STOP-HAE: A Phase 3, Randomized, Double-Blind, Placebo-Controlled Study of siRNA Targeting of Prekallikrein With ADX-324 in Participants With Hereditary Angioedema
Evaluate the efficacy safety, pharmacokinetics (PK), pharmacodynamics (PD), and health-related quality of life (HRQoL) measures in participants with Type I and Type II HAE.
• Age ≥18 years at the time of signing informed consent.
• Have a documented diagnosis of HAE-1/HAE-2 (Type I or II)
• Experience ≥1 Investigator-confirmed HAE attack in the first 4 weeks of Screening or ≥2 Investigator-confirmed HAE attacks in 8 weeks of Screening.
• Have access to, and the ability to use, at least one acute HAE therapy to treat HAE attacks (eg, plasma-derived or recombinant C1-INH concentrate or a BK2-receptor antagonist) that has previously been shown to be effective for the participant.
• Participants must be deemed medically appropriate for on-demand treatment as the sole medicinal management for their HAE during the study, per Investigator.
• Women of childbearing potential must have a negative serum pregnancy test during Screening and a negative urine pregnancy test on Study Day 1 before study drug administration and must agree to use acceptable contraceptive methods if engaged in sexual activity of childbearing potential (refer to Section 13.2.2) from the time of signing the informed consent form (ICF) until the EOS Visit or 1 month after study drug administration, whichever is longer.