A Survey of Recombinant ADAMTS13 in Participants With Congenital Thrombotic Thrombocytopenic Purpura

Special Drug Use Surveillance of ADZYNMA Intravenous 1500 (All-Case Investigation)

This study is a survey in Japan of recombinant ADAMTS13 used to treat or to prevent participants with congenital thrombotic thrombocytopenic purpura (cTTP). The study sponsor will not be involved in how the participants are treated but will provide instructions on how the clinics will record what happens during the study.

The main aim of the study is to check for side effects related from recombinant ADAMTS13 and to check if recombinant ADAMTS13 improves or prevents cTTP.

During the study, participants with cTTP will take recombinant ADAMTS13 intravenous injection according to their clinic's standard practice. The study doctors will check for side effects from recombinant ADAMTS13 for 18 months.

Study Overview

• Status: Recruiting
• Conditions: Thrombotic Thrombocytopenic Purpura (TTP)
• Intervention / Treatment: Drug: Recombinant ADAMTS13

• Study Type: Observational
• Enrollment (Estimated): 40

Contacts and Locations

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

Study Locations

• Japan
  • Tokyo, Japan
    • Recruiting
    • Takeda Selected Site

Participation Criteria

Eligibility Criteria

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

• Sampling Method: Probability Sample

Study Population

The population of this survey are all participants who meet the inclusion/exclusion criteria.

Description

Inclusion Criteria:

- All participants with congenital thrombotic thrombocytopenic purpura (cTTP), treated with recombinant ADAMTS13

Exclusion Criteria:

- None

Study Plan

How is the study designed?

Number of groups / cohorts

1

Cohorts and Interventions

Group / Cohort	Intervention / Treatment
Recombinant ADAMTS13; Participants will receive recombinant ADAMTS13 intravenous injection.	Drug: Recombinant ADAMTS13; Recombinant ADAMTS13, Intravenous injection; Other Names: TAK-755; ADZYNMA Intravenous 1500

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
Number of Participants who Experience at Least One Treatment-Emergent Adverse Events (TEAE)	Up to 18 Months

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Percent Change in Observed Platelet Count from Baseline at the End of Treatment	Percent change in platelet count from baseline at the end of treatment will be reported.	Up to 18 Months
Percent Change in Observed ADAMTS13 Activity from Baseline at the End of Treatment	Percent change in ADAMTS13 activity from baseline at the end of treatment will be reported.	Up to 18 Months
Percent Change in Observed ADAMTS13 Inhibitor from Baseline at the End of Treatment	Percent change in ADAMTS13 inhibitor from baseline at the end of treatment will be reported.	Up to 18 Months
Number of Participants with Thrombotic Thrombocytopenic Purpura (TTP) Events on Periodic Replacement Therapy	Number of participants with TTP events on periodic replacement therapy will be reported.	Up to 18 Months

Collaborators and Investigators

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

Publications and helpful links

Helpful Links

• To obtain more information on the study, click here/on this link

Study record dates

Study Major Dates

• Study Start (Actual): May 30, 2024
• Primary Completion (Estimated): September 30, 2032
• Study Completion (Estimated): September 30, 2032

Study Registration Dates

• First Submitted: May 29, 2024
• First Submitted That Met QC Criteria: May 29, 2024
• First Posted (Actual): June 4, 2024

Study Record Updates

• Last Update Posted (Estimated): October 7, 2025
• Last Update Submitted That Met QC Criteria: October 2, 2025
• Last Verified: October 1, 2025

More Information

Terms related to this study

• 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-755-4005; jRCT2031240130 (Registry Identifier: jRCT)

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