Open-label Berotralstat Access to HAE Patients Previously Enrolled in Berotralstat Studies
This study, called APeX-A, is for people with Hereditary Angioedema (HAE). If you’ve already been in certain berotralstat studies (Study 302, 204, or 304), this trial allows you to continue receiving this medicine. The main goal is to understand the long-term safety and how well people tolerate berotralstat when taken over a longer period. This is especially helpful in countries where berotralstat isn't readily available yet. Adults and teenagers (12 years and older) will take a 150 mg tablet once a day, while younger children will receive a dose based on their weight. You'll have regular check-ups every 24 weeks to get more medicine and for safety monitoring.
At a glance
What is this study about?
This new study, called APeX-A, is designed for individuals living with Hereditary Angioedema (HAE) who have previously participated in other research studies involving a medication called berotralstat. HAE is a rare genetic condition that causes sudden, severe swelling episodes in different parts of the body.
The main purpose of the APeX-A study is to allow people who have already benefited from berotralstat access to continue taking it. This is particularly important in places where the medication isn't yet available for general use. By continuing to take berotralstat, researchers can gather more information about its long-term safety and how well people cope with the treatment over time. This helps build a complete picture of the medicine's effects.
Ultimately, the findings from this study will help regulators and doctors better understand berotralstat's profile. This long-term safety data is crucial for ensuring that effective treatments for HAE are not only available but also well-understood for their effects over many years of use, helping patients manage their condition with greater confidence.
Key takeaways
- This study is for people with HAE who previously took berotralstat in other studies.
- It provides ongoing access to berotralstat to help prevent HAE attacks.
- The main goal is to check the long-term safety of the medicine.
- Participants will have regular check-ups every 24 weeks.
- Some participants might stay in the study for up to 10 years.
Who may be eligible?
To join this study, you must have already taken part in one of these previous berotralstat studies: Study 302, 204, or 304. Both men and women are welcome to participate, and if you are under 18, your parent or legal guardian will need to give their permission.
It's important that your study doctor believes you would continue to benefit from taking berotralstat. If you are able to become pregnant, you will need to use effective contraception throughout the study.
You cannot join if you are pregnant or breastfeeding, or if you are already taking other medications specifically designed to prevent HAE attacks long-term. You also can't be taking part in another trial for a different investigational medicine.
- Have you already been in a berotralstat study (Study 302, 204, or 304)?
- Do you feel you still benefit from berotralstat?
- Are you NOT pregnant or breastfeeding?
- Are you NOT taking other long-term HAE prevention medicines?
- Are you NOT in another trial for an experimental medicine?
This is a guide only — the research team will confirm whether you can take part.
What does participation involve?
If you join this study, you'll continue to take berotralstat by mouth once a day. You'll need to visit the clinic about every 24 weeks (roughly every 6 months). At these visits, you'll receive more of your medication, and the study team will carefully check your health and how you're feeling to make sure the treatment is safe for you. The total time you can stay in the study varies depending on which previous study you were in. For those from Studies 302 and 204, you could be in this study for up to 10 years. If you were in Study 304, you can stay until you turn 16 or for up to 5 years, whichever comes first, or until you can get berotralstat through another way.
Potential risks and benefits
Locations (16)
- Study CenterOttawa, Canada
- Study CenterBrno, Czechia
- Study CenterPilsen, Czechia
- Study CenterGrenoble, France
- Study CenterMarseille, France
- Study CenterParis, France
- Study CenterFrankfurt, Germany
- Study CenterSkopje, North Macedonia
- Study CenterKrakow, Poland
- Study CenterMartin, Slovakia
- Study CenterCape Town, South Africa
- Study CenterDaegu, South Korea
+4 more sites — see the official record for the full list.
Common questions
What is Hereditary Angioedema (HAE)?
HAE is a rare condition that causes sudden, severe swelling attacks in various parts of the body.
What is berotralstat?
Berotralstat is a medication used to help prevent HAE attacks. It's taken as a pill once a day.
Why is this study important?
It allows people who have already been helped by berotralstat to continue taking it, especially where it's not widely available, and helps us understand its long-term safety.
How often will I need to visit the clinic?
You'll need to visit the clinic about every 24 weeks (every 6 months) for check-ups and to get more medication.
Can I stop taking part in the study?
Yes, you can choose to leave the study at any time. This won't affect your future medical care.
How to find out more
Always speak to your GP or specialist before deciding to take part in a study.
Interested in taking part?
Discussion
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