Special Drug Use Surveillance of ADZYNMA Intravenous 1500 (All-Case Investigation)
Trial Snapshot
- Phase
- Not Applicable
- Status
- Recruiting
- Sponsor
- Enrollment
- 40
- Locations
- 1
- Primary Endpoint
- Number of Participants who Experience at Least One Treatment-Emergent Adverse Events (TEAE)
Study Overview
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.
Study Design
- Study Type
- Observational
- Observational Model
- Cohort
- Time Perspective
- Prospective
Eligibility Criteria
- Sex
- All
- Accepts Healthy Volunteers
- No
Inclusion Criteria
- •- All participants with congenital thrombotic thrombocytopenic purpura (cTTP), treated with recombinant ADAMTS13
Exclusion Criteria
- Not provided
Arms & Interventions
Recombinant ADAMTS13
Participants will receive recombinant ADAMTS13 intravenous injection.
Intervention: Recombinant ADAMTS13 (Drug)
Outcomes
Primary Outcomes
Number of Participants who Experience at Least One Treatment-Emergent Adverse Events (TEAE)
Time Frame: Up to 18 Months
Secondary Outcomes
- 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)