Phase 3 Trial to Evaluate the Efficacy and Safety of Aficamten Compared to Placebo in Adults With Symptomatic nHCM (ACACIA-HCM)
|
The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details. |
| ClinicalTrials.gov Identifier: NCT06081894 |
|
Recruitment Status :
Recruiting
First Posted : October 13, 2023
Last Update Posted : April 15, 2024
|
Sponsor:
Cytokinetics
Information provided by (Responsible Party):
Cytokinetics
- Study Details
- Tabular View
- No Results Posted
- Disclaimer
- How to Read a Study Record
Brief Summary:
This clinical trial will study the effects of aficamten (versus placebo) on the quality of life, exercise capacity, and clinical outcomes of patients with non-obstructive hypertrophic cardiomyopathy.
| Condition or disease | Intervention/treatment | Phase |
|---|---|---|
| Symptomatic Non-Obstructive Hypertrophic Cardiomyopathy | Drug: Aficamten Drug: Placebo | Phase 3 |
| Study Type : | Interventional (Clinical Trial) |
| Estimated Enrollment : | 420 participants |
| Allocation: | Randomized |
| Intervention Model: | Parallel Assignment |
| Masking: | Double (Participant, Investigator) |
| Primary Purpose: | Treatment |
| Official Title: | A Phase 3, Multi-Center, Randomized, Double-Blind Trial to Evaluate the Efficacy and Safety of Aficamten Compared to Placebo in Adults With Symptomatic Non-Obstructive Hypertrophic Cardiomyopathy |
| Actual Study Start Date : | August 30, 2023 |
| Estimated Primary Completion Date : | June 2026 |
| Estimated Study Completion Date : | September 2026 |
Resource links provided by the National Library of Medicine
MedlinePlus related topics:
Cardiomyopathy
| Arm | Intervention/treatment |
|---|---|
|
Experimental: Aficamten
Participants in this arm will receive a single daily oral dose of 5 mg, 10 mg, 15 mg, or 20 mg of aficamten with dose levels guided by echocardiography assessments, for up to 72 weeks.
|
Drug: Aficamten
Oral Tablet |
|
Placebo Comparator: Placebo
Participants in this arm will receive placebo, for up to 72 weeks.
|
Drug: Placebo
Oral Tablet |
Primary Outcome Measures :
- Change in Kansas City Cardiomyopathy Questionnaire - Clinical Summary Score (KCCQ-CSS) [ Time Frame: Baseline to Week 36 ]Effect of aficamten compared with placebo on participant health status
Secondary Outcome Measures :
- Change in composite of two Z-scores of CPET parameters (pVO2 and VE/VCO2 slope) [ Time Frame: Baseline to Week 36 ]Effect of aficamten compared with placebo on global exercise capacity based on maximal and sub-maximal exercise performance
- Proportion of participants with ≥ 1 class improvement in NYHA Functional Class [ Time Frame: Baseline to Week 36 ]Effect of aficamten compared with placebo on NYHA Functional Classification
- Change in NT-proBNP [ Time Frame: Baseline to Week 36 ]Effect of aficamten compared with placebo on a biomarker of cardiac wall stress
- Change in LAVI [ Time Frame: Baseline to Week 36 ]Effect of aficamten compared with placebo on echocardiographic measures of structural remodeling
- Time to first CV event (CV death; heart transplantation or left ventricular assist device; aborted sudden cardiac death; non-fatal stroke; heart failure hospitalization; or cardiac arrhythmia requiring treatment or hospitalization) [ Time Frame: Baseline to End of Study, Week 72 ]Effect of aficamten compared with placebo on cardiovascular events
Information from the National Library of Medicine
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.
| Ages Eligible for Study: | 18 Years to 85 Years (Adult, Older Adult) |
| Sexes Eligible for Study: | All |
| Accepts Healthy Volunteers: | No |
Criteria
Inclusion Criteria:
- 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 ≥ 30 and ≤ 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
Exclusion Criteria:
-
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.
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): NCT06081894
Contacts
| Contact: Cytokinetics MD | 650-624-2929 | medicalaffairs@cytokinetics.com |
Locations
Show 27 study locations
Sponsors and Collaborators
Cytokinetics
Investigators
| Study Director: | Cytokinetics MD | Cytokinetics |
| Responsible Party: | Cytokinetics |
| ClinicalTrials.gov Identifier: | NCT06081894 |
| Other Study ID Numbers: |
CY 6033 2023-505797-15-00 ( Other Identifier: EU CTR Number ) |
| First Posted: | October 13, 2023 Key Record Dates |
| Last Update Posted: | April 15, 2024 |
| Last Verified: | December 2023 |
| Individual Participant Data (IPD) Sharing Statement: | |
| Plan to Share IPD: | No |
| Studies a U.S. FDA-regulated Drug Product: | Yes |
| Studies a U.S. FDA-regulated Device Product: | No |
Keywords provided by Cytokinetics:
|
CK-3773274 CK-274 Aficamten Symptomatic Non-Obstructive Hypertrophic Cardiomyopathy |
nHCM ACACIA-HCM ACACIA |
Additional relevant MeSH terms:
|
Cardiomyopathies Cardiomyopathy, Hypertrophic Hypertrophy Heart Diseases Cardiovascular Diseases |
Pathological Conditions, Anatomical Aortic Stenosis, Subvalvular Aortic Valve Stenosis Aortic Valve Disease Heart Valve Diseases |