ClinicalTrials.gov record NCT07429942

A Study to Learn More About the Treatment of People With Congenital Thrombotic Thrombocytopenic Purpura (cTTP) Who Received Recombinant ADAMTS13 (rADAMTS13) as Part of the Early Access Program

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

Treatment and Management of Patients With Congenital Thrombotic Thrombocytopenic Purpura (cTTP): An International, Multi-center Retrospective Chart Review of Patients in the Early Access Program (EAP) Treated With Recombinant ADAMTS13 (rADAMTS13)

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.

StatusNot yet recruitingThe study has been registered but has not started enrolling. Sites and contacts may not be listed yet.

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

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 is a retrospective chart review of people with congenital TTP who received recombinant ADAMTS13 (rADAMTS13) through an early access program. The study collects existing medical-record data to describe health outcomes, treatment use, laboratory measures, pregnancies, and hospitalizations before and during rADAMTS13 treatment. No new treatments or procedures are given for the study.

What it is studying. The study is trying to describe the clinical picture and care of people with cTTP before and after they received rADAMTS13. It will look at counts of acute and subacute TTP episodes, blood and biochemical measures (for example platelet count, hemolysis markers, LDH, creatinine, proteinuria), ADAMTS13 activity and antibodies, treatment patterns (why and how rADAMTS13 was given, dose and frequency), pregnancies and outcomes, and hospital stays as recorded in medical charts.

What the researchers hope to learn. To describe how cTTP affects people treated with rADAMTS13 and to document treatment patterns, laboratory and biomarker measures, pregnancy outcomes, reasons for starting or stopping treatment, and hospital days. Results aim to improve understanding of real-world use and outcomes after rADAMTS13 in this patient group.

What this study is about

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

Congenital thrombotic thrombocytopenic purpura (cTTP) is a rare blood disorder that some people are born with. It is caused by inherited changes in the ADAMTS13 gene that reduce the body's ability to produce the ADAMTS13 enzyme. ADAMTS13 normally cleaves ultra-large multimers of a protein called von Willebrand factor (VWF). In cTTP, low ADAMTS13 activity allows these ultra-large VWF multimers to build up and promote blood clot formation in small blood vessels. These clots can restrict blood flow to vital organs and lead to serious complications. Recombinant ADAMTS13 (rADAMTS13) is a manufactured form of human ADAMTS13 designed to replace the missing enzyme and restore ADAMTS13 activity. This study aims to describe the impact of cTTP on participants before and after treatment with rADAMTS13. It will also evaluate participants' health outcomes after treatment and describe treatment patterns before and after rADAMTS13, including whether treatment was used to prevent or treat TTP episodes, how often it was given, the amount received, and others. In addition, the study will describe pregnancies and outcomes for the mother and baby before and during treatment with rADAMTS13. Only data already available in the medical records of the people who received rADAMTS13 through Takeda's early access program (EAP) for cTTP will be collected and reviewed in this study.

Interventions being studied

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

Other: No Intervention

Eligibility

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

Inclusion criteria

for the rADAMTS13 EAP are:
Participants of any age can participate who have a confirmed diagnosis of severe congenital ADAMTS13 deficiency or cTTP.
Participants must be on preventative or prophylactic treatment for cTTP or must have had at least one TTP event in the past.
Participants must have no other treatment options available (this includes other clinical studies for cTTP).
The inclusion criteria for this retrospective chart review are:
Pediatric and adult participants (no age restrictions) with cTTP, treated with rADAMTS13 via the EAP, who received at least two administrations of rADAMTS13 and who have provided consent (or the legal guardians) to participate in this retrospective chart review.
As per local regulations, evidence of a personally signed (or signed by a legally acceptable representative) and dated informed consent form/informed assent form (ICF/IAF) indicating that the participant (or their legal guardian) has been informed of all pertinent aspects of the retrospective chart review or an approval to process data without informed consent granted by an institutional review board/independent ethics committee (IRB/IEC)) Participants included in the EAP who were/are transitioned to the commercially available product will have their data abstracted for the duration of their participation in the EAP as well as when they received the commercially available product until the end of chart abstraction.
There are no additional

Exclusion criteria

for this chart review.

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.

This is a retrospective chart review using existing medical records only. Study staff will collect information already in the record — for example treatment dates and doses of rADAMTS13, lab results (platelets, ADAMTS13 activity, LDH, kidney measures), symptoms, hospital days, and pregnancy outcomes before (up to 6 months) and during rADAMTS13 treatment. No new treatments, study procedures, or invasive tests are required by the study. Participation requires prior consent for chart review or an IRB/IEC approval to use records without consent.

How the study is structured. A retrospective observational cohort chart review: researchers will collect and analyze existing medical-record data from participants treated with rADAMTS13 in the Early Access Program. Data cover the period before rADAMTS13 started (up to 6 months per the protocol) and during treatment, with some outcomes followed until the end of chart abstraction; no study-assigned treatments or interventions are given.

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 registry says recruitment has not started. When is it expected to open, and can I register interest now?
No sites are listed on the registry yet. Where will the study take place?
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: Sep 10, 2026 (estimated)
Primary completion: Oct 30, 2026 (estimated)
Study completion: Oct 30, 2026 (estimated)
Target enrollment: 94 participants (estimated)
Last updated on registry: Aug 28, 2026
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

Study locations have not been posted on ClinicalTrials.gov yet. This study is not yet recruiting, so sites may be added when recruitment begins. The central study contact above may be able to tell you about planned sites.

Terms used on this page · explanations by MedicalUpdates.ai

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.
informed consent form: The process of explaining a study, including its risks and what participation involves, so that a person can decide freely whether to take part. Consent is usually documented in writing, and a participant can choose to leave the study later.
assent: A child's or dependent adult's agreement to take part, given alongside the formal consent of a parent or legal representative.
legal guardian: A person who is legally allowed to make decisions, including consent to research, on behalf of someone who cannot consent for themselves.
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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