All studies
Active not recruitingPHASE3INTERVENTIONAL

Study of Oral Deucrictibant Extended-Release Tablet for Prophylaxis Against Angioedema Attacks in Adolescents and Adults With HAE

This research study is investigating an oral tablet called deucrictibant. It's designed for people aged 12 and older who have Hereditary Angioedema (HAE) and experience recurrent swelling attacks. The main goal is to find out if taking this tablet once a day can help prevent these attacks from happening. In this study, some participants will receive the new drug, while others will receive a placebo (a tablet with no active medicine), to fairly assess the drug's effectiveness and safety. It's a 'double-blind' study, meaning neither you nor your doctor will know whether you're taking deucrictibant or the placebo, ensuring unbiased results. Your participation would last about 6 months, plus a follow-up period, and involve regular check-ups and keeping a daily diary.

At a glance

Status
Active not recruiting
Phase
PHASE3
Sponsor
Pharvaris Netherlands B.V.
Enrolment target
81
Start
01 Dec 2024
Estimated completion
01 Aug 2026

What is this study about?

This research is looking into a new oral tablet called deucrictibant for individuals with Hereditary Angioedema, often shortened to HAE. HAE is a rare genetic condition that causes sudden and unpredictable swelling attacks, which can be painful and sometimes life-threatening. The purpose of this study is to see if taking deucrictibant once a day can help prevent these attacks from happening in adults and young people aged 12 and over.

To properly test the new tablet, some participants will be given deucrictibant, while others will receive a 'placebo' – a tablet that looks the same but contains no active medicine. This comparison is standard practice in medical research to understand if the new drug has a real effect. The study is also 'double-blind', meaning neither you nor your study doctor will know whether you are taking deucrictibant or the placebo, which helps make the results as accurate as possible. We want to find out how well deucrictibant works and if it is safe to use.

This study is a 'Phase 3' trial, which means it's one of the final stages of testing before a new medicine might be considered for wider use. It's an important step in potentially offering a new treatment option for managing HAE and improving the lives of those affected by this condition. Your involvement would help researchers gather vital information about this potential new preventative treatment.

Key takeaways

  • The study tests a new daily tablet (deucrictibant) to prevent HAE attacks.
  • It involves adults and children aged 12 and over with HAE.
  • Participants will receive either deucrictibant or a placebo; neither you nor your doctor will know which.
  • The study lasts about 6-7 months and involves regular check-ups and a daily diary.
  • You must have had at least 3 HAE attacks in the 3 months before starting.
  • You can stop participating at any time if you wish.

Who may be eligible?

To be considered for this study, you must be 12 years old or older and have a confirmed diagnosis of Hereditary Angioedema (HAE). You should have experienced at least three HAE attacks in the three months leading up to the study's first visit. It's also important that you have reliable access to your usual HAE emergency treatments, should you need them during the study.

There are also some reasons why you might not be able to join. For example, if your angioedema is due to a cause other than HAE, or if you've recently taken part in another clinical trial with an experimental drug. You can't have taken deucrictibant before, or certain other medications like ACE inhibitors or specific HAE preventative treatments recently. Pregnant or breastfeeding women, or those planning to become pregnant, are also unable to take part. We also need to check your liver and kidney health, and ensure there are no other significant medical conditions that might make participating unsafe for you.

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.

  1. Are you 12 years old or older?
  2. Do you have a confirmed diagnosis of Hereditary Angioedema (HAE)?
  3. Have you experienced at least 3 HAE attacks in the last 3 months?
  4. Are you able to use your usual emergency HAE treatments if needed?
  5. Are you not currently pregnant or breastfeeding, and not planning to become pregnant during the study?
  6. Have you not used certain other HAE preventative medications recently?
Answer every question to see your result.

What does participation involve?

If you join this study, your involvement would last for approximately 6 to 7 months in total. This includes an initial check-up period to confirm you meet all the study requirements. Once enrolled, you would take a study tablet (either deucrictibant or a placebo) once daily for 24 weeks, which is about six months. Throughout this time, you'll have regular visits to the clinic for assessments to check on your health and how the treatment is working. You will also be asked to keep a daily electronic diary to record information about any HAE attacks you experience and how you are feeling. After the 24 weeks of treatment, there will be a follow-up period of up to 4 weeks. Some participants may also have the option to continue into a separate, longer-term study if they wish.

Potential risks and benefits

Participating in a clinical trial may offer potential benefits, such as access to a new experimental treatment that isn't yet widely available, and close medical monitoring. Many people also find satisfaction in contributing to medical research that could help others with their condition. However, there are also potential risks; you might experience side effects from the study drug, or the treatment might not be effective for you. There is also the possibility of experiencing HAE attacks while in the study, as some participants will receive a placebo. You will always have the right to withdraw from the study at any time, for any reason, without it affecting your usual medical care.

Locations (56)

Some site locations are approximate. We're improving this — please verify with the trial team before travelling.
  • Study Site
    Verified postcode
    Little Rock, United States
  • Study Site
    Verified postcode
    San Diego, United States
  • Study site
    Verified postcode
    Santa Monica, United States
  • Study site
    Verified postcode
    Walnut Creek, United States
  • Study Site
    Verified postcode
    Chevy Chase, United States
  • Study Site
    Verified postcode
    St Louis, United States
  • Study Site
    Verified postcode
    Corrientes, Argentina
  • Study Site
    Verified postcode
    San Martin, Argentina
  • Study Site
    Verified postcode
    Santo André, Brazil
  • Study Site
    Verified postcode
    Sofia, Bulgaria
  • Study Site
    Verified postcode
    Sofia, Bulgaria
  • Study Site
    Verified postcode
    Edmonton, Canada

Common questions

What is Hereditary Angioedema (HAE)?

HAE is a rare genetic condition that causes sudden and often painful swelling in various parts of the body, including the face, hands, feet, airways, or stomach.

What is a placebo?

A placebo is a 'dummy' tablet that looks just like the study drug but contains no active medicine. It's used to compare against the real drug to see if the new medicine has a genuine effect.

What does 'double-blind' mean?

Double-blind means that neither you nor your study doctor will know whether you are receiving the new drug or the placebo. This helps ensure the study results are fair and unbiased.

How long would I need to take part in the study?

Your participation in the study would last for about 6 to 7 months, including an initial screening, 24 weeks of taking the study drug, and a follow-up period.

Can I stop participating in the study if I want to?

Yes, you have the right to withdraw from the study at any time, for any reason, without it affecting your future medical care.

How to find out more

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

Discussion

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