Active clinical trials for "Angioedemas, Hereditary"

This is a non-interventional treatment Registry of Hereditary Angioedema (HAE) patients treated with C1 inhibitor, either plasma-derived (pdC1INH) or the recombinant human form (rhC1INH / Ruconest), to observe adverse events and insufficient efficacy, and to assess the immunological profile following single and repeated treatment with Ruconest.

The expanded access program allows people to gain access to an unlicensed treatment on compassionate grounds. Lanadelumab, also known as TAK-743, is a medicine to help prevent angioedema attacks. This expanded access program enables these participants with a high unmet medical need to continue receiving lanadelumab during the interim period between completion of either the SHP643-301 (NCT04070326; SPRING study) or the TAK-743-3001 (NCT04444895) study and potential licensure of lanadelumab for the respective age group and/or treatment.

This expanded access program will provide access to berotralstat for eligible participants with hereditary angioedema in the U.S.

Since 1963 Hereditary AngioEdema (HAE) is considered an autosomal dominant disorder (Donaldson and Evans), characterized by a quantitative and/or qualitative deficit of C1 esterase inhibitor (C1-INH), which affects approximately 1:50.000 individuals in the general population. From this period the link between HAE and psychiatry was interrupted, however genetic issues could not comprehensively explain the clinical evolution of the disease. Clinical studies show an evident gap between genotype and phenotype of HAE. For this still controversial question, we have designed this cross-sectional study in order to establish the relationship between HAE clinical manifestations and neurobiological/psychopatological parameters.