Extension Study of ADX-324 in HAE

• Research type

  Research Study

• Full title

  A Phase 3 Extension Study of siRNA Targeting of Prekallikrein With ADX-324 in Participants With Hereditary Angioedema

• IRAS ID

  1013805

• Contact name

  Donald Fong

• Contact email

  cstorgard@adarx.com

• Sponsor organisation

  ADARx Pharmaceuticals Inc.

• Clinicaltrials.gov Identifier

  NCT07428499)

• 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, larynx, extremities, 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 This is a Phase 3 extension study. At the End of Study (EOS) Visit in the core .study, participants will be assessed for study eligibility for this extension study.
  All eligible participants in the main study will be enrolled on the extension study and administered ADX-324 for up to 3 years. Participants who received ADX-324 in the core study will continue to receive the same dosing regimen as in the core study (ie, ADX-324 300 mg every 6 months [Q6M] in Group 1 or 240 mg every 3 months [Q3M] in Group 2). Participants who received placebo 1.5 mL in the core study will receive ADX-324 300 mg Q6M (Group 1) and those who received placebo 1.2 mL in the core study will receive 240 mg Q3M (Group 2). In order to maintain the blind in the core study, all participants will receive an injection Q3M; participants in the Q6M regimen will alternate between ADX-324 and placebo Q3M while participants in the Q3M group will receive ADX-324 Q3M as outlined.

• REC name

  South Central - Hampshire B Research Ethics Committee

• REC reference

  26/SC/0102

• Date of REC Opinion

  12 May 2026

• REC opinion

  Further Information Favourable Opinion