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."