A Phase 3, Randomized, Double-blind, Placebo-controlled Study to Evaluate the Efficacy and Safety of Orally Administered Deucrictibant Extended-release Tablet for Prophylaxis Against Angioedema Attacks in Adolescents and Adults with Hereditary Angioedema
Hereditary angioedema (HAE) is a rare condition that causes sudden, painful swelling under the skin and inside the body. Current treatments often involve injections, which can be difficult to manage. This Phase 3 clinical trial is testing whether a new preventative medicine called deucrictibant can stop attacks before they start. Deucrictibant is a long-acting tablet taken by mouth. During the 24-week study period, researchers will compare it against a dummy tablet (placebo) to see how well it works and check for side effects. Neither you nor the study team will know which tablet you receive. Doctors will track how many attacks participants experience and check improvements in everyday quality of life. If swelling attacks do occur during the study, standard rescue medications are provided to treat them quickly and safely.
At a glance
What is this study about?
Hereditary angioedema, often shortened to HAE, is a long-term genetic condition. People with HAE experience unpredictable attacks of severe swelling in places like the hands, feet, face, gut, and throat. These attacks can be painful, frightening, and at times dangerous if the airways swell. Many current treatments require injections under the skin or into a vein, which can be inconvenient and uncomfortable.
This study looks at a new drug called deucrictibant. It is designed to work as an 'extended-release' tablet, meaning it slowly releases medicine into your body throughout the day. By blocking a specific chemical pathway responsible for swelling, the tablet aims to keep swelling attacks from happening in the first place.
Because this is a Phase 3 study, the drug has already been tested in smaller groups. Researchers now need to confirm how safe and effective it is in a larger, broader group of people. If the results are positive, this medicine could offer people living with HAE a much simpler daily option for preventing attacks without the need for needles.
Throughout the study, you will have regular contact with a specialist healthcare team. Your safety is closely monitored through routine health checks, and you will always have access to standard on-demand emergency treatments if an attack does break through.
Key takeaways
- Tests a new daily preventative tablet (deucrictibant) for hereditary angioedema (HAE).
- Aims to reduce the number and severity of sudden, painful swelling attacks.
- Lasts for 24 weeks of treatment, comparing the active tablet to an inactive dummy pill (placebo).
- Emergency rescue medication is provided in case you experience an attack during the trial.
- Participation is voluntary, and you can withdraw at any time without losing your usual care.
Who may be eligible?
This trial is open to adults aged 18 and older who have been diagnosed with hereditary angioedema. To take part, you should have a documented history of regular swelling attacks so that researchers can measure whether the study medicine reduces how often they happen.
You will need to be in generally stable health apart from your HAE. The study team will review your medical history and current medications to make sure the trial is safe for you.
You may not be able to take part if you have certain other severe medical conditions, such as major kidney or liver problems, or if you are taking specific medications that could interact with the study drug. Pregnant or breastfeeding women are usually unable to join clinical trials of this kind.
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 18 or older.
- I have a confirmed medical diagnosis of hereditary angioedema (HAE).
- I still experience occasional swelling attacks.
- I am able and willing to attend regular clinic visits over roughly six months.
- I am willing to take a daily tablet and keep a diary of my symptoms.
What does participation involve?
Taking part in the trial lasts around six months (24 weeks of treatment), plus a short screening period before you start and a follow-up period at the end. You will be randomly assigned to take either deucrictibant tablets or matching dummy tablets (placebo) once daily by mouth.
You will visit the clinic regularly for health check-ups. These visits will include routine blood and urine tests, heart rhythm checks (ECGs), and blood pressure measurements. You will also be asked to complete simple surveys about your quality of life, daily activities, and how well your swelling is controlled.
You will keep a diary of any symptoms or swelling episodes. If you experience an attack, you will have access to standard fast-acting rescue medicine (such as icatibant injections) to treat it immediately.
Potential risks and benefits
Locations (10)
- —UnverifiedFrance
- —UnverifiedBulgaria
- —UnverifiedSlovakia
- —UnverifiedHungary
- —UnverifiedIreland
- —UnverifiedPoland
- —UnverifiedRomania
- —UnverifiedItaly
- —UnverifiedSpain
- —UnverifiedGermany
Common questions
What is a placebo and why is it used?
A placebo is a dummy tablet that looks identical to the real study drug but contains no active medicine. It helps researchers prove whether any improvements or side effects are truly caused by the new drug.
What happens if I have a swelling attack during the trial?
Your safety comes first. You will be given proven rescue medicine (such as icatibant) to keep at home so you can treat any breakthrough swelling attacks straight away.
Will taking part cost me anything?
No. The study medication, clinic visits, and health tests are provided free of charge. Most trials also reimburse reasonable travel expenses for hospital visits.
Can I carry on taking my normal medicines?
The study doctor will review all the medicines you currently take. Some routine medications are allowed, but you may need to avoid certain treatments while on the study.
Can I stop taking part if I change my mind?
Yes. You can leave the study at any point without giving a reason. Your regular doctor will ensure you continue to receive the best available standard treatment.
How to find out more
Always speak to your GP or specialist before deciding to take part in a study.
Interested in taking part?
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