CSL Behring enrolls first patient in phase III study of C1-INH for prevention of HAE attacks

CSL Behring today announced it has enrolled the first patient in COMPACT, an international phase III study of a volume-reduced, subcutaneous formulation of C1-esterase inhibitor (C1-INH) concentrate in patients with frequent hereditary angioedema (HAE) attacks (NCT01912456). This phase of the COMPACT program will assess the efficacy and safety of a new formulation of the CSL Behring C1-INH concentrate in preventing hereditary angioedema attacks when the therapy is administered twice weekly under the skin (i.e., subcutaneously) of patients diagnosed with HAE.

COMPACT is an acronym for Clinical Studies for Optimal Management in Preventing Angioedema with Low-Volume Subcutaneous C1-inhibitor Replacement Therapy).

"To date, COMPACT has shown that various doses of this volume-reduced formulation of C1-INH concentrate are well tolerated when administered at a single infusion site twice weekly," 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. "We also observed a dose-dependent, physiologically relevant increase in functional C1-INH plasma levels. From a clinical perspective, these results are intriguing and could lead to a more convenient option for people with HAE."

The COMPACT phase III, double-blind, randomized, placebo-controlled, cross-over study enrolls adolescent and adult patients with HAE types I or II who have frequent attacks. The study will measure the number of hereditary angioedema attacks that subjects experience while receiving each treatment. Subjects will be able to take on-demand medication for acute attacks at any time during the study.

"The COMPACT study is an important demonstration of the commitment CSL Behring has to the HAE community," said Russell Basser, Senior Vice President of Clinical Research and Development at CSL Behring. "CSL Behring has been a leader in this area for decades, so we are confident that our current efforts to develop a safe, effective and convenient new treatment option for HAE patients will be successful."