CSL Behring commences C1-INH phase I/II study in hereditary angioedema

CSL Behring announced today that it has initiated an international phase I/II study of a volume-reduced, subcutaneous formulation of C1-esterase inhibitor (C1-INH) concentrate in patients with hereditary angioedema (HAE). Part of the COMPACT (Clinical Studies for Optimal Management in Preventing Angioedema with Low-Volume Subcutaneous C1-inhibitor Replacement Therapy) program, the study will evaluate the pharmacokinetics, pharmacodynamics and safety of various doses of this presentation of C1-INH.

"We're enormously pleased that a range of therapy options is now available to treat HAE," said Bruce Zuraw, MD, Professor of Medicine at the University of California, San Diego, USA, and Chairman of the Steering Committee for the COMPACT program. "Yet, we know that a significant number of patients would prefer a preventive therapy that does not require intravenous infusions and that might offer fewer side effects than currently available treatments. We are confident this study will guide us in the development of this novel method of using C1-esterase inhibitor replacement for the routine prevention of HAE attacks."

The open-label study will enroll adult patients with HAE type I or II. After an initial intravenous injection of C1-INH, each participant will be assigned to receive a single subcutaneous injection of the volume-reduced formulation of C1-INH twice a week for four weeks. Subjects will participate in two such periods with two different doses of volume-reduced C1-INH. Throughout the study, researchers will monitor C1-INH levels in the blood, as well as assess the safety and tolerability of the formulation at different doses.

"As a pioneer in the development of subcutaneous immunoglobulin therapy, we are excited to apply our expertise and experience in the treatment of hereditary angioedema," said Russell Basser, Senior Vice President of Clinical Research and Development at CSL Behring. "Subcutaneous administration of C1-esterase inhibitor will represent another important advance for patients suffering from the frequent and often debilitating attacks of HAE."

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CSL Behring

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