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Enrolling by invitationPHASE2, PHASE3INTERVENTIONAL

Extension Study of Oral PHA-022121 for Acute Treatment of Angioedema Attacks in Patients With Hereditary Angioedema

This research is an ongoing study for individuals aged 12 and older who have a health condition called Hereditary Angioedema (HAE). HAE causes sudden, unpredictable swelling attacks in different parts of the body, which can sometimes be life-threatening if it affects the throat. The study is testing a new medicine, deucrictibant, which is taken by mouth to treat these attacks when they happen. The main goal is to understand how safe this medicine is over a longer period and how well it works to stop or reduce HAE attacks. This is an extension study, meaning it includes people who have already been part of earlier studies looking into this medication, as well as some new participants with a specific type of HAE.

At a glance

Status
Enrolling by invitation
Phase
PHASE2, PHASE3
Sponsor
Pharvaris Netherlands B.V.
Enrolment target
150
Start
28 Dec 2022
Estimated completion
01 Jun 2027
Hereditary AngioedemaHereditary Angioedema Type IHereditary Angioedema Type IIHereditary Angioedema Types I and IIHereditary Angioedema AttackHereditary Angioedema With C1 Esterase Inhibitor DeficiencyHereditary Angioedema - Type 1Hereditary Angioedema - Type 2Hereditary Angioedema - Type 3C1 Esterase Inhibitor [C1-INH] DeficiencyC1 Esterase Inhibitor DeficiencyC1 Esterase Inhibitor, Deficiency ofC1 Inhibitor DeficiencyHereditary Angioedema Type IIIHereditary Angioedema (HAE)Hereditary Angioedema Type I and II

What is this study about?

This study is an important next step in understanding a new medicine called deucrictibant for people who experience Hereditary Angioedema (HAE) attacks. HAE is a condition that causes sudden, often painful, swelling attacks. These attacks can happen in various parts of the body, like the hands, feet, stomach, or face. Sometimes, the swelling can occur in the throat, which can be very serious and even life-threatening if it blocks breathing. Currently, treatments for HAE attacks are available, but doctors are always looking for new, easier ways to manage the condition.

Deucrictibant is a tablet designed to be taken by mouth when an HAE attack starts. This study is called an 'extension study' because it builds on previous research. It allows people who have already started taking deucrictibant in earlier studies to continue using it, helping researchers gather more long-term information. It also includes a small group of new participants who have a specific type of HAE (HAE-nC1INH) and haven't tried deucrictibant before.

The main purpose of this research is to see how safe deucrictibant is when used over a longer period and how well it helps to stop or relieve HAE attacks. By learning more about this medicine, doctors hope to offer more options for people living with HAE, potentially making it easier to manage their condition and improve their quality of life. The study design involves different parts, including some participants continuing treatment from earlier studies without knowing if they are getting the active drug or a dummy pill (Part A), and others receiving the active drug openly (Part B).

Key takeaways

  • This study is for people aged 12 and up with Hereditary Angioedema (HAE).
  • It's testing a new oral medicine, deucrictibant, for long-term use to treat HAE attacks.
  • The study aims to understand the medicine's safety and how well it works over time.
  • Many participants will have been in previous deucrictibant studies.
  • There are careful checks to ensure participant safety and monitor for side effects.

Who may be eligible?

To join this study, you must be at least 12 years old. If you're under 18, your parent or guardian will need to give their permission, and you'll also need to agree to take part.

Most participants in this study will have already been part of previous research on deucrictibant (Study C201 or Study C306). You would have received at least one dose of the study medicine in those earlier studies to be considered. There's also a chance for some adults (18 years or older) with a specific type of HAE called HAE Type 3 (HAE-nC1INH) who haven't tried deucrictibant before to join, provided they meet certain medical criteria confirmed by blood tests and genetic information.

It's important that you (and your parent/guardian if you are an adolescent) are willing and able to follow all the study instructions. If you are a woman who could become pregnant, you'll need to agree to specific pregnancy tests and use effective birth control throughout the study and for a short time after finishing the medicine.

Quick self-check
  • Are you 12 years old or older?
  • Do you have Hereditary Angioedema (HAE)?
  • Have you participated in previous deucrictibant studies (C201 or C306)? (If not, are you an adult with HAE Type 3 and interested in new treatment options?)
  • Are you willing to attend regular hospital visits and follow study instructions?
  • If you are a woman who could get pregnant, are you willing to use birth control and have pregnancy tests?

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

What does participation involve?

If you join this study, you will be taking deucrictibant by mouth when you experience an HAE attack. The study has different parts. If you're coming from Study PHA022121-C201, you might continue in a 'double-blind' part (Part A), which means neither you nor your study doctor will know if you're taking the active medicine or a placebo (a dummy pill). Everyone moving into 'Part B' will know they are receiving the active deucrictibant medicine.

Over the course of the study, you'll have regular hospital visits to check your health, assess how well the medicine is working, and monitor for any side effects. These visits will involve discussions with the study team, physical examinations, and possibly blood tests or other assessments. You will also be asked to keep track of your HAE attacks and when you take the study medicine. The total duration of your participation will vary depending on which part of the study you join and your individual needs, but it's a long-term study designed to gather information on the medicine's effects over time.

Potential risks and benefits

Taking part in this study could potentially offer benefits, such as access to a new oral medicine to treat your HAE attacks, which might be more convenient than existing treatments. However, there's no guarantee that deucrictibant will work for everyone, or that it will be better than your current treatment. As with any medicine, there are potential risks, including side effects from deucrictibant, though the study aims to carefully monitor and understand these. You will be told about all known risks before you decide to join. Remember, your participation is completely voluntary, and you have the right to withdraw from the study at any time, for any reason, without it affecting your medical care.

Locations (63)

  • Study site
    Birmingham, United States
  • Study site
    Scottsdale, United States
  • Study site
    Little Rock, United States
  • Study site
    San Diego, United States
  • Study site
    Santa Monica, United States
  • Study site
    Walnut Creek, United States
  • Study site
    Colorado Springs, United States
  • Study site
    Chevy Chase, United States
  • Study site
    Boston, United States
  • Study site
    Detroit, United States
  • Study site
    St Louis, United States
  • Study site
    Hershey, United States

+51 more sites — see the official record for the full list.

Common questions

What is Hereditary Angioedema (HAE)?

HAE is a rare genetic condition that causes unpredictable attacks of severe swelling in different body parts, including the throat, making it hard to breathe.

What is deucrictibant?

Deucrictibant is a new medicine that comes in a tablet form and is being tested to treat HAE attacks when they happen.

Who can join this study?

You must be at least 12 years old and have HAE. Most participants will have been in a previous deucrictibant study, though some new adults with a specific HAE type might qualify.

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

In some parts of the study, neither you nor your doctor will know if you're getting the active medicine or a dummy pill. In other parts, everyone will receive the active medicine.

What does 'long-term on-demand treatment' mean?

It means the medicine is taken when an HAE attack occurs, and the study is observing its safety and effectiveness over a longer period of time.

How to find out more

Official trial record

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

Interested in taking part?

Register your interest

Share your details and the research team for "Extension Study of Oral PHA-022121 for Acute Treatment of An…" will contact you if you may be eligible. Always speak to your GP before agreeing to take part.

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