Clinical Trial: Open-label, Long-term Safety Study of Avoralstat in Subjects With Hereditary Angioedema
Study Status: Terminated
Recruit Status: Terminated
Study Type: Interventional
Official Title: OPuS-4: An Open-label Study to Evaluate the Long-term Safety of Avoralstat in Subjects With Hereditary Angioedema
Brief Summary: This is an open-label study designed to evaluate the long-term safety of prophylactic avoralstat (500 mg three times daily) when given to approximately 150 patients with hereditary angioedema (HAE) for a duration of up to 72 weeks. The study will also evaluate the long-term efficacy and impact on quality of life of avoralstat prophylactic treatment.
Detailed Summary:
Sponsor: BioCryst Pharmaceuticals
Current Primary Outcome: Proportion of subjects who discontinue due to a treatment-emergent adverse event, who experience a treatment-emergent serious AE, who experience a Grade 3 or 4 AE and who experience Grade 3 or 4 laboratory abnormalities [ Time Frame: Up to 72 weeks ]
Original Primary Outcome: Same as current
Current Secondary Outcome:
- Angioedema attack rate (subject-reported HAE attacks normalized for time on study) [ Time Frame: Up to 72 weeks ]
- Durability in response (assessed as subject-reported HAE attacks) [ Time Frame: change over time through 72 weeks ]
- Proportion of subjects who discontinue avoralstat with a reason of lack of efficacy [ Time Frame: Up to 72 weeks ]
- Quality of life as determined by the EQ-5D-5L [ Time Frame: Up to 72 weeks ]
- Quality of life as determined by the Angioedema Quality of Life Questionnaire [ Time Frame: Up to 72 weeks ]
Original Secondary Outcome: Same as current
Information By: BioCryst Pharmaceuticals
Dates:
Date Received: January 20, 2016
Date Started: December 2015
Date Completion:
Last Updated: February 15, 2016
Last Verified: February 2016
