cTTP patients treated with recombinant ADAMTS13: what happened after the early access program

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This is an observational, retrospective review of people with congenital ADAMTS13 deficiency treated with rADAMTS13 via an early access program. The study describes disease impact and health outcomes before and after rADAMTS13, and characterizes treatment patterns around its use for prevention or acute episodes. It includes participants of all ages with confirmed severe cTTP who had at least two administrations and provided consent, across multiple European centers. No active intervention is performed as part of the study; data are collected from patient records. The aim is to understand real-world use, safety signals, and potential benefits in this rare condition.

Why it matters

Who can join: the full rules

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.

Where it's running

• SAMODZIELNY PUBLICZNY SZPITAL KLINICZNY NR 1 IM. PROF. STANISLAWA SZYSZKO Slskiego UM w Katowicach
  Zabrze, Poland Recruiting
• Universitaetsklinikum Koeln
  Cologne, North Rhine-Westphalia, Germany Not yet recruiting
• Charite Universitatsmedizin Berlin
  Berlin, Germany Withdrawn
• Uniwersytecki Szpital Dzieciecy w Krakowie - Management Office
  Krakow, Poland Not yet recruiting
• SP ZOZ Szpital Uniwersytecki w Krakowie
  Krakow, Poland Not yet recruiting
• Santa Familia PTG Lodz
  Lodz, Poland Not yet recruiting

Study contacts

• Study coordinator: Takeda Contact
• Email: medinfoUS@takeda.com
• Phone: +1-877-825-3327

Similar studies

Other studies for the same condition or treatment. Recruiting studies are listed first.

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• Registry for patients with Thrombotic Thrombocytopenic Purpura in Turkey

  This study is collecting information from patients with Thrombotic Thrombocytopenic Purpura in Turkey to see how the disease is treated and how it affects their health over time.

  Recruiting Turkish Hematology Association · Ankara · NCT05950750

How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.