CLINICAL TRIAL: ICATIBANT FOR THE TREATMENT OF HEREDITARY ANGIOEDEMA

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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.