New drug ADZYNMA tracked for safety in rare blood disorder

NCT ID NCT06441578

Recruiting now Disease control Sponsor: Takeda Source: ClinicalTrials.gov ↗

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.

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Conditions

The condition(s) this trial relates to.

congenital thrombotic thrombocytopenic purpura thrombotic thrombocytopenic purpura

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

THROMBOTIC THROMBOCYTOPENIC PURPURA (TTP)

Contacts and locations

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Study contacts

Contact

Email: •••••@•••••

Locations

Takeda selected site
RECRUITING

Tokyo, Japan

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