Health TrialsbyHealthPanel

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

ConditionThrombotic Thrombocytopenic Purpura (TTP)
Trial statusRecruiting
Trial phaseNot listed
Trial typeObservational
Biological sexAll
AgeNot listed
SponsorTakeda

About this trial

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.

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

None

Disqualifiers

None

Trial population

The population of this survey are all participants who meet the inclusion/exclusion criteria.

Trial design

Design model

Cohort

Time perspective

Prospective

Treatments tested in this trial

  • Recombinant ADAMTS13

    Drug

    Recombinant ADAMTS13, Intravenous injection

Treatment groups

40 Participants
are divided into 1 treatment group
Group A: Recombinant ADAMTS131 intervention

Trial outcomes

Primary outcomes

1

Number of Participants who Experience at Least One Treatment-Emergent Adverse Events (TEAE)

Time frame
Up to 18 Months

Secondary outcomes

1

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.

Time frame
Up to 18 Months
2

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.

Time frame
Up to 18 Months
3

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.

Time frame
Up to 18 Months
4

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.

Time frame
Up to 18 Months

Other outcomes

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

Takeda

Lead sponsor

Source ClinicalTrials.gov
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