BACKGROUND The presence and extent of left ventricular hypertrophy (LVH) is a major determinant of symptoms in patients with hypertrophic cardiomyopathy (HCM). There is increasing evidence to suggest that myocardial energetic impairment represents a central mechanism leading to LVH in HCM. There is currently a significant unmet need for disease-modifying therapy that regresses LVH in HCM patients. Perhexiline, a potent carnitine palmitoyl transferase-1 (CPT-1) inhibitor, improves myocardial energetics in HCM, and has the potential to reduce LVH in HCM. OBJECTIVE The primary objective is to evaluate the effects of perhexiline treatment on the extent of LVH, in symptomatic HCM patients with at least moderate LVH. METHODS/DESIGN RESOLVE-HCM is a prospective, multicenter double-blind placebo-controlled randomized trial enrolling symptomatic HCM patients with at least moderate LVH. Sixty patients will be randomized to receive either perhexiline or matching placebo. The primary endpoint is change in LVH, assessed utilizing cardiovascular magnetic resonance (CMR) imaging, after 12-months treatment with perhexiline. SUMMARY RESOLVE-HCM will provide novel information on the utility of perhexiline in regression of LVH in symptomatic HCM patients. A positive result would lead to the design of a Phase 3 clinical trial addressing long-term effects of perhexiline on risk of heart failure and mortality in HCM patients.

中文翻译：

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哌克昔林消退症状性肥厚型心肌病患者左心室肥厚的随机对照试验（RESOLVE-HCM 试验）。


背景左心室肥厚（LVH）的存在和程度是肥厚型心肌病（HCM）患者症状的主要决定因素。越来越多的证据表明，心肌能量损害是 HCM 中导致 LVH 的核心机制。目前对于使 HCM 患者的 LVH 消退的疾病修饰疗法的需求尚未得到满足。 Perhexiline 是一种有效的肉碱棕榈酰转移酶 1 (CPT-1) 抑制剂，可改善 HCM 中的心肌能量，并有可能降低 HCM 中的 LVH。目的 主要目的是评估哌可昔林治疗对至少中度 LVH 的有症状 HCM 患者的 LVH 程度的影响。方法/设计 RESOLVE-HCM 是一项前瞻性、多中心、双盲、安慰剂对照随机试验，招募至少患有中度 LVH 的有症状 HCM 患者。 60 名患者将被随机分配接受哌克昔林或匹配的安慰剂。主要终点是使用哌克西林治疗 12 个月后利用心血管磁共振 (CMR) 成像评估的 LVH 变化。总结 RESOLVE-HCM 将提供关于哌可昔林在有症状 HCM 患者 LVH 消退中的效用的新信息。积极的结果将导致设计一项 3 期临床试验，以解决 perhexiline 对 HCM 患者心力衰竭和死亡率风险的长期影响。