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

NCT07353099 | Recruiting

• 2 other identifiers: TAK-755-4007EUPAS1000000870
• Study Type: observational
• Target: 50
• Locations: 2 countries
• Sites: 7

Brief 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. 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.

Conditions

Thrombotic Thrombocytopenic Purpura (TTP)

Keywords

Drug Therapy

Outcome Measures

Primary Outcomes (2)

• 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).

  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.

  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

  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

  Up to 7 days after each administration of Adzynma

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

  From start of the study drug administration up to 5.5 years

• Gestational age at the Time of Infant Birth

  At birth

• Number of Participants With Spontaneous Abortion

  Up to 5.5 years

Study Arms (1)

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.

Other: No Intervention

Interventions

No Intervention OTHER

This is a non-interventional study.

All Participants

Eligibility Criteria

• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

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.

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Takeda: lead

Study Sites (7)

AKH - Medizinische Universitaet Wien

Vienna, 1090, Austria

RECRUITING

Universitatsklinikum Bonn

Bonn, 53127, Germany

NOT YET RECRUITING

Universitaetsklinikum Koeln

Cologne, 50937, Germany

NOT YET RECRUITING

Universitaetsklinikum Jena

Jena, 7743, Germany

NOT YET RECRUITING

Universitaetsklinikum Leipzig

Leipzig, 4103, Germany

NOT YET RECRUITING

Klinikum Rechts der Isar der Technischen Universitaet Muenchen

München, 81675, Germany

NOT YET RECRUITING

LMU Klinikum

München, Germany

NOT YET RECRUITING

Related Links

• Click here for more information about this trial in easy-to-understand language.
• Click here to ask Takeda's chatbot for comprehensive and easy-to-understand information about clinical trials - even across products and indications - in your local language.

MeSH Terms

Conditions

Purpura, Thrombotic Thrombocytopenic

Condition Hierarchy (Ancestors)

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

Study Officials

• Study Director

  Takeda

  STUDY DIRECTOR

Central Study Contacts

Takeda Contact

CONTACT

+1-877-825-3327; medinfoUS@takeda.com

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: RETROSPECTIVE
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: January 13, 2026
• First Posted: January 20, 2026
• Study Start: May 26, 2026
• Primary Completion (Estimated): April 1, 2030
• Study Completion (Estimated): April 1, 2030
• Last Updated: June 11, 2026

Record last verified: 2026-06

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL, SAP, ICF, CSR
• Access Criteria: IPD from eligible studies will be shared with qualified researchers according to the criteria and process described on https://vivli.org/ourmember/takeda/. For approved requests, the researchers will be provided access to anonymized data (to respect patient privacy in line with applicable laws and regulations) and with information necessary to address the research objectives under the terms of a data sharing agreement.

Locations

AU (1); DE (6)