RecruitingPhase 3NCT06960213

STOP-HAE: A Phase 3 Study of ADX-324 in HAE

STOP-HAE: A Phase 3, Randomized, Double-Blind, Placebo-Controlled Study of siRNA Targeting of Prekallikrein With ADX-324 in Participants With Hereditary Angioedema


Sponsor

ADARx Pharmaceuticals, Inc.

Enrollment

90 participants

Start Date

Aug 28, 2025

Study Type

INTERVENTIONAL

Conditions

Summary

This study will evaluate the efficacy and safety of ADX-324 in participants with Type 1 or Type 2 hereditary angioedema. The study will also evaluate safety, pharmacokinetics (PK), pharmacodynamics (PD), and health-related quality of life measures.


Eligibility

Min Age: 18 Years

Inclusion Criteria4

  • 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
  • Able to use at least one acute therapy to treat HAE attacks (such as a plasma-derived or recombinant C1-INH concentrate or a BK2-receptor antagonist)

Exclusion Criteria9

  • Concurrent diagnosis of another form of recurrent angioedema (such as acquired angioedema, HAE with normal C1-INH (previously known as HAE Type III), idiopathic angioedema, or recurrent angioedema associated with urticaria).
  • Any clinically significant renal disease
  • Any clinically significant hepatic disease
  • Have used any of the following for long-term prevention of HAE attacks:
  • C1-INH agent (CINRYZE, HAEGARDA, RUCONEST) within 2 weeks prior to Screening.
  • Berotralstat (ORLADEYO) within 3 weeks prior to Screening.
  • Lanadelumab (TAKHZYRO) within 8 weeks prior to the Screening.
  • Androgen use within 12 weeks prior to Screening.
  • Received prior treatment with any RNA/DNA-based therapy for HAE or intolerant to any prior RNA/DNA-based therapy for any condition, excluding vaccines.

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Interventions

DRUGADX-324

siRNA duplex oligonucleotide

DRUGPlacebo

saline


Locations(14)

ADARx Clinical Site

Little Rock, Arkansas, United States

ADARx Clinical Site

Walnut Creek, California, United States

ADARx Clinical Site

Wheaton, Maryland, United States

ADARx Clinical Site

Detroit, Michigan, United States

ADARx Clinical Site

St Louis, Missouri, United States

ADARx Clinical Site

Las Vegas, Nevada, United States

ADARx Clinical Site

New York, New York, United States

ADARx Clinical Site

Cincinnati, Ohio, United States

ADARx Clinical Site

Columbus, Ohio, United States

ADARx Clinical Site

Toledo, Ohio, United States

ADARx Clinical Site

Ottawa, Ontario, Canada

ADARx Clinical Site

Hradec Králové, Czechia

ADARx Clinical Site

Budapest, Hungary

ADARx Clinical Site

Madrid, Spain

View Full Details on ClinicalTrials.gov

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NCT06960213


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