Phase 3 Study of Intravenous Human Plasma-Derived C1 Esterase Inhibitor Concentrate for Treating and Preventing Acute Hereditary Angioedema Attacks in Patients With Congenital C1-INH Deficiency

What this Trial Is About

Researchers are evaluating an intravenous human plasma-derived C1 esterase inhibitor C1-INH concentrate in people with congenital C1-INH deficiency who experience acute hereditary angioedema attacks. This phase 3, randomized, double-blind, placebo-controlled study aims to assess the effectiveness and safety of this treatment both for managing attacks and preventing attacks before medical procedures. Participants receive either OCTA-C1-INH, a purified concentrate of human C1-INH given as a slow intravenous injection at a dose of 20 IUkg body weight, or a placebo injection of saline solution. The treatment is administered after the first qualifying angioedema attack. Both blinded and open-label participants receive OCTA-C1-INH, while only blinded participants receive the placebo. The study uses a parallel group design to compare these interventions. During the study, participants symptoms are closely monitored, focusing on the time to clear symptom relief within 4 hours after injection. Researchers assess symptom severity changes and response rates to the treatment. Participants must comply with all study procedures and are evaluated through clinical assessments and safety monitoring throughout the trial, which lasts until the study completion in June 2027.

Study of IV Human Plasma-derived C1 Esterase Inhibitor Concentrate in Patients With Congenital C1-INH Deficiency for Treatment and Pre-procedure Preventing of Acute Hereditary Angioedema Attacks