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A Phase 3 trial to measure how safe and effective CK-3773274 is in Adults with obstructive hypertrophic cardiomyopathy (oHCM)

Phase 1
Conditions
obstructive hypertrophic cardiomyopathy (oHCM)
MedDRA version: 27.0Level: PTClassification code 10020871Term: Hypertrophic cardiomyopathySystem Organ Class: 10010331 - Congenital, familial and genetic disorders
Therapeutic area: Diseases [C] - Cardiovascular Diseases [C14]
Registration Number
EUCTR2021-003536-92-PL
Lead Sponsor
Cytokinetics, Incorporated
Brief Summary

Not available

Detailed Description

Not available

Recruitment & Eligibility

Status
ot Recruiting
Sex
All
Target Recruitment
282
Inclusion Criteria

- Males and females between 18 and 85 years of age, inclusive, at screening.
- Body mass index <35 kg/m^2.
- Diagnosed with HCM per the following criteria:
a. Has LV hypertrophy and non-dilated LV chamber in the absence of other cardiac disease and
b. Has an end-diastolic LV wall thickness as measured by the echocardiography core laboratory of:
• =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
- Has resting LVOT-G =30 mmHg and post-Valsalva LVOT-G =50 mmHg during screening as determined by the echocardiography core laboratory
- LVEF =60% at screening as determined by the echocardiography core laboratory.
- New York Heart Association (NYHA) Functional Class II or III at screening
- Hemoglobin =10 g/dL at screening.
- Respiratory exchange ratio (RER) =1.05 and pVO2 =90% predicted on the screening CPET per the core laboratory.
- Patients on beta-blockers, verapamil, diltiazem, or disopyramide should have been on a stable doses for >6 weeks prior to randomization and anticipate remaining on the same medication regimen during the trial. Patients treated with disopyramide must also be concomitantly treated with a beta blocker and/or calcium channel blocker.
Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 169
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 113

Exclusion Criteria

- Known or suspected infiltrative, genetic or storage disorder causing cardiac hypertrophy that mimics oHCM (eg, Noonan syndrome, Fabry disease, amyloidosis).
- Significant valvular heart disease (per investigator judgment).
a) Moderate-severe valvular aortic stenosis.
b) Moderate-severe mitral regurgitation not due to systolic anterior motion of the mitral valve.
- History of LV systolic dysfunction (LVEF <45%) or stress cardiomyopathy at any time during their clinical course.
- Inability to exercise on a treadmill or bicycle (eg, orthopedic limitations).
- Has been treated with septal reduction therapy (surgical myectomy or percutaneous alcohol septal ablation) or has plans for either treatment during the trial period.
- Documented paroxysmal atrial fibrillation during the screening period.
- Paroxysmal or permanent atrial fibrillation is only excluded IF:
• rhythm restoring treatment (eg, direct-current cardioversion, atrial fibrillation ablation procedure, or antiarrhythmic therapy)has been required =6 months prior to screening.
• rate control and anticoagulation have not been achieved for at least 6 months prior to screening.

- History of syncope or sustained ventricular tachyarrhythmia with exercise within 6 months prior to screening.
- Has received prior treatment with CK-3773274 or mavacamten.
Exclusion Criteria for CMR Sub-study
- Inability to tolerate CMR.
- Has an implantable cardioverter-defibrillator (ICD).
- Has a cardiac pacemaker.

Study & Design

Study Type
Interventional clinical trial of medicinal product
Study Design
Not specified
Primary Outcome Measures
NameTimeMethod
Main Objective: - To evaluate the effect of CK-3773274 on exercise capacity in patients with symptomatic oHCM;Secondary Objective: - To evaluate the effect of CK-3773274 on patient health status<br>- To evaluate the effect of CK-3773274 on New York Heart Association (NYHA) Functional Classification<br>- To evaluate the effect of CK-3773274 on post-Valsalva left ventricular outflow tract gradients (LVOT-G)<br>- To evaluate the effect of CK-3773274 on exercise capacity<br>- To evaluate the effect of CK-3773274 on duration of eligibility for septal reduction therapy<br><br>Safety<br>To evaluate the safety and tolerability profile of CK-3773274 in patients with symptomatic oHCM;Primary end point(s): • Change in peak oxygen uptake (pVO2) by cardiopulmonary exercise testing (CPET);Timepoint(s) of evaluation of this end point: from baseline to Week 24
Secondary Outcome Measures
NameTimeMethod
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