Hereditary AngioedemaPhase 3APeX-ANCT04933721
Open-label Berotralstat Access to HAE Patients Previously Enrolled in Berotralstat Studies
A Phase 3 study testing berotralstat in people with Hereditary Angioedema and HAE, run by BioCryst Pharmaceuticals at 16 sites in 11 countries.
Trial summary
This is a phase 3b open-label study providing access to berotralstat for HAE patients who were previously enrolled in berotralstat studies. Read more about this study
- Who is it for?
- Ages 2 years and older
- Conditions the study is for
- Hereditary Angioedema, HAE
- What is being tested?
- berotralstat
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 139 participants
- Where
- 16 sitesin 11 countries
- Study dates
- 23 Jul 2021to Aug 2031
- Phase
- Phase 3
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 2 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- 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
Who cannot join
- 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.
Trial locations and contacts
16 sites in 11 countries. Contact the study team or a site near you to ask about taking part.
16 sites in 11 countries
South Korea3 sites
Study Center
Daegu
Study Center
Gwangju
Study Center
Seoul
About this study
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
This is a phase 3b open-label study providing access to berotralstat for HAE patients who were previously enrolled in berotralstat studies.
Read the 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.
- Conditions
- Hereditary Angioedema, HAE
- Being tested
- berotralstat
- Keywords
- prophylaxis
- Medical subject headings
- Angioedemas, Hereditary
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
berotralstatDrug
Also called Orladeyo
BCX7353 capsules or granules administered orally once daily
| Study group | Role | What they receive |
|---|---|---|
| BCX7353 capsules or granules once daily | Experimental | Berotralstat (BCX7353) capsules or granules orally administered once daily. Receives: Drug: berotralstat |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number and proportion of subjects with a treatment-related TEAE
Measured: 240 weeks
Number and proportion of subjects who experience a serious adverse event (SAE)
Measured: 240 weeks
Number and proportion of subjects who experience a treatment-related Grade 3 or 4 TEAE
Measured: 240 weeks
Number and proportion of subjects who experience a treatment-related Grade 3 or 4 treatment-emergent chemistry abnormality
Measured: 240 weeks
Number and proportion of subjects who discontinue due to a TEAE
Measured: 240 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted22 Jun 2021
- Study start23 Jul 2021
- Primary completionAug 2031 (estimated)
- Study completionAug 2031 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Hereditary AngioedemaStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Prevention
- Allocation
- Not applicable
- Intervention model
- Single group
- Model description
- Open-label berotralstat (BCX7353) orally administered QD.
- Masking
- None (open label)
- Enrolment
- 139 participants (estimated)
Sponsor and investigators
- Lead sponsor
- BioCryst Pharmaceuticals
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Vesna Grivcheva-Panoska, MD, PhD, Principal investigator, PHI University Clinic of Dermatology
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04933721
- Sponsor's protocol ID
- BCX7353-312
- Acronym
- APeX-A
- Status last verified
- Dec 2025
- First submitted
- 14 Jun 2021
- Last updated
- 23 Dec 2025
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 23 Dec 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT04933721
- Acronym / sponsor ID
- APeX-A / BCX7353-312
- Conditions
- Hereditary Angioedema, HAE
- Keywords
- prophylaxis
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Enrolling by invitation
- Enrolment
- 139 participants (estimated)
- Lead sponsor
- BioCryst PharmaceuticalsIndustry (pharma, biotech, devices)
- Study start
- 23 Jul 2021 (actual)
- Primary completion
- Aug 2031 (estimated)
- Study completion
- Aug 2031 (estimated)
- First posted
- 22 Jun 2021 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Prevention
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
- Model description
- Open-label berotralstat (BCX7353) orally administered QD.
| Arm | Type | Receives |
|---|---|---|
| BCX7353 capsules or granules once daily | Experimental | Drug: berotralstatBerotralstat (BCX7353) capsules or granules orally administered once daily. |
Who may qualify
- Ages
- 2 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (4 listed)
- 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 (4 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| berotralstatAlso: OrladeyoBCX7353 capsules or granules administered orally once daily | Drug | BCX7353 capsules or granules once daily |
Primary outcome
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
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 23 Dec 2025.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Vesna Grivcheva-Panoska, MD, PhD principal investigator
PHI University Clinic of Dermatology
16 sites in 11 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing berotralstat in people with Hereditary Angioedema and HAE, run by BioCryst Pharmaceuticals at 16 sites in 11 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 4 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: enrolling by invitation, as of the sponsor's last update on 23 Dec 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 16 sites in France, South Korea, Czechia, Canada, Germany and 6 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Number and proportion of subjects with a treatment-related TEAE (time frame: 240 weeks). The study lists 5 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 23 Dec 2025.