Sunday, November 13
Randomized, Double-Blind, Placebo-Controlled Trial of Recombinant Human C1 Inhibitor for Prophylaxis of Hereditary Angioedema Attacks 1:30 PM - 1:45 PM
Introduction: Recombinant human C1 inhibitor (rhC1INH) is an approved, on-demand treatment for hereditary angioedema (HAE) attacks. Open-label data suggest that rhC1INH may prevent HAE attacks. Methods: Patients =13 years of age with functional C1INH levels <50% of normal and history of =4 HAE attacks during the preceding 3 months were included in this phase 2, double-blind, 3-period crossover study. Patients received intravenous rhC1INH 50 IU/kg (max, 4200 IU) once and twice weekly and placebo in three 4-week treatment periods that were separated by 1 week. With the crossover design, all patients received each of the dosing regimens. Attack symptoms were recorded daily. The number of HAE attacks per 4-week treatment phase (primary endpoint) and percentage of patients with clinical response (=50% reduction in number of attacks from treatment with placebo to treatment with rhC1INH; secondary endpoint) were determined. Adverse events (AEs) and immunogenicity were also assessed. Results: Thirty-two patients were randomized to treatment. Mean number of HAE attacks was significantly reduced with rhC1INH twice weekly (P < 0.0001) and once weekly (P = 0.0004) versus placebo (Figure). Most patients (74.2%; 95% CI, 57-86) who received rhC1INH twice weekly and 41.9% (95% CI, 26-59) who received rhC1INH once weekly had =50% reduction in number of HAE attacks. No patients withdrew because of AEs and no thrombotic or thromboembolic events, drug hypersensitivity or anaphylactic reactions, or neutralizing antibodies were reported. Conclusion: rhC1INH provided clinically relevant reductions in HAE attack frequency and was well-tolerated. Data support the continued development of rhC1INH for HAE prophylaxis.
Presenting Author: Marco Cicardi, MD
Author.: Marc Andrew Riedl, MD, MS
Author.: Vesna Grivcheva Panovska, MD
Author.: Dumitru Moldovan, MD
Author.: James Baker, MD
Author.: William Yang, MD, FAAAAI
Author.: Avner Reshef, MD
Author.: Sladjana Andrejevic, MD
Author.: Richard Lockey, MD
Author.: Roman Hakl, MD
Author.: Shmuel Kivity, MD
Author.: Luca Bellizzi, MD
Author.: Joseph R. Harper, PharmD
Author.: Anurag Relan, MD
Format: Oral
Category: Angioedema/Urticaria
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