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

JRCT ID: jRCT2031240130

Registered date:30/05/2024

Basic Information

• Recruitment status: Recruiting
• Health condition(s) or Problem(s) studied: Thrombotic Thrombocytopenic Purpura
• Date of first enrollment: 30/05/2024
• Target sample size: 40
• Study type: Observational

Outcome(s)

Primary Outcome

1.Number of Participants who Experience at Least One Treatment-Emergent Adverse Events (TEAE)
Time Frame: Up to 18 Months

Secondary Outcome

1.Percent Change in Observed Platelet Count from Baseline at the End of Treatment
Time Frame: Up to 18 Months
Percent change in platelet count from baseline at the end of treatment will be reported.

2.Percent Change in Observed ADAMTS13 Activity from Baseline at the End of Treatment
Time Frame: Up to 18 Months
Percent change in ADAMTS13 activity from baseline at the end of treatment will be reported.

3.Percent Change in Observed ADAMTS13 Inhibitor from Baseline at the End of Treatment
Time Frame: Up to 18 Months
Percent change in ADAMTS13 inhibitor from baseline at the end of treatment will be reported.

4.Number of Participants with Thrombotic Thrombocytopenic Purpura (TTP) Events on Periodic Replacement Therapy
Time Frame: Up to 18 Months
Number of participants with TTP events on periodic replacement therapy will be reported.

Key inclusion & exclusion criteria

• Age minimum: Not applicable
• Age maximum: Not applicable
• Gender: Both
• Include criteria: All participants with congenital thrombotic thrombocytopenic purpura (cTTP), treated with recombinant ADAMTS13.
• Exclude criteria: Not Applicable

Related Information

• Primary Sponsor: Contact for Clinical Trial Information
• Secondary ID(s): NCT06441578