NTLA-2002 in Adults With Hereditary Angioedema (HAE)
This research study is looking into a new treatment called NTLA-2002 for adults who have Hereditary Angioedema (HAE) Type 1 or Type 2. HAE is a condition that causes sudden swelling attacks. The main goals of this study are to check if NTLA-2002 is safe, how well your body tolerates it, and if it helps reduce or prevent HAE attacks. Researchers will also be looking at how the medicine works in your body over time. This is an early stage of testing for NTLA-2002, meaning it's one of the first times it's being given to people. If you're an adult with HAE and are interested in new treatment options, this study might be for you.
At a glance
What is this study about?
Hereditary Angioedema (HAE) is a rare genetic condition that causes unpredictable and often severe swelling attacks. These attacks can affect various parts of the body, including the skin, gut, and airways, and can be very painful and even life-threatening if they affect the throat. Current treatments help manage these attacks, but researchers are always looking for new and more effective ways to prevent them.
This study is investigating a new potential medicine called NTLA-2002. It's an early-stage clinical trial, which means it’s one of the first times this medicine is being tested in people. The main purpose is to carefully check if NTLA-2002 is safe for people with HAE. We also want to understand if it’s well-tolerated, meaning if people can take it without too many unwanted side effects. Beyond safety, the study aims to see if NTLA-2002 has a positive effect on HAE, helping to control or prevent new swelling attacks.
Researchers will also be monitoring how the medicine moves through your body and how your body reacts to it over time. This information is crucial for understanding how NTLA-2002 works and for deciding if it's a promising treatment that should be studied further in larger groups of people. Your participation would provide valuable information to help advance knowledge and potential treatments for HAE.
Key takeaways
- This study is testing a new medicine (NTLA-2002) for Hereditary Angioedema (HAE).
- It's an early-stage study, focused on safety and how well the medicine is tolerated.
- Adults with HAE Type 1 or Type 2 can be considered for participation.
- You might receive the new medicine or a 'dummy' treatment.
- Participation involves regular clinic visits and health checks.
- You can stop taking part at any time without affecting your care.
Who may be eligible?
To be able to take part in this study, you must be an adult aged 18 or over and have been diagnosed with Hereditary Angioedema (HAE) Type 1 or Type 2. You’ll also need to be able to show that you've had HAE attacks and have access to and know how to use at least one medicine to treat your sudden swelling attacks.
There are also some reasons why you might not be able to join. For example, if you have another type of regular swelling condition that isn't HAE, or if you know you've had a bad reaction to similar types of medicines in the past. If the study doctor thinks that taking part in the study could put your safety at risk, or if you're not able to follow the study's instructions, then you wouldn't be able to participate. You also can't be taking part in another medical study at the same time.
- Are you 18 years old or older?
- Have you been diagnosed with Hereditary Angioedema (HAE) Type 1 or Type 2?
- Do you currently have a way to treat your HAE attacks (e.g., medication)?
- Are you able to regularly attend study visits?
- Are you willing to not participate in other medical studies during this one?
- Do you have good overall health, according to your study doctor?
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, you'll receive doses of either NTLA-2002 or a 'dummy' treatment (normal saline, which is just saltwater). Both are given directly into your vein. The study team will regularly check your health to see how you're doing, gather blood and urine samples, and record any side effects you might experience. This helps them understand the safety and effects of the treatment.
You'll have regular hospital visits for assessments and to receive the treatment. The total length of your participation is not specified but will involve several visits over a period of time. You'll also need to be willing to follow all the study procedures carefully and avoid joining any other medical studies while this one is ongoing.
Potential risks and benefits
Locations (9)
- Clinical Trial SiteCampbelltown, Australia
- Clinical Trial SiteGrenoble, France
- Clinical Trial SiteLille, France
- Clinical Trial SiteParis, France
- Clinical Trial SiteBerlin, Germany
- Clinical Trial SiteFrankfurt, Germany
- Clinical Trial SiteAmsterdam, Netherlands
- Clinical Trial SiteAuckland, New Zealand
- Clinical Trial SiteCambridge, United Kingdom
Common questions
What is Hereditary Angioedema (HAE)?
HAE is a rare inherited condition that causes sudden and painful swelling attacks in various parts of the body, such as the skin, throat, or gut.
What is NTLA-2002?
NTLA-2002 is an experimental medicine being tested in this study to see if it can help adults with Hereditary Angioedema.
Will I definitely receive the new medicine?
In this study, you might receive NTLA-2002 or a 'dummy' treatment called normal saline, which is a saltwater solution. This is common in studies to help researchers compare the effects.
How will the medicine be given?
Both NTLA-2002 and the 'dummy' treatment will be given to you directly into a vein (intravenously).
Can I stop participating if I want to?
Yes, you are free to leave the study at any time, for any reason, without it affecting your usual medical care.
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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