An Open-label Study to Evaluate the Long-term Safety and Efficacy of CSL312 (Garadacimab) in the Prophylactic Treatment of Hereditary Angioedema
This study is testing a new medicine called garadacimab for people who have hereditary angioedema (HAE). HAE is a rare condition that causes sudden swelling attacks. This research is a 'Phase III' study, which means the medicine has already been tested in smaller groups and is now being looked at in a larger group of people to confirm its long-term safety and effectiveness. The main goal is to understand how safe गरडाकिमाब is, by counting any side effects people might have. Researchers also want to see if it helps reduce the number of HAE attacks people experience, how often they need emergency treatment, and how severely they are affected. This ongoing study is open to both men and women aged 18 and over.
At a glance
What is this study about?
This study is all about understanding a new medicine called garadacimab, which is being investigated as a way to prevent attacks in people with hereditary angioedema (HAE). HAE is a very rare genetic condition that causes sudden, unpredictable swelling deeper under the skin, often in the face, hands, feet, or tummy. These swelling attacks can be painful and, in some cases, serious if they affect the airways.
The main aim of this research is to see how safe garadacimab is when used over a longer period. The researchers will be carefully monitoring participants for any side effects, also known as 'adverse events', to ensure the medicine is well-tolerated. They'll be looking at how many people experience side effects, how often they occur, and whether any are serious. This information is really important for understanding the overall safety of the treatment.
Beyond safety, the study also wants to find out how well garadacimab works to prevent HAE attacks. They'll be looking at things like the total number of attacks people have, whether the medicine reduces attacks by a certain percentage (like 50%, 70%, or even 100% – meaning no attacks at all), and if people need less emergency treatment for their attacks. They'll also be checking how many moderate or severe attacks people have and how satisfied participants feel with the treatment. All of this helps build a full picture of whether garadacimab could be a helpful long-term option for people living with HAE.
Key takeaways
- This study is testing a new medicine called garadacimab for hereditary angioedema (HAE).
- It aims to find out if the medicine is safe and if it helps prevent HAE attacks over a long period.
- Anyone aged 18 or older with HAE can potentially join, regardless of gender.
- Participation involves regular clinic visits to monitor your health and the treatment's effects.
- You can stop participating in the study at any time without affecting your medical care.
Who may be eligible?
This study is looking for adults who are at least 18 years old. There is no upper age limit, so older adults can also take part. The study is open to both men and women.
To be considered for the study, you must have a confirmed diagnosis of hereditary angioedema (HAE). More specific details about your health condition and any other medications you are taking would be discussed in detail by the study team to ensure the research is right for you, and you are right for the research.
It's important to remember that these are just the basic requirements. The study team will review your full medical history to check if you meet all the other criteria that help make sure the study is safe and effective for everyone involved. They will also make sure the study results are as clear and accurate as possible.
- Are you 18 years old or older?
- Do you have a diagnosis of hereditary angioedema (HAE)?
- Are you able to attend regular clinic appointments?
- Are you comfortable with taking a new, investigational medication?
This is a guide only — the research team will confirm whether you can take part.
What does participation involve?
If you decide to take part in this study, you would first have a screening visit to make sure you meet all the requirements. This would likely involve a review of your medical history, a physical exam, and some blood tests. After that, you would start receiving the study medication, garadacimab. The researchers will explain exactly how often and how you would take or receive this medication.
Throughout the study, you would have regular visits to the clinic. These visits are important for the study team to monitor your health, check for any side effects, and see how well the medicine is working. This might include more physical exams, blood tests, and discussions about any HAE attacks you've had since your last visit. You might also be asked to complete questionnaires about your quality of life and how you feel about the treatment.
The study is designed to look at the long-term effects of garadacimab, so your participation would likely last for an extended period, potentially several years. The team will provide you with a detailed schedule of visits and what to expect at each one. There will also be follow-up contact, even after you stop taking the study medication, to ensure your health is still stable.
Potential risks and benefits
Locations (5)
- —Netherlands
- —Germany
- —Hungary
- —Czechia
- —Spain
Common questions
What is hereditary angioedema (HAE)?
HAE is a rare genetic condition causing sudden and unpredictable episodes of swelling in different parts of the body, like the hands, feet, face, or tummy.
What is garadacimab?
Garadacimab is a new medicine being tested to see if it can help prevent HAE attacks.
What does 'Phase III' mean for a drug trial?
Phase III means the medicine has already been tested in smaller groups and is now being studied in a larger group of people to confirm its safety and how well it works long-term.
How long will I need to be in the study if I take part?
This is a long-term study, so your participation could last for an extended period, possibly several years, to assess the long-term effects.
Will I still receive my usual HAE care if I join the study?
Joining the study should not stop you from receiving your regular medical care for HAE. The study team will work with your doctor.
How to find out more
Always speak to your GP or specialist before deciding to take part in a study.
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