A Phase 3 interventional study of berotralstat in Hereditary Angioedema and HAE, sponsored by BioCryst Pharmaceuticals. Enrolling by invitation at 16 sites in 11 countries. Open to participants aged 2 Years and older. Per ClinicalTrials.gov, last updated 2025-12-23.
Sponsored by BioCryst Pharmaceuticals · Phase 3, Interventional, and Prevention
This is a phase 3b open-label study providing access to berotralstat for HAE patients who were previously enrolled in berotralstat studies.
APeX-A, 7353-312, is a single-arm, open-label, multicenter study. The study will be conducted in countries where berotralstat is not available either commercially or via another mechanism and is planned in countries where pediatric participants have been enrolled in BCX7353-304 (Study 304). Adult and adolescent (≥12 years) participants will receive berotralstat 150 mg administered orally once daily (QD). Pediatric participants will receive a weight-appropriate dose. The study will assess the long term safety and tolerability of berotralstat.
Eligible subjects will be enrolled directly into Study BCX7353-312 (Study 312) from Studies 302, 204, and 304. Subjects will receive berotralstat administered orally once daily (QD). Subjects will return to the clinic every 24 weeks for drug dispensation and safety monitoring. Participants form Studies 302 and 204 may remain on study for up to 480 weeks (approximately 10 years). Participants from Study 304 may remain in the study until 16 years of age and have access to berotralstat via another mechanism; or up to 5 years, whichever comes first.
Safety and tolerability will be evaluated through assessment of serious adverse events (SAEs) and treatment-related, treatment-emergent adverse events (TEAEs). Up to 139 subjects are planned to enroll.
171 studies on the registry are indexed under Angioedemas, Hereditary; 24 are open to participants now.
This study's planned enrollment of 139 is above the median of 48 across 116 interventional studies indexed under Angioedemas, Hereditary.
Browse Angioedemas, Hereditary studies →BioCryst Pharmaceuticals is the lead sponsor of 54 studies on the registry; 1 is open to participants now.
Of its 10 completed or terminated interventional studies of FDA-regulated products, 8 (80%) have results posted.
Counted across the registry records on this site, refreshed daily.
Key Inclusion Criteria:
Key Exclusion Criteria:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Contraception
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Investigational drug or device
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Berotralstat (BCX7353) capsules or granules orally administered once daily.
Drug: berotralstat
BCX7353 capsules or granules administered orally once daily
Also known as: Orladeyo
Number and proportion of subjects with a treatment-related TEAE
Time frame: 240 weeks
Number and proportion of subjects who experience a serious adverse event (SAE)
Time frame: 240 weeks
Number and proportion of subjects who experience a treatment-related Grade 3 or 4 TEAE
Time frame: 240 weeks
Number and proportion of subjects who experience a treatment-related Grade 3 or 4 treatment-emergent chemistry abnormality
Time frame: 240 weeks
Number and proportion of subjects who discontinue due to a TEAE
Time frame: 240 weeks
Plan to share: No
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Studies of the drug tested here: Berotralstat