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Enrolling by invitation NCT04933721

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

Phase III Interventional Hereditary Angioedema HAE

For patients and families

In plain language

An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.

What is being studied
The protocol lists: berotralstat.
Who it may be relevant to
Registry conditions: Hereditary Angioedema, HAE. Basic parameters: from 2 years · All.
What needs checking
Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
Where it takes place
Canada, Czechia, France, Germany, North Macedonia +6
Next step
Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →

Unsure about the terms? Read our patient guide →

Official title

An Open-label Study to Provide Berotralstat Access to Subjects With Type 1 and 2 Hereditary Angioedema Who Were Previously Enrolled in Berotralstat Studies

Overview

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

Detailed description

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.

Interventions

  • Drug berotralstat
    BCX7353 capsules or granules administered orally once daily

Primary outcome measures

  • 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]

Eligibility criteria

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

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.

Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.

Healthy volunteers: No

Study design

Allocation
N/A
Model
Single group
Masking
Open label
Primary purpose
Prevention

Study locations

France · 3 centers
  • Study Center — Grenoble
  • Study Center — Marseille
  • Study Center — Paris
South Korea · 3 centers
  • Study Center — Daegu
  • Study Center — Gwangju
  • Study Center — Seoul
Czechia · 2 centers
  • Study Center — Brno
  • Study Center — Pilsen
Canada · 1 center
  • Study Center — Ottawa
Germany · 1 center
  • Study Center — Frankfurt
North Macedonia · 1 center
  • Study Center — Skopje
Poland · 1 center
  • Study Center — Krakow
Slovakia · 1 center
  • Study Center — Martin
South Africa · 1 center
  • Study Center — Cape Town
Spain · 1 center
  • Study Center — Madrid
United Kingdom · 1 center
  • Study Center — Bristol

Identifiers

NCT: NCT04933721 · BCX7353-312

Primary sources (government registries)

View this study on ClinicalTrials.gov ↗