Berotralstat for Hereditary Angioedema
(APeX-A Trial)
What You Need to Know Before You Apply
What is the purpose of this trial?
This trial aims to provide continued access to berotralstat, a medication for people with hereditary angioedema (HAE), a condition that causes sudden and severe swelling. It is intended for those who participated in previous berotralstat studies and found the treatment beneficial. Participants should not use other long-term treatments for HAE attacks. As a Phase 3 trial, this study represents the final step before FDA approval, offering participants a chance to contribute to the potential availability of this treatment for others.
Do I need to stop my current medications to join the trial?
Yes, you will need to stop taking other medications for long-term prevention of HAE attacks and any other investigational drugs during the study.
Is there any evidence suggesting that berotralstat is likely to be safe for humans?
Research has shown that berotralstat is generally safe for people with hereditary angioedema (HAE). Long-term studies found that it effectively reduces the number of HAE attacks. Importantly, these studies identified no major safety issues. Participants took berotralstat daily, and it was well-tolerated across all age groups, including children, teenagers, and adults. This consistent safety record makes berotralstat a promising option for managing HAE.12345
Why do researchers think this study treatment might be promising for hereditary angioedema?
Berotralstat is unique because it offers a once-daily oral option for managing hereditary angioedema (HAE), which is a departure from the injections or intravenous treatments commonly used for this condition. Its mechanism of action targets the kallikrein-kinin system, which helps prevent the excessive buildup of bradykinin, a protein that contributes to swelling in HAE. Researchers are excited about berotralstat because it simplifies treatment with a convenient oral form, potentially improving patient adherence and quality of life.
What is the effectiveness track record for berotralstat in treating hereditary angioedema?
Research shows that berotralstat helps reduce the number of hereditary angioedema (HAE) attacks. Participants in this trial will receive berotralstat, which studies have shown results in a 29-100% drop in attack frequency. Many also experienced less severe attacks. Studies confirm that patients who switched to berotralstat managed their symptoms well and felt more satisfied with their treatment. Overall, berotralstat has consistently proven safe and effective for people of all ages, leading to a better quality of life for those with HAE.34567
Who Is on the Research Team?
Vesna Grivcheva-Panoska, MD, PhD
Principal Investigator
PHI University Clinic of Dermatology
Are You a Good Fit for This Trial?
This trial is for men and women with Hereditary Angioedema (HAE) who were already part of previous berotralstat studies. They must be able to give written consent, benefit from ongoing berotralstat treatment, and use effective contraception.Inclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Treatment
Participants receive berotralstat 150 mg orally once daily, with pediatric participants receiving a weight-appropriate dose
Follow-up
Participants are monitored for safety and effectiveness after treatment
What Are the Treatments Tested in This Trial?
Interventions
- berotralstat
Trial Overview
The study is an open-label phase 3b trial that allows participants continued access to the medication berotralstat, which they received in earlier trials. It aims to provide further data on its long-term usage.
How Is the Trial Designed?
1
Treatment groups
Experimental Treatment
Berotralstat (BCX7353) capsules or granules orally administered once daily.
Find a Clinic Near You
Who Is Running the Clinical Trial?
BioCryst Pharmaceuticals
Lead Sponsor
Citations
Long-term safety and efficacy of once-daily berotralstat in ...
Treatment with berotralstat led to improvements in HAE attack rates and Angioedema Quality of Life Questionnaire scores up to week 96, with greater improvements ...
Berotralstat effectiveness and safety in patients with ...
Five patients showed a response to berotralstat, observed as a 29-100% reduction in attack rates. Three patients experienced a reduction in attack severity from ...
Hereditary angioedema outcomes in US patients switched ...
Patients switching to berotralstat monotherapy maintained good control of their HAE symptoms and reported improved treatment satisfaction.
4.
ir.biocryst.com
ir.biocryst.com/news-releases/news-release-details/biocryst-presents-new-data-long-term-efficacy-and-safetyBioCryst Presents New Data on the Long-term Efficacy and ...
—Data across pediatric, adolescent, and adult populations demonstrate sustained reductions in HAE attacks and consistent safety profile—.
Hereditary angioedema outcomes in US patients switched ...
Patients switching to berotralstat monotherapy maintained good control of their HAE symptoms and reported improved treatment satisfaction.
Berotralstat effectiveness and safety in patients with ...
Long-term prophylactic berotralstat was effective in reducing HAE attacks for five out of six patients with HAE-nC1-INH. No significant safety ...
NCT03472040 | A Long Term Safety Study of BCX7353 in ...
This is an open-label study to evaluate the long term safety and effectiveness of oral treatment with BCX7353 in preventing acute angioedema attacks.
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