Study of Re-Treatment With ALXN2220 in Patients With ATTR-CM
This is a study for people with a heart condition called Amyloid Transthyretin Cardiomyopathy (ATTR-CM). If you previously took part in an earlier study (NI006-101) and received the medicine ALXN2220, this trial might be for you. We are looking at giving ALXN2220 again to see how it affects your body and how safe it is over 48 weeks. Researchers will also be checking for any changes in your heart's structure and how well it is working. We'll also be observing what happened to your heart during the time you weren't taking the medicine after the first study. This is a follow-up to understand the long-term effects of this treatment.
At a glance
What is this study about?
This research study is about a heart condition called Amyloid Transthyretin Cardiomyopathy (ATTR-CM). In this condition, an abnormal protein builds up in the heart, making it stiff and harder for it to pump blood. The study is specifically designed for people who have taken part in a previous research trial (called Study NI006-101) where they received a medicine called ALXN2220.
The main goal of this new study is to understand more about how ALXN2220 works when it's given again to people who have previously used it. We also want to confirm how safe it is. Researchers will be carefully checking how the medicine affects your body, including any changes to your heart's structure and how well it functions. They will also look at how you feel and your overall health during the study.
Another important part of this study is to look at what has happened to participants' hearts since they finished the previous study and stopped taking ALXN2220. By re-treating with the medicine, we hope to gain a better understanding of how ALXN2220 can help manage ATTR-CM over a longer period and whether taking breaks from the medication has a significant impact on the condition.
Key takeaways
- This study is for people who have previously taken part in a specific ATTR-CM medicine trial (ALXN2220).
- It aims to see how safe ALXN2220 is and how it affects the body when given again.
- Researchers will monitor your heart and overall health over 48 weeks.
- It's an important follow-up to understand longer-term effects of the treatment.
- Participation involves regular clinic visits for checks and medication.
Who may be eligible?
To be able to join this study, everyone must have taken part in a previous study called NI006-101 and received at least one dose of the medicine ALXN2220. This means it's not open to people who haven't participated in that specific earlier trial.
There are also some reasons why you might not be able to join. For example, if you know you are allergic or intolerant to proteins or any part of the study medicine. Also, if you had to stop the previous study because of a serious or dangerous side effect from the medicine, you wouldn't be able to join this one.
Finally, if you have any new health conditions or existing conditions that are not under control since you finished the last study, and these might make it unsafe for you to take part, the study team would need to consider this 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.
- Did I receive at least one dose of ALXN2220 in Study NI006-101?
- Do I have any known allergies to medicines or any part of the study drug?
- Did I have to stop the previous study because of a serious or dangerous side effect?
- Do I have any new or uncontrolled health problems since the last study?
What does participation involve?
If you join this study, you will go through a 'Screening Period' first to make sure it's safe and suitable for you. After that, you will enter the 'Active Treatment Period' which will last for 48 weeks (about 11 months). During this time, you will regularly receive the medicine ALXN2220. The study team will ask you to visit the clinic regularly for check-ups. These visits will involve various assessments, such as blood tests, heart scans, and tests to see how well your heart is working. They will also ask you questions about how you are feeling and any changes in your health. The medical team will explain the exact schedule of visits and what each visit will involve.
Potential risks and benefits
Locations (6)
- Hôpital Henri MondorVerified postcodeCréteil, France
- CHU de Rennes - Hôpital PontchaillouVerified postcodeRennes, France
- CHU Toulouse - Hôpital RangueilVerified postcodeToulouse, France
- Universitätsklinikum HeidelbergVerified postcodeHeidelberg, Germany
- University Medical Center GroningenVerified postcodeGroningen, Netherlands
- Hospital Universitario Puerta de Hierro MajadahondaVerified postcodeMajadahonda, Spain
Common questions
What is ATTR-CM?
ATTR-CM is a heart condition where an abnormal protein builds up in the heart, making it stiff and harder for it to pump blood properly.
Who can join this study?
Only people who previously took part in an earlier study (NI006-101) and received the medicine ALXN2220 can join this one.
What is ALXN2220?
ALXN2220 is a medicine being tested for ATTR-CM. This study is looking at re-treating people with it.
How long will the study last?
After an initial check-up period, the active treatment part of the study will last for 48 weeks (about 11 months).
Can I stop taking part if I change my mind?
Yes, you are free to leave the study at any time, for any reason, without affecting your medical care.
How to find out more
Always speak to your GP or specialist before deciding to take part in a study.
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