Thrombotic Thrombocytopenic Purpura (TTP)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
An observational study of Thrombotic Thrombocytopenic Purpura (TTP), run by Takeda at 8 sites in 2 countries.
Trial 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. Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Thrombotic Thrombocytopenic Purpura (TTP)
- How many people
- 94 participants
- Where
- 8 sitesin 2 countries
- Study dates
- 24 Sep 2026to 30 Oct 2026
- Phase
- Observational
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Study population: Adult and pediatric participants treated with rADAMTS13 via the EAP at participating centers will be included in this study.
Who may be able to join
- 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.
Who cannot join
None listed.
Trial locations and contacts
8 sites in 2 countries. Contact the study team or a site near you to ask about taking part.
8 sites in 2 countries
Poland6 sites, 6 recruiting
- Not yet recruiting
Uniwersytecki Szpital Dzieciecy w Krakowie - Management Office
Krakow, 30-663
Site Contact, +48123339220, pietrys_d@usdk.pl
Danuta Pietrys
- Not yet recruiting
SP ZOZ Szpital Uniwersytecki w Krakowie
Krakow, 30-688
Site Contact, +48124003308, jzdziarska@su.krakow.pl
Joanna Zdziarska
- Not yet recruiting
Santa Familia PTG Lodz
Lodz, 90-302
Site Contact, +48604753686, michalwitkowski13@gmail.com
Michal Witkowski
- Not yet recruiting
Department Cardiology Medical University Lodz
Lodz, 93513
Site Contact, +4842 279 25 07, wojciech.mlynarski@umed.lodz.pl
Wojciech Mlynarski
- Not yet recruiting
Instytut Hematologii i Transfuzjologii
Warsaw, 02-776
Site Contact, +48223496158, jwindyga@ihit.waw.pl
Jerzy Windyga
- Recruiting
SAMODZIELNY PUBLICZNY SZPITAL KLINICZNY NR 1 IM. PROF. STANISLAWA SZYSZKO Slskiego UM w Katowicach
Zabrze, 41-800
Site Contact, +48618850500, mszczepanska@sum.edu.pl
Maria Szczepanska
Germany2 sites, 1 recruiting
- Withdrawn
Charite Universitatsmedizin Berlin
Berlin, 13353
- Not yet recruiting
Universitaetsklinikum Koeln
Cologne, North Rhine-Westphalia, 50937
Site Contact, +4922147830966, lucas.kuehne@uk-koeln.de
Lucas Kuehne
About this study
Official title: Treatment and Management of Patients With Congenital Thrombotic Thrombocytopenic Purpura (cTTP): An International, Multi-center Retrospective Chart Review of Patients in the Early Access Program (EAP) Treated With Recombinant ADAMTS13 (rADAMTS13)
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.
Read more
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.
- Being tested
- No Intervention
- Keywords
- Drug Therapy
- Medical subject headings
- Purpura, Thrombotic Thrombocytopenic
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
No InterventionOther
This is a non-interventional study.
| Study group | Role | What they receive |
|---|---|---|
| Participants with cTTP | Data of participants who have received rADAMTS13 for the treatment of cTTP will be collected retrospectively from electronic medical records (eMR) for before (up to 6 months) and after initiation of rADAMTS13 treatment.
Participant enrollment and data collection for this study is expected to be completed by the last quarter of 2026. Receives: Other: No Intervention |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of cTTP Acute and Subacute Episodes Before and During Treatment with rADAMTS13
Acute episodes will be defined as those with clinically overt features of thrombotic microangiopathy, including thrombocytopenia, microangiopathic hemolytic anemia, and/or organ dysfunction, often requiring urgent therapeutic intervention. Subacute or non-overt episodes will be defined as episodes with laboratory evidence of thrombocytopenia or hemolysis without significant clinical symptoms or organ involvements, often detected on routine monitoring.
Measured: Up to 6 months prior to the initiation of rADAMTS13 administration and continuing during rADAMTS13 administration
Changes in Hematological Measures (Platelet Count) Before and During Treatment with rADAMTS13
Changes in hematological measures like platelet count (thrombocytopenia) will be reported.
Measured: Up to 6 months prior to the initiation of rADAMTS13 administration and continuing during rADAMTS13 administration
Changes in Hematological Measures (Microangiopathic Hemolytic Anemia [MAHA]) Before and During Treatment with rADAMTS13
Changes in hematological measures like MAHA will be reported.
Measured: Up to 6 months prior to the initiation of rADAMTS13 administration and continuing during rADAMTS13 administration
Changes in Biochemical Measures (Lactate Dehydrogenase [LDH]) Before and During Treatment with rADAMTS13
Changes in Biochemical measures like LDH will be reported.
Measured: Up to 6 months prior to the initiation of rADAMTS13 administration and continuing during rADAMTS13 administration
Changes in Biochemical Measures (Proteinuria) Before and During Treatment with rADAMTS13
Changes in Biochemical measures like Proteinuria will be reported.
Measured: Up to 6 months prior to the initiation of rADAMTS13 administration and continuing during rADAMTS13 administration
Changes in Biochemical Measures (Serum creatinine) Before and During Treatment with rADAMTS13
Changes in Biochemical measures like serum creatinine will be reported.
Measured: Up to 6 months prior to the initiation of rADAMTS13 administration and continuing during rADAMTS13 administration
Changes in Biomarker Measures Before and During Treatment with rADAMTS13
Changes in biomarker measures like ADAMTS13 activity level, total ADAMTS13 neutralizing/binding antibodies will be reported.
Measured: Up to 6 months prior to the initiation of rADAMTS13 administration and continuing during rADAMTS13 administration
Number of Participants with cTTP Symptoms Before and During Treatment with rADAMTS13
Number of participants with cTTP symptoms like abdominal pain, fatigue/lethargy, fever, bruising/purpura, neurological symptoms/stroke episodes, renal signs, thrombocytopenia, upper respiratory tract infections, headache/migraine, dizziness, diarrhea, nausea will be assessed using participant's electronic medical record (eMR).
Measured: Up to 6 months before initiation of rADAMTS13 administration and continuing during rADAMTS13 administration
Secondary outcomes (13)
Number of Reasons for rADAMTS13 Early Access Request
Number of reasons for early access request will be described.
Measured: Up to 6 months before initiation of rADAMTS13 administration and continuing during rADAMTS13 administration
Number of Treatment Initiation Characteristics at the First Dose of rADAMTS13
Treatment initiation characteristics includes first rADAMTS13 treatment, participants weight at initiation of treatment, starting dose of rADAMTS13, other dose than specified in summary of product characteristics for prophylaxis or acute treatment, starting prophylaxis dose, frequency and duration will be reported.
Measured: From first dose of rADAMTS13 until EAP discontinuation (up to 6 months)
Number of Treatment Changes During rADAMTS13 Treatment
Treatment changes during rADAMTS13 treatment (for each treatment episode) includes treatment purpose, treatment dates, location, doses and duration, any change(s) to dose, dosing frequency or duration, including dates and the reason(s), additional cTTP prophylaxis treatments will be reported.
Measured: From first dose of rADAMTS13 until EAP discontinuation (up to 6 months)
Peak Activity Level of ADAMTS13
Peak ADAMTS13 activity level i.e., highest level post-infusion will be reported.
Measured: Up to 6 months before initiation of rADAMTS13 administration and continuing during rADAMTS13 administration
Trough Activity Level of ADAMTS13
Trough ADAMTS13 activity level i.e., lowest level before next dose will be reported.
Measured: Up to 6 months before initiation of rADAMTS13 administration and continuing during rADAMTS13 administration
Time of Sample Collection Relative to Infusion
Time of sample collection relative to infusion (1-hour pre infusion, 6 hours post infusion etc) will be reported.
Measured: Up to 6 months before initiation of rADAMTS13 administration and continuing during rADAMTS13 administration
Duration of Treatment and EAP Discontinuation With rADAMTS13
Duration of treatment and EAP Discontinuation with rADAMTS13 will be reported.
Measured: Up to end of treatment (up to 18 months)
Number of Reasons for Treatment and EAP Discontinuation Before and During Treatment with rADAMTS13
Number of reasons for treatment and EAP discontinuation before and during treatment with rADAMTS13 will be reported.
Measured: Up to end of treatment (up to 18 months)
Number of Participants Showing Response as per Laboratory Assessments After Treatment with rADAMTS13
Number of participants showing response as per laboratory assessments after treatment with radamts13 will be reported.
Measured: After rADAMTS13 administration
Number of Instances of Pregnancy and its Corresponding Outcome Before and During Treatment with rADAMTS13
Number of instances of pregnancy and its corresponding Outcome, including pregnancies coinciding with exposure to rADAMTS13 will be reported.
Measured: Up to 6 months prior to the initiation of rADAMTS13 administration and continuing during rADAMTS13 administration
Duration of Infusion During Treatment with rADAMTS13
Duration of infusion during treatment with rADAMTS13 will be reported.
Measured: From First dose of rADAMTS13 until EAP discontinuation (Up to 6 months)
Number of Days Spent in Hospital
Number of days spent by participants in hospital will be reported.
Measured: From first dose of rADAMTS13 until EAP discontinuation (Up to 6 months)
Number of Treatment Related Reasons for Hospitalization
Number of treatment related reason for hospitalization will be reported.
Measured: From first dose of rADAMTS13 until EAP discontinuation (Up to 6 months)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted24 Feb 2026
- Study start24 Sep 2026
- Primary completion30 Oct 2026 (estimated)
- Study completion30 Oct 2026 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Thrombotic Thrombocytopenic Purpura (TTP)Studies of the same condition, open and under way.
- More trials run by TakedaThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Retrospective
- Enrolment
- 94 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Takeda
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Study Director, Study director, Takeda
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07429942
- Sponsor's protocol ID
- TAK-755-4008
- Status last verified
- Oct 2026
- First submitted
- 28 Jan 2026
- Last updated
- 20 Oct 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Yes: Takeda provides access to the de-identified individual participant data (IPD) for eligible studies to aid qualified researchers in addressing legitimate scientific objectives (Takeda's data sharing commitment is available on https://clinicaltrials.takeda.com/takedas-commitment?commitment=5). These IPDs will be provided in a secure research environment following approval of a data sharing request, and under the terms of a data sharing agreement.