ICATIBANT FOR THE TREATMENT OF HEREDITARY ANGIOEDEMA

这个子项目是许多研究子项目中的一个 由NIH/NCRR资助的中心赠款提供的资源。子项目和 研究者（PI）可能从另一个NIH来源获得了主要资金， 因此可以在其他CRISP条目中表示。所列机构为 研究中心，而研究中心不一定是研究者所在的机构。 遗传性血管性水肿（HAE）是一种常染色体显性遗传疾病，临床特征为皮肤、喉和胃肠道水肿的复发性和自限性发作。 皮肤血管性水肿的特征是面部、四肢或生殖器的毁容，但非炎症性和无痛性肿胀。当出现在胃肠道区域时，症状更严重，包括伴有恶心，呕吐和腹泻的急性腹痛。最严重的HAE发作导致喉水肿，引起上呼吸道阻塞，如果未诊断和/或未治疗，可能导致窒息死亡。 主要目的是评估缓激肽（BK）拮抗剂Icatibant与安慰剂相比在缓解遗传性血管性水肿（HAE）受试者中中度至极重度急性皮肤和/或腹部水肿发作引起的症状方面的疗效。 次要目的是评估几乎完全缓解的反应时间、总体结局、每种症状的严重程度以及皮下（s.c.）在患有皮肤和/或腹部水肿发作的HAE受试者中比较艾替班特与安慰剂。 反复皮下注射治疗的有效性和安全性。还将在开放标签扩展阶段评估艾替班特。 此外，在首次发作的双盲治疗后，将评估艾替班特的相对药物经济学影响和与艾替班特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个月开放标签治疗后评估并与历史数据进行比较。 将探索发生喉水肿发作的受试者的疗效和安全性，直至研究期间有上呼吸道症状的受试者可用。 这里收集的信息将为治疗的影响提供初步证据。

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.