PURPOSE OF THE STUDY.
To determine if functional levels of C1 inhibitor activity would provide effective prophylaxis against attacks of hereditary angioedema (HAE).
Patients who were 12 years or older with type 1 or 2 HAE and had 4 or more attacks in a consecutive 2-month period within 3 months before screening.
This was an international, prospective, multicenter, randomized, double-blind, placebo-controlled, dose-ranging, phase 3 trial to evaluate the efficacy and safety of self-administered subcutaneous CSL830. Patients were randomly assigned to 1 of 4 treatment sequences in a crossover design consisting of two 16-week treatment periods using either 40 IU or 60 IU of CSL830 per kilogram of body weight twice weekly or a placebo. The primary efficacy end point was the number of attacks of angioedema, and the secondary end point was the portion of patients who had a response of >50% reduction in attacks.
Of the 90 patients who underwent randomization, 78 completed the trial. Both doses compared with the placebo reduced the rate of attacks of HAE: 40 IU, −2.42 attacks per month (95% confidence interval, −3.38 to −1.46); and 60 IU, −3.51 attacks per month (95% confidence interval, −4.12 to −2.81). Response rates were 76% for 40 IU and 90% for 60 IU. The need for rescue medication was reduced from 5.5 uses per month in the placebo group to 1.3 uses per month in the 40 IU group and 0.32 uses per month in the 60 IU group.
This study highlights that self-administration of subcutaneous CSL830 was safe and showed long-term prevention of HAE. Of patients, >50% had no moderate-to-severe attacks while receiving CSL830.
This study helps to understand the effectiveness of a self-administered product in decreasing the significant burden of attacks in this rare disease.
- Copyright © 2017 by the American Academy of Pediatrics