Efficacy and Safety of Immunosuppression, Caplacizumab and Plasma Infusion Without Therapeutic Plasma Exchange in Immune-mediated Thrombotic Thrombocytopenic Purpura
This research is exploring a new, less invasive approach to treating a rare blood condition called immune thrombotic thrombocytopenic purpura (iTTP). Currently, patients need daily plasma exchange (PEX), which can be time-consuming and have side effects. This trial aims to see if a treatment combination of immunosuppressive medicines, caplacizumab (a medicine that stops blood clots), and plasma infusions (without daily PEX) is just as effective and safe. By potentially removing the need for daily PEX, this new method could make treatment much easier for patients, reduce hospital stays, and lower the risk of common complications associated with the standard treatment.
At a glance
What is this study about?
Immune thrombotic thrombocytopenic purpura, or iTTP, is a rare but serious blood condition. In iTTP, your body mistakenly attacks a protein called ADAMTS13, which is important for controlling blood clotting. This leads to tiny clots forming in your blood vessels, which can cause problems in various organs and lower your platelet count.
The usual treatment for iTTP involves daily plasma exchange (PEX). This is a procedure similar to dialysis, where some of your blood is taken out, treated, and then returned. While PEX is effective, it’s a big undertaking. It takes a lot of time, often means spending more time in hospital, and can come with risks and side effects like problems with the catheter used or reactions to the plasma. Because of this, doctors are always looking for ways to make treatment better and easier for patients.
This study is investigating a new treatment approach that aims to avoid daily plasma exchange. It combines medicines that calm down the immune system (immunosuppressive agents), a special drug called caplacizumab that helps prevent new clots, and plasma infusions (giving plasma directly into your bloodstream, but not as part of a daily exchange). The goal is to see if this combination can effectively treat iTTP without the need for the invasive and time-consuming daily plasma exchange. If successful, this could significantly improve the treatment experience for people with iTTP, making it safer, more comfortable, and leading to shorter hospital stays.
Key takeaways
- This study is testing a new way to treat iTTP without daily plasma exchange.
- It combines immune-calming drugs, a clot-preventing medicine (Caplacizumab), and plasma infusions.
- The goal is to make iTTP treatment less invasive and potentially safer.
- Participation involves receiving study treatments and having regular check-ups.
- This could reduce hospital time and complications associated with standard treatment.
Who may be eligible?
To join this study, generally, you must be an adult aged 18 or older. You would need to have been diagnosed with iTTP based on specific clinical and lab tests. This diagnosis usually involves having a certain level of platelets (tiny blood cells involved in clotting) and creatinine (a waste product in your blood) after other causes have been ruled out. If you've had iTTP before, the exact criteria might be slightly different.
There are also some important reasons why you might not be able to join. For example, if your platelet count is too high before starting plasma treatment, or if your condition is thought to be a different type of blood disorder. Additionally, if you have other serious health issues like uncontrolled cancer, severe neurological problems (like significant seizures or a coma), or if you are pregnant, you would not be able to take part. Women who could become pregnant would need to be using effective contraception.
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.
- Are you 18 years old or older?
- Have you been diagnosed with iTTP (or a similar past episode)?
- Are you currently not pregnant?
- Are you able to use effective contraception if you're a woman who could become pregnant?
- Do you have a platelet count 100 G/L or less before starting plasma treatment?
- Do you not have other serious conditions like uncontrolled cancer or severe neurological problems?
What does participation involve?
This study is designed to evaluate a new treatment approach, so if you were to participate, you would receive a combination of medicines: immunosuppressive drugs to calm your immune system, caplacizumab (a drug to help prevent blood clots), and plasma infusions (where you receive plasma directly into your bloodstream). The frequency and duration of these treatments would be guided by the study doctors, and they would monitor your condition closely.
Throughout the study, you would attend regular appointments for check-ups, blood tests, and to assess how you are responding to the treatment. These visits are important for the doctors to understand how well the new approach is working and to ensure your safety. While the exact total duration isn't specified, typical clinical trials for new treatments involve several weeks or months of active treatment and follow-up. You would receive detailed information about the schedule before deciding to join.
Potential risks and benefits
Locations (30)
- Chu AmiensVerified postcodeAmiens, France· Recruiting
- Chu AngersVerified postcodeAngers, France· Recruiting
- Chru BesanonVerified postcodeBesançon, France· Recruiting
- Chu BobignyVerified postcodeBobigny, France· Recruiting
- Hopital Jean VerdieVerified postcodeBondy, France· Recruiting
- Chu BordeauxVerified postcodeBordeaux, France· Recruiting
- Chu Clermont-FerrandVerified postcodeClermont-Ferrand, France· Recruiting
- Chu LilleVerified postcodeLille, France· Recruiting
- Chu LimogesVerified postcodeLimoges, France· Not yet recruiting
- Chu Edouard HerriotVerified postcodeLyon, France· Recruiting
- Ap-Hm La ConceptionVerified postcodeMarseille, France· Recruiting
- Chu MontpellierVerified postcodeMontpellier, France· Recruiting
Common questions
What is iTTP?
iTTP (immune thrombotic thrombocytopenic purpura) is a rare blood disorder where your immune system attacks a protein, leading to tiny blood clots and low platelet counts.
What is 'plasma exchange' and why is it usually given?
Plasma exchange is a procedure similar to dialysis that removes harmful substances from your blood. It's normally used daily for iTTP to remove problem proteins and provide healthy ones.
What is Caplacizumab?
Caplacizumab is a medicine that helps prevent the tiny blood clots that cause problems in iTTP.
Will I still get plasma if I join this trial?
Yes, you will still receive plasma, but it will be given as infusions (directly into your bloodstream) instead of daily plasma exchange.
Who is running this study?
The study title is about 'Efficacy and Safety of Immunosuppression, Caplacizumab and Plasma Infusion Without Therapeutic Plasma Exchange in Immune-mediated Thrombotic Thrombocytopenic Purpura'. It is a clinical trial being conducted by medical researchers.
How to find out more
Ygal Benhamou, Pr
Always speak to your GP or specialist before deciding to take part in a study.
Interested in taking part?
Discussion
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