A Phase II, Double-blind, Placebo-controlled, Randomized, Dose-ranging, Parallel Group Study to Evaluate the Safety and Efficacy of PHA-022121 Administered Orally for Prophylaxis Against Angioedema Attacks in Patients with Hereditary Angioedema due to C1-Inhibitor Deficiency (Type I or Type II)
This research is looking at a new medicine called PHA-022121 for people who suffer from hereditary angioedema (HAE), specifically Type 1 or Type 2. The main goal is to find out if this medicine, taken by mouth, can safely and effectively reduce the number of painful swelling attacks that HAE causes. Participants will be split into groups; some will receive the new medicine, and others will receive a placebo (a dummy pill with no active ingredient). We will compare how many attacks occur in each group over a period of about three months. This will help doctors understand if PHA-022121 could be a helpful treatment to prevent HAE attacks.
At a glance
What is this study about?
This study is about finding better ways to prevent swelling attacks for people with a condition called hereditary angioedema (HAE) Type 1 or Type 2. HAE is a rare genetic condition that causes sudden, painful swelling attacks, most often in the skin, gut, or airways. These attacks can be very uncomfortable, sometimes dangerous, and can significantly affect daily life.
The new medicine being tested is called PHA-022121, and it's given as a tablet. The main aim of this study is to see if taking PHA-022121 regularly can reduce how often these swelling attacks happen. It's a 'Phase II' study, which means it’s still in the earlier stages of testing. We are looking for the right dose and to make sure it's safe.
To make sure we get clear results, some participants will receive the new medicine, and others will receive a 'placebo' (a dummy pill that looks the same but has no active ingredients). This is important so we can fairly compare the effects of the new medicine against not taking it. By carefully tracking the number and severity of attacks in both groups, researchers can figure out if PHA-022121 is effective and safe enough to continue testing.
Key takeaways
- Tests a new medicine (PHA-022121) for hereditary angioedema (HAE).
- Aims to prevent HAE swelling attacks.
- Compares PHA-022121 to a dummy pill (placebo).
- Participation lasts about three months in the first stage.
- For adults aged 18 and over with HAE Type 1 or 2.
- Close medical checks and monitoring throughout.
Who may be eligible?
This study is designed for adults aged 18 and over who have been diagnosed with Hereditary Angioedema (HAE) Type 1 or Type 2. Both men and women can take part.
To join, you'll need to meet specific health requirements that the study doctors will check. For example, they will look at your medical history, your current health, and how often you typically experience HAE attacks. You will also need to be able to attend regular appointments and follow the study instructions carefully.
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.
- Are you 18 years old or older?
- Have you been diagnosed with Hereditary Angioedema (HAE) Type 1 or Type 2?
- Are you able to take medicine by mouth?
- Are you able to attend regular study appointments?
What does participation involve?
If you decide to take part in this study, you would primarily be involved for about three months in the first part. You would be asked to take either the study medicine (PHA-022121) or a placebo (a dummy pill) by mouth every day. You would have regular visits to the clinic where doctors and nurses would check your health, ask about your HAE attacks, and monitor any side effects. You would also need to record any HAE attacks you experience during this time. There may be further stages to the study after the initial three months, but these would be discussed with you in detail.
Potential risks and benefits
Locations (6)
- —UnverifiedPoland
- —UnverifiedAustria
- —UnverifiedItaly
- —UnverifiedIreland
- —UnverifiedBulgaria
- —UnverifiedGermany
Common questions
What is hereditary angioedema (HAE)?
Hereditary angioedema (HAE) is a rare genetic condition that causes sudden and painful swelling, often in the face, hands, feet, stomach, or throat. These attacks can be uncomfortable and sometimes serious.
What is PHA-022121?
PHA-022121 is a new medicine being tested to see if it can help prevent HAE attacks. It's taken as a tablet by mouth.
What is a 'placebo'?
A placebo is a dummy pill that looks exactly like the study medicine but doesn't contain any active drug. Giving some people a placebo allows researchers to fairly compare the effects of the real medicine.
How long will I be in the study?
The first main part of the study where you take the medicine or placebo will last about three months (84 days). There might be options for longer-term participation, but this would be explained fully.
Will I know if I'm getting the real medicine or the placebo?
No, this is a 'double-blind' study, meaning neither you nor your study doctor will know whether you are receiving PHA-022121 or the placebo. This helps ensure fair and unbiased results.
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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