RecruitingPHASE3INTERVENTIONAL

A Study of Navenibart in Participants With Hereditary Angioedema

This research study, called a Phase 3 trial, is looking at a new medicine called navenibart for people who have Hereditary Angioedema (HAE). HAE is a rare condition that causes sudden swelling attacks. The main goal is to find out if navenibart can safely and effectively stop these swelling attacks from happening in both adults and teenagers. Some participants will receive navenibart, while others will receive a placebo (a dummy treatment with no active medicine). This helps researchers compare the effects fairly. It’s a ‘double-blind’ study, meaning neither the participants nor the doctors will know who is getting the actual medicine and who is getting the placebo.

At a glance

Status

Recruiting

Phase

PHASE3

Sponsor

Astria Therapeutics, Inc.

Enrolment target

145

Start

20 Mar 2025

Estimated completion

01 Sep 2027

Hereditary Angioedema (HAE)

What is this study about?

This important study is testing a new treatment called navenibart for a condition known as Hereditary Angioedema, often shortened to HAE. HAE is a genetic condition that causes sudden, unpredictable swelling attacks in different parts of the body, such as the skin, stomach, or throat. These attacks can be painful, debilitating, and sometimes even life-threatening, especially if the swelling affects the airways.

The main aim of this study is to see if navenibart can help prevent these swelling attacks from happening. Researchers are also very keen to understand if the medicine is safe. They will compare navenibart with a 'placebo' – which looks just like the real medicine but contains no active ingredients. This is a standard way to find out if the new medicine really works better than no treatment.

This study is a 'Phase 3' trial, which means it's one of the final steps before a new medicine might be approved for wider use. It involves many medical centres and participants, carefully looking at how navenibart works in adults and teenagers who have HAE type 1 or type 2. If successful, navenibart could offer a new way to help manage HAE and improve the lives of those affected.

Key takeaways

This study is testing a new medicine, navenibart, for Hereditary Angioedema (HAE).
Its main goal is to prevent HAE swelling attacks in adults and teenagers.
Participants will receive either navenibart or a placebo (dummy treatment).
It's a 'Phase 3' study, aiming to confirm safety and effectiveness.
Regular clinic visits and self-monitoring of HAE attacks will be required.

Who may be eligible?

To join this study, you must have a confirmed diagnosis of Hereditary Angioedema (HAE), either type 1 or type 2. This means your doctor will have found evidence of HAE from your medical history and specific blood tests.

During a short period before the main study, called a 'run-in' period, you would have needed to experience at least two HAE swelling attacks. This helps the researchers understand your typical attack pattern before you start any new treatment.

There are also some reasons why you wouldn't be able to join. For example, if you have other types of swelling conditions that mimic HAE, or if you've recently used certain other medicines for preventing HAE attacks. You would need to stop these other preventative medicines for a set time before the study begins, under the guidance of the study doctors.

Could this study suit you?

Answer these quick questions to see if you may be eligible. This is a guide only — the research team makes the final call.

Do you have a doctor-confirmed diagnosis of Hereditary Angioedema (HAE) Type 1 or Type 2?
Have you had at least two HAE attacks recorded by a doctor very recently?
Are you aged 12 years or older?
Do you currently have any other swelling conditions like HAE type 3?
Have you recently used certain other medicines to prevent HAE attacks (your doctor can advise on specific timeframes)?

Answer every question to see your result.

What does participation involve?

If you join the study, you will receive either the new medicine, navenibart, or a placebo, given as an injection under the skin. You won't know which one you're getting, and neither will your study doctor – this is to make sure the results are as fair and unbiased as possible. Over the course of the study, you'll have regular visits to the clinic so the medical team can monitor your health, check for any side effects, and see how many HAE attacks you experience. You will also need to keep a record of your HAE attacks at home. The total duration of your participation in this study will be explained in detail by the study team, including how often you'll need to attend appointments and how long the treatment period will last, along with any follow-up checks.

Potential risks and benefits

Taking part in this study could offer the potential benefit of receiving an experimental treatment that might reduce your HAE attacks. However, there's no guarantee the treatment will work for you, and you might receive a placebo. As with any medication, there's a risk of side effects, which will be carefully monitored by the study team. All potential risks and side effects will be fully explained before you decide to participate. Remember, you can choose to leave the study at any time, for any reason, without it affecting your usual medical care.

Locations (87)

Some site locations are approximate. We're improving this — please verify with the trial team before travelling.

Site 9
Verified postcode
Birmingham, United States· Recruiting
Site 2
Verified postcode
Scottsdale, United States· Recruiting
Site 3
Verified postcode
Little Rock, United States· Recruiting
Site 26
Verified postcode
San Diego, United States· Recruiting
Site 14
Verified postcode
San Diego, United States· Recruiting
Site 4
Verified postcode
Santa Monica, United States· Recruiting
Site 1
Verified postcode
Walnut Creek, United States· Recruiting
Site 8
Verified postcode
Centennial, United States· Recruiting
Site 11
Verified postcode
Colorado Springs, United States· Recruiting
Site 25
Verified postcode
Tampa, United States· Recruiting
Site 69
Verified postcode
Weston, United States· Recruiting
Site 43
Verified postcode
Overland Park, United States· Withdrawn

Common questions

What is Hereditary Angioedema (HAE)?

HAE is a rare genetic condition that causes sudden, unpredictable swelling in different parts of your body, like your skin, bowels, or throat.

What is a 'Phase 3' clinical trial?

It's one of the final stages of testing a new medicine, involving many people, to confirm its safety and effectiveness before it might be made available to everyone.

What is a 'placebo'?

A placebo looks exactly like the study medicine but contains no active ingredients. It helps researchers understand if the new medicine is truly effective.

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

No, neither you nor your study doctor will know. This is called 'double-blind' and helps ensure the study results are accurate and fair.

What is 'navenibart'?

Navenibart is the name of the new investigational medicine being tested in this study to prevent HAE attacks.

How to find out more

Claire VanEenwyk, MD

alphaorbit_general@astriatx.com 1-617-349-1971 Official trial record

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