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

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

Status

Recruiting

Phases

Unknown

Study type

Observational

Source

ClinicalTrials.gov

Registry ID

NCT06441578

Enrollment

Registered

2024-06-04

Start date

2024-05-30

Completion date

2032-09-30

Last updated

2025-10-07

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Thrombotic Thrombocytopenic Purpura (TTP)

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.

Interventions

DRUGRecombinant ADAMTS13

Recombinant ADAMTS13, Intravenous injection

Study design

Observational model

COHORT

Time perspective

PROSPECTIVE

Eligibility

Sex/Gender

ALL

Healthy volunteers

Inclusion criteria

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

Exclusion criteria

\- None

Design outcomes

Primary

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

Secondary

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

Countries

Japan

Contacts

Primary ContactTakeda Contact

medinfoUS@takeda.com+1-877-825-3327

Outcome results

None listed

Source: ClinicalTrials.gov · Data processed: Feb 4, 2026