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A Study in Children and, Adults With Congenital Thrombotic Thrombocytopenic Purpura (cTTP) Treated With Adzynma

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NCT07353099 on ClinicalTrials.gov ↗
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In plain words

What the trial is, and whether it could suit you

Congenital thrombotic thrombocytopenic purpura (cTTP) is a rare blood disorder that some people are born with. It is caused by a problem in a gene passed down from parents to children, which affects the body's ability to produce a enzyme called ADAMTS13. This enzyme helps to cut down a larger form of protein called von Willebrand Factor (VWF). People with cTTP have low levels of ADAMTS13. Without ADAMTS13, large forms of vWF build up and cause blood clots in small blood vessels. These clots can block blood flow to vital organs, causing serious health problems. Adzynma is a human ADAMTS13 protein made in the laboratory. It works the same way as natural ADAMTS13 does and may provide higher levels of ADAMTS13. The main aim of this study is to learn more about the risk of children and adults with cTTP treated with Adzynma developing antibodies that prevent Adzynma from working properly (cal

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📋 Eligibility rules (as published)
Inclusion criteria: 1. Is diagnosed with cTTP. 2. Had received commercially available Adzynma treatment for cTTP within the eligibility period of which there must be a recorded date and dose of at least one Adzynma administration in their medical chart. 3. Provides a signed informed consent form (informed consent form \[ICF\]; or assent and consent forms, if applicable), in accordance with local ethical and institutional requirements. Exclusion criteria: 1. Has a history or presence of a functional ADAMTS13 inhibitor (that is, neutralizing antibodies to ADAMTS13/rADAMTS13). 2. Has concurrent use of an investigational drug or is enrolled in another clinical trial at the time of index Adzynma infusion.
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What it tests

  • No Intervention
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Where

7 sites
AKH - Medizinische Universitaet Wien
Vienna, Austria
Universitatsklinikum Bonn
Bonn, Germany
Universitaetsklinikum Koeln
Cologne, Germany
Universitaetsklinikum Jena
Jena, Germany
Universitaetsklinikum Leipzig
Leipzig, Germany
Klinikum Rechts der Isar der Technischen Universitaet Muenchen
München, Germany
LMU Klinikum
München, Germany
🤝 Taking part is your choice, and you can leave at any time. Only the trial team can confirm you are eligible — always decide together with your own doctor.
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