RecruitingPHASE3INTERVENTIONAL

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

This research study, called STOP-HAE, is investigating a new medicine named ADX-324 for adults who have Hereditary Angioedema (HAE) Type 1 or Type 2. HAE is a rare condition that causes sudden swelling attacks. The main goals of the study are to see how well ADX-324 works to prevent these attacks and to make sure it is safe to use. Researchers will also learn how the medicine acts in the body and how it might improve the daily lives of people living with HAE. This is a Phase 3 study, meaning it's one of the final steps before a new medicine can potentially be approved for wider use.

At a glance

Status

Recruiting

Phase

PHASE3

Sponsor

ADARx Pharmaceuticals, Inc.

Enrolment target

Start

28 Aug 2025

Estimated completion

31 Dec 2027

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

What is this study about?

Hereditary Angioedema (HAE) is a rare inherited condition that can cause unpredictable and often painful episodes of swelling in different parts of the body, like the hands, feet, stomach, or face. These swelling attacks can be very disruptive to daily life and, in some cases, can be life-threatening if they affect the throat.

This study is looking at an investigational medicine called ADX-324. An investigational medicine means it's still being tested and is not yet available for general use. The researchers want to find out if ADX-324 can effectively reduce the number and severity of HAE attacks in people who have Type 1 or Type 2 HAE. They will also be closely watching for any side effects to understand the medicine's safety.

Alongside checking how well ADX-324 works and if it's safe, the study will also look at other important things. This includes understanding how the medicine behaves in the body (pharmacokinetics and pharmacodynamics) and how it affects a person's overall quality of life. The aim is to find better ways to manage HAE and improve the lives of those affected by it.

Key takeaways

This study is testing a new medicine called ADX-324 for HAE Type 1 and 2.
The main goals are to see if ADX-324 prevents HAE attacks and if it's safe.
Participants will receive either ADX-324 or a placebo.
Regular clinic visits and health monitoring will be part of the study.
Taking part is voluntary, and you can withdraw at any time.
This is a Phase 3 study, a key step towards potential new treatments.

Who may be eligible?

To join this study, you need to be at least 18 years old and have a confirmed diagnosis of Hereditary Angioedema (HAE) Type 1 or Type 2. You should also have experienced at least one HAE attack in the past month, or at least two attacks in the past two months, that were confirmed by your doctor. It's also important that you are able to use at least one of the standard treatments for an acute HAE attack, such as C1-INH concentrates or a BK2-receptor antagonist.

There are also some things that would mean you couldn't join the study. For example, if you have another type of angioedema (swelling condition) that isn't HAE Type 1 or 2, like HAE with normal C1-INH or swelling linked to hives. You also can't have serious kidney or liver problems. If you've recently used certain long-term preventative HAE medicines within specific timeframes before the study started, you might not be able to participate. This includes certain C1-INH agents, berotralstat, lanadelumab, or androgen-based medications.

Finally, if you've previously been treated with any HAE medicine that works by affecting your RNA/DNA, or if you had problems with such a treatment for any other condition (excluding vaccines), you would not be able to take part in this study.

Quick self-check

Are you 18 years old or older?
Do you have a confirmed diagnosis of Hereditary Angioedema (HAE) Type 1 or Type 2?
Have you had at least one HAE attack in the last month, or two in the last two months, confirmed by your doctor?
Are you able to use your usual acute (on-demand) HAE treatments?
Do you have severe kidney or liver problems?
Have you recently used certain long-term preventative HAE medicines (your doctor can advise on specific timings for these)?

This is a guide only — the research team will confirm whether you can take part.

What does participation involve?

If you decide to take part in the study, you would receive either the investigational medicine ADX-324 or a placebo. A placebo looks like the study medicine but contains no active ingredients, and it's used to compare the effects of the actual medicine. You wouldn't know whether you're receiving ADX-324 or the placebo, nor would your study doctor, as this helps ensure fair and unbiased results. You would be asked to attend regular clinic visits where the study team would monitor your health, check for any HAE attacks, and take blood samples. You would also be asked about your general health and how your HAE affects your daily life. The total duration of your participation in this study would involve regular assessments and follow-up over a period of time, which the study team will explain in detail.

Potential risks and benefits

Taking part in a clinical trial offers potential benefits, such as access to a new investigational medicine that may help manage your HAE attacks. You would also receive close medical attention and monitoring throughout the study. However, there are also potential risks. The investigational medicine, ADX-324, might cause side effects, some of which may be unpleasant or serious, even if they are rare. Because you might receive the placebo, there's a chance you won't get any benefit from the investigational medicine. It's important to remember that participating is entirely voluntary, and you have the right to withdraw from the study at any time, for any reason, without it affecting your usual care.

Locations (51)

ADARx Clinical Site
Litchfield Park, United States· Recruiting
ADARx Clinical Site
Little Rock, United States· Recruiting
ADARx Clinical Site
San Diego, United States· Recruiting
ADARx Clinical Site
Walnut Creek, United States· Recruiting
ADARx Clinical Site
Orlando, United States· Recruiting
ADARx Clinical Site
Chevy Chase, United States· Recruiting
ADARx Clinical Site
Wheaton, United States· Recruiting
ADARx Clinical Site
Detroit, United States· Recruiting
ADARx Clinical Site
St Louis, United States· Recruiting
ADARx Clinical Site
Las Vegas, United States· Recruiting
ADARx Clinical Site
New York, United States· Recruiting
ADARx Clinical Site
Cincinnati, United States· Recruiting

+39 more sites — see the official record for the full list.

Common questions

What is Hereditary Angioedema (HAE)?

HAE is a rare genetic condition causing sudden, painful swelling attacks in various body parts, which can be serious.

What is ADX-324?

ADX-324 is a new medicine being tested to see if it can help prevent HAE attacks. It's not yet approved for general use.

What does 'Phase 3 Study' mean?

Phase 3 means this is one of the final steps in testing a new medicine. It involves more people and aims to confirm how well it works and if it's safe.

Will I know if I'm getting the real medicine or a placebo?

No, this is a 'blinded' study, meaning neither you nor your study doctor will know if you're getting ADX-324 or the inactive placebo. This helps make the study results accurate.

Can I leave the study if I change my mind?

Yes, you can choose to leave the study at any time, for any reason, without it affecting your medical care.

How to find out more

Lupe Gallegos

lgallegos@adarx.com 877-232-7974 Official trial record

Always speak to your GP or specialist before deciding to take part in a study.