Active not recruitingOBSERVATIONAL

Retrospective Analysis of the Efficiency of Caplacizumab in the Treatment of aTTP

This study is a look back at how a medicine called Caplacizumab (known as Cablivi®) has been used in Germany to treat a rare and serious blood clotting condition called acquired Thrombotic Thrombocytopenic Purpura (aTTP). Researchers want to understand why doctors prescribe it and how effective it has been for patients in real-world situations, not just in controlled trials. The study also hopes to find clues about which patients might respond best to Caplacizumab. By doing this, the goal is to improve how aTTP is treated in the future, helping patients get better care and potentially reducing treatment costs.

At a glance

Status

Active not recruiting

Sponsor

University of Cologne

Enrolment target

350

Start

25 Mar 2021

Estimated completion

12 Dec 2034

Acquired Thrombotic Thrombocytopenic Purpura

What is this study about?

This research study is looking at a condition called acquired Thrombotic Thrombocytopenic Purpura (aTTP). This is a serious, rare blood disorder where tiny blood clots form in small blood vessels throughout the body. These clots can block blood flow to organs like the brain, heart, and kidneys, which can cause serious health problems. The study focuses on a medicine called Caplacizumab, which is sold under the brand name Cablivi®.

The main aim of this study is to understand how Caplacizumab is being used by doctors in Germany and how well it works for patients with aTTP in everyday medical practice. It's an "observational" study, meaning researchers will look at existing patient records rather than trying new treatments. They want to see if the medicine helps patients get better and recover from their aTTP episodes.

The researchers are also interested in identifying certain signs or factors that might predict how a patient will respond to Caplacizumab. By gathering this information, they hope to develop better ways to treat aTTP in the future. The overall goal is to improve patient care, reduce complications, and potentially make treatments more efficient and cost-effective.

Key takeaways

The study looks at how a medicine called Caplacizumab treats a serious blood condition called aTTP.
It aims to improve future treatment of aTTP by understanding current practices in Germany.
Participation involves researchers reviewing your past medical records only.
There are no new treatments, visits, or tests required for this study.
The goal is to improve patient outcomes and potentially reduce treatment costs for aTTP.

Who may be eligible?

To be part of this study, you would have needed to meet certain conditions at the time of your aTTP diagnosis and treatment.

Firstly, you must have had a confirmed diagnosis of an active episode of acquired Thrombotic Thrombocytopenic Purpura (aTTP). You also must have received at least one dose of the medicine Caplacizumab (Cablivi®), given either into a vein or under the skin. You needed to be 18 years old or older and have been able to understand and sign a consent form, agreeing for your medical information to be used in the study.

You would not have been included if your Thrombotic Thrombocytopenic Purpura was hereditary (meaning you were born with it), rather than acquired. Also, if you were unable to provide informed consent, you could not have participated.

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 you have a confirmed diagnosis of acquired Thrombotic Thrombocytopenic Purpura (aTTP)?
Were you treated with at least one dose of Caplacizumab (Cablivi®)?
Were you 18 years old or older at the time of your treatment?
Was your TTP acquired (not hereditary)?
Did you provide consent for your medical information to be used (or are able to now)?

Answer every question to see your result.

What does participation involve?

If you meet the criteria for this study, your involvement would be retrospective, meaning it looks back at your medical history. You wouldn't need to do anything new. Researchers will review your existing medical records from when you were treated for an aTTP episode. This means there are no special visits, extra tests, or new medications for you to take as part of the study. Your medical information would be collected from your past treatment, and there is no ongoing follow-up required from you. The study simply involves researchers examining information that's already in your medical file.

Potential risks and benefits

Since this is a retrospective study, meaning researchers are looking at past medical records, there are no direct physical risks or benefits to you for participating. You will not receive any new treatments or undergo additional tests. The potential benefit is that the information gathered from your and other patients' experiences could help doctors understand aTTP better and develop improved treatment plans for future patients, potentially leading to better outcomes and fewer complications. You have the right to withdraw your consent for your medical information to be used in the study at any time.

Locations (1)

University Hospital of Cologne
Verified postcode
Cologne, Germany

Common questions

What is acquired Thrombotic Thrombocytopenic Purpura (aTTP)?

It's a rare and serious blood condition where abnormal blood clots form in small blood vessels, which can harm organs like your brain and kidneys.

What is Caplacizumab (Cablivi®)?

It's a medicine used to treat aTTP by helping to stop these harmful blood clots from forming.

Will I have to take new medicine or visits for this study?

No, this study looks at past medical records. You won't need to take any new medicine or attend extra appointments.

Who is running this study?

This study is a 'national, prospective, multi-centre observational study' in Germany, meaning it's conducted across several hospitals in Germany to look at real-world treatment practices.

Why is this study important?

It helps doctors understand how Caplacizumab works in real life and aims to improve how aTTP is treated in the future, which could lead to better patient care.