Clinical evidence
3.1.1HAELO
31 evidence topics · 12 sources
3.1.1.2Location and study date
First participant dosed: announcement date
Follow-up at the February 10, 2026 data cutoff
Phase 3 HAELO: study design
3.1.1.4Eligibility criteria
Registry inclusion criteria
Registry excluded HAE population
Phase 3 HAELO: randomization and dose
Optional crossover for the placebo group
3.1.1.6.1Rationale for using monthly HAE attack rate as the primary endpoint
Primary clinical outcome: HAE attack frequency during weeks 5–28
Validation of outcomes instruments: AE-QoL minimal clinically important difference
Validation of outcomes instruments: AE-QoL content validation and psychometric evaluation
Validation of outcomes instruments: AE-QoL content validation in adults and adolescents
Validation of outcomes instruments: AE-QoL psychometric validation
3.1.1.7Statistical analysis description
3.1.1.7.1Number of participants (Planned and analyzed)
Originally planned enrollment
Registry actual enrollment
3.1.1.7.2Description of analysis sets
Analysis after initiation of long-term prophylaxis
3.1.1.8.1Participant disposition
Reported treatment groups
3.1.1.8.2Baseline characteristics
Phase 3 baseline characteristics
3.1.1.8.3Efficacy results
Phase 3 HAELO: primary endpoint, weeks 5–28
Phase 3 HAELO: relative attack-rate reduction
Phase 3 HAELO: attack-free status, weeks 5–28
Phase 3 HAELO: additional attack-related secondary endpoints
3.1.1.8.4Health-related quality of life and patient-reported outcomes
Phase 3 HAELO: AE-QoL secondary endpoint
Week 28 AE-QoL assessments
Phase 3 HAELO: safety through week 28
Phase 3 HAELO: infusion-related reactions
Phase 3 HAELO: infusion-related reaction severity and duration
Phase 3 HAELO: hepatic laboratory findings
Summary
The randomized comparison enrolled 80 participants, with 52 receiving lonvo-z and 28 receiving placebo. Median follow-up was 7.5 months. This follow-up does not establish lifetime durability or exclude uncommon delayed adverse effects. Attack data after initiation of long-term prophylaxis were excluded from the reported attack-rate analysis. Week 28 AE-QoL data were available for 44 participants receiving lonvo-z and 20 receiving placebo, fewer than the randomized populations. The placebo-controlled design does not establish comparative effectiveness against an active prophylactic treatment. Eligibility excluded HAE with normal C1 inhibitor.