International study by CSL Behring is world’s first placebo-controlled phase III trial to examine subcutaneous administration of C1-INH for use in treating rare, sometimes life-threatening, condition

KING OF PRUSSIA, PA, USA I February 20, 2014 I 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.”

Additional information about the COMPACT trial and participating centers can be found here:

About Hereditary Angioedema

Hereditary angioedema due to decreased C1-esterase inhibitor (C1-INH) is caused by mutations in SERPING1, the gene coding for C1-INH. It is inherited in an autosomal dominant manner. Symptoms of HAE include recurring episodes of edema, or swelling, in the hands, feet, the face, the abdomen, and/or the larynx. Patients who have abdominal attacks of HAE can experience episodes of severe pain, diarrhea, nausea, and vomiting caused by swelling of the intestinal wall. HAE attacks that involve the face and larynx can result in airway closure, asphyxiation, and, if untreated, death. Diagnosis of HAE requires a blood test to confirm low or abnormal levels of C1-INH.  For more information about HAE, please visit or

CSL Behring currently has licensed a C1-inhibitor product in Australia, Canada, Europe, Japan, the United States and several other countries in Asia and South America for treatment of acute attacks of HAE.

About CSL Behring

CSL Behring is a leader in the plasma protein therapeutics industry. Committed to saving lives and improving the quality of life for people with rare and serious diseases, the company manufactures and markets a range of plasma-derived and recombinant therapies worldwide. CSL Behring therapies are indicated for the treatment of coagulation disorders including hemophilia and von Willebrand disease, primary immune deficiencies, hereditary angioedema and inherited respiratory disease. The company’s products are also used in cardiac surgery, organ transplantation, burn treatment and to prevent hemolytic disease of the newborn. CSL Behring operates one of the world’s largest plasma collection networks, CSL Plasma. CSL Behring is a subsidiary of CSL Limited (CSL.AX), a biopharmaceutical company headquartered in Melbourne, Australia. For information: