Clinical trial NCT04933721: Open-label Berotralstat Access to HAE Patients Previously Enrolled in Berotralstat Studies
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
Study type: INTERVENTIONAL
Overall status: ACTIVE_NOT_RECRUITING
Phase: PHASE3
Conditions: Hereditary Angioedema
HAE
Keywords: prophylaxis
Lead sponsor: BioCryst Pharmaceuticals
Sponsor class: INDUSTRY
First posted: 2021-06-22
Enrollment: 139
Sex: ALL
Minimum age (years): 2
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Intervention model description: Open-label berotralstat (BCX7353) orally administered QD.
Primary purpose: PREVENTION

Brief summary: 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.

Eligibility criteria: 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 outcomes:
measure: Number and proportion of subjects with a treatment-related TEAE
timeFrame: 240 weeks
measure: Number and proportion of subjects who experience a serious adverse event (SAE)
timeFrame: 240 weeks
measure: Number and proportion of subjects who experience a treatment-related Grade 3 or 4 TEAE
timeFrame: 240 weeks
measure: Number and proportion of subjects who experience a treatment-related Grade 3 or 4 treatment-emergent chemistry abnormality
timeFrame: 240 weeks
measure: Number and proportion of subjects who discontinue due to a TEAE
timeFrame: 240 weeks
Secondary outcomes:
Other outcomes:
Locations:
city: Brno
country: Czechia
facility: Study Center
city: Plzen
country: Czechia
facility: Study Center
city: Grenoble
country: France
facility: Study Center
city: Paris
country: France
facility: Study Center
city: Daegu
country: Korea, Republic of
facility: Study Center
city: Gwangju
country: Korea, Republic of
facility: Study Center
city: Seoul
country: Korea, Republic of
facility: Study Center
city: Skopje
country: North Macedonia
facility: Study Center
city: Kraków
country: Poland
facility: Study Center
city: Martin
country: Slovakia
facility: Study Center
city: Cape Town
country: South Africa
facility: Study Center
Study officials:
affiliation: PHI University Clinic of Dermatology
name: Vesna Grivcheva-Panoska, MD, PhD
role: PRINCIPAL_INVESTIGATOR
References: