Study of Oral Deucrictibant XR Tablet for Prophylaxis and Deucrictibant IR Capsule for On-Demand Treatment of Angioedema Attacks in Adults With Acquired Angioedema Due to C1 Inhibitor Deficiency
This study is for adults aged 18 and over who have a rare condition called acquired angioedema (AAE) due to C1 inhibitor deficiency. We're looking at a new medicine called deucrictibant. The study aims to see if an extended-release tablet version of deucrictibant can prevent swelling attacks. It also investigates if a different, immediate-release capsule version can effectively treat attacks once they start. To do this, some people will receive the new medicine, while others will get a placebo (a dummy pill with no active ingredient). The trial is divided into three main parts: two where participants don't know if they're getting the active drug or placebo, and one where everyone receives the active drug. This helps us understand both the benefits and safety of deucrictibant for AAE.
At a glance
What is this study about?
This study is focused on a health condition called acquired angioedema (AAE), which happens when your body's C1 inhibitor doesn't work correctly. This can cause sudden, unpredictable swelling attacks in different parts of the body, like the face, hands, feet, or tummy. These attacks can be very uncomfortable and even serious.
Researchers are testing a new medicine called deucrictibant. The goal is to see if this medicine can help people with AAE in two important ways: first, by preventing swelling attacks from happening in the first place, and second, by treating attacks quickly when they do occur. They are comparing the new medicine to a 'placebo', which looks exactly like the study medicine but has no active ingredients. This helps them understand if the new drug is truly working or if improvements are due to other factors.
The study has a few stages. Initially, some participants will try the extended-release tablet to see if it prevents attacks over several weeks. Later, participants will test the immediate-release capsule to see if it can stop a swelling attack once it has started. Finally, there's an open-label part where all participants will receive the active medicine, allowing for longer-term safety observation. This careful approach helps the research team gather all the necessary information to determine if deucrictibant is a safe and effective treatment option for acquired angioedema.
Key takeaways
- This study is testing a new medicine for acquired angioedema (AAE).
- It aims to see if the medicine can prevent attacks and treat them when they happen.
- Some people will receive the new medicine, others a dummy pill (placebo).
- Participation involves different treatment phases, some for prevention, some for on-demand treatment.
- Regular health monitoring and recording information about your attacks will be required.
- You can leave the study at any time you wish.
Who may be eligible?
To join this study, you must be 18 years or older and have been diagnosed with acquired angioedema (AAE) that is due to a C1 inhibitor deficiency. You should have experienced AAE attacks before the study starts. For some parts of the study, your AAE condition needs to be stable and well-managed, meaning you have ways to handle any attacks that occur.
There are also reasons why you might not be able to join. For example, if you've recently taken part in another clinical trial with a new medicine, or if you are pregnant, planning to get pregnant, or breastfeeding. People with certain other health problems, like significant liver or kidney issues, severe problems with your stomach or gut that might affect how your body takes in medicine, or a history of epilepsy, might also not be suitable. It’s also important that you don't have problems with alcohol or drug abuse, as this could interfere with the study.
Women who could become pregnant must agree to use effective birth control methods during the study and have pregnancy tests as required. If you are unable to easily record information on an electronic device, you might also not be able to take part.
- Are you 18 years old or older?
- Have you been diagnosed with acquired angioedema (AAE) due to C1-inhibitor deficiency?
- Do you have stable AAE and ways to manage your attacks?
- Are you not pregnant, planning to be pregnant, or breastfeeding?
- Have you avoided other new investigational drugs in the last 30 days?
- Are you able to use an electronic device to record your health information?
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, the study will involve several stages. First, there's a 'screening' period to confirm you're a good fit for the study. After that, you'll enter one of the treatment parts.
In the first part, about 24 participants will be randomly chosen to receive either the deucrictibant extended-release tablet or a placebo for 12 weeks. Neither you nor the study team will know which you are getting. After this, you'll move to the second part. New participants might also join directly into this second part. In this stage, you'll receive either the immediate-release deucrictibant capsule or a placebo to treat two AAE attacks, but again, you won't know which one. This is done in a special 'crossover' way, meaning you might get both at different times for different attacks. Finally, if you complete the second part, you might join a third, 'open-label' part, where all participants will openly receive the immediate-release deucrictibant capsule to treat any attacks. Throughout the study, you'll be asked to record information about your attacks and your health using an electronic device. The total duration of your participation will depend on which parts of the study you join and for how long.
Potential risks and benefits
Locations (17)
- Study SiteWalnut Creek, United States· Recruiting
- Study SiteWalnut Creek, United States· Recruiting
- Study SiteSt Louis, United States· Recruiting
- Study SiteSofia, Bulgaria· Recruiting
- Study SiteEdmonton, Canada· Recruiting
- Study SiteParis, France· Recruiting
- Study SiteBerlin, Germany· Recruiting
- Study SiteFrankfurt am Main, Germany· Recruiting
- Study SiteMunich, Germany· Recruiting
- Study SiteBudapest, Hungary· Recruiting
- Study ListMilan, Italy· Recruiting
- Study SiteBasel, Switzerland· Recruiting
+5 more sites — see the official record for the full list.
Common questions
What is acquired angioedema (AAE)?
It's a rare condition that causes sudden and unpredictable swelling attacks in various body parts due to an issue with a protein called C1 inhibitor.
What is a 'placebo'?
A placebo is a dummy pill that looks exactly like the study medicine but doesn't contain any active drug. It helps researchers see if the real medicine is effective.
Will I know if I'm getting the study drug or the placebo?
In some parts of the study, neither you nor your doctor will know if you're getting deucrictibant or the placebo. This is called 'double-blind'.
How long will I be in the study?
The length of your participation will depend on which parts of the study you qualify for and join, with some initial treatment periods lasting 12 weeks.
Can I stop participating in the study at any time?
Yes, you are free to leave the study at any point, and doing so will not affect your future medical care.
How to find out more
Pharvaris Clinical Team
Always speak to your GP or specialist before deciding to take part in a study.
Interested in taking part?
Discussion
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