ClinicalTrials.gov record NCT07353099

A Study in Children and, Adults With Congenital Thrombotic Thrombocytopenic Purpura (cTTP) Treated With Adzynma

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

A Post-Authorization Safety Study (PASS) to Further Evaluate Real-World Safety in Patients With Congenital Thrombotic Thrombocytopenic Purpura (cTTP) Treated With Adzynma

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
LocationAustria, Germany
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 observational study that reviews existing medical records of children and people with congenital TTP (cTTP) who received commercially available Adzynma. The study aims to record allergic (hypersensitivity) reactions within about 7 days after each infusion and up to 6 months after the first infusion, the development of neutralizing antibodies within 6 months, longer-term safety over several years, and pregnancy and infant outcomes. Only data already in patients' medical charts will be collected. No new treatments or procedures are given as part of this study.

What it is studying. The study is collecting real-world medical-record data to learn how often people treated with Adzynma develop neutralizing antibodies within 6 months, have allergic (hypersensitivity) reactions shortly after treatment, and to describe longer-term safety and pregnancy/infant outcomes.

What the researchers hope to learn. To describe the frequency of neutralizing antibodies after Adzynma and the timing of allergic reactions; to capture long-term safety events, treatment-emergent adverse events, and pregnancy and infant outcomes among people treated with Adzynma.

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 a problem in a gene passed down from parents to children, which affects the body's ability to produce a enzyme called ADAMTS13. This enzyme helps to cut down a larger form of protein called von Willebrand Factor (VWF). People with cTTP have low levels of ADAMTS13. Without ADAMTS13, large forms of vWF build up and cause blood clots in small blood vessels. These clots can block blood flow to vital organs, causing serious health problems. Adzynma is a human ADAMTS13 protein made in the laboratory. It works the same way as natural ADAMTS13 does and may provide higher levels of ADAMTS13. The main aim of this study is to learn more about the risk of children and adults with cTTP treated with Adzynma developing antibodies that prevent Adzynma from working properly (called neutralizing antibodies) within 6 months after the first treatment and to understand the risk of allergic reactions within 7 days of the first treatment with Adzynma. Other aims are to better understand how safe treatment with Adzynma is over a longer period of time (called long-term safety) in children and adults with cTTP and to gather information about pregnancies and babies of women who have received Adzynma while pregnant. Only data already available in the medical records of the people who received Adzynma for the treatment of cTTP in normal clinical practice will be reviewed and collected during 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

  • Is diagnosed with cTTP.
  • 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.
  • 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

  • Has a history or presence of a functional ADAMTS13 inhibitor (that is, neutralizing antibodies to ADAMTS13/rADAMTS13).
  • Has concurrent use of an investigational drug or is enrolled in another clinical trial at the time of index Adzynma infusion.

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 allow the study team to review and collect information already in their medical records about their Adzynma treatment and health outcomes. No study drug or extra medical procedures are given by the study; this is a retrospective, non‑interventional review. The record review will cover safety outcomes (for example hypersensitivity reactions, neutralizing antibody results, treatment‑emergent adverse events) and pregnancy/infant outcomes when present, for about five years of available records. Participants must sign an informed consent form (or assent and consent where applicable).

How the study is structured. A retrospective, observational cohort study: researchers will review and record information already present in medical records for people who received commercially available Adzynma for cTTP. Data collection covers approximately 5 years of records and includes adverse events, antibody test results if recorded, and pregnancy/baby outcomes. There is no study treatment or additional testing performed by the study team.

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 Austria, Germany. 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 26, 2026
Primary completion
Apr 1, 2030 (estimated)
Study completion
Apr 1, 2030 (estimated)
Target enrollment
50 participants (estimated)
Last updated on registry
Oct 2, 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.

Participating sites (7)

Terms used on this page · explanations by MedicalUpdates.ai

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.
investigational drug
A treatment that is being tested and has not been approved for this use. It may be approved for a different condition. Being studied does not mean it works or is available outside the study.
clinical trial
A study in which researchers assign participants to one or more interventions, such as a drug, procedure, device, behavioural programme or other approach, to measure their effects.
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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