Expanded Access Program of TAK-755 for Congenital Thrombotic Thrombocytopenic Purpura (cTTP) (NCT05770219) | Clinical Trial Compass
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Expanded Access Program of TAK-755 for Congenital Thrombotic Thrombocytopenic Purpura (cTTP)
Plain-language summary
The expanded access program allows people to gain access to unlicensed treatment on compassionate grounds. TAK-755 also known as rADAMTS13, is a medicine that treats people born with severe congenital or hereditary thrombotic thrombocytopenic purpura (cTTP). This expanded access program enables continued access to those participants who have no other treatment options available for cTTP.
Who can participate
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion criteria
. Participant or legally authorized representative has provided signed informed consent (≥18 years of age) and/or assent form (signed by legal representative if participants is \<18 years of age).
. Participant has a documented diagnosis of severe congenital ADAMTS13 deficiency, defined by:
. Confirmed molecular genetic testing, or
. ADAMTS13 activity and a negative test for ADAMTS13 antibodies.
. Participant is currently on a prophylactic regimen or has a documented history of at least 1 TTP event.
. Standard of Care (SoC) is considered inadequate, or the participant is intolerant to SoC\*.
Exclusion criteria
. Participant is currently eligible to enter into an interventional clinical trial for cTTP.
. There is a suitable alternative commercially available treatment for the treatment of cTTP.
. Participant has been diagnosed with any other thrombotic microangiopathies (TMAs) like disorder (microangiopathic hemolytic anemia), including acquired TTP.
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
. Participant has a known hypersensitivity/allergies/intolerance or contraindications to TAK-755 or its excipients, such as hamster proteins.
. Participant has a medical history or presence of a functional ADAMTS13 inhibitor.
. Participant has a medical history of genetic or acquired immune deficiency that would interfere with the assessment of product immunogenicity, including Participants who are human immunodeficiency virus (HIV)-positive with an absolute cluster of differentiation 4 (CD4) count \<200 per millimeter cube (\<200/mm\^3) or who are receiving chronic immunosuppressive drugs