APeX-2
12 evidence topics · 3 sources
Objective
Treatment evaluation objective
Location and study date
Study design
Eligibility criteria
Run-in attack requirement
Treatment
Berotralstat 110-mg and 150-mg regimens: route and administration
Study outcomes
Rationale for using monthly HAE attack rate as the primary endpoint
Not applicable.
Statistical analysis description
Number of participants (Planned and analyzed)
Description of analysis sets
Results
Participant disposition
Treatment exposure
| Population | Participants |
|---|---|
| Received at least one dose | “120” |
Baseline characteristics
Median baseline attack rate during the prospective run-in period
Efficacy results
Health-related quality of life and patient-reported outcomes
No evidence found.
Safety results
Serious treatment-emergent adverse events considered drug related
Study limitations
Summary
The study randomized 121 participants and treated 120. The efficacy population was intent-to-treat, whereas the reported treated populations included 41 participants receiving 110 mg, 40 receiving 150 mg, and 39 receiving placebo. The 24-week comparison evaluates ongoing oral prophylaxis and does not provide a direct comparison with lonvo-z. The absence of drug-related serious adverse events in this sample does not exclude uncommon risks.