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NCT07429942

A Study to Learn More About the Treatment of People With Congenital Thrombotic Thrombocytopenic Purpura (cTTP) Who Received Recombinant ADAMTS13 (rADAMTS13) as Part of the Early Access Program

Not yet recruiting Last updated 13 March 2026
What this trial tests

trial testing No Intervention in Thrombotic Thrombocytopenic Purpura (TTP) in 94 participants. Not yet recruiting.

Timeline
7 April 2026
Primary endpoint
1 May 2026
1 May 2026

Quick facts

Lead sponsorTakeda
StatusNot yet recruiting
Study typeOBSERVATIONAL
Enrollment94
Start date7 April 2026
Primary completion1 May 2026
Estimated completion1 May 2026

Drugs / interventions tested

Conditions studied

Sponsor

Takeda — full company profile →

Who can join

Eligibility, any sex, with Thrombotic Thrombocytopenic Purpura (TTP). Patients with the condition only — healthy volunteers not accepted.

Sponsor's own description

Congenital thrombotic thrombocytopenic purpura (cTTP) is a rare blood disorder that some people are born with. It is caused by inherited changes in the ADAMTS13 gene that reduce the body's ability to produce the ADAMTS13 enzyme. ADAMTS13 normally cleaves ultra-large multimers of a protein called von Willebrand factor (VWF). In cTTP, low ADAMTS13 activity allows these ultra-large VWF multimers to build up and promote blood clot formation in small blood vessels. These clots can restrict blood flow to vital organs and lead to serious complications. Recombinant ADAMTS13 (rADAMTS13) is a manufactured form of human ADAMTS13 designed to replace the missing enzyme and restore ADAMTS13 activity. This study aims to describe the impact of cTTP on participants before and after treatment with rADAMTS13. It will also evaluate participants' health outcomes after treatment and describe treatment patterns before and after rADAMTS13, including whether treatment was used to prevent or treat TTP episodes, how often it was given, the amount received, and others. In addition, the study will describe pregnancies and outcomes for the mother and baby before and during treatment with rADAMTS13. Only data already available in the medical records of the people who received rADAMTS13 through Takeda's early access program (EAP) for cTTP will be collected and reviewed in this study.

Publications & conference data

No peer-reviewed publications indexed yet for this trial.

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Other trials of No Intervention

Trials testing the same drug.

Other recruiting trials for Thrombotic Thrombocytopenic Purpura (TTP)

Currently open trials in the same condition.

Other Takeda trials

Trials by the same sponsor.

Verify against primary sources

Data sources for this page

Drug Landscape aggregates and links these public records for informational use only. Always verify against the primary source before clinical or regulatory decisions. Canonical URL: https://druglandscape.com/trial/NCT07429942.

Primary sources · FDA · ClinicalTrials.gov · EMA · SEC EDGAR · ChEMBL · Wikidata · full sourcing