By InvitationPhase 3

Open-label Berotralstat Access to HAE Patients Previously Enrolled in Berotralstat Studies

Canada, Czechia, France139 participantsStarted 2021-07-23

Plain-language summary

This is a phase 3b open-label study providing access to berotralstat for HAE patients who were previously enrolled in berotralstat studies.

Who can participate

Age range

2 Years

Sex

ALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

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.

Questions worth asking your doctor

Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.

1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?

Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.

Questions for the trial coordinator

The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.

1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?

A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.

What they're measuring

Number and proportion of subjects with a treatment-related TEAE

Timeframe: 240 weeks

Number and proportion of subjects who experience a serious adverse event (SAE)

Timeframe: 240 weeks

Number and proportion of subjects who experience a treatment-related Grade 3 or 4 TEAE

Timeframe: 240 weeks

Number and proportion of subjects who experience a treatment-related Grade 3 or 4 treatment-emergent chemistry abnormality

Timeframe: 240 weeks

Number and proportion of subjects who discontinue due to a TEAE

Timeframe: 240 weeks

Trial details

NCT IDNCT04933721

SponsorBioCryst Pharmaceuticals

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2031-08

View on ClinicalTrials.gov More Hereditary Angioedema trials

Who can participate

Age range

2 Years

Sex

ALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

What they're measuring

Number and proportion of subjects with a treatment-related TEAE

Timeframe: 240 weeks

Number and proportion of subjects who experience a serious adverse event (SAE)

Timeframe: 240 weeks

Number and proportion of subjects who experience a treatment-related Grade 3 or 4 TEAE

Timeframe: 240 weeks

Number and proportion of subjects who experience a treatment-related Grade 3 or 4 treatment-emergent chemistry abnormality

Timeframe: 240 weeks

Number and proportion of subjects who discontinue due to a TEAE

Timeframe: 240 weeks