Clinical trial NCT05414175: A Study of Mavacamten in Obstructive Hypertrophic Cardiomyopathy
Official title: A Phase 3, Open-label, Single Arm, Clinical Study to Evaluate Efficacy, Safety and Tolerability of Mavacamten in Adults With Symptomatic Obstructive Hypertrophic Cardiomyopathy
Study type: INTERVENTIONAL
Overall status: ACTIVE_NOT_RECRUITING
Phase: PHASE3
Conditions: Cardiomyopathy, Hypertrophic Obstructive
Keywords: Mavacamten
Obstructive Hypertrophic Cardiomyopathy (HCM)
Lead sponsor: Bristol-Myers Squibb
Sponsor class: INDUSTRY
First posted: 2022-06-10
Enrollment: 38
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: yes
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: The purpose of this study is to evaluate the effectiveness, safety, and tolerability of a 30-week course of mavacamten and the long-term effects of mavacamten in Japanese participants with symptomatic obstructive hypertrophic cardiomyopathy (HCM).


Eligibility criteria: Inclusion Criteria:

* Age 18 and greater, body weight ≥ 35kg
* Has adequate acoustic windows to enable accurate transthoracic echocardiograms (TTEs)
* Diagnosed with obstructive hypertrophic cardiomyopathy consistent with current American College of Cardiology Foundation/American Heart Association, European Society of Cardiology, and Japanese Circulation Society guidelines
* Has documented left ventricular ejection fraction (LVEF) ≥60% NYHA Class II or III

Exclusion Criteria:

* Known infiltrative or storage disorder causing cardiac hypertrophy that mimics oHCM, such as Fabry disease, amyloidosis, or Noonan syndrome with LV hypertrophy
* History of syncope or sustained ventricular tachyarrhythmia with exercise within 6 months prior to Screening
* History of resuscitated sudden cardiac arrest (at any time) or known history of appropriate implantable cardioverter defibrillator (ICD) discharge for life-threatening ventricular arrhythmia within 6 months prior to Screening
* Paroxysmal atrial fibrillation with atrial fibrillation present at the time of Screening.
* Persistent or permanent atrial fibrillation not on anticoagulation for at least 4 weeks prior to Screening and/or not adequately rate controlled within 6 months prior to Screening
* Treatment (within 14 days prior to Screening) or planned treatment during the study with cibenzoline, disopyramide or ranolazine
* Treatment (within 14 days prior to Screening) or planned treatment during the study with a combination of beta blockers and verapamil or a combination of beta blockers and diltiazem
* Has been successfully treated with invasive septal reduction (surgical myectomy or percutaneous alcohol septal ablation \[ASA\]) within 6 months prior to Screening or plans to have either of these treatments during the study
* ICD placement within 2 months prior to Screening or planned ICD placement during the study
* Has a history or evidence of any other clinically significant disorder, condition, or disease that, in the opinion of the investigator, would pose a risk to participant safety or interfere with the study evaluation procedures, or completion
* Prior treatment with cardiotoxic agents such as doxorubicin or similar

Other protocol-defined inclusion/exclusion criteria apply

Primary outcomes:
measure: Change From Baseline in Post-exercise Left Ventricular Outflow Tract (LVOT) Peak Gradient at Week 30
timeFrame: At Baseline and Week 30
Secondary outcomes:
measure: Change From Baseline in Kansas City Cardiomyopathy Questionnaire 23-item Version (KCCQ-23) Clinical Summary Score (CSS) at Week 30
timeFrame: At Baseline and Week 30
measure: Percentage of Participants With at Least 1 Class Improvement in New York Heart Association (NYHA) Functional Class From Baseline to Week 30
timeFrame: Baseline and at Week 30
measure: Change From Baseline in N-terminal Pro B-type Natriuretic Peptide (NT-proBNP) at Week 30
timeFrame: At baseline and week 30
measure: Change From Baseline in Cardiac Troponin I at Week 30
timeFrame: At baseline and week 30
measure: Change From Baseline in Cardiac Troponin T at Week 30
timeFrame: At baseline and week 30
Other outcomes:
Locations:
city: Uwajima
country: Japan
facility: Local Institution - 0020
state: Ehime
city: Himeji-Shi
country: Japan
facility: Local Institution - 0026
state: Hyogo
city: Kobe-shi
country: Japan
facility: Local Institution - 0016
state: Hyogo
city: Tsukuba-Shi
country: Japan
facility: Local Institution - 0017
state: Ibaraki
city: Kanazawa-shi
country: Japan
facility: Local Institution - 0023
state: Ishikawa
city: Yokohama
country: Japan
facility: Local Institution - 0019
state: Kanagawa
city: Nankoku-shi
country: Japan
facility: Local Institution - 0015
state: Kochi
city: Tsu-Shi
country: Japan
facility: Local Institution - 0028
state: MIE
city: Sendai
country: Japan
facility: Local Institution - 0027
state: Miyagi
city: Suita-Shi
country: Japan
facility: Local Institution - 0011
state: Osaka
city: Suita
country: Japan
facility: Local Institution - 0014
state: Osaka
city: Hamamatsu-city
country: Japan
facility: Local Institution - 0012
state: Shizuoka
city: Bunkyo-Ku
country: Japan
facility: Local Institution - 0010
state: Tokyo
city: Chuo-Ku
country: Japan
facility: Local Institution - 0009
state: Tokyo
city: Fuchu-Shi
country: Japan
facility: Local Institution - 0003
state: Tokyo
city: Itabashi-Ku
country: Japan
facility: Local Institution - 0001
state: Tokyo
city: Koto-Ku
country: Japan
facility: Local Institution - 0007
state: Tokyo
city: Shinjuku-Ku
country: Japan
facility: Local Institution - 0005
state: Tokyo
city: Shinjuku-ku
country: Japan
facility: Local Institution - 0013
state: Tokyo
city: Osaka
country: Japan
facility: Local Institution - 0018
Study officials:
affiliation: Bristol-Myers Squibb
name: Bristol-Myers Squibb
role: STUDY_DIRECTOR
References: