NCT07023614招募中2 期
A Global Phase 2b, Randomized, Double-Blinded, Placebo-Controlled Trial to Evaluate the Efficacy and Safety of Ninerafaxstat in Patients With Symptomatic Non-Obstructive Hypertrophic Cardiomyopathy - FORTITUDE-HCM
干预措施
相关药物
试验速览
- 阶段
- 2 期
- 状态
- 招募中
- 入组人数
- 165
- 试验地点
- 68
- 主要终点
- Change in Kansas City Cardiomyopathy Questionnaire Clinical Summary Score (KCCQ-CSS)
研究概览
简要总结
FORTITUDE-HCM is a global, multicenter, double-blind, parallel-group, placebo-controlled Phase 2b study that will assess the efficacy and safety of ninerafaxstat compared to placebo on top of Standard of Care in patients with symptomatic nHCM
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- Triple (Participant, Care Provider, Investigator)
入排标准
- 年龄范围
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Select Inclusion Criteria:
- •Has a clinical diagnosis of HCM consistent with current American College of Cardiology/American Heart Association and European Society of Cardiology Guideline definitions
- •Has had confirmation of nHCM by the echocardiography core laboratory based on screening rest and exercise stress echocardiography
- •New York Heart Association (NYHA) functional Class II or III at screening
- •Functional limitation as defined by a screening CPET
排除标准
- •Has a known or suspected infiltrative, genetic, or storage disorder causing cardiac hypertrophy that mimics nHCM
- •Has any other condition judged by the investigator to be the primary cause of dyspnea, exercise intolerance, and/or angina
- •Has an inability to exercise on a treadmill or bicycle (eg, orthopedic limitations)
- •Has any medical condition that precludes upright exercise stress testing
- •Other protocol-defined inclusion and exclusion criteria apply.
研究组 & 干预措施
Ninerafaxstat
Experimental
干预措施: Ninerafaxstat 200mg MR (Drug)
Placebo
Placebo Comparator
干预措施: Placebo (Drug)
结局指标
主要结局
Change in Kansas City Cardiomyopathy Questionnaire Clinical Summary Score (KCCQ-CSS)
时间窗: Baseline to Week 12
The Clinical Summary Score is a merging of symptom frequency and symptom burden domains into a total symptom score combined with the physical limitation domain. Scoring ranges from 0 to 100, with higher scores indicating better health status.
次要结局
- Change in ventilatory efficiency (VE/VCO2 slope) during cardiopulmonary exercise testing (CPET)(Baseline to Week 12)
- Change in the Kansas City Cardiomyopathy Questionnaire Total Symptom Score (KCCQ-TSS)(Baseline to Week 12)
- Change in the Kansas City Cardiomyopathy Questionnaire Physical Limitations Score (KCCQ-PLS)(Baseline to Week 12)
- Change in the Kansas City Cardiomyopathy Questionnaire Overall Summary Score (KCCQ-OSS)(Baseline to Week 12)
研究者
研究点 (68)
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相关资讯
Imbria's Ninerafaxstat Wins FDA Orphan Drug Designation for Non-Obstructive Hypertrophic Cardiomyopathy- Imbria Pharmaceuticals received FDA Orphan Drug Designation for ninerafaxstat, a first-in-class partial fatty acid oxidation inhibitor for symptomatic non-obstructive hypertrophic cardiomyopathy (nHCM).
- The designation provides seven years of potential market exclusivity upon approval, tax credits for clinical trials, and PDUFA fee waivers, complementing existing patents extending to 2038 and beyond.
- Ninerafaxstat shifts cardiac metabolism from fatty acids to glucose for more efficient energy production, representing a differentiated approach from myosin inhibitors that target contractility.
- Topline data from the ongoing Phase 2b FORTITUDE-HCM trial are anticipated in the first half of 2027, with no FDA-approved therapies currently available for nHCM.2 months agoImbria Pharmaceuticals Secures Extended Series B Funding to Advance First-in-Class Cardiovascular Therapy Ninerafaxstat- Imbria Pharmaceuticals announced AXA IM Alts' participation in an upsized Series B financing round to support clinical development of ninerafaxstat, a first-in-class cardiovascular therapy targeting cardiac energy metabolism.
- The company is advancing ninerafaxstat through the ongoing FORTITUDE-HCM Phase 2b clinical trial for symptomatic non-obstructive hypertrophic cardiomyopathy, a debilitating condition with no currently approved treatments.
- Cardiovascular disease remains the world's leading cause of mortality, responsible for more than 20 million deaths annually, highlighting the significant unmet medical need that ninerafaxstat aims to address.10 months agoImbria Pharmaceuticals Partners with Patient Advocacy Group to Accelerate Recruitment for Novel Heart Disease Trial- Imbria Pharmaceuticals has partnered with the Hypertrophic Cardiomyopathy Association to accelerate patient recruitment for the FORTITUDE-HCM Phase 2b trial evaluating ninerafaxstat in non-obstructive hypertrophic cardiomyopathy.
- The global trial is enrolling approximately 165 patients with symptomatic nHCM, a debilitating condition affecting 250,000-400,000 patients in the United States with no currently approved treatments.
- Ninerafaxstat represents a first-in-class cardiovascular therapy that optimizes mitochondrial energy production by shifting cardiac metabolism from fatty acids to glucose without affecting heart rate or blood pressure.
- The partnership will leverage HCMA's outreach programs and patient communications to connect eligible patients with trial enrollment resources and increase awareness of this potential treatment option.11 months ago