Recruiting NCT06960213
STOP-HAE: A Phase 3 Study of ADX-324 in HAE
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- The protocol lists: ADX-324, Placebo.
- Who it may be relevant to
- Registry conditions: Hereditary Angioedema, HAE, Hereditary Angioedema - Type 1, Hereditary Angioedema - Type 2. Basic parameters: from 18 years · All.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- United States, Argentina, Australia, Austria, Belgium +14
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
Unsure about the terms? Read our patient guide →
Official 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
Overview
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.
Interventions
- Drug ADX-324
siRNA duplex oligonucleotide - Drug Placebo
saline
Primary outcome measures
- Efficacy of ADX-324 in preventing HAE attacks [Time frame: Day 22 to Week 25]
Secondary outcome measures (5)
- Efficacy of ADX-324 in preventing HAE attacks [Time frame: Day 22 to Week 25]
- Evaluate the effects of ADX-324 on the quality and pattern of HAE attacks [Time frame: Day 22 to Week 25]
- Evaluate the effects of ADX-324 on the quality and pattern of HAE attacks [Time frame: Day 22 to Week 25]
- Evaluate the effects of ADX-324 on the quality and pattern of HAE attacks [Time frame: Day 22 to Week 25]
- Evaluate the effects of ADX-324 on the quality and pattern of HAE attacks [Time frame: Day 22 to Week 25]
Eligibility criteria
Inclusion criteria
- 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 criteria
- 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.
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: No
Study design
- Allocation
- Randomized
- Model
- Parallel assignment
- Masking
- Quadruple blind
- Primary purpose
- Treatment
Study locations
United States · 15 centers
- ADARx Clinical Site — Litchfield Park
- ADARx Clinical Site — Little Rock
- ADARx Clinical Site — San Diego
- ADARx Clinical Site — Walnut Creek
- ADARx Clinical Site — Orlando
- ADARx Clinical Site — Chevy Chase
- ADARx Clinical Site — Wheaton
- ADARx Clinical Site — Detroit
- … and 7 more centers
France · 4 centers
- ADARx Clinical Site — Nice
- ADARx Clinical Site — Tours
- ADARx Clinical Site — Montpellier
- ADARx Clinical Site — Paris
United Kingdom · 4 centers
- ADARx Clinical Site — Cambridge
- ADARx Clinical Site — Cardiff
- ADARx Clinical Site — London
- ADARx Clinical Site — Southampton
Canada · 3 centers
- ADARx Clinical Site — Edmonton
- ADARx Clinical Site — Ottawa
- ADARx Clinical Site — Montreal
China · 3 centers
- ADARx Clinical Site — Beijing
- ADARx Clinical Site — Harbin
- ADARx Clinical Site — Zhengzhou
Israel · 3 centers
- ADARx Clinical Site — Ramat Gan
- ADARx Clinical Site — Ashkelon
- ADARx Clinical Site — Tel Aviv
Poland · 3 centers
- ADARx Clinical Site — Krakow
- ADARx Clinical Site — Lodz
- ADARx Clinical Site — Wroclaw
Argentina · 2 centers
- ADARx Clinical Site — Capital Federal
- ADARx Clinical Site — Buenos Aires
Croatia · 2 centers
- ADARx Clinical Site — Split
- ADARx Clinical Site — Zagreb
Czechia · 2 centers
- ADARx Clinical Site — Hradec Králové
- ADARx Clinical Site — Prague
Spain · 2 centers
- ADARx Clinical Site — Barcelona
- ADARx Clinical Site — Madrid
Australia · 1 center
- ADARx Clinical Site — Adelaide
Austria · 1 center
- ADARx Clinical Site — Vienna
Belgium · 1 center
- ADARx Clinical Site — Edegem
Bulgaria · 1 center
- ADARx Clinical Site — Sofia
Germany · 1 center
- ADARx Clinical Site — Tübingen
Hong Kong · 1 center
- ADARx Clinical Site — Hong Kong
Hungary · 1 center
- ADARx Clinical Site — Budapest
Taiwan · 1 center
- ADARx Clinical Site — Taichung
Identifiers
NCT: NCT06960213 · ADX-324-301