STDY OF EFFICACY&SAFETY OF RCMBNNT C1 INHBTR 4 TRTMNT OF ACUTEATCKS OF ANGIOEDEM

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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 is a genetic disease in which patients are susceptible to attacks of swelling under the skin of the face, hands, feet, buttocks, genitalia and intestines. If the attacks affect the tongue or throat, there is a risk of suffocation. The attacks may be brought on by trauma such an injury or dental work and may accompany a menstrual period. Some attacks have no known cause. Patients with hereditary angioedema are not able to make a protein called C1 inhibitor (C1 INH). In most patients the attacks can be prevented by treatment with low doses of male hormones (androgens). Some patients, especially young women, are not able to use androgens because they can cause acne, growth of facial hair and weight gain. Androgens cannot be used during pregnancy. Androgen treatment stimulates the body to make C1 inhibitor. In the United States, treatment of acute attacks involves use of narcotics for pain and supportive treatments such as replacement of fluids. For some patients, infusions of fresh frozen blood plasma are beneficial. In Europe, the C1 inhibitor is commercially available and is the standard of care for treatment of acute attacks in patients with hereditary angioedema. The purpose of this research study is to study the safety and clinical effects of IV infusions of C1 inhibitor in patients with hereditary angioedema during acute attacks that affect the intestines, genitalia and/or face and throat. The goals are to learn which doses are effective and how fast the drug works. These results will be compared with those observed in patients who get an inactive IV infusion (placebo). The long term goal of the study which will be done in 15-20 centers in the Country is to get information for an application to the U.S. Food and Drug Administration for approval of C1 inhibitor as a treatment for hereditary angioedema in the United States