Clinical Trial: Efficacy and Safety of BCX7353 to Prevent Angioedema Attacks in Subjects With Hereditary Angioedema
Study Status: Recruiting
Recruit Status: Recruiting
Study Type: Interventional
Official Title: A Randomized, Double-blind, Placebo-controlled, Dose-ranging, Parallel-group Study to Evaluate the Efficacy, Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of BCX7353 as a Preventative T
Brief Summary: This 3-part study will evaluate the safety and efficacy of an oral treatment, BCX7353, in preventing angioedema attacks in subjects with hereditary angioedema (HAE). In Part 1 of the study, eligible subjects will be randomized to receive oral BCX7353 or placebo for 4 weeks. Assuming successful completion of Part 1, additional subjects will be randomized in Part 2 to one of 2 lower doses of BCX7353 or placebo. Part 3 will enroll additional subjects into one of three doses of BCX7353 or placebo. The study will compare the number of acute attacks in each treatment group, as well as a number of other clinical and pharmacologic outcomes, and the safety and tolerability of each dose of BCX7353 compared to placebo.
Detailed Summary:
Sponsor: BioCryst Pharmaceuticals
Current Primary Outcome: Number of confirmed HAE attacks [ Time Frame: 28 days ]
Original Primary Outcome: Same as current
Current Secondary Outcome:
- Incidence and severity of adverse events and laboratory abnormalities [ Time Frame: 28 days ]
- Quality of Life, as measured by the Angioedema Quality of Life Questionnaire [ Time Frame: 28 days ]
- Quality of Life, as measured by the Depression, Anxiety, Stress Scales (DASS) questionnaire [ Time Frame: 28 days ]
- Plasma concentrations of BCX7353 at steady state [ Time Frame: 28 days ]
Original Secondary Outcome: Same as current
Information By: BioCryst Pharmaceuticals
Dates:
Date Received: August 10, 2016
Date Started: August 2016
Date Completion: April 2017
Last Updated: April 17, 2017
Last Verified: April 2017
