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

NCT06960213 | Recruiting Phase 3 DMC FDA Drug

• 1 other identifier: ADX-324-301
• Study Type: interventional
• Target: 90
• Locations: 18 countries
• Sites: 49

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.

Conditions

Hereditary Angioedema; HAE; Hereditary Angioedema - Type 1; Hereditary Angioedema - Type 2

Keywords

HAE; Hereditary angioedema; onvuzosiran

Outcome Measures

Primary Outcomes (1)

• Efficacy of ADX-324 in preventing HAE attacks

  The time-normalized number of Investigator-confirmed HAE attacks per month for ADX-324 Dose Level 1 vs placebo

  Day 22 to Week 25

Secondary Outcomes (5)

• Efficacy of ADX-324 in preventing HAE attacks

  Day 22 to Week 25

• Evaluate the effects of ADX-324 on the quality and pattern of HAE attacks

  Day 22 to Week 25

• Evaluate the effects of ADX-324 on the quality and pattern of HAE attacks

  Day 22 to Week 25

• Evaluate the effects of ADX-324 on the quality and pattern of HAE attacks

  Day 22 to Week 25

• Evaluate the effects of ADX-324 on the quality and pattern of HAE attacks

  Day 22 to Week 25

Study Arms (3)

ADX-324 Dose Level 1

EXPERIMENTAL

Drug: ADX-324

ADX-324 Dose Level 2

EXPERIMENTAL

Drug: ADX-324

Placebo

PLACEBO COMPARATOR

Drug: Placebo

Interventions

ADX-324 DRUG

siRNA duplex oligonucleotide

Also known as: siRNA

ADX-324 Dose Level 1; ADX-324 Dose Level 2

Placebo DRUG

saline

Also known as: Saline

Placebo

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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)

You may not qualify if:

• 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.

Sponsors & Collaborators

• ADARx Pharmaceuticals, Inc.: lead

Study Sites (50)

ADARx Clinical Site

Litchfield Park, Arizona, 85340, United States

RECRUITING

ADARx Clinical Site

Little Rock, Arkansas, 72205, United States

RECRUITING

ADARx Clinical Site

San Diego, California, 92122, United States

RECRUITING

ADARx Clinical Site

Walnut Creek, California, 94598, United States

RECRUITING

ADARx Clinical Site

Orlando, Florida, 32807, United States

RECRUITING

ADARx Clinical Site

Chevy Chase, Maryland, 20815, United States

RECRUITING

ADARx Clinical Site

Wheaton, Maryland, 20902, United States

RECRUITING

ADARx Clinical Site

Detroit, Michigan, 48202, United States

RECRUITING

ADARx Clinical Site

St Louis, Missouri, 63141, United States

RECRUITING

ADARx Clinical Site

Las Vegas, Nevada, 89128, United States

RECRUITING

ADARx Clinical Site

New York, New York, 10029, United States

RECRUITING

ADARx Clinical Site

Cincinnati, Ohio, 45236, United States

RECRUITING

ADARx Clinical Site

Columbus, Ohio, 43235, United States

RECRUITING

ADARx Clinical Site

Toledo, Ohio, 43617, United States

RECRUITING

ADARx Clinical Site

Hershey, Pennsylvania, 17033, United States

NOT YET RECRUITING

ADARx Clinical Site

Capital Federal, Buenos Aires, C1425BEN, Argentina

NOT YET RECRUITING

ADARx Clinical Site

Buenos Aires, Buenos Aires F.D., C1035AAT, Argentina

NOT YET RECRUITING

ADARx Clinical Site

Adelaide, South Australia, 5000, Australia

NOT YET RECRUITING

ADARx Clinical Site

Vienna, 1090, Austria

RECRUITING

ADARx Clinical Site

Edegem, Antwerpen, 2650, Belgium

NOT YET RECRUITING

ADARx Clinical Site

Sofia, 1000, Bulgaria

RECRUITING

ADARx Clinical Site

Edmonton, Alberta, T6G 2R3, Canada

NOT YET RECRUITING

ADARx Clinical Site

Ottawa, Ontario, K1H 1E4, Canada

RECRUITING

ADARx Clinical Site

Montreal, Quebec, H2W 1R7, Canada

NOT YET RECRUITING

ADARx Clinical Site

Beijing, 100730, China

NOT YET RECRUITING

ADARx Clinical Site

Harbin, 150001, China

NOT YET RECRUITING

ADARx Clinical Site

Zhengzhou, 450003, China

NOT YET RECRUITING

ADARx Clinical Site

Split, 21000, Croatia

RECRUITING

ADARx Clinical Site

Zagreb, 10000, Croatia

RECRUITING

ADARx Clinical Site

Hradec Králové, 500 05, Czechia

RECRUITING

ADARx Clinical Site

Prague, 15000, Czechia

RECRUITING

ADARx Clinical Site

Nice, Alpes-Maritimes, 06300, France

NOT YET RECRUITING

ADARx Clinical Site

Tours, Indre-et-Loire, 37000, France

NOT YET RECRUITING

ADARx Clinical Site

Montpellier, 34295, France

NOT YET RECRUITING

ADARx Clinical Site

Paris, 75012, France

NOT YET RECRUITING

ADARx Clinical Site

Tübingen, Baden-Wurttemberg, 72076, Germany

NOT YET RECRUITING

ADARx Clinical Site

Hong Kong, 111, Hong Kong

NOT YET RECRUITING

ADARx Clinical Site

Budapest, 1088, Hungary

RECRUITING

ADARx Clinical Site

Ramat Gan, Tel Aviv, 5262100, Israel

NOT YET RECRUITING

ADARx Clinical Site

Ashkelon, 7830604, Israel

NOT YET RECRUITING

ADARx Clinical Site

Tel Aviv, 64239, Israel

NOT YET RECRUITING

ADARx Clinical Site

Lodz, 92-213, Poland

RECRUITING

ADARx Clinical Site

Wroclaw, 50-556, Poland

RECRUITING

ADARx Clinical Site

Barcelona, 08907, Spain

RECRUITING

ADARx Clinical Site

Madrid, 28007, Spain

RECRUITING

ADARx Clinical Site

Taichung, 40705, Taiwan

RECRUITING

ADARx Clinical Site

Cambridge, CB2 0QQ, United Kingdom

RECRUITING

ADARx Clinical Site

Cardiff, CF14 4YS, United Kingdom

RECRUITING

ADARx Clinical Site

London, E1 1BB, United Kingdom

RECRUITING

ADARx Clinical Site

Southampton, SO16 6YD, United Kingdom

RECRUITING

MeSH Terms

Conditions

Angioedemas, Hereditary

Interventions

RNA, Small Interfering; Sodium Chloride

Condition Hierarchy (Ancestors)

Angioedema; Vascular Diseases; Cardiovascular Diseases; Hereditary Complement Deficiency Diseases; Primary Immunodeficiency Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Urticaria; Skin Diseases, Vascular; Skin Diseases; Skin and Connective Tissue Diseases; Hypersensitivity, Immediate; Hypersensitivity; Immune System Diseases; Immunologic Deficiency Syndromes

Intervention Hierarchy (Ancestors)

RNA, Antisense; Antisense Elements (Genetics); Nucleic Acids, Nucleotides, and Nucleosides; RNA; Nucleic Acids; RNA, Small Untranslated; RNA, Untranslated; Chlorides; Hydrochloric Acid; Chlorine Compounds; Inorganic Chemicals; Sodium Compounds

Study Officials

• Lauge Farnaes, MD

  ADARx Pharmaceuticals, Inc.

  STUDY DIRECTOR

Central Study Contacts

Lupe Gallegos

CONTACT

877-232-7974; lgallegos@adarx.com

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Masking Details: Double blinded
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: Randomized, double-blind, placebo-controlled study

Study Record Dates

• First Submitted: April 7, 2025
• First Posted: May 7, 2025
• Study Start: August 28, 2025
• Primary Completion (Estimated): June 30, 2027
• Study Completion (Estimated): December 31, 2027
• Last Updated: April 22, 2026

Record last verified: 2026-04

Locations

CA (3); HU (1); ES (2); US (15); CZ (2); TW (1); FR (4); IL (3); GB (4); BE (1); BU (1); HK (1); PL (2); AR (2); AU (1); CN (3); CR (2); DE (1)