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New study tracks safety of adzynma for rare blood clot disorder

NCT ID NCT07353099

Knowledge-focused Sponsor: Takeda Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now This study
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This study looks at the real-world safety of Adzynma, a lab-made protein, in people with congenital thrombotic thrombocytopenic purpura (cTTP), a rare inherited blood disorder. Researchers will review medical records of about 50 children and adults to see how many develop allergic reactions or antibodies that stop the drug from working. The goal is to better understand long-term safety and effects during pregnancy.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance
Adzynma (lab-made ADAMTS13 protein)
What this could lead to
If successful, this study will provide crucial safety data on Adzynma, helping doctors better manage cTTP patients.
What could go wrong
This is an observational study, not a trial testing new effects. It only reviews existing medical records, so it cannot prove new benefits or risks beyond what is already known.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Participants

About 50 people

The number the study aims to enrol. It can still change while the study runs.

Started

May 2026

Expected to finish

Apr 2030

An estimate. End dates often move.

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Who is studied

Pediatric and adult participants who have received Adzynma for the treatment of cTTP, either prophylactically or as on-demand therapy for acute episodes-including those who became pregnant during Adzynma treatment.

Ages

Children (under 18), adults (18 to 64) and older adults (65 and over)

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

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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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)

How to take part

Only the study team decides who joins. These are the ways to reach them.

  1. The study's own enquiry address

    This study publishes an address for enquiries. See it below .

  2. The places running it

    7 sites in 2 countries. The list below names each one and where it is.

  3. The official record

    ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.

    Open the record ↗

  4. A doctor treating you

    A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.

Contacts and locations

Study contacts

  • Contact

    Email: •••••@•••••

Locations

  • AKH - Medizinische Universitaet Wien

    RECRUITING

    Vienna, 1090, Austria

  • Klinikum Rechts der Isar der Technischen Universitaet Muenchen

    NOT_YET_RECRUITING

    München, 81675, Germany

  • LMU Klinikum

    NOT_YET_RECRUITING

    München, Germany

  • Universitaetsklinikum Jena

    NOT_YET_RECRUITING

    Jena, 7743, Germany

  • Universitaetsklinikum Koeln

    NOT_YET_RECRUITING

    Cologne, 50937, Germany

  • Universitaetsklinikum Leipzig

    NOT_YET_RECRUITING

    Leipzig, 4103, Germany

  • Universitatsklinikum Bonn

    NOT_YET_RECRUITING

    Bonn, 53127, Germany

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