Efficacy and Safety of BCX7353 to Prevent Angioedema Attacks in Subjects With Hereditary Angioedema

• Status: Completed
• Trial end date: 2017-08-01
• Target enrollment: 0
• Participant gender: All

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.

• Phase: Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: BioCryst Pharmaceuticals
• Treatments: Berotralstat

Criteria

Key Inclusion Criteria:

• A clinical diagnosis of HAE type I or II

• Documented HAE attacks within a defined calendar period

• Access to acute attack medications

• Sexually active women of child-bearing potential and sexually active men must utilize effective contraception

Key Exclusion Criteria:

• Women who are pregnant or breast-feeding

• Any clinical condition or medical history that would interfere with the subject's safety or ability to participate in the study

• Use of C1INH, androgens or tranexamic acid for prophylaxis of HAE attacks

• History of or current alcohol or drug abuse

• Infection with hepatitis B, hepatitis C or HIV

• Participation in any other investigational drug study currently or within the last 30 days