Active not recruitingPHASE3INTERVENTIONAL

A Study of TAK-755 in Participants With Congenital Thrombotic Thrombocytopenic Purpura

This study is researching a medicine called TAK-755 for a condition known as Thrombotic Thrombocytopenic Purpura (TTP), which some people are born with. In TTP, harmful blood clots can form, blocking blood flow to vital organs. The study wants to find out if TAK-755 can either prevent these life-threatening flare-ups or help treat them when they occur. Researchers will also be carefully monitoring for any long-term effects of the medicine. Participants will receive TAK-755 either regularly to prevent flare-ups, or when a flare-up happens. The study includes both adults and children, aiming to gather more information on how TAK-755 works over time.

At a glance

Status

Active not recruiting

Phase

PHASE3

Sponsor

Takeda

Enrolment target

Start

14 Apr 2021

Estimated completion

16 Mar 2027

Thrombotic Thrombocytopenic Purpura (TTP)

What is this study about?

Thrombotic Thrombocytopenic Purpura, often shortened to TTP, is a rare problem where tiny blood clots form in small blood vessels throughout your body. These clots can stop oxygen-rich blood from getting to important organs like your brain, kidneys, and heart, which can cause serious health issues. The clotting process also uses up important blood cells called platelets, which help stop bleeding. This means that people with TTP might also experience bleeding problems, like purple bruises under the skin.

TTP happens because a special protein in the blood, called ADAMTS13, isn't working properly or is missing. ADAMTS13 normally helps control blood clotting. The medicine being studied, TAK-755, aims to replace this missing protein. The main goal of this study is to see how safe TAK-755 is when used for a longer time, and if it can prevent or treat TTP flare-ups, which are also called 'acute TTP events'.

Researchers are also interested in seeing how TAK-755 works for people who have been on previous TTP studies, as well as those new to this treatment. The study is especially focused on people who were born with this type of TTP. By carefully observing participants, doctors hope to learn more about how TAK-755 can help manage this condition and improve their quality of life.

Key takeaways

This study is testing a medicine called TAK-755 for TTP, a rare blood clotting condition.
It aims to prevent or treat TTP flare-ups and monitor the medicine's long-term safety.
Treatment involves regular infusions, possibly at home for some, for up to 3 years.
Both adults and children up to 70 years old may be eligible.
The study helps understand how to better manage TTP.
Your health and safety will be carefully monitored throughout the study.

Who may be eligible?

This study is looking for people aged from newborns up to 70 years old. You might be able to join if you have been diagnosed with a severe lack of the ADAMTS13 protein, which causes TTP. Importantly, you should not be experiencing a severe TTP flare-up when you start the study.

The study is primarily for people who have already completed a previous TAK-755 study. However, some people who are new to TAK-755 treatment might also be able to join. If you are female and could become pregnant, you'll need to have a negative pregnancy test and agree to use effective birth control throughout the study. Males must also use effective contraception.

More detailed checks will be done by the study team to confirm if this study is suitable for you.

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 between 0 and 70 years old?
Do you have a diagnosis of severe congenital ADAMTS-13 deficiency (a specific type of TTP)?
Are you currently NOT having a severe TTP flare-up (meaning your platelet count isn't too low and another marker, LDH, isn't too high)?
If you are female and could become pregnant, are you willing to use effective birth control and have pregnancy tests?
Are you willing and able to follow all study instructions and attend appointments?

Answer every question to see your result.

What does participation involve?

If you take part in this study, you'll receive the study medicine, TAK-755, which is given directly into a vein. There are two main ways the treatment is given:

1. **To prevent flare-ups (prophylactic group):** You'll receive TAK-755 either every week or every other week. You might be able to have this treatment at home with help from a carer or even give it to yourself, if certain conditions are met and it's allowed where you live. This treatment could last for up to about 3 years. You'll need to visit the clinic at least every 12 weeks for check-ups. 2. **To treat flare-ups when they happen (on-demand group):** If you have a TTP flare-up, you'll receive TAK-755 daily until your flare-up gets better. About 4 weeks after the flare-up has improved, you'll have a follow-up visit at the clinic.

The study involves regular check-ups to monitor your health and how the medicine is working. The total study could last around 6 years, but your individual participation length will depend on which group you're in. Around 77 people will participate in total.

Potential risks and benefits

Taking part in any medical study has potential benefits and risks. A potential benefit of this study is that TAK-755 might help prevent TTP flare-ups or effectively treat them when they occur, which could improve your health and quality of life. The study aims to gather more information about how this medicine works over a longer period. As with all medicines, there's a risk of side effects, which the study team will monitor very closely. It's also possible that TAK-755 might not work for everyone. You are free to withdraw from the study at any time, for any reason, without it affecting your usual medical care.

Locations (27)

Some site locations are approximate. We're improving this — please verify with the trial team before travelling.

Childrens Healthcare of Atlanta
Verified postcode
Atlanta, United States
Roswell Park Comprehensive Cancer Center
Verified postcode
Buffalo, United States
Duke University Medical Center
Verified postcode
Durham, United States
Mid Ohio Heart Clinic Inc
Verified postcode
Dublin, United States
University of Oklahoma
Verified postcode
Oklahoma City, United States
AKH - Medizinische Universität Wien
Verified postcode
Vienna, Austria
Beijing Children's Hospital
Verified postcode
Beijing, China
Peking Union Medical College Hospital
Verified postcode
Beijing, China
Tongji Hospital Affiliated to Tongji Medicine University
Verified postcode
Wuhan, China
The First Affiliated Hospital of Soochow University
Verified postcode
Suzhou, China
Institute of Hematology and Hospital of Blood Disease
Verified postcode
Tianjin, China
Hôpital Necker - Enfants Malades
Verified postcode
Paris, France

Common questions

What is TTP?

TTP is a rare condition where tiny blood clots form in small blood vessels, which can cause serious health problems in organs like the brain, kidneys, and heart.

What is TAK-755 and how does it work?

TAK-755 is a medicine that replaces a missing protein called ADAMTS13, which helps control blood clotting. The aim is to prevent or treat TTP flare-ups.

How long will I be in the study?

Depending on your treatment plan, you could be in the study for up to about 3 years for preventing flare-ups, or for shorter periods if you're treating flare-ups when they happen. The overall study is expected to last around 6 years.

Can children join this study?

Yes, children from newborns up to 17 years old, as well as adults up to 70 years old, might be able to participate if they meet the other eligibility criteria.

Where will I receive the treatment?

Treatment is given into a vein. For preventing flare-ups, it might be in a clinic or potentially at home. For treating flare-ups, it will likely be in a clinic setting.