Phase 3
Completed N=387
A Long Term Safety Study of BCX7353 in Hereditary Angioedema
Hereditary Angioedema · HAE · Prophylaxis
Source: ClinicalTrials.gov NCT03472040 ↗
Enrolled (actual)
387
Serious AEs
11.1%
Results posted
Jun 2023
Primary outcomePrimary: Safety & Tolerability — 94; 240; 58; 119 Participants
◆ Published Evidence
Emerging
13citations · ~3 / year
Shared decision-making in the management of hereditary angioedema: An analysis of patient and physician perspectives.
Summary
This is an open-label study to evaluate the long term safety and effectiveness of oral treatment with BCX7353 in preventing acute angioedema attacks in patients with Type I and Type II Hereditary Angioedema (HAE).
Linked Publications (5)
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Shared decision-making in the management of hereditary angioedema: An analysis of patient and physician perspectives.
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Hereditary angioedema outcomes in US patients switched from injectable long-term prophylactic medication to oral berotralstat.
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Assessment of HAE prophylaxis transition from androgen therapy to berotralstat: A subset analysis of the APeX-S trial.
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A review of berotralstat for the treatment of hereditary angioedema.
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Long-term safety and efficacy of once-daily berotralstat in patients with hereditary angioedema: APeX-S final results.
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Safety & Tolerability |
94; 240; 58; 119; 23; 20 | — |
| SECONDARY Incidence of Acute Attacks of Angioedema in Subjects During Treatment |
95; 231; 87; 214; 59; 99 | — |
| SECONDARY The Durability of Response to Treatment |
1.276; 1.079; 1.005; 0.694; 1.121; 0.591 | — |
| SECONDARY Patient Reported Quality of Life (QoL) During Treatment |
-8.85; -10.38; -11.20; -10.39; -11.92; -11.47 | — |
| SECONDARY Patient's Satisfaction With Medication During Long Term Administration of Berotralstat |
-4.1; -1.3; -0.6; -3.6; 1.5; -0.1 | — |
Eligibility Criteria
Key Inclusion Criteria
- Subjects with HAE Type I or II who either have participated in a previous BCX7353 study or, in selected countries, in the opinion of the Investigator are expected to derive benefit from an oral treatment for the prevention of angioedema attacks.
- Access to appropriate medication for treatment of acute attacks
- Acceptable effective contraception
- Written informed consent
Key Exclusion Criteria
- Pregnancy or breast-feeding
- Any clinically significant medical condition or 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
- Any laboratory parameter abnormality that, in the opinion of the Investigator, is clinically significant and relevant for this study
- Discontinuation of study drug due to a hypersensitivity reaction BCX7353 in a prior study
- Severe hypersensitivity to multiple medicinal products or severe hypersensitivity/ anaphylaxis with unclear etiology
- Unacceptable noncompliance in a previous BCX7353 study (if applicable) as assessed by the Sponsor or Investigator
- Investigational drug exposure, other than BCX7353, within 30 days prior to the screening visit (or baseline if no screening visit)
Data sourced from ClinicalTrials.gov (NCT03472040) and the linked publication. Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication. Informational only — not medical advice.