ClinicalTrials.gov record NCT06441578

A Survey of Recombinant ADAMTS13 in Participants With Congenital Thrombotic Thrombocytopenic Purpura

Record copied from ClinicalTrials.gov: Oct 10, 2026AI summaries generated: Jul 27, 2026

Special Drug Use Surveillance of ADZYNMA Intravenous 1500 (All-Case Investigation)

About this page. This page shows the study's official ClinicalTrials.gov record ↗, with added help to make it easier to understand. We explain medical terms, highlight key details in plain language, and suggest questions you may want to ask the study team.

The record is periodically copied from ClinicalTrials.gov and may lag behind changes. AI summaries are checked by software, not a clinician.Read moreShow less

The official record is written by the study sponsor and published on ClinicalTrials.gov. It may not always reflect the latest changes. Before making any decisions, please confirm important information with the study team and your own healthcare team.

Where you see "AI summary", AI was used to create a plain-language explanation and software checked it against the source. It has not been reviewed by a clinician, so it may contain mistakes or leave out important details.

StatusRecruitingThe study is currently enrolling participants. Only the study team can say whether a particular person can join.

Study typeObservationalA study in which researchers do not assign participants to a treatment or other intervention. They observe or measure what happens, which may include study visits, tests, questionnaires or a review of medical records.

SexAll

LocationJapan

AI summaryWhat this study is about, in plain languageShow ▾Hide ▴

AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.

This prospective observational survey follows people with congenital thrombotic thrombocytopenic purpura (cTTP) who are treated with recombinant ADAMTS13 (ADZYNMA). Participants receive the intravenous drug according to their clinic's usual care; the study records side effects and laboratory measures for up to 18 months. The study sponsor provides instructions for how clinics record data but does not direct individual treatment.

What it is studying. The study is testing the safety of recombinant ADAMTS13 (counting treatment-emergent adverse events) and measuring changes in platelet counts, ADAMTS13 activity and inhibitors, and the occurrence of TTP events during treatment.

What the researchers hope to learn. To collect safety data on recombinant ADAMTS13 and report changes in platelet counts, ADAMTS13 activity and inhibitors, and the number of TTP events during treatment.

What this study is about

The study's own summary, as published on ClinicalTrials.gov. Underlined terms have a short explanation.

This study is a survey in Japan of recombinant ADAMTS13 used to treat or to prevent participants with congenital thrombotic thrombocytopenic purpura (cTTP). The study sponsor will not be involved in how the participants are treated but will provide instructions on how the clinics will record what happens during the study. The main aim of the study is to check for side effects related from recombinant ADAMTS13 and to check if recombinant ADAMTS13 improves or prevents cTTP. During the study, participants with cTTP will take recombinant ADAMTS13 intravenous injection according to their clinic's standard practice. The study doctors will check for side effects from recombinant ADAMTS13 for 18 months.

Interventions being studied

Being studied in this trial does not mean the treatment or procedure is approved or available for this condition.

Drug: Recombinant ADAMTS13

Eligibility

The criteria exactly as listed on ClinicalTrials.gov. Only the study team can say whether they apply to you.

Inclusion criteria

All participants with congenital thrombotic thrombocytopenic purpura (cTTP), treated with recombinant ADAMTS13

Exclusion criteria

None

Study description and what participation involves

The registry record has no detailed description beyond the summary above.

AI summaryWhat participation may involve, in plain languageShow ▾Hide ▴

AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.

Participants receive recombinant ADAMTS13 by intravenous injection as part of their clinic's usual care. Study doctors will monitor and record treatment-emergent side effects and clinical measures (including platelet counts, ADAMTS13 activity and inhibitor levels, and TTP events) for up to 18 months. The sponsor provides instructions on how clinics should record what happens but does not direct individual treatment decisions.

How the study is structured. A prospective observational cohort: people with cTTP who receive recombinant ADAMTS13 as part of routine care are followed and their outcomes are recorded for up to 18 months. No study-assigned treatment or randomization; clinics follow usual practice while collecting safety and lab data per study instructions.

Questions you could ask

These questions are suggestions for a conversation with the study team or your own clinician, not advice.

This is an observational study, so the study itself gives no treatment. What would taking part involve for me, and would it change my usual care?
The listed sites are in Japan. Is any of them reachable for me, and does the study help with travel?
Which of the eligibility criteria would need to be checked with tests before I could join?

More on thrombotic thrombocytopenic purpura

Clinical trials18 trials listed for thrombotic thrombocytopenic purpuraEvery trial the registry lists for thrombotic thrombocytopenic purpura, with recruitment status, phase and sites, updated from ClinicalTrials.gov.See all trials →Research updatesWhat changed in thrombotic thrombocytopenic purpura researchLatest monthly update: September 2026. Includes: Caplacizumab use. New research summarized each month in plain language, with links to every source paper.Read the latest update →

Important dates

Study start: May 30, 2024
Primary completion: Sep 30, 2032 (estimated)
Study completion: Sep 30, 2032 (estimated)
Target enrollment: 40 participants (estimated)
Last updated on registry: Oct 7, 2025
Data verified: Oct 10, 2026

Sponsor and research team

The lead sponsor funds and oversees the trial. A pharmaceutical or biotech company sponsor typically means the study is testing that company's drug or device; a hospital, university, or government agency usually indicates investigator-initiated or publicly funded research.

Lead sponsor: Takeda

Central study contact

If you're interested in participating, the central contact can answer general questions about the study — including eligibility requirements and how to find a recruiting site near you. Actual eligibility is determined by the site's medical team.

Takeda Contact 📞 +1-877-825-3327 ✉ medinfoUS@takeda.com

Participating sites (1)

Takeda selected siteRecruiting
Tokyo · Japan

Terms used on this page · explanations by MedicalUpdates.ai

study sponsor: The organisation or person that initiates the study and has authority over it. The sponsor may be a company, hospital, university, government agency or individual, and is not necessarily the organisation that funds the study.
Inclusion Criteria: The conditions a person must meet to be considered for the study, for example a confirmed diagnosis, an age range or a test result.
Exclusion Criteria: The conditions that rule a person out even if they meet the inclusion criteria, for example other illnesses, certain medicines or pregnancy.

The study record on this page is reproduced from ClinicalTrials.gov, a database of the U.S. National Library of Medicine, where it was submitted by the study sponsor. MedicalUpdates.ai does not own this content, is not affiliated with or endorsed by ClinicalTrials.gov, NIH, or the U.S. Government, and does not verify the record. Plain-language notes, the glossary and the questions above are added by this site; text in the boxes marked "AI summary" was written by an AI model and may contain errors. Registry records change over time. Check the original record for the current version and discuss eligibility or participation with a qualified healthcare professional.

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