterios de inclusión

Between 18-85 years of age
Body mass index \< 40 kg/m2
Diagnosed with nHCM and has a screening echocardiogram with the following:
End-diastolic left ventricular (LV) wall thickness:
≥ 15 mm in one or more myocardial segments OR
≥ 13 mm in one or more wall segments and a known disease-causing gene mutation or positive family history of HCM AND
Resting LVOT-G \< 30 mmHg AND Valsalva LVOT-G \< 50 mmHg AND
LVEF ≥ 60%
Participants with a history of intracavitary obstruction are eligible.
NYHA class II or III
Respiratory exchange ratio of ≥ 1.00 at screening by cardiopulmonary exercise testing (CPET) and predicted peak oxygen uptake (pVO2) ≤ 90% for age and sex
KCCQ-CSS score of ≤ 85
NT-proBNP of:
NT-pro BNP ≥ 300 pg/mL or NT-proBNP ≥ 900 pg/mL if in atrial fibrillation or atrial flutter OR
For Black participants, an NT-pro BNP ≥ 225 pg/mL or NT-proBNP ≥ 675 pg/mL if in atrial fibrillation or atrial flutter

Criterios de exclusión

Significant valvular heart disease (per Investigator judgment)
Moderate or severe valvular aortic stenosis or fixed subaortic obstruction
Moderate or severe mitral regurgitation
Known or suspected infiltrative, genetic or storage disorder causing cardiac hypertrophy that mimics nHCM (eg, Noonan syndrome, Fabry disease, amyloidosis)
Known current unrevascularized coronary artery stenosis of ≥ 70% or documented history of myocardial infarction.
History of LV systolic dysfunction (LVEF \< 45%) or stress cardiomyopathy
Inability to exercise on a treadmill or bicycle (eg, orthopedic limitations)
Documented room air oxygen saturation reading \< 90% at screening or history of significant chronic obstructive pulmonary disease or severe/significant pulmonary hypertension
History of syncope, symptomatic ventricular arrhythmia, or sustained ventricular tachyarrhythmia with exercise within 3 months prior to screening
History of resistant hypertension (persistently elevated blood pressure despite maximal doses of 3 or more classes of medications for hypertension control)
Screening diastolic blood pressure ≥ 100 mmHg
Received prior treatment with aficamten
Received treatment with mavacamten within 3 months prior to screening (must be discussed with the medical monitor prior to screening)
Undergone septal reduction therapy \< 6 months prior to screening
Is being considered for or is likely to be considered for heart transplant listing or left ventricular assist device placement during the study period
Paroxysmal or permanent atrial fibrillation is excluded only if:
rhythm restoring treatment (e.g., direct-current cardioversion, atrial fibrillation ablation procedure, or antiarrhythmic therapy) has been required ≤ 3 months prior to screening
rate control and anticoagulation have not been achieved for at least 3 months prior to screening.

Intervenciones

drug

Aficamten

Oral Tablet

drug

Placebo

Oral Tablet

Ubicaciones

Hospital Italiano de Buenos Aires Juan Domingo Peron

Buenos Aires, Argentina

ICBA - Instituto Cardiovascular de Buenos Aires

Buenos Aires, Argentina

Hospital Privado Centro Medico de Cordoba S.A

Córdoba, Argentina

Patrocinadores

PrincipalCytokinetics

ColaboradorSanofi

ColaboradorBayer

Aviso: La información de este ensayo proviene de fuentes públicas y tiene carácter exclusivamente informativo. No constituye asesoramiento médico, regulatorio ni de ningún otro tipo. Las traducciones son automatizadas y no poseen validez oficial. Consultá siempre con un profesional de la salud. Ver Términos de Uso.

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