berotralstat: BCX7353 capsules or granules administered orally once daily
Study summary
This is a phase 3b open-label study providing access to berotralstat for HAE patients who were previously enrolled in berotralstat studies.
Eligibility
Sex
ALL
Min age
2 Years
Max age
—
Healthy volunteers
No
Key Inclusion Criteria:
* Males and females currently enrolled in BioCryst-sponsored Study 302, 204, or 304
* Participant or parent/legally designated representative (for participants \<18 years of age) able to provide written informed consent.
* Would benefit from continued berotralstat treatment
* Acceptable effective contraception
Key Exclusion Criteria:
* Pregnancy or breast-feeding
* Any condition or situation, including medical history that, in the opinion of the investigator or sponsor, would interfere with the subject's safety or ability to participate in the study
* Use of other medications for long-term prophylaxis of HAE attacks at the Baseline visit or any time during the study.
* Use of any other investigational medicinal product at the Baseline visit or any time during the study.
Primary outcome measure(s)
Number and proportion of subjects with a treatment-related TEAE — 240 weeks
Number and proportion of subjects who experience a serious adverse event (SAE) — 240 weeks
Number and proportion of subjects who experience a treatment-related Grade 3 or 4 TEAE — 240 weeks
Number and proportion of subjects who experience a treatment-related Grade 3 or 4 treatment-emergent chemistry abnormality — 240 weeks
Number and proportion of subjects who discontinue due to a TEAE — 240 weeks
This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
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