STOP-HAE: A Phase 3 Study of ADX-324 in HAE
Research type
Research Study
Full title
STOP-HAE: A Phase 3, Randomized, Double-Blind, Placebo-Controlled Study of siRNA Targeting of Prekallikrein With ADX-324 in Participants With Hereditary Angioedema
IRAS ID
1012410
Contact name
Chris Storgard
Contact email
Sponsor organisation
ADARx Pharmaceuticals, Inc.
Clinicaltrials.gov Identifier
Research summary
Hereditary angioedema (HAE) is a rare genetic disorder that affects approximately 1 in 50,000 people worldwide. HAE is characterised by recurrent and unpredictable attacks of swelling typically involving the face, tongue, throat, arms, legs, gastrointestinal tract, and/or genitalia that can be painful, debilitating, and life threatening. Current treatments for HAE help manage sudden attacks or reduce how often they occur. However, the most effective treatment often involve frequent injection or infusions, which can be a burden for patients. There is a clear need for a highly effective treatment that minimises treatment burden. ADARx Pharmaceuticals, Inc. is developing ADX-324, a new drug, to help prevent HAE attacks. It works by reducing a protein in the body that is involved in swelling, thereby reducing HAE attacks. This study will evaluate how effective ADX-324 is in preventing HAE attacks compared with a placebo (a similar looking 'drug' with no active medications) in participants with HAE. Participants will be randomly assigned to 1 of 3 treatment groups. 1) ADX-324 300mg on day 1 (D1) + Placebo on Week 13 (W13), 2) ADX-324 240mg D1 + ADX-324 240mg W13, 3) Placebo D1 + Placebo W13. After the start of study drug administration, participants will return to the hospital for 7 post-dose visits at 2- or 4-week intervals for effectiveness and safety tests for 6 months. The study ends upon completion of the 6 month visits.
REC name
North East - Tyne & Wear South Research Ethics Committee
REC reference
25/NE/0161
Date of REC Opinion
16 Oct 2025
REC opinion
Further Information Favourable Opinion