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Purpose

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.

Conditions

Eligibility

Eligible Ages
Over 18 Years
Eligible Sex
All
Accepts Healthy Volunteers
No

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: 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.

Study Design

Phase
Phase 3
Study Type
Interventional
Allocation
Randomized
Intervention Model
Parallel Assignment
Intervention Model Description
Randomized, double-blind, placebo-controlled study
Primary Purpose
Treatment
Masking
Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Masking Description
Double blinded

Arm Groups

ArmDescriptionAssigned Intervention
Experimental
ADX-324 Dose Level 1
  • Drug: ADX-324
    siRNA duplex oligonucleotide
    Other names:
    • siRNA
Experimental
ADX-324 Dose Level 2
  • Drug: ADX-324
    siRNA duplex oligonucleotide
    Other names:
    • siRNA
Placebo Comparator
Placebo
  • Drug: Placebo
    saline
    Other names:
    • Saline

Recruiting Locations

ADARx Clinical Site
Litchfield Park, Arizona 85340

ADARx Clinical Site
Little Rock, Arkansas 72205

ADARx Clinical Site
San Diego, California 92122

ADARx Clinical Site
Walnut Creek, California 94598

ADARx Clinical Site
Orlando, Florida 32807

ADARx Clinical Site
Chevy Chase, Maryland 20815

ADARx Clinical Site
Wheaton, Maryland 20902

ADARx Clinical Site
Detroit, Michigan 48202

ADARx Clinical Site
St Louis, Missouri 63141

ADARx Clinical Site
Las Vegas, Nevada 89128

ADARx Clinical Site
New York, New York 10029

ADARx Clinical Site
Cincinnati, Ohio 45236

ADARx Clinical Site
Columbus, Ohio 43235

ADARx Clinical Site
Toledo, Ohio 43617

ADARx Clinical Site
Hershey, Pennsylvania 17033

More Details

Status
Recruiting
Sponsor
ADARx Pharmaceuticals, Inc.

Study Contact

Lupe Gallegos
877-232-7974
lgallegos@adarx.com

Notice

Study information shown on this site is derived from ClinicalTrials.gov (a public registry operated by the National Institutes of Health). The listing of studies provided is not certain to be all studies for which you might be eligible. Furthermore, study eligibility requirements can be difficult to understand and may change over time, so it is wise to speak with your medical care provider and individual research study teams when making decisions related to participation.