CLINICAL TRIAL: ICATIBANT FOR THE TREATMENT OF HEREDITARY ANGIOEDEMA

这个子项目是许多研究子项目中利用 资源由NIH/NCRR资助的中心拨款提供。子项目和 调查员(PI)可能从NIH的另一个来源获得了主要资金， 并因此可以在其他清晰的条目中表示。列出的机构是 该中心不一定是调查人员的机构。 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、喉和胃肠道反复发作的自限性水肿症。皮肤血管性水肿的特征是面部、四肢或生殖器肿胀，面容受损，但不瘙痒，无痛。当出现在胃肠道时，症状更严重，包括急性腹痛并伴有恶心、呕吐和腹泻。最严重的HAE发作会导致喉部浮肿，导致上呼吸道阻塞，如果不诊断和/或不治疗，可能会导致窒息死亡。 主要目的是评估缓激肽(BK)拮抗剂icatibant与安慰剂在遗传性血管水肿(HAE)患者中重度急性皮肤和/或腹部水肿发作所致症状缓解方面的疗效。 次要目标是评估几乎完全缓解的反应时间比率、总体结果、每种症状的严重性以及皮下注射(S.C.)的安全性和耐受性。在患有皮肤和/或腹部水肿发作的HAE患者中，与安慰剂进行比较。 S.C.重复治疗的有效性和安全性。在开放标签扩展阶段也将对icatibant进行评估。此外，在首次发作的双盲治疗后，将评估icatibant的相对药物经济学影响，以及在icatibant治疗下与水肿症发作相关的经济负担，并与安慰剂进行比较；在开放标签治疗6个月后，评估并与历史数据进行比较。 在研究期间，只要有上呼吸道症状的受试者可以使用，将探索其在经历喉部浮肿发作的受试者中的有效性和安全性。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.