A Survey of Recombinant ADAMTS13 in Participants With Congenital Thrombotic Thrombocytopenic Purpura
Special Drug Use Surveillance of ADZYNMA Intravenous 1500 (All-Case Investigation)
2 other identifiers
observational
40
1 country
1
Brief Summary
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.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P25-P50 for all trials
Started May 2024
Longer than P75 for all trials
1 active site
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
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Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
May 29, 2024
CompletedStudy Start
First participant enrolled
May 30, 2024
CompletedFirst Posted
Study publicly available on registry
June 4, 2024
CompletedPrimary Completion
Last participant's last visit for primary outcome
September 30, 2032
ExpectedStudy Completion
Last participant's last visit for all outcomes
September 30, 2032
October 7, 2025
October 1, 2025
8.3 years
May 29, 2024
October 2, 2025
Conditions
Outcome Measures
Primary Outcomes (1)
Number of Participants who Experience at Least One Treatment-Emergent Adverse Events (TEAE)
Up to 18 Months
Secondary Outcomes (4)
Percent Change in Observed Platelet Count from Baseline at the End of Treatment
Up to 18 Months
Percent Change in Observed ADAMTS13 Activity from Baseline at the End of Treatment
Up to 18 Months
Percent Change in Observed ADAMTS13 Inhibitor from Baseline at the End of Treatment
Up to 18 Months
Number of Participants with Thrombotic Thrombocytopenic Purpura (TTP) Events on Periodic Replacement Therapy
Up to 18 Months
Study Arms (1)
Recombinant ADAMTS13
Participants will receive recombinant ADAMTS13 intravenous injection.
Interventions
Recombinant ADAMTS13, Intravenous injection
Eligibility Criteria
The population of this survey are all participants who meet the inclusion/exclusion criteria.
You may qualify if:
- \- All participants with congenital thrombotic thrombocytopenic purpura (cTTP), treated with recombinant ADAMTS13
You may not qualify if:
- \- None
Contact the study team to confirm eligibility.
Sponsors & Collaborators
- Takedalead
Study Sites (1)
Takeda selected site
Tokyo, Japan
Related Links
MeSH Terms
Conditions
Condition Hierarchy (Ancestors)
Study Officials
- STUDY DIRECTOR
Study Director
Takeda
Central Study Contacts
Study Design
- Study Type
- observational
- Observational Model
- COHORT
- Time Perspective
- PROSPECTIVE
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
May 29, 2024
First Posted
June 4, 2024
Study Start
May 30, 2024
Primary Completion (Estimated)
September 30, 2032
Study Completion (Estimated)
September 30, 2032
Last Updated
October 7, 2025
Record last verified: 2025-10
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.
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.