An open-label, long-term, safety and tolerability study of SAR445088 in participants with cold agglutinin disease previously treated with SAR445088 or never treated with SAR445088
This study is investigating a new medicine called riliprubart for people who have a rare blood condition called cold agglutinin disease (AIHA). This condition causes the body's immune system to mistakenly attack and destroy its own red blood cells. The main goal of this particular study is to check how safe the medicine is when taken over a longer period and whether people can tolerate it well. We are looking for any side effects that might happen. Some people in the study will have taken riliprubart before, and others will be trying it for the first time. The study will also look at how the medicine affects certain blood levels and how it works in the body.
At a glance
What is this study about?
This study is about a new medication called riliprubart, which is being tested for a condition called autoimmune haemolytic anaemia, specifically a type known as cold agglutinin disease. This is a rare condition where your body's defence system, called the immune system, mistakenly attacks and destroys your own red blood cells. Red blood cells are important because they carry oxygen around your body. When they are destroyed too quickly, it can lead to anaemia, making you feel tired and weak.
The main purpose of this study is to understand the safety of riliprubart over a long time. Researchers want to know if it causes any unwanted effects, often called side effects, and how well people can cope with taking the medicine. Some people who join this study will have already taken riliprubart in a previous study, while others will be trying it for the first time. This helps researchers compare the effects in different groups.
Beyond safety, the study will also look at how riliprubart affects your body. This includes checking things like your red blood cell levels (haemoglobin), yellow pigment levels (bilirubin), and other markers in your blood that show how your condition is progressing. They will also look at how much of the medicine stays in your body and if your body develops any reactions to it. All this information helps doctors understand if riliprubart could be a helpful treatment for people with cold agglutinin disease.
Key takeaways
- This study is for adults with cold agglutinin disease.
- It tests a new medicine called riliprubart.
- The main focus is to check how safe the medicine is long-term.
- It also looks at how the medicine affects blood levels and how your body uses it.
- Participation involves regular clinic visits and blood tests.
- You can stop participating at any time.
Who may be eligible?
To be considered for this study, you must be an adult, aged 18 years or older. The study is open to both men and women.
Whether you have been part of an earlier study for riliprubart or not will be part of the decision-making process. Some participants will have taken the medicine before, and others will be new to it. Your doctor will carefully review your medical history and current health to see if this study is suitable for you.
There will be specific health criteria that you need to meet, or not meet, to join the study. These are put in place to ensure your safety and that the study results are as clear as possible. It's important to discuss all your medical conditions and medicines with the study team.
- Are you an adult aged 18 years or older?
- Have you been diagnosed with cold agglutinin disease?
- Are you able to attend regular clinic appointments and blood tests?
- Are you willing to take a new study medication?
- Are you comfortable discussing your full medical history with the study team?
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 will be given the study medication, riliprubart, which you will take for a period of time. You will need to attend regular visits to the clinic, where the study team will monitor your health closely. These visits will involve various assessments, such as blood tests to check your red blood cell count, liver and kidney function, and other markers related to your condition and the study medication. You may also have physical examinations and answer questions about how you are feeling.
The frequency of visits and the types of tests will be clearly explained by the study team. They will also monitor you for any side effects and adjust your care as needed. After you finish taking the study medicine, there will likely be a follow-up period where you will have further appointments to ensure your health remains stable. The total duration of your participation, including treatment and follow-up, will vary and will be fully explained before you agree to join.
Potential risks and benefits
Locations (3)
- —Germany
- —Netherlands
- —Italy
Common questions
What is cold agglutinin disease?
It's a rare blood condition where your immune system mistakenly attacks and destroys your own red blood cells, which can cause anaemia.
What is riliprubart?
It's a new medicine being tested to see if it can help people with cold agglutinin disease by stopping their immune system from destroying red blood cells.
What is the main goal of this study?
The main goal is to check how safe riliprubart is when taken over a long time and to see if people taking it have any side effects.
Will I get the medicine if I join?
Yes, if you join the study, you will receive the study medication, riliprubart, as part of your participation.
Can I stop participating in the study if I want to?
Yes, you have the right to withdraw from the study at any time, for any reason, without it affecting your regular medical care.
How to find out more
Always speak to your GP or specialist before deciding to take part in a study.
Discussion
Community discussion
Powered by our forum at community.patient.info. Please be respectful — this is not medical advice.