BCX7353-312
Research type
Research Study
Full title
An open-label study to provide berotralstat access to subjects with type I and II hereditary angioedema who were previously enrolled in berotralstat studies
IRAS ID
1011988
Contact name
Emilia Crane
Contact email
Sponsor organisation
BioCryst Pharmaceuticals Inc
Eudract number
2024-511285-37
Clinicaltrials.gov Identifier
Research summary
The BCX7353-312 study will test the long-term safety of berotralstat taken once a day in preventing attacks of hereditary angioedema (HAE) in patients.
This study will include patients who have previously taken part in Berotralstat studies, in the UK this will be the BCX7353-304 study. Patients will receive berotralstat 150mg capsules or granules, this will depend on age and weight. Berotralstat is licensed in the UK Orladeyo® capsules for use in HAE patients aged 12 years or older. This study plans to recruit approximately 139 participants. The primary objective of the study is to evaluate the long-term safety of long-term prophylactic treatment in HAE. The primary endpoints for assessing the objective will be the number and proportion of participants with treatment emergent adverse events (TEAE) and serious adverse events (SAE). The total duration of the trial will be until 16 years of age and has access to berotralstat through another mechanism or up to 5 years, whichever comes first.
The duration participants will be in the trial is 240 weeks, with 10 visits, every 24 weeks, throughout the study, all of which will be at the hospital. For participants who reach 240 weeks or who stop their participation prior to the end-of-study, a follow-up visit will be conducted 3 weeks after their last dose of study drug. A follow-up visit is not required for participants who discontinue the study because they are receiving berotralstat via another mechanism.REC name
Wales REC 3
REC reference
25/WA/0194
Date of REC Opinion
7 Aug 2025
REC opinion
Further Information Favourable Opinion