AN OPEN LABEL, SINGLE ARM, EXPLORATIVE TRIAL TO EVALUATE EFFICACY AND SAFETY OF BEROTRALSTAT IN PREVENTING ANGIOEDEMA ATTACKS IN SUBJECTS WITH ACQUIRED ANGIOEDEMA DUE TO C1 INHIBITOR DEFICIENCY (AAE)
Acquired angioedema is a rare condition that causes sudden, uncomfortable swelling attacks in places such as the face, hands, feet, gut, or throat. It happens when the body lacks an active blood protein called C1 inhibitor, which normally keeps swelling under control. This clinical trial is testing a medicine called Orladeyo (berotralstat). Orladeyo comes as a capsule taken once a day by mouth. It is already approved for people with inherited swelling disorders, but doctors want to see if it also works well for people who developed the condition later in life. Everyone in the study will take the capsule for 24 weeks (around six months). Doctors will track how often attacks happen compared to before starting the medication. They will also monitor whether the attacks become milder and check for any side effects.
At a glance
What is this study about?
Acquired angioedema (AAE) is a rare disorder that causes unexpected bouts of severe swelling beneath the skin and inside the body. Unlike hereditary angioedema, which is passed down through families, the acquired form usually develops in adulthood. It occurs when the body's supply of a protective protein called C1 inhibitor becomes depleted or stops working properly. This can lead to painful swelling in the stomach, limbs, face, or airway.
In this clinical trial, researchers are exploring whether a medicine called Orladeyo (berotralstat) can help prevent these attacks. Orladeyo works by blocking a specific enzyme in the blood called plasma kallikrein. When C1 inhibitor levels are too low, this enzyme becomes overly active and triggers fluid leakage into tissues. By blocking it with a daily capsule, doctors hope to stop swelling attacks before they start.
Currently, people with acquired angioedema have very limited options for long-term prevention and often rely on emergency treatments when an attack strikes. Testing an easy-to-take oral capsule is an important step forward. If the trial is successful, it could provide a much simpler way for patients to manage their condition and reduce trips to hospital.
Key takeaways
- Tests a daily oral capsule (Orladeyo 150 mg) to prevent swelling attacks.
- Aimed at adults aged 18 and older with acquired angioedema (AAE).
- There is no placebo; every participant receives the active medication.
- Treatment lasts for 24 weeks (around six months) alongside close health monitoring.
- Participants can continue to use rescue medication for sudden attacks if needed.
Who may be eligible?
This study is designed for adults aged 18 and over who have an established diagnosis of acquired angioedema linked to low or poorly working C1 inhibitor. Both men and women can take part.
Because the study focuses specifically on the 'acquired' form of the condition, it is not suitable for people with hereditary angioedema (the type inherited from parents). Participants will also need to have a history of active swelling attacks so researchers can tell whether the treatment makes a difference.
Before you can join, the trial doctor will review your full medical history and perform standard blood tests. They will make sure that your organs are working well and check that Orladeyo is safe to take alongside your other daily medicines.
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 been diagnosed with acquired angioedema (not the inherited form).
- I still experience regular swelling attacks.
- I can swallow a capsule once daily.
- I am willing to track my symptoms in a daily study diary.
- I can attend regular hospital visits for check-ups over several months.
What does participation involve?
Taking part in the trial lasts around seven to eight months in total. It begins with a short observation period where the study team records how many swelling attacks you normally experience. After this, you will take one 150 mg Orladeyo capsule by mouth each day for 24 weeks (about six months). Throughout the study, you will have regular clinic visits and phone check-ups with the medical team. You will be asked to keep a simple diary recording any swelling attacks, how severe they were, and whether you needed your usual emergency rescue medication. Doctors will also carry out routine blood tests and physical exams to ensure you stay safe.
Potential risks and benefits
Locations (1)
- —UnverifiedItaly
Common questions
Will I definitely receive the study medicine?
Yes. This is an 'open-label, single-arm' trial, meaning there is no dummy drug (placebo). Every participant receives the active Orladeyo capsules.
How do I take the medicine?
Orladeyo is taken as a single capsule once a day by mouth, usually with food.
Can I still use my normal rescue medicine if I have an attack?
Yes. You will still have access to your usual emergency medications to treat any sudden swelling attacks that occur during the study.
Does it cost anything to take part?
No. The study medication, clinic visits, and medical tests are provided free of charge. Travel expenses to attend visits are also often covered.
What happens if I change my mind?
You are free to leave the trial at any time, for any reason. Your regular doctors will continue to look after you just as before.
How to find out more
Always speak to your GP or specialist before deciding to take part in a study.
Discussion
Community discussion
Powered by our forum at community.patient.info. Please be respectful — this is not medical advice.