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

Recruiting

• Conditions: Thrombotic Thrombocytopenic Purpura (TTP)
• Interventions: Drug: Recombinant ADAMTS13

• Registration Number: NCT06441578
• Lead Sponsor: Takeda

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.

Detailed Description

Not available

Basic Information
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Recruitment & Eligibility
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Study & Design
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Trial Locations

Study Timeline

• Study First Submitted: 2024-05-29
• Study First Submitted QC: 2024-05-29
• Study Start: 2024-05-30
• Study First Posted: 2024-06-04
• Status Verified: 2024-07
• Last Update Submitted: 2024-07-01
• Last Update Posted: 2024-07-03
• Primary Completion: 2032-09-30
• Study Completion: 2032-09-30

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 40

Inclusion Criteria

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

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Exclusion Criteria

• None

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Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Arm && Interventions

Group	Intervention	Description
Recombinant ADAMTS13	Recombinant ADAMTS13	Participants will receive recombinant ADAMTS13 intravenous injection.

Primary Outcome Measures

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

Secondary Outcome Measures

Name	Time	Method
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.

Trial Locations

Locations (1)

Takeda selected site; 🇯🇵 Tokyo, Japan