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 Type
OBSERVATIONAL
Enrollment
40
Recombinant ADAMTS13, Intravenous injection
Takeda selected site
Tokyo, Japan
RECRUITINGNumber of Participants who Experience at Least One Treatment-Emergent Adverse Events (TEAE)
Time frame: Up to 18 Months
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.
Time frame: 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.
Time frame: 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.
Time frame: 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.
Time frame: Up to 18 Months
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