CSL Behring Reaches Primary Endpoint in Clinical Study of C1-INH for Treatment of Hereditary ...(ver 20 years in Germany Austria Switzerl...),Health

"The successful completion of this trial confirms longstanding observations made from the administration of more than 300,000 treatments of C1-INH concentrate in Europe. We are delighted that it will now be submitted for registration in the UK," said Dr. Hilary Longhurst, Consultant Immunologist, Barts and The London NHS Trust, London, England, and investigator of the I.M.P.A.C.T. trial.

Study design and results

HAE is caused by the partial absence of functional C1 inhibitor in the blood. The objective of I.M.P.A.C.T. was to assess whether replacing the C1 protein missing in patients with HAE through treatment with C1-INH can address the underlying mechanism of the disease.

I.M.P.A.C.T. evaluated in a prospective and double-blind fashion whether different doses of human pasteurized C1-INH concentrate lead to faster relief of acute symptoms of abdominal and facial attacks compared with placebo in patients with HAE. It is also the largest dose comparison study ever done in HAE. To be eligible for inclusion in the study, patients had to be six years of age or older, with a diagnosis of C1-INH deficiency and a documented history of facial or abdominal attacks.

The primary objective was to show that C1-INH shortens the time to onset of relief of symptoms of abdominal or facial HAE attacks. The primary endpoint was time between start of C1-INH administration and onset of relief of symptoms from abdominal or facial attacks as determined by the subject's assessment. The median time to onset of relief of symptoms in the 20 U/kg b.w. C1-INH treatment group was 30 minutes, compared to 1.5 hours for patients who received placebo.

The secondary endpoint, that is, the proportion of subjects with increased intensity of clinical HAE symptoms between two and four hours aft
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