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

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.

Study Overview

• Status: Not yet recruiting
• Conditions: Thrombotic Thrombocytopenic Purpura (TTP)
• Intervention / Treatment: Other: No Intervention

• Study Type: Observational
• Enrollment (Estimated): 50

Contacts and Locations

• Study Contact: Name: Takeda Contact; Phone Number: +1-877-825-3327; Email: medinfoUS@takeda.com

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child; Adult; Older Adult
• Accepts Healthy Volunteers: No

• 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.

Description

Inclusion criteria:

1. Is diagnosed with cTTP.
2. 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.
3. 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:

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

Study Plan

How is the study designed?

Number of groups / cohorts

1

Cohorts and Interventions

Group / Cohort	Intervention / Treatment
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; This is a non-interventional study.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
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 Outcome Measures

Outcome Measure	Measure Description	Time Frame
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.	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.	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.	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.	At birth
Number of Participants With Spontaneous Abortion	Spontaneous abortion is defined as pregnancy loss before 20 weeks of gestation.	Up to 5.5 years
Number of Participants With Stillbirth	Stillbirth is defined as a fetal death after 20 weeks of gestation.	Up to 5.5 years
Number of Participants With Induced Abortion	Number of participants with induced abortion will be reported.	Up to 5.5 years
Number of Participants With Live Birth	Number of participants with live birth will be reported.	Up to 5.5 years
Number of Participants With any Pregnancy Related Complications	Number of participants with any pregnancy related complications will be reported.	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.	At birth
Number of Infants Categorized According to Year of Birth	Number of infants categorized according to year of birth will be reported.	Up to 5.5 years
Number of Infants With Normal Birth Weight	Number of infants with normal birth weight will be reported.	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.	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.	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).	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.	Up to 5.5 years

Collaborators and Investigators

• Sponsor: Takeda
• Investigators: Study Director: Study Director, Takeda

Publications and helpful links

Helpful Links

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• Click here for more information about this trial in easy-to-understand language.

Study record dates

Study Major Dates

• Study Start (Estimated): May 31, 2026
• Primary Completion (Estimated): April 1, 2030
• Study Completion (Estimated): April 1, 2030

Study Registration Dates

• First Submitted: January 13, 2026
• First Submitted That Met QC Criteria: January 13, 2026
• First Posted (Actual): January 20, 2026

Study Record Updates

• Last Update Posted (Actual): May 13, 2026
• Last Update Submitted That Met QC Criteria: May 8, 2026
• Last Verified: May 1, 2026

More Information

Terms related to this study

• Keywords: Drug Therapy
• Additional Relevant MeSH Terms: Cytopenia; Pathologic Processes; Hemorrhage; Skin Manifestations; Hematologic Diseases; Blood Coagulation Disorders; Blood Platelet Disorders; Thrombotic Microangiopathies; Purpura, Thrombocytopenic; Purpura; Thrombocytopenia; Thrombophilia; Pathological Conditions, Signs and Symptoms; Signs and Symptoms; Hemic and Lymphatic Diseases; Purpura, Thrombotic Thrombocytopenic
• Other Study ID Numbers: TAK-755-4007; EUPAS1000000870 (Other Identifier: EU PAS)

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: YES
• IPD Plan Description: 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.
• IPD Sharing 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.
• IPD Sharing Supporting Information Type: STUDY_PROTOCOL; SAP; ICF; CSR

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No