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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT06081894




Registration number
NCT06081894
Ethics application status
Date submitted
31/08/2023
Date registered
13/10/2023

Titles & IDs
Public title
Phase 3 Trial to Evaluate the Efficacy and Safety of Aficamten Compared to Placebo in Adults With Symptomatic nHCM
Scientific 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
Secondary ID [1] 0 0
2023-505797-15-00
Secondary ID [2] 0 0
CY 6033
Universal Trial Number (UTN)
Trial acronym
ACACIA-HCM
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Symptomatic Non-Obstructive Hypertrophic Cardiomyopathy 0 0
Condition category
Condition code
Cardiovascular 0 0 0 0
Other cardiovascular diseases

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - Aficamten
Treatment: Drugs - Placebo

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.

Placebo comparator: Placebo - Participants in this arm will receive placebo, for up to 72 weeks.


Treatment: Drugs: Aficamten
Oral Tablet

Treatment: Drugs: Placebo
Oral Tablet
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Change in Kansas City Cardiomyopathy Questionnaire - Clinical Summary Score (KCCQ-CSS)
Timepoint [1] 0 0
Baseline to Week 36
Secondary outcome [1] 0 0
Change in composite of two Z-scores of CPET parameters (pVO2 and VE/VCO2 slope)
Timepoint [1] 0 0
Baseline to Week 36
Secondary outcome [2] 0 0
Proportion of participants with = 1 class improvement in NYHA Functional Class
Timepoint [2] 0 0
Baseline to Week 36
Secondary outcome [3] 0 0
Change in NT-proBNP
Timepoint [3] 0 0
Baseline to Week 36
Secondary outcome [4] 0 0
Change in LAVI
Timepoint [4] 0 0
Baseline to Week 36
Secondary outcome [5] 0 0
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)
Timepoint [5] 0 0
Baseline to End of Study, Week 72

Eligibility
Key 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
Minimum age
18 Years
Maximum age
85 Years
Sex
Both males and females
Can healthy volunteers participate?
No
Key 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.

Study design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Blinded (masking used)
Who is / are masked / blinded?
The people receiving the treatment/s


The people analysing the results/data
Intervention assignment
Parallel
Other design features
Phase
Phase 3
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
30/08/2023
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
1/09/2026
Actual
Sample size
Target
420
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
QLD,SA
Recruitment hospital [1] 0 0
The Prince Charles Hospital - Chermside
Recruitment hospital [2] 0 0
Flinders Medical Centre Cardiology Research - Adelaide
Recruitment postcode(s) [1] 0 0
4032 - Chermside
Recruitment postcode(s) [2] 0 0
5042 - Adelaide
Recruitment outside Australia
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Oxford

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Cytokinetics
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Cytokinetics MD
Address 0 0
Cytokinetics
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Cytokinetics MD
Address 0 0
Country 0 0
Phone 0 0
650-624-2929
Fax 0 0
Email 0 0
medicalaffairs@cytokinetics.com
Contact person for scientific queries

Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
No
No/undecided IPD sharing reason/comment


What supporting documents are/will be available?

No Supporting Document Provided



Results publications and other study-related documents

No documents have been uploaded by study researchers.


Results not provided in https://clinicaltrials.gov/study/NCT06081894