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Recruiting NCT06441578

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

— Observational Thrombotic Thrombocytopenic Purpura (TTP)

For patients and families

In plain language

An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.

What is being studied
The protocol lists: Recombinant ADAMTS13.
Who it may be relevant to
Registry conditions: Thrombotic Thrombocytopenic Purpura (TTP). Basic parameters: No limits · All.
What needs checking
Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
Where it takes place
Japan
Next step
Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →

Unsure about the terms? Read our patient guide →

Official title

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

Overview

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.

Interventions

  • Drug Recombinant ADAMTS13
    Recombinant ADAMTS13, Intravenous injection

Primary outcome measures

  • Number of Participants who Experience at Least One Treatment-Emergent Adverse Events (TEAE) [Time frame: Up to 18 Months]
Secondary outcome measures (4)
  • Percent Change in Observed Platelet Count from Baseline at the End of Treatment [Time frame: Up to 18 Months]
  • Percent Change in Observed ADAMTS13 Activity from Baseline at the End of Treatment [Time frame: Up to 18 Months]
  • Percent Change in Observed ADAMTS13 Inhibitor from Baseline at the End of Treatment [Time frame: Up to 18 Months]
  • Number of Participants with Thrombotic Thrombocytopenic Purpura (TTP) Events on Periodic Replacement Therapy [Time frame: Up to 18 Months]

Eligibility criteria

Inclusion criteria

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

Exclusion criteria

\- None

Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.

Healthy volunteers: No

Study design

Observational model
Cohort

Study locations

Japan · 1 center
  • Takeda selected site — Tokyo

Identifiers

NCT: NCT06441578 · TAK-755-4005 · jRCT2031240130

Primary sources (government registries)

View this study on ClinicalTrials.gov ↗