Long-Term Follow-Up (LTFU) of Subjects Treated With NTLA 2002
This study is a long-term check-up for individuals who have already had a treatment called NTLA-2002 as part of a previous medical trial. The main goal is to watch how these people are getting on over a longer period. Researchers want to understand if the treatment continues to have effects, good or bad, and how their health changes after receiving NTLA-2002 for hereditary angioedema. It’s an observational study, meaning participants won't receive new treatment but will be monitored to see the long-term impact of the treatment they've already had. It helps gather important information about the safety and effectiveness of NTLA-2002 over many years.
At a glance
What is this study about?
This study is designed to keep an eye on people who have previously been treated with a new medicine called NTLA-2002. This treatment was given as part of an earlier clinical trial for a condition called hereditary angioedema (HAE). HAE is a rare genetic condition that causes sudden swelling in different parts of the body, which can be painful and sometimes dangerous.
The main purpose of this study is to understand the long-term effects of NTLA-2002. Researchers want to see how participants are doing many years after receiving the treatment. This includes looking at their general health, how often they experience swelling attacks, and if there are any lasting side effects from the medication. By tracking these things over a longer time, the study helps to build a more complete picture of how NTLA-2002 works and its overall impact.
This type of follow-up study is very important because it helps scientists and doctors learn about the lasting benefits and potential risks of new treatments once they've been given. The information gathered from this study will help ensure that new medicines are safe and effective in the long run for people living with hereditary angioedema. Participants in this study will not receive new doses of NTLA-2002; they will simply be monitored.
Key takeaways
- This study is observing people who have already received a new treatment (NTLA-2002) for Hereditary Angioedema (HAE).
- It aims to understand the long-term effects of the treatment over many years.
- Participants will not receive new medication during this study.
- Your involvement helps gather important safety and effectiveness information for HAE treatments.
- You must have been in a previous NTLA-2002 trial and agree to the follow-up.
- Your contribution is valuable for future patients with HAE.
Who may be eligible?
To be part of this follow-up study, you must have already participated in an earlier study where you received the NTLA-2002 treatment. This means you would have been given either a full or partial dose of the medicine during that previous trial.
Also, you need to agree to join this specific follow-up study by signing a consent form. This form explains everything about the study, and your agreement shows you understand what's involved and are happy to take part.
Finally, you must be willing and able to come to all the study appointments and follow the instructions given by the study team. There are no other specific health reasons that would stop you from taking part, as long as you meet these main points.
- Did you take part in an earlier study where you received the NTLA-2002 treatment?
- Are you happy to sign a consent form to join this new follow-up study?
- Are you willing and able to attend the required study appointments?
- Can you follow the instructions given by the study team?
This is a guide only — the research team will confirm whether you can take part.
What does participation involve?
Taking part in this study means you will attend regular check-up appointments. During these visits, the study team will monitor your health and gather information about your hereditary angioedema. This might include asking about your symptoms, how often you have swelling attacks, and if you've experienced any changes in your health since your last visit or since receiving the NTLA-2002 treatment.
You won't be given any new medication in this study, as it's purely to observe the long-term effects of the treatment you received previously. The study team will tell you the exact schedule of visits and what each appointment will involve, including any tests or questions they might ask. The total length of your participation will depend on the study plan, which aims to follow people for a long time to understand lasting effects.
Potential risks and benefits
Locations (7)
- Campbelltown HospitalCampbelltown, Australia
- Centre National de Reference - GrenobleGrenoble, France
- Hôpital Claude HuriezLille, France
- Charite-Universitätsmedizin BerlinBerlin, Germany
- University of Amsterdam Academic Medical CenterAmsterdam, Netherlands
- New Zealand Clinical ResearchAuckland, New Zealand
- Cambridge University Hospitals NHS Foundation Trust, Addenbrooke's HospitalCambridge, United Kingdom
Common questions
What is hereditary angioedema (HAE)?
HAE is a rare genetic condition that causes unpredictable and often painful swelling in various parts of the body, like the face, hands, feet, or airway.
What is NTLA-2002?
NTLA-2002 is a new type of gene-editing treatment that was tested in earlier clinical trials for hereditary angioedema.
Will I receive more treatment in this study?
No, this study is a 'follow-up' to check on people who have already received NTLA-2002. You will not be given any new doses of the treatment.
What will I have to do if I join?
You will attend scheduled appointments where doctors will ask about your health and HAE symptoms. You'll also need to agree to take part by signing a form.
Why is this study important?
It helps researchers understand the long-term benefits and any lasting side effects of NTLA-2002, which is crucial for the future of HAE treatment.
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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