ICATIBANT FOR THE TREATMENT OF HEREDITARY ANGIOEDEMA

Icatibant 用于治疗遗传性血管性水肿

• 批准号: 7604975
• 负责人: Gerald J Gleich
• 金额: $ 1.04万
• 依托单位: UNIVERSITY OF UTAH
• 依托单位国家: 美国
• 财政年份: 2007
• 资助国家: 美国
• 起止时间: 2007-03-01 至 2008-02-29
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/7604975
• 关键词: Abdomen; Abdominal Pain; Acute; Angioneurotic Edema; Area; Asphyxia; Bradykinin; Cessation of life; Computer Retrieval of Information on Scientific Projects Database; Cutaneous; Data; Diarrhea; Disease; Double-Blind Method; Economic Burden; Edema; Face; Funding; Gastrointestinal tract structure; Grant; Institution; Label; Laryngeal Edema; Larynx; Lead; Limb structure; Nausea and Vomiting; Obstruction; Outcome; Painless; Pharmaceutical Economics; Phase; Placebos; Rate; Reaction Time; Recurrence; Relative (related person); Research; Research Personnel; Resources; Safety; Severities; Skin; Source; Swelling; Symptoms; United States National Institutes of Health; experience; gastrointestinal; hereditary angioneurotic edema; icatibant; subcutaneous

This subproject is one of many research subprojects utilizing the resources provided by a Center grant funded by NIH/NCRR. The subproject and investigator (PI) may have received primary funding from another NIH source, and thus could be represented in other CRISP entries. The institution listed is for the Center, which is not necessarily the institution for the investigator. Hereditary angioedema (HAE) is an autosomal dominant disorder clinically characterized by recurrent and self-limiting episodes of edema of the skin, larynx, and gastrointestinal tract. Cutaneous angioedema is marked by disfiguring, but non-pruritic and painless, swelling of the face, extremities, or genitals. When presenting in the gastrointestinal area, the symptoms are more severe, including acute abdominal pain accompanied by nausea, vomiting, and diarrhea. The most serious of HAE attacks results in laryngeal edema, causing obstruction of the upper airways that may lead to death by asphyxiation if undiagnosed and/or untreated. The primary objective is to assess the efficacy of the bradykinin (BK) antagonist Icatibant compared with placebo on the onset of relief of symptoms resulting from moderate to very severe acute cutaneous and/or abdominal edema attacks in subjects with hereditary angioedema (HAE). The Secondary objectives are to assess the rate of response time to almost complete relief, global outcome, severity of each symptom, and safety and tolerability of subcutaneous (s.c.) Icatibant compared with placebo in subjects with HAE suffering from cutaneous and/or abdominal edema attacks. The efficacy and safety of repeated treatments with s.c. Icatibant will also be assessed during the open label extension phase. In addition, the relative pharmacoeconomic impact of Icatibant, and the economic burden associated with onset of an edema attack under Icatibant treatment will be assessed and compared with placebo after the double blind treatment of the first attack, and assessed and compared with historical data after 6 months open label treatment. The efficacy and safety in subjects experiencing laryngeal edema attacks will be explored, to the extent that subjects with symptoms of the upper airway become available during the study period. The information collected here will provide preliminary evidence of the impact of tretment.

该子项目是利用该技术的众多研究子项目之一 资源由 NIH/NCRR 资助的中心拨款提供。子项目及 研究者 (PI) 可能已从 NIH 的另一个来源获得主要资金， 因此可以在其他 CRISP 条目中表示。列出的机构是 对于中心来说，它不一定是研究者的机构。 遗传性血管性水肿（HAE）是一种常染色体显性遗传性疾病，临床特征为皮肤、喉部和胃肠道水肿的反复发作和自限性发作。 皮肤血管性水肿的特点是面部、四肢或生殖器出现毁容，但无瘙痒和无痛肿胀。当出现在胃肠道区域时，症状更加严重，包括急性腹痛并伴有恶心、呕吐和腹泻。最严重的 HAE 发作会导致喉部水肿，导致上呼吸道阻塞，如果未经诊断和/或未经治疗，可能会导致窒息死亡。 主要目的是评估缓激肽 (BK) 拮抗剂艾替替班与安慰剂相比，对遗传性血管性水肿 (HAE) 受试者中度至极重度急性皮肤和/或腹部水肿发作引起的症状缓解的疗效。 次要目标是评估患有皮肤和/或腹部水肿发作的 HAE 受试者与安慰剂相比，几乎完全缓解的反应时间、总体结果、每种症状的严重程度以及皮下 (s.c.) 艾替替班的安全性和耐受性。 皮下注射重复治疗的有效性和安全性艾替班特还将在开放标签延伸阶段进行评估。 此外，将在首次发作双盲治疗后评估艾替替班的相对药物经济学影响，以及与艾替替班治疗下水肿发作相关的经济负担，并与安慰剂进行比较，并在 6 年后与历史数据进行评估和比较。几个月的开放标签治疗。 将探讨经历喉部水肿发作的受试者的有效性和安全性，以便在研究期间出现上呼吸道症状的受试者。 这里收集的信息将提供治疗影响的初步证据。