Hereditary Angioedema (HAE)Phase 3NCT07428499
Phase 3 Extension Study of ADX-324 in Participants With Hereditary Angioedema (HAE)
A Phase 3 study testing ADX-324 Dose Level 1 and ADX-324 Dose Level 2 in people with Hereditary Angioedema (HAE) and Hereditary Angioedema - Type 1, run by ADARx Pharmaceuticals, Inc. at 48 sites in 19 countries.
Trial summary
Study ADX-324-302 is an extension study for participants who complete the Phase 3 ADX-324-301 trial. The extension study will provide information about the safety and efficacy of additional dosing of ADX-324 in participants with Type I and Type II hereditary angioedema (HAE). Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Hereditary Angioedema (HAE), Hereditary Angioedema - Type 1, Hereditary Angioedema - Type 2
- What is being tested?
- ADX-324 Dose Level 1 and ADX-324 Dose Level 2
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 90 participants
- Where
- 48 sitesin 19 countries
- Study dates
- 2 Apr 2026to 30 Jun 2030
- Phase
- Phase 3
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Have a documented diagnosis of HAE (Type I or II)
- Completed Study ADX-324-301
- Have access to an acute therapy to treat HAE attacks (such as plasma derived or recombinant C1-INH concentrate or a BK2-receptor antagonist)
Who cannot join
- A negative reaction to study drug in ADX-324-301
Trial locations and contacts
48 sites in 19 countries. Contact the study team or a site near you to ask about taking part.
48 sites in 19 countries
United States15 sites, 15 recruiting
- Not yet recruiting
ADARx Clinical Site
Chevy Chase, Maryland, 20815
- Not yet recruiting
ADARx Clinical Site
Cincinnati, Ohio, 45236
- Not yet recruiting
ADARx Clinical Site
Columbus, Ohio, 43235
- Not yet recruiting
ADARx Clinical Site
Detroit, Michigan, 48202
- Not yet recruiting
ADARx Clinical Site
Hershey, Pennsylvania, 17033
- Recruiting
ADARx Clinical Site
Las Vegas, Nevada, 89128
- Not yet recruiting
ADARx Clinical Site
Litchfield Park, Arizona, 85340
- Not yet recruiting
ADARx Clinical Site
Little Rock, Arkansas, 72205
- Recruiting
ADARx Clinical Site
New York, New York, 10029
- Not yet recruiting
ADARx Clinical Site
Orlando, Florida, 32807
- Not yet recruiting
ADARx Clinical Site
San Diego, California, 92122
- Not yet recruiting
ADARx Clinical Site
St Louis, Missouri, 63141
- Not yet recruiting
ADARx Clinical Site
Toledo, Ohio, 43617
- Not yet recruiting
ADARx Clinical Site
Walnut Creek, California, 94598
- Not yet recruiting
ADARx Clinical Site
Wheaton, Maryland, 20902
France4 sites, 4 recruiting
- Not yet recruiting
ADARx Clinical Site
Montpellier, 34295
- Not yet recruiting
ADARx Clinical Site
Nice, Alpes-Maritimes, 06300
- Not yet recruiting
ADARx Clinical Site
Paris, 75012
- Not yet recruiting
ADARx Clinical Site
Tours, Indre-et-Loire, 37000
United Kingdom4 sites, 4 recruiting
- Not yet recruiting
ADARx Clinical Site
Cambridge, CB2 0QQ
- Not yet recruiting
ADARx Clinical Site
Cardiff, CF14 4YS
- Not yet recruiting
ADARx Clinical Site
London, E1 1BB
- Not yet recruiting
ADARx Clinical Site
Southampton, SO16 6YD
Canada3 sites, 3 recruiting
- Not yet recruiting
ADARx Clinical Site
Edmonton, Alberta, T6G 2R3
- Not yet recruiting
ADARx Clinical Site
Montreal, Quebec, H2W 1R7
- Not yet recruiting
ADARx Clinical Site
Ottawa, Ontario, K1H 1E4
China3 sites, 3 recruiting
- Not yet recruiting
ADARx Clinical Site
Beijing, 100730
- Not yet recruiting
ADARx Clinical Site
Harbin, 150001
- Not yet recruiting
ADARx Clinical Site
Zhengzhou, 450003
Argentina2 sites, 2 recruiting
- Not yet recruiting
ADARx Clinical Site
Buenos Aires, Buenos Aires F.D., C1035AAT
- Not yet recruiting
ADARx Clinical Site
Capital Federal, Buenos Aires, C1425BEN
Croatia2 sites, 2 recruiting
- Not yet recruiting
ADARx Clinical Site
Split, 21000
- Not yet recruiting
ADARx Clinical Site
Zagreb, 10000
Czechia2 sites, 2 recruiting
- Recruiting
ADARx Clinical Site
Hradec Králové, 500 05
- Not yet recruiting
ADARx Clinical Site
Prague, 15000
Israel2 sites, 2 recruiting
- Not yet recruiting
ADARx Clinical Site
Ashkelon, 7830604
- Not yet recruiting
ADARx Clinical Site
Tel Aviv, 5262100
Spain2 sites, 2 recruiting
- Not yet recruiting
ADARx Clinical Site
Barcelona, 08907
- Not yet recruiting
ADARx Clinical Site
Madrid, 28007
Australia1 site, 1 recruiting
- Not yet recruiting
ADARx Clinical Site
Adelaide, South Australia, 5000
Austria1 site, 1 recruiting
- Not yet recruiting
ADARx Clinical Site
Vienna, 1090
Belgium1 site, 1 recruiting
- Not yet recruiting
ADARx Clinical Site
Edegem, Antwerpen, 2650
Bulgaria1 site, 1 recruiting
- Not yet recruiting
ADARx Clinical Site
Sofia, 1000
Germany1 site, 1 recruiting
- Not yet recruiting
ADARx Clinical Site
Tübingen, Baden-Wurttemberg, 72076
Hong Kong1 site, 1 recruiting
- Not yet recruiting
ADARx Clinical Site
Hong Kong, 111
Hungary1 site, 1 recruiting
- Not yet recruiting
ADARx Clinical Site
Budapest, 1088
Taiwan1 site, 1 recruiting
- Not yet recruiting
ADARx Clinical Site
Taichung, 40705
About this study
Official title: A Phase 3 Extension Study of siRNA Targeting of Prekallikrein With ADX-324 in Participants With Hereditary Angioedema
Study ADX-324-302 is an extension study for participants who complete the Phase 3 ADX-324-301 trial. The extension study will provide information about the safety and efficacy of additional dosing of ADX-324 in participants with Type I and Type II hereditary angioedema (HAE). The study will also include pharmacodynamic (PD), pharmacokinetic (PK), and health-related quality of life (HRQoL) measurements.
- Conditions
- Hereditary Angioedema (HAE), Hereditary Angioedema - Type 1, Hereditary Angioedema - Type 2, HAE
- Being tested
- ADX-324 Dose Level 1, ADX-324 Dose Level 2
- Keywords
- Hereditary angioedema, HAE, ADX-324, onvuzosiran
- Medical subject headings
- Angioedemas, Hereditary
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ADX-324 Dose Level 1Drug
siRNA duplex oligonucleotide
ADX-324 Dose Level 2Drug
siRNA duplex oligonucleotide
| Study group | Role | What they receive |
|---|---|---|
| ADX-324 Dose Level 1 | Experimental | Not described Receives: Drug: ADX-324 Dose Level 1 |
| ADX-324 Dose Level 2 | Experimental | Not described Receives: Drug: ADX-324 Dose Level 2 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Safety of ADX-324 in Participants with HAE
Incidence and severity of treatment-emergent adverse events
Measured: Month 36
Secondary outcomes (1)
Efficacy of ADX-324 in Preventing HAE Attacks
Number of Investigator-confirmed HAE attacks per month
Measured: Month 36
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted23 Feb 2026
- Study start2 Apr 2026
- Primary completion30 Jun 2030 (estimated)
- Study completion30 Jun 2030 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Hereditary Angioedema (HAE)Studies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- Double blind
- Who is masked
- Participant, Investigator
- Enrolment
- 90 participants (estimated)
Sponsor and investigators
- Lead sponsor
- ADARx Pharmaceuticals, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Lauge Farnaes, MD, Study director, ADARx Pharmaceuticals
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07428499
- Sponsor's protocol ID
- ADX-324-302
- Status last verified
- Sep 2026
- First submitted
- 19 Feb 2026
- Last updated
- 4 Sep 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Undecided
Study at a glance
From the registry record, last updated by the sponsor on 4 Sep 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 3 Extension Study of siRNA Targeting of Prekallikrein With ADX-324 in Participants With Hereditary Angioedema
- ClinicalTrials.gov ID
- NCT07428499
- Acronym / sponsor ID
- ADX-324-302
- Conditions
- Hereditary Angioedema (HAE), Hereditary Angioedema - Type 1, Hereditary Angioedema - Type 2, HAE
- Keywords
- Hereditary angioedema, HAE, ADX-324, onvuzosiran
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 90 participants (estimated)
- Lead sponsor
- ADARx Pharmaceuticals, Inc.Industry (pharma, biotech, devices)
- Study start
- 2 Apr 2026 (actual)
- Primary completion
- 30 Jun 2030 (estimated)
- Study completion
- 30 Jun 2030 (estimated)
- First posted
- 23 Feb 2026 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Study ADX-324-302 is an extension study for participants who complete the Phase 3 ADX-324-301 trial. The extension study will provide information about the safety and efficacy of additional dosing of ADX-324 in participants with Type I and Type II hereditary angioedema (HAE). The study will also include pharmacodynamic (PD), pharmacokinetic (PK), and health-related quality of life (HRQoL) measurements.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- Double blind (participant, investigator)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| ADX-324 Dose Level 1 | Experimental | Drug: ADX-324 Dose Level 1 |
| ADX-324 Dose Level 2 | Experimental | Drug: ADX-324 Dose Level 2 |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (3 listed)
- Have a documented diagnosis of HAE (Type I or II)
- Completed Study ADX-324-301
- Have access to an acute therapy to treat HAE attacks (such as plasma derived or recombinant C1-INH concentrate or a BK2-receptor antagonist)
Key exclusion criteria (1 listed)
- A negative reaction to study drug in ADX-324-301
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| ADX-324 Dose Level 1siRNA duplex oligonucleotide | Drug | ADX-324 Dose Level 1 |
| ADX-324 Dose Level 2siRNA duplex oligonucleotide | Drug | ADX-324 Dose Level 2 |
Primary outcome
Safety of ADX-324 in Participants with HAE
Time frame: Month 36
Incidence and severity of treatment-emergent adverse events
1 secondary outcome
Efficacy of ADX-324 in Preventing HAE Attacks
Time frame: Month 36
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 4 Sep 2026.
- Individual participant dataSharing planned: Undecided
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Lauge Farnaes, MD study director
ADARx Pharmaceuticals
48 sites in 19 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing ADX-324 Dose Level 1 and ADX-324 Dose Level 2 in people with Hereditary Angioedema (HAE) and Hereditary Angioedema - Type 1, run by ADARx Pharmaceuticals, Inc. at 48 sites in 19 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 3 inclusion and 1 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 4 Sep 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 48 sites in United States, France, United Kingdom, Canada, China and 14 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Lupe Gallegos (877-232-7974, info@adarx.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Safety of ADX-324 in Participants with HAE (time frame: Month 36). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 4 Sep 2026.