Skip to main content
OpenTrials
Recruiting

NCT Number: NCT07353099

A Study in Children and, Adults 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.

Recruiting

Interested in participating?

Request Info

Key information

Conditions

Thrombotic Thrombocytopenic Purpura (TTP) Blood Coagulation Disorders Blood Platelet Disorders Cytopenia Hematologic Diseases Hemic and Lymphatic Diseases Hemorrhage Pathologic Processes Pathological Conditions, Signs and Symptoms Purpura Purpura, Thrombocytopenic Purpura, Thrombotic Thrombocytopenic Signs and Symptoms Skin Manifestations Thrombocytopenia Thrombophilia Thrombotic Microangiopathies

Sex eligibility

All sexes

Study type

Observational

Primary location

AKH - Medizinische Universitaet Wien, Vienna, Austria

Loading trial locations.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

No intervention

Other

This is a non-interventional study.

Primary outcomes

  1. Number of Participants With Hypersensitivity Reactions Following the Index Adzynma Infusions

    Time frame: Up to 6 months following the index Adzynma infusion

    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).

  2. Number of Participants With Neutralizing Antibodies to ADAMTS13 Following the Index Adzynma Infusions

    Time frame: Up to 6 months following the index Adzynma infusion

    Number of participants with neutralizing antibodies to ADAMTS13 following the index Adzynma infusions will be reported.

Secondary outcomes

  1. Number of Participants With Long Term Safety Risk of Hypersensitivity Reactions Following the Index Adzynma Infusion

    Time frame: Up to 6 months of follow-up time from the index Adzynma infusion

    Number of participants with long term safety risk of hypersensitivity reactions following the index Adzynma infusion will be reported.

  2. Number of Participants With Risk of Hypersensitivity Reactions After Each Administration of Adzynma Following the Index Infusion

    Time frame: Up to 7 days after each administration of Adzynma

    Number of participants with risk of hypersensitivity reactions after each administration of Adzynma following the index infusion will be reported.

  3. Number of Participants With Treatment-emergent Adverse Events (TEAEs)

    Time frame: From start of the study drug administration up to 5.5 years

    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.

  4. Gestational age at the Time of Infant Birth

    Time frame: At birth

    Gestational age at the time of infant birth will be reported.

  5. Number of Participants With Spontaneous Abortion

    Time frame: Up to 5.5 years

    Spontaneous abortion is defined as pregnancy loss before 20 weeks of gestation.

  6. Number of Participants With Stillbirth

    Time frame: Up to 5.5 years

    Stillbirth is defined as a fetal death after 20 weeks of gestation.

  7. Number of Participants With Induced Abortion

    Time frame: Up to 5.5 years

    Number of participants with induced abortion will be reported.

  8. Number of Participants With Live Birth

    Time frame: Up to 5.5 years

    Number of participants with live birth will be reported.

  9. Number of Participants With any Pregnancy Related Complications

    Time frame: Up to 5.5 years

    Number of participants with any pregnancy related complications will be reported.

  10. 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

    Time frame: 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.

  11. Number of Infants Categorized According to Year of Birth

    Time frame: Up to 5.5 years

    Number of infants categorized according to year of birth will be reported.

  12. Number of Infants With Normal Birth Weight

    Time frame: Up to 5.5 years

    Number of infants with normal birth weight will be reported.

  13. Number of Infants With Small for Gestational Age (SGA)

    Time frame: Up to 5.5 years

    Small for gestational age is defined as birth size (weight, length or head circumference) less than the 10th centile for sex and gestational age.

  14. Number of Infants With Congenital Anomaly Detected at the Time of Birth

    Time frame: At birth

    Number of infants with congenital anomaly detected at the time of birth will be reported.

  15. Number of Participants by Breastfeeding Status While Receiving Adzynma

    Time frame: Up to 5.5 years

    Breastfeeding status during Adzynma treatment will be assessed and categorized based on response (Yes, No, or Unknown).

  16. Number of Infant Categorized by Growth and Development Outcomes

    Time frame: Up to 5.5 years

    Number of infants categorized by growth and development outcomes (normal, abnormal [specify], or unknown) will be reported.

Study contacts

Contact information is provided by the study sponsor or research team.

Takeda Contact

CONTACT

[email protected]

+1-877-825-3327

Sponsors and collaborators

Lead sponsor

Takeda

Industry

Registry information

Official study title

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

Important dates

Study start
2026
Primary completion
2030
Study completion
2030
First posted
Jan 20, 2026
Registry last updated
Jun 11, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

Related clinical trials

Published trials that share one or more normalized conditions with this study.