Clinical evidence
3.1.2ITL-2002-CL-001
34 evidence topics · 12 sources
Phase 1/2 registry objectives
Phase 1 primary objectives
3.1.2.2Location and study date
Phase 1 durability analysis: data cutoff
3.1.2.4Eligibility criteria
Phase 1/2 registry inclusion criteria
Phase 1/2 registry exclusion criterion
3.1.2.6.1Rationale for using monthly HAE attack rate as the primary endpoint
Phase 2 primary endpoint: weeks 1–16
Pooled 50-mg analysis: composite outcome
3.1.2.7Statistical analysis description
3.1.2.7.1Number of participants (Planned and analyzed)
Registry actual enrollment across phases
Phase 2 randomized and treated populations
3.1.2.7.2Description of analysis sets
Phase 2 attack-rate analysis
Phase 2 paired AE-QoL analysis
Pooled 50-mg analysis: time origin
3.1.2.8.1Participant disposition
Pooled 50-mg analysis population
3.1.2.8.2Baseline characteristics
Phase 2 baseline characteristics
3.1.2.8.3Efficacy results
Phase 1 follow-up: EAACI 2025
Phase 2: mean monthly attack rate, weeks 1–16
Phase 2: difference versus placebo
Phase 2: 50-mg group complete response
Pooled phase 1/2 and long-term follow-up: ACAAI 2025, August 29, 2025 data cutoff
Pooled phase 1/2 and long-term follow-up: median follow-up, AAAAI 2026
Pooled phase 1/2 and long-term follow-up: attack-free status and prophylaxis, AAAAI 2026
3.1.2.8.4Health-related quality of life and patient-reported outcomes
Phase 2 patient-reported outcomes: AAAAI 2025
Phase 2 AE-QoL total score: change from baseline to week 16
Phase 2 safety: October 2024 report
Phase 2 safety: placebo-arm exception in the same report
Pooled phase 1/2 and long-term follow-up: serious pulmonary embolism
Summary
Phase 1 was open-label and included 10 participants across three dose cohorts. Phase 2 randomized 27 participants, including six assigned to placebo. The reported confidence intervals for the differences in monthly attack rate versus placebo crossed zero, so the point estimates alone do not demonstrate a statistically conclusive difference. The AE-QoL analysis included only participants with paired assessments and had nine, nine, and four participants in the 25-mg, 50-mg, and placebo groups, respectively. The pooled 50-mg analyses combine phase 1/2 and long-term follow-up observations. Becoming attack-free and prophylaxis-free during follow-up is not the same outcome as remaining attack-free throughout follow-up. Seven participants had less than six months of follow-up after becoming attack-free and prophylaxis-free. These pooled observations do not establish lifetime durability.
Pooled phase 1/2 and long-term follow-up: follow-up limitation