A phase 3b, prospective, open-label, multicenter, single treatment arm, continuation study of the safety and efficacy of TAK-755 (rADAMTS-13, also know as BAX930/SHP655) in the prophylactic and on-demand treatment of subjects with severe congenital thrombotic thrombocytopenic purpura (cTTP; Upshaw-Schulman Syndrome, or hereditary thrombotic thrombocytopenic purpura)
This study is a long-term look at a medicine called TAK-755, also known as rADAMTS-13. It's for people who have a rare, inherited blood clotting disorder called congenital thrombotic thrombocytopenic purpura (cTTP). The main goal is to understand the long-term safety of TAK-755. Researchers also want to see how well it works to prevent TTP episodes in people who take it regularly, as a preventative measure. They will compare new patients to those who have already been in an earlier study. This is a Phase 3 study, meaning it's a key step in testing new treatments to see if they should be approved for wider use.
At a glance
What is this study about?
This study is looking into a medicine called TAK-755 for people with a rare, inherited blood condition called congenital thrombotic thrombocytopenic purpura, or cTTP for short. Because it's passed down through families, it's sometimes called hereditary TTP. People with cTTP have problems with their blood clotting system, which can lead to serious health issues. TAK-755 is designed to help correct this problem.
The main focus of this study is to see how safe TAK-755 is over a longer period. While previous studies might have looked at its immediate effects, this one wants to gather more information about its safety when used for an extended time. Besides safety, the researchers are also very interested in how well TAK-755 can prevent sudden, serious attacks of TTP when people take it regularly, as a preventative treatment.
This is an ongoing study, which means it builds on previous research. It's a 'Phase 3' study, which is an important stage where new medicines are tested in a larger group of people to confirm their safety and how well they work before they can be considered for wider use. By gathering more information, this study aims to help doctors understand if TAK-755 could be a good long-term treatment option for people with cTTP.
Key takeaways
- Targets inherited blood clotting disorder (cTTP).
- Focuses on long-term safety of TAK-755.
- Also assesses how well TAK-755 prevents TTP episodes.
- Open to adults aged 18 and older.
- Is a continuation study; some participants may have been in previous trials.
Who may be eligible?
To be able to join this study, participants need to be at least 18 years old, and there is no upper age limit. The study is open to both men and women.
The main requirement is that you must have a diagnosis of severe congenital thrombotic thrombocytopenic purpura (cTTP), also known as Upshaw-Schulman Syndrome or hereditary thrombotic thrombocytopenic purpura. This means your condition is inherited and can be quite serious.
Some participants might be new to TAK-755, while others may have already taken part in a previous study involving this medicine. The study will look at both groups to get a full picture.
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?
- Do you have a diagnosis of severe congenital thrombotic thrombocytopenic purpura (cTTP) (also known as Upshaw-Schulman Syndrome or hereditary TTP)?
- Are you able to attend regular clinic visits and follow study instructions?
- Have you discussed your interest in this study with your doctor?
What does participation involve?
If you decide to take part in this study, you'll be given the study medicine, TAK-755. You'll take it either regularly to prevent TTP episodes (prophylactic treatment) or when you have symptoms of an episode (on-demand treatment), as decided by the study doctors.
Throughout the study, you'll have regular visits to the clinic. At these visits, doctors and nurses will carry out various tests and checks to monitor your health, how the medicine is affecting you, and if you've had any TTP events. This will include blood tests and physical examinations. The total duration of your participation will depend on the study's design, as it's a continuation study following up on previous research.
Potential risks and benefits
Locations (6)
- —UnverifiedFrance
- —UnverifiedGermany
- —UnverifiedAustria
- —UnverifiedItaly
- —UnverifiedSpain
- —UnverifiedPoland
Common questions
What is congenital TTP?
Congenital TTP is a rare, inherited blood disorder that can cause serious problems with blood clotting and lead to low platelet counts and red blood cell damage.
What is TAK-755?
TAK-755 is a medicine that aims to help people with congenital TTP by replacing a missing protein that helps control blood clotting.
Why is this study being done?
This study is primarily to learn more about the long-term safety of TAK-755 and how well it prevents TTP episodes in people with congenital TTP.
Who can take part in this study?
Adults aged 18 or over, with a diagnosis of severe congenital TTP, can potentially join this study.
Will I have to pay to be in the study?
No, you will not have to pay for the study medicine or the medical tests directly related to the study.
How to find out more
Always speak to your GP or specialist before deciding to take part in a study.
Discussion
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