EUCTR2017-002530-23-BE进行中（未招募）1 期
A Randomized, Double blind, Placebo controlled Clinical Study to Evaluate Mavacamten (MYK-461) in Adults with Symptomatic Obstructive Hypertrophic Cardiomyopathy - EXPLORER-HCM
适应症
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 入组人数
- 220
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional clinical trial of medicinal product
入排标准
- 性别
- All
入选标准
- •1.Able to understand and comply with the study procedures, understand the risks involved in the study, and provide written informed consent according to federal, local, and institutional guidelines before the first study specific procedure
- •2.Is at least 18 years old at Screening
- •3.Body weight is greater than 45 kg at Screening
- •4.Has adequate acoustic windows to enable accurate TTEs (Refer to Echocardiography Site Instruction Manual)
- •5.Diagnosed with oHCM consistent with current AACF/AMA and ESC guidelines, ie, satisfy both criteria below (criteria to be documented by the echocardiography core laboratory):
- •A.Has unexplained left ventricular (LV) hypertrophy with nondilated ventricular chambers in the absence of other cardiac (eg, hypertension, aortic stenosis) or systemic disease and with maximal LV wall thickness =15 mm (or =13 mm with positive family history of hypertrophic cardiomyopathy [HCM]), as determined by core lab interpretation and B.Has LVOT peak gradient =50 mmHg during Screening as assessed by echocardiography at rest, after Valsalva maneuver, or postexercise
- •(confirmed by echocardiography core laboratory interpretation)
- •6.Has documented left ventricular ejection fraction (LVEF) =55% by echocardiography core laboratory read of Screening TTE at rest 7. Has LVOT gradient with Valsalva maneuver at Screening TTE of =30
- •mmHg, determined by echocardiography core laboratory 8.Has (NYHA) functional Class II or III symptoms at Screening 9.Has documented oxygen saturation at rest =90% at Screening
- •10.Is able to perform an upright CPET and has a respiratory exchange ratio (RER) =1.0 at Screening per central reading; if the RER is between 0.91 and 1.0, the participant may be enrolled only if it is determined by the central CPET laboratory that peak exercise has been achieved in the subject (the only permitted reasons for subpeak performance are [1] a decrease in systolic blood pressure or [2] severe angina as described in the CPET Laboratory Manual)
- •11.Female participants must not be pregnant or lactating and, if sexually active, must use one of the following highly effective birth control methods from the Screening visit through 3 months after the last dose of investigational medicinal product (IMP).
- •combined (estrogen- and progestogen-containing) hormonal contraception associated with inhibition of ovulation or progestogenonly hormonal contraception associated with inhibition of ovulation by oral, implantable, or injectable route of administration
- •intrauterine device (IUD)
- •intrauterine hormone-releasing system (IUS)
- •bilateral tubal occlusion
- •Female is surgically sterile for 6 months or postmenopausal for 1 year. Permanent
- •sterilization includes hysterectomy, bilateral oophorectomy, bilateral salpingectomy, and/or documented bilateral tubal occlusion at least 6 months prior to Screening. Females are considered
- •postmenopausal if they have had amenorrhea for at least 1 year or more following
- •cessation of all exogenous hormonal treatments and follicle stimulating hormone levels are in the postmenopausal range.
- •Male partners must also use a contraceptive (eg, barrier, condom or vasectomy)
- •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 187
- •F.1.3 Elderly (>=65 years) yes
- •F.1.3.1 Number of subjects for this age range 33
排除标准
- •1.Previously participated in a clinical study with mavacamten
- •2.Hypersensitivity to any of the components of the mavacamten
- •formulation
- •3.Participated in a clinical trial in which the participant received any
- •investigational drug (or is currently using an investigational device)
- •within 30 days of Screening, or at least 5x the respective elimination half life (whichever is longer) 4.Infiltrative or storage disorder causing CH that mimics oHCM, such as Fabry disease, amyloidosis, or Noonan syndrome with LVH
- •5.Medical condition that precludes upright exercise stress testing
- •6.History of syncope within 6 months prior to Screening or history of
- •sustained ventricular tachyarrhythmia with exercise within 6 months
- •prior to Screening
- •7.History of resuscitated sudden CA (at any time) or history of
- •appropriate ICD discharge/shock for life-threatening VA within 6 months prior to Screening (Note: history of anti-tachycardia pacing (ATP) within 6 months or ever is allowed)
- •8.Has paroxysmal, intermittent AF with AF present per the investigator's evaluation of the participant's ECG at time of Screening
- •9.Has persistent/permanent AF not on anticoagulation for at least 4
- •weeks to Screening &/or not adequately rate controlled within 6 months prior to Screening
- •10.Current treatment (within 14 days to Screening) or planned
- •treatment during the study with disopyramide or ranolazine
- •11.Current treatment (within 14 days prior to Screening) or planned
- •treatment during the study with a combination of ß-blockers and
- •verapamil or a combination of ß-blockers and diltiazem
- •12.Individuals on ß-blockers, verapamil, or diltiazem, any dose
- •adjustment of that medication <14 days to Screening or any anticipated change in treatment regimen using these medications during the study
- •13.(Note: Prev. #13 was removed entirely) Successfully treated with ISR (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
- •14.ICD placement or pulse generator change within 2 months prior to
- •Screening or planned new ICD placement during study (pulse generator changes, if needed during the study, are allowed)
- •15.Has QT interval with Fridericia correction (QTcF) >500 ms at
- •screening or other ECG abnormality considered by investigator to pose
- •risk to participant safety (eg, second-degree atrioventricular block type II)
- •16.Documented OCAD (>70% stenosis in one or more epicardial
- •coronary arteries) or history of MI
- •17.Moderate or severe (as per investigator's judgment) AVS at
- •18.Acute or serious comorbid condition (eg, major infection or
- •hematologic, renal, metabolic, gastrointestinal, or endocrine
- •dysfunction) that, in the judgment of the investigator, could lead to
- •premature termination of study participation or interfere with the
- •measurement or interpretation of the efficacy & safety assessments in
- •19.Has pulmonary disease that limits exercise capacity or systemic
- •arterial oxygen saturation
- •20.History of malignant disease within 10 years of Screening: see
- •details in protocol
- •21.Has safety laboratory parameters (chemistry, hematology,
- •coagulation, & urinalysis) outside normal limits (according to the central
- •laboratory reference range) at Screening as assessed by the central
- •laboratory; however, participant with safety laboratory parameters
- •outside normal limits may be included if he or she meets all of the
- •following crit
研究者
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