New drug ADZYNMA tracked for safety in rare blood disorder
NCT ID NCT06441578
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This study monitors 40 people in Japan with congenital thrombotic thrombocytopenic purpura (cTTP) who are receiving ADZYNMA (recombinant ADAMTS13) as part of their routine care. Researchers will track side effects and check if the drug improves platelet counts and ADAMTS13 activity over 18 months. The goal is to see how well the drug works in everyday medical practice.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
Active substance
ADZYNMA (recombinant ADAMTS13)
What this could lead to
If successful, this could confirm that ADZYNMA is a safe and effective way to manage congenital TTP and prevent its complications.
What could go wrong
This is a small, observational study in Japan with only 40 participants, so results may not apply to everyone. It is not designed to prove the drug works, only to monitor real-world use.
Disclaimer
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the original study
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Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
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Study contacts
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
Locations
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Takeda selected site
RECRUITINGTokyo, Japan