RecruitingPHASE3INTERVENTIONAL

Long-Term, Open-label Study of Oral Deucrictibant Extended-Release Tablet for Prophylaxis Against Angioedema Attacks in Adolescents and Adults With HAE

This research study, called a Phase 3 trial, is for people aged 12 and older who have a rare condition called Hereditary Angioedema (HAE). The study is testing a new tablet, deucrictibant, taken once a day, to see if it can help prevent angioedema attacks (sudden, painful swelling). We want to find out if this treatment is safe and effective over a long period. Participants will take the tablet for about two and a half years, use an electronic diary, and have regular check-ups. This study builds on earlier research and aims to provide a new option for managing HAE, helping people live with fewer attacks.

At a glance

Status

Recruiting

Phase

PHASE3

Sponsor

Pharvaris Netherlands B.V.

Enrolment target

170

Start

01 Feb 2025

Estimated completion

01 Dec 2028

Hereditary Angioedema (HAE)

What is this study about?

This study is about a new once-a-day tablet called deucrictibant for individuals, including teenagers and adults aged 12 and older, who have a condition called Hereditary Angioedema (HAE). HAE is a rare genetic disorder that causes episodes of severe swelling, which can be painful, disfiguring, and sometimes life-threatening if it affects the throat. The main goal of this study is to see if taking deucrictibant regularly can safely and effectively reduce how often these swelling attacks happen over a long period.

This is a 'Phase 3' study, which means the treatment has already been tested in smaller groups of people and shown some promise. Now, it's being tested in a larger group to confirm its benefits and understand its side effects over a longer time. The study is 'open-label,' which means both you and your study doctor will know you are taking the deucrictibant tablet.

Finding new ways to prevent HAE attacks is very important for improving the quality of life for people living with this condition. This research could potentially offer a new preventative treatment option, helping individuals to manage their HAE more effectively and experience fewer unexpected and distressing swelling episodes.

Key takeaways

This study is testing a new tablet, deucrictibant, for people with Hereditary Angioedema (HAE).
The goal is to see if taking it daily can prevent HAE swelling attacks.
It's for individuals aged 12 and older.
Participation involves taking the tablet daily, keeping an electronic diary, and having regular check-ups for about 2.5 years.
This could potentially offer a new long-term treatment option for HAE.

Who may be eligible?

To join this study, you must be 12 years old or older and have a confirmed diagnosis of Hereditary Angioedema (HAE). If you haven't been in a previous study with deucrictibant, you should have experienced at least one HAE attack in the three months before the study starts. It's also important that you have reliable access to your usual emergency treatments for HAE attacks and are able to use them if needed.

There are also some reasons why you might not be able to join. For example, if you have any other type of angioedema that isn't HAE, or if you've recently taken part in another drug study. You also can't join if you've had gene therapy, or if you had to stop a previous deucrictibant study because of safety concerns or difficulty following the rules.

Additionally, certain medications, like some blood pressure drugs (ACE inhibitors) or hormone-containing medications (like some contraceptives), would mean you couldn't join if you've used them recently. If you are pregnant, planning to become pregnant, or breastfeeding, you would also not be able to participate. The study team will check your kidney and liver health, and any other serious health conditions could also affect whether you can take part.

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.

I am aged 12 or older.
I have been diagnosed with Hereditary Angioedema (HAE).
I have access to my usual emergency HAE treatments.
I am not pregnant or breastfeeding, and I don't plan to become pregnant during the study.
I haven't taken certain other medications or been in another drug study recently.

Answer every question to see your result.

What does participation involve?

If you decide to take part, the study will last for about two and a half years (around 130 weeks), with a final check-up four weeks after you finish the medication. You will be taking one deucrictibant extended-release tablet by mouth once a day. Before starting, there will be a screening period to make sure this study is right for you, unless you're moving directly from a previous deucrictibant study.

Throughout the study, you'll have regular appointments at the clinic for safety checks, which will include blood tests and other examinations. You'll also need to complete an electronic diary every day to record any HAE attacks or other experiences you have. Additionally, you'll be asked to fill out questionnaires at certain times during the study. This helps the researchers understand how the treatment is working and how you are feeling.

Potential risks and benefits

Taking part in this study could offer some potential benefits, such as receiving a new investigational treatment that might reduce your HAE attacks. You'll also receive careful monitoring and support from a dedicated study team. However, like all medications, deucrictibant might have side effects, and some blood tests or procedures you undergo could cause discomfort. Since this is a research study, there's no guarantee the treatment will work for you or that you won't experience side effects. You are free to withdraw from the study at any time, for any reason, without it affecting your medical care.

Locations (62)

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

Study Site
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Birmingham, United States· Recruiting
Study Site
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Scottsdale, United States· Recruiting
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Little Rock, United States· Recruiting
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San Diego, United States· Recruiting
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Santa Monica, United States· Recruiting
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Walnut Creek, United States· Recruiting
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Wheaton, United States· Recruiting
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St Louis, United States· Recruiting
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Las Vegas, United States· Recruiting
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Hershey, United States· Recruiting
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Dallas, United States· Recruiting
Study Site
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Milwaukee, United States· Recruiting

Common questions

What is Hereditary Angioedema (HAE)?

HAE is a rare genetic condition that causes sudden and painful swelling attacks in different parts of the body, like the face, hands, feet, tummy, or throat. These attacks can be very serious.

What is a 'Phase 3' study?

A Phase 3 study means this new medicine has already been through some tests and is now being studied in a larger group of people to confirm its safety and how well it works over a longer time.

Will I know if I'm getting the drug?

Yes, this is an 'open-label' study, meaning you and your study doctor will both know you are taking the deucrictibant tablet every day.

How long will I be in the study?

The study treatment period is about two and a half years (130 weeks), followed by a final check-up within four weeks after you stop taking the medication.

What if I have an HAE attack during the study?

It's important that you still have access to and can use your usual emergency treatments for HAE attacks while you are taking part in this study. The study team can provide more details if you join.

How to find out more

Pharvaris Clinical Team

clinicaltrials@pharvaris.com 0031 712-036-410 Official trial record

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