HAE, Hereditary Angioedema, Hereditary Angioedema - Type 1, Hereditary Angioedema - Type 2
Conditions
Keywords
HAE, Hereditary angioedema, onvuzosiran
Brief 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.
Interventions
DRUGADX-324
siRNA duplex oligonucleotide
DRUGPlacebo
saline
Sponsors
ADARx Pharmaceuticals, Inc.
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
QUADRUPLE (Subject, Caregiver, Investigator, Outcomes Assessor)
Masking description
Double blinded
Intervention model description
Randomized, double-blind, placebo-controlled study
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
Key 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) Key
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: 1. C1-INH agent (CINRYZE, HAEGARDA, RUCONEST) within 2 weeks prior to Screening. 2. Berotralstat (ORLADEYO) within 3 weeks prior to Screening. 3. Lanadelumab (TAKHZYRO) within 8 weeks prior to the Screening. 4. 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.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Efficacy of ADX-324 in preventing HAE attacks | Day 22 to Week 25 | The time-normalized number of Investigator-confirmed HAE attacks per month for ADX-324 Dose Level 1 vs placebo |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Efficacy of ADX-324 in preventing HAE attacks | Day 22 to Week 25 | The time-normalized number of Investigator-confirmed HAE attacks per month for ADX-324 Dose Level 2 vs placebo |
| Evaluate the effects of ADX-324 on the quality and pattern of HAE attacks | Day 22 to Week 25 | The time-normalized number of Investigator-confirmed HAE attacks requiring acute HAE therapy per month for ADX-324 vs placebo. |
Countries
Argentina, Australia, Austria, Belgium, Brazil, Bulgaria, Canada, China, Croatia, Czechia, France, Germany, Hong Kong, Hungary, Israel, Italy, Poland, Serbia, South Africa, Spain, Taiwan, Turkey (Türkiye), United Kingdom, United States
Contacts
CONTACTLupe Gallegos
STUDY_DIRECTORLauge Farnaes, MD
ADARx Pharmaceuticals, Inc.
Outcome results
None listed