Phase 2 Study of Sildenafil for the Treatment of Lymphatic Malformations

西地那非治疗淋巴管畸形的 2 期研究

• 批准号: 8612453
• 负责人: Anna Lee Bruckner
• 金额: $ 39.73万
• 依托单位: STANFORD UNIVERSITY
• 依托单位国家: 美国
• 财政年份: 2014
• 资助国家: 美国
• 起止时间: 2014-09-10 至 2020-02-29
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/8612453
• 关键词: Phase; Study; Sildenafil; Treatment; Lymphatic

Project Summary Lymphatic malformations (LMs) are localized areas of abnormal development of the lymphatic system that commonly occur in the head and neck of children. LMs are considered a rare or orphan disease which causes complications including pain, ulceration, secondary infection, infiltration of other organs, and death. Current therapies involve surgical excision or methods of chemical or physical destruction of portions of lesions. No therapies are uniformly effective and all have the risk of significant adverse events. We recently witnessed almost complete resolution of a LM lesion in a child who was treated with sildenafil oral therapy for pulmonary arterial hypertension. We have subsequently evaluated additional subjects who improved with sildenafil. The goal of this clinical research trial is to document the benefit or absence of benefit of sildenafil therapy for LMs and identify which type of patient will benefit from sildenafil. This study is a double-blind placebo-controlled trial which involves precise documentation of volume changes associated with therapy or placebo by using MRI segmentation techniques. We will also observe and document the clinical response to sildenafil or placebo using clinical evaluation scores and surveys. The results of the study should identify characteristics of LM lesions which may suggest a beneficial response to sildenafil therapy. Sildenafil has very low risk of side effects in the dosage used in this trial. Documentation of an effective response of LMs to sildenafil will accelerate the interest in, and the ability to understand, the mechanisms of LM formation and treatment.

项目概要 淋巴管畸形 (LM) 是淋巴管异常发育的局部区域。 淋巴系统，常见于儿童的头颈部。 LM 被认为是 罕见或孤儿疾病，会导致并发症，包括疼痛、溃疡、继发性 感染、浸润其他器官并导致死亡。目前的治疗方法包括手术切除或 对病变部分进行化学或物理破坏的方法。没有统一的治疗方法 有效且都有发生重大不良事件的风险。我们最近目睹了几乎 接受西地那非口服疗法治疗的儿童的 LM 病变完全消退 肺动脉高压。我们随后评估了其他受试者 用西地那非改善。该临床研究试验的目的是记录益处或 西地那非治疗对 LM 没有益处，并确定哪种类型的患者会受益 来自西地那非。这项研究是一项双盲安慰剂对照试验，涉及精确的 使用 MRI 记录与治疗或安慰剂相关的体积变化 分割技术。我们还将观察并记录西地那非的临床反应 或使用临床评估分数和调查的安慰剂。研究结果应确定 LM 病变的特征可能表明对西地那非治疗有有益的反应。 本试验中使用的剂量西地那非的副作用风险非常低。的文档 LM 对西地那非的有效反应将加速人们对西地那非的兴趣和能力 了解 LM 形成和治疗的机制。