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Clinical Trials in Poland / NCT04933721
Enrolling by invitation Phase 3

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

NCT04933721 · tracked via the Priya Life Science Poland tracker
Sponsor
BioCryst Pharmaceuticals
Phase
Phase 3
Started
2021-07-23
Last updated
2025-12-23

Condition(s) studied

Hereditary AngioedemaHAE

Investigational drug(s) / intervention(s)

berotralstat →

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

Trial sites (16)

FacilityCityRegionStatus
Study Center Ottawa Canada
Study Center Brno Czechia
Study Center Pilsen Czechia
Study Center Grenoble France
Study Center Marseille France
Study Center Paris France
Study Center Frankfurt Germany
Study Center Skopje North Macedonia
Study Center Krakow Poland
Study Center Martin Slovakia
Study Center Cape Town South Africa
Study Center Daegu South Korea
Study Center Gwangju South Korea
Study Center Seoul South Korea
Study Center Madrid Spain
Study Center Bristol United Kingdom

More BioCryst Pharmaceuticals trials in Poland

Berotralstat Treatment in Children With Hereditary Angioedema Active, not recruiting

Other trials for the same condition

Berotralstat Treatment in Children With Hereditary Angioedema Active, not recruiting
Extension Study of Oral PHA-022121 for Acute Treatment of Angioedema Attacks in Patients With Hereditary Angioedema Enrolling by invitation
STOP-HAE: A Phase 3 Study of ADX-324 in HAE Recruiting
A Study to Assess the Long-Term Safety and Efficacy of Donidalorsen in the Prophylactic Treatment of Hereditary Angioedema (HAE) Active, not recruiting
Phase 3 Extension Study of ADX-324 in Participants With Hereditary Angioedema (HAE) Recruiting
Official registry record

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.

View NCT04933721 on ClinicalTrials.gov ↗ ← All trials in Poland