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Phase 3 Trial to Evaluate the Efficacy and Safety of Aficamten Compared to Placebo in Adults With Symptomatic oHCM (SEQUOIA-HCM) (SEQUOIA-HCM)

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ClinicalTrials.gov Identifier: NCT05186818
Recruitment Status : Completed
First Posted : January 11, 2022
Last Update Posted : January 8, 2024
Sponsor:
Collaborator:
Ji Xing Pharmaceuticals (Shanghai) Co., Ltd.
Information provided by (Responsible Party):
Cytokinetics

Study Description
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Brief Summary:
The purpose of this study is to evaluate the efficacy and safety of aficamten (CK-3773274) in adults with symptomatic hypertrophic cardiomyopathy and left ventricular outflow tract obstruction

Condition or disease Intervention/treatment Phase
Obstructive Hypertrophic Cardiomyopathy (oHCM) Drug: CK-3773274 (5 mg, 10 mg, 15 mg and 20 mg) Drug: Placebo to match CK-3773274 Phase 3

Study Design
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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 282 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Double (Participant, Investigator)
Primary Purpose: Treatment
Official Title: A Phase 3, Multi-Center, Randomized, Double-blind, Placebo-controlled Trial to Evaluate the Efficacy and Safety of CK-3773274 in Adults With Symptomatic Hypertrophic Cardiomyopathy and Left Ventricular Outflow Tract Obstruction
Actual Study Start Date : February 1, 2022
Actual Primary Completion Date : December 1, 2023
Actual Study Completion Date : December 1, 2023

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Cardiomyopathy

Arms and Interventions
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Arm Intervention/treatment
Experimental: CK-3773274 up to 20 mg
Patients will receive doses of 5 mg, 10 mg, 15 mg or 20 mg of CK-3773274 with dose levels guided by echocardiography assessments for up to 24 weeks
 Drug: CK-3773274 (5 mg, 10 mg, 15 mg and 20 mg)
CK-3773274 tablets administered orally
Placebo Comparator: Placebo to match CK-3773274
Patients will receive placebo for up to 24 weeks
 Drug: Placebo to match CK-3773274
Placebo administered orally


Outcome Measures
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Primary Outcome Measures :
  1. Change in peak oxygen uptake (pVO2) by cardiopulmonary exercise testing (CPET) [ Time Frame: Baseline to Week 24 ]
    Effect of CK-3773274 on exercise capacity in patients with symptomatic oHCM


Secondary Outcome Measures :
  1. Change in Kansas City Cardiomyopathy Questionnaire - Clinical Summary Score (KCCQ-CSS) [ Time Frame: Baseline to Week 12 and Week 24 ]
    Effect of CK-3773274 on patient health status

  2. Proportion of patients with ≥1 class improvement in New York Heart Association (NYHA) Functional Class [ Time Frame: Baseline to Week 12 and Week 24 ]
    Effect of CK-3773274 on NYHA Functional Classification

  3. Change in post-Valsalva left ventricular outflow tract gradients (LVOT-G) [ Time Frame: Baseline to Week 12 and Week 24 ]
    Effect of CK-3773274 on post-Valsalva LVOT-G

  4. Proportion of patients with post-Valsalva LVOT G <30 mmHg [ Time Frame: Baseline to Week 12 and Week 24 ]
    Effect of CK-3773274 on post-Valsalva LVOT-G

  5. Change in total workload during CPET [ Time Frame: Baseline to Week 24 ]
    Effect of CK-3773274 on exercise capacity

  6. Duration of eligibility for septal reduction therapy (SRT) [ Time Frame: Baseline to Week 24 ]
    Effect of CK-3773274 on duration of eligibility for septal reduction therapy


Eligibility Criteria
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Ages Eligible for Study:   18 Years to 85 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Key Inclusion Criteria:

  • Males and females between 18 and 85 years of age, inclusive, at screening.
  • Body mass index <35 kg/m2.

  • Diagnosed with HCM per the following criteria:

    • Has LV hypertrophy and non-dilated LV chamber in the absence of other cardiac disease and

    • 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.
  • NYHA Functional Class II or III at screening.
  • Hemoglobin ≥10g/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 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.

Key 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).

    • Moderate-severe valvular aortic stenosis.
    • 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.
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT05186818


Locations
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Sponsors and Collaborators
Cytokinetics
Ji Xing Pharmaceuticals (Shanghai) Co., Ltd.
Investigators
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Study Director: Cytokinetics MD Cytokinetics
More Information
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Responsible Party: Cytokinetics
ClinicalTrials.gov Identifier: NCT05186818    
Other Study ID Numbers: CY 6031
2021-003536-92 ( EudraCT Number )
First Posted: January 11, 2022    Key Record Dates
Last Update Posted: January 8, 2024
Last Verified: January 2024

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Cytokinetics:
Obstructive Hypertrophic Cardiomyopathy
oHCM
CK-3773274
CK-274
 Aficamten
SEQUOIA-HCM
CY 6031
SEQUOIA
Additional relevant MeSH terms:
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Cardiomyopathies
Cardiomyopathy, Hypertrophic
Hypertrophy
Heart Diseases
Cardiovascular Diseases
 Pathological Conditions, Anatomical
Aortic Stenosis, Subvalvular
Aortic Valve Stenosis
Aortic Valve Disease
Heart Valve Diseases