Thrombotic Thrombocytopenic Purpura (TTP)NCT07353099
A Study in Children and, Adults With Congenital Thrombotic Thrombocytopenic Purpura (cTTP) Treated With Adzynma
An observational study of Thrombotic Thrombocytopenic Purpura (TTP), run by Takeda at 7 sites in 2 countries.
Trial summary
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. Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Thrombotic Thrombocytopenic Purpura (TTP)
- How many people
- 50 participants
- Where
- 7 sitesin 2 countries
- Study dates
- 26 May 2026to 1 Apr 2030
- Phase
- Observational
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Study population: 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.
Who may be able to join
- 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.
Who cannot join
- 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.
Trial locations and contacts
7 sites in 2 countries. Contact the study team or a site near you to ask about taking part.
7 sites in 2 countries
Germany6 sites, 6 recruiting
- Recruiting
Universitatsklinikum Bonn
Bonn, 53127
Site Contact, heiko.ruehl@ukbonn.de
Ruehl Heiko
- Not yet recruiting
Universitaetsklinikum Koeln
Cologne, 50937
Site Contact, lucas.kuehne@uk-koeln.de
Kuehne Lucas
- Not yet recruiting
Universitaetsklinikum Jena
Jena, 7743
Site Contact, karim.kentouche@med.uni-jena.de
Kentouche Karim
- Recruiting
Universitaetsklinikum Leipzig
Leipzig, 4103
Site Contact, maria.weise@medizin.uni-leipzig.de
Weise Maria
- Not yet recruiting
Klinikum Rechts der Isar der Technischen Universitaet Muenchen
München, 81675
Site Contact, matthiaschristoph.braunisch@mri.tum.de
Braunisch Matthias
- Not yet recruiting
LMU Klinikum
München
Site Contact, ulf.schoenermarck@med.uni-muenchen.de
Schoenermarck Ulf
Austria1 site, 1 recruiting
- Recruiting
AKH - Medizinische Universitaet Wien
Vienna, 1090
Site Contact, johannes.thaler@meduniwien.ac.at
Johannes Thaler
About this study
Official title: A Post-Authorization Safety Study (PASS) to Further Evaluate Real-World Safety in Patients With Congenital Thrombotic Thrombocytopenic Purpura (cTTP) Treated With Adzynma
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.
- Being tested
- No Intervention
- Keywords
- Drug Therapy
- Medical subject headings
- Purpura, Thrombotic Thrombocytopenic
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
No InterventionOther
This is a non-interventional study.
| Study group | Role | What they receive |
|---|---|---|
| All Participants | Data of participants who have received Adzynma for the treatment of cTTP will be collected retrospectively from medical records for approximately 5 years. Receives: Other: No Intervention |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Participants With Hypersensitivity Reactions Following the Index Adzynma Infusions
Index infusion is defined as the first Adzynma infusion date recorded in the medical record using commercially available Adzynma (excluding doses received in clinical studies or early access program).
Measured: Up to 6 months following the index Adzynma infusion
Number of Participants With Neutralizing Antibodies to ADAMTS13 Following the Index Adzynma Infusions
Number of participants with neutralizing antibodies to ADAMTS13 following the index Adzynma infusions will be reported.
Measured: Up to 6 months following the index Adzynma infusion
Secondary outcomes (16)
Number of Participants With Long Term Safety Risk of Hypersensitivity Reactions Following the Index Adzynma Infusion
Number of participants with long term safety risk of hypersensitivity reactions following the index Adzynma infusion will be reported.
Measured: Up to 6 months of follow-up time from the index Adzynma infusion
Number of Participants With Risk of Hypersensitivity Reactions After Each Administration of Adzynma Following the Index Infusion
Number of participants with risk of hypersensitivity reactions after each administration of Adzynma following the index infusion will be reported.
Measured: Up to 7 days after each administration of Adzynma
Number of Participants With Treatment-emergent Adverse Events (TEAEs)
TEAE is defined as any event emerging or manifesting at or after the initiation of treatment with a study intervention or medicinal product or any existing event that worsens in either intensity or frequency following exposure to the study intervention or medicinal product.
Measured: From start of the study drug administration up to 5.5 years
Gestational age at the Time of Infant Birth
Gestational age at the time of infant birth will be reported.
Measured: At birth
Number of Participants With Spontaneous Abortion
Spontaneous abortion is defined as pregnancy loss before 20 weeks of gestation.
Measured: Up to 5.5 years
Number of Participants With Stillbirth
Stillbirth is defined as a fetal death after 20 weeks of gestation.
Measured: Up to 5.5 years
Number of Participants With Induced Abortion
Number of participants with induced abortion will be reported.
Measured: Up to 5.5 years
Number of Participants With Live Birth
Number of participants with live birth will be reported.
Measured: Up to 5.5 years
Number of Participants With any Pregnancy Related Complications
Number of participants with any pregnancy related complications will be reported.
Measured: Up to 5.5 years
Number of Participants Categorized According to Gestational age of Greater Than or Equal to (>=) 37 Gestational Weeks and Less Than (<) 37 Gestational Weeks at Birth
Number of participants categorized according to gestational age of greater than or equal to (>=) 37 gestational weeks and less than (<) 37 gestational weeks at birth will be reported.
Measured: At birth
Number of Infants Categorized According to Year of Birth
Number of infants categorized according to year of birth will be reported.
Measured: Up to 5.5 years
Number of Infants With Normal Birth Weight
Number of infants with normal birth weight will be reported.
Measured: Up to 5.5 years
Number of Infants With Small for Gestational Age (SGA)
Small for gestational age is defined as birth size (weight, length or head circumference) less than the 10th centile for sex and gestational age.
Measured: Up to 5.5 years
Number of Infants With Congenital Anomaly Detected at the Time of Birth
Number of infants with congenital anomaly detected at the time of birth will be reported.
Measured: At birth
Number of Participants by Breastfeeding Status While Receiving Adzynma
Breastfeeding status during Adzynma treatment will be assessed and categorized based on response (Yes, No, or Unknown).
Measured: Up to 5.5 years
Number of Infant Categorized by Growth and Development Outcomes
Number of infants categorized by growth and development outcomes (normal, abnormal [specify], or unknown) will be reported.
Measured: Up to 5.5 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted20 Jan 2026
- Study start26 May 2026
- Primary completion1 Apr 2030 (estimated)
- Study completion1 Apr 2030 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Thrombotic Thrombocytopenic Purpura (TTP)Studies of the same condition, open and under way.
- More trials run by TakedaThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Retrospective
- Enrolment
- 50 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Takeda
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Study Director, Study director, Takeda
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07353099
- Sponsor's protocol ID
- TAK-755-4007
- Other IDs
- EUPAS1000000870
- Status last verified
- Oct 2026
- First submitted
- 13 Jan 2026
- Last updated
- 2 Oct 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Yes: Takeda provides access to the de-identified individual participant data (IPD) for eligible studies to aid qualified researchers in addressing legitimate scientific objectives (Takeda's data sharing commitment is available on https://clinicaltrials.takeda.com/takedas-commitment?commitment=5). These IPDs will be provided in a secure research environment following approval of a data sharing request, and under the terms of a data sharing agreement.