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
| NCT ID | NCT07429942 |
| Status | Recruiting |
| Phase | — |
| Sponsor | Takeda |
| Condition | Thrombotic Thrombocytopenic Purpura (TTP) |
| Study Type | OBSERVATIONAL |
| Enrollment | 94 participants |
| Start Date | 2026-09-24 |
| Primary Completion | 2026-10-30 |
Eligibility & Interventions
Eligibility Fast-Check
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What to Expect as a Participant
This is an observational study. You will not receive an experimental treatment; researchers will collect data based on your existing condition or standard treatment.
This trial targets 94 participants in total. It began in 2026-09-24 with a primary completion date of 2026-10-30.
⚠ This information is for research awareness only. Always consult your physician before joining any clinical trial. Participation is voluntary and you may withdraw at any time.
Brief Summary
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.
Eligibility Criteria
Inclusion criteria for the rADAMTS13 EAP are: * Participants of any age can participate who have a confirmed diagnosis of severe congenital ADAMTS13 deficiency or cTTP. * Participants must be on preventative or prophylactic treatment for cTTP or must have had at least one TTP event in the past. * Participants must have no other treatment options available (this includes other clinical studies for cTTP). The inclusion criteria for this retrospective chart review are: * Pediatric and adult participants (no age restrictions) with cTTP, treated with rADAMTS13 via the EAP, who received at least two administrations of rADAMTS13 and who have provided consent (or the legal guardians) to participate in this retrospective chart review. * As per local regulations, evidence of a personally signed (or signed by a legally acceptable representative) and dated informed consent form/informed assent form (ICF/IAF) indicating that the participant (or their legal guardian) has been informed of all pertinent aspects of the retrospective chart review or an approval to process data without informed consent granted by an institutional review board/independent ethics committee (IRB/IEC)) Participants included in the EAP who were/are transitioned to the commercially available product will have their data abstracted for the duration of their participation in the EAP as well as when they received the commercially available product until the end of chart abstraction. There are no additional exclusion criteria for this chart review.
Contact & Investigator
Study Director
STUDY DIRECTOR
Takeda
Frequently Asked Questions
Who can join the NCT07429942 clinical trial?
This trial is open to participants of all sexes, studying Thrombotic Thrombocytopenic Purpura (TTP). Full inclusion and exclusion criteria are listed in the Eligibility Criteria section. Always confirm your eligibility with the research team before applying.
Is NCT07429942 currently recruiting?
Yes, NCT07429942 is actively recruiting participants. Contact the research team at medinfoUS@takeda.com for enrollment information.
Where is the NCT07429942 trial being conducted?
This trial is being conducted at Cologne, Germany, Berlin, Germany, Krakow, Poland, Krakow, Poland and 4 additional locations.
Who is sponsoring the NCT07429942 clinical trial?
NCT07429942 is sponsored by Takeda. The principal investigator is Study Director at Takeda. The trial plans to enroll 94 participants.