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

Thrombotic Thrombocytopenic Purpura (TTP) Clinical Trial

Official title:

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

Clinical Trial Details — Status: Not yet recruiting

Administrative data

• NCT number: NCT06441578
• Other study ID #: TAK-755-4005
• Status: Not yet recruiting
• Start date: May 30, 2024
• Est. completion date: September 30, 2032

Study information

• Verified date: May 2024
• Source: Takeda
• Contact: Takeda Contact
• Phone: +1-877-825-3327
• Email: medinfoUS[@]takeda.com
• Is FDA regulated: No
• Study type: Observational

Clinical Trial 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.

Recruitment information / eligibility

• Status: Not yet recruiting
• Enrollment: 40
• Est. completion date: September 30, 2032
• Est. primary completion date: September 30, 2032
• Accepts healthy volunteers: No
• Gender: All
• Age group: N/A and older

Eligibility

Inclusion Criteria:

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

Exclusion Criteria:

• None

Related Conditions & MeSH terms

• Purpura
• Purpura, Thrombocytopenic
• Purpura, Thrombotic Thrombocytopenic
• Thrombotic Thrombocytopenic Purpura (TTP)

Intervention

Drug:
• Recombinant ADAMTS13
Recombinant ADAMTS13, Intravenous injection

Locations

Country	Name	City	State
n/a

Sponsors (1)

Lead Sponsor	Collaborator
Takeda

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Number of Participants who Experience at Least One Treatment-Emergent Adverse Events (TEAE)		Up to 18 Months
Secondary	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.	Up to 18 Months
Secondary	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.	Up to 18 Months
Secondary	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.	Up to 18 Months
Secondary	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.	Up to 18 Months