Acquired Thrombotic Thrombocytopenic PurpuraPREDICT2020NCT05876221
Platelet Response to Caplacizumab in the Treatment of Acquired Thrombotic Thrombocytopenic Purpura
An observational study of Acquired Thrombotic Thrombocytopenic Purpura, run by University of Cologne at 1 site in Germany.
Trial summary
The interpretation of platelet counts has to be revaluated in the light of caplacizumab. By effectively blocking platelet binding sites on VWF-multimers, the nanobody leads to a rapid normalization of the platelet count within 3 to 4 days. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Acquired Thrombotic Thrombocytopenic Purpura
- What is being tested?
- Cablivi
- How many people
- 223 participants
- Where
- 1 siteGermany
- Study dates
- 30 Oct 2023to 30 Mar 2025
- Phase
- Observational
- Are there results?
- None posted in the registry
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 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Study population: In this trial will be enclosed iTTP patients treated with caplacizumab from: * the German real-world cohort of iTTP patients treated with caplacizumab in the years 2018 to 2020 , and * the respective TITAN and HERCULES trial cohorts.
Who may be able to join
- Confirmed diagnosis of autoimmune thrombotic thrombocytopenic purpura
- Treatment with at least a single dose of caplacizumab, either i.v. or s.c.
- Male or female patients older than 18 years of age
Who cannot join
- Hereditary thrombotic thrombocytopenic purpura
Trial locations and contacts
1 site in 1 country.
About this study
The interpretation of platelet counts has to be revaluated in the light of caplacizumab. By effectively blocking platelet binding sites on VWF-multimers, the nanobody leads to a rapid normalization of the platelet count within 3 to 4 days. Most importantly, caplacizumab uncouples platelet counts from ADAMTS13 activity and thereby launches unprecedented thrombocyte dynamics, with potential pitfalls for over- and undertreatment.
A relevant number of patients responds to caplacizumab with a brisk increase in platelet count, followed by a marked dip of platelets (patient on the left). This may mislead treating physicians into re-intensifying therapy, with a respective risk for adverse side-effects and complications. Taken together, these observations call for reliable descriptions and the identification of predictive parameters to predict the platelet response upon administration of caplacizumab in a large patient cohort. Here, PREDICT-2020 is designed as a retrospective study to specifically address the following aspects:
- Identifying and describing clusters of platelet responses to caplacizumab
- Identifying potential pitfalls for treating physicians
- Predicting the individual thrombocyte response
- Correlating platelet responses with individual patient outcome
- Being tested
- Cablivi
- Keywords
- caplacizumab, platelet responses, aTTP, iTTP
- 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.
CabliviDrug
Patients with immune Thrombotic Thrombocytopenic Purpura, who have been treated with caplacizumab (Cablivi®)
| Study group | Role | What they receive |
|---|---|---|
| aTTP-Patients | Patients with immune Thrombotic Thrombocytopenic Purpura, who have been treated with caplacizumab (Cablivi®) Receives: Drug: Cablivi |
What is being measured
The primary outcome is the main question the study is designed to answer.
Reliable description and prediction of platelet responses to caplacizumab
Reliable description and prediction of platelet responses to caplacizumab employing mathematic modelling algorithms
Measured: Enrollment
Secondary outcomes (3)
Determination of different clusters of platelet responses to caplacizumab
It will be hypothesized the existence of different clusters of caplacizumab responders during the first weeks of therapy, when ADAMTS13 activity typically is still <10%. A detailed cluster analysis and description of thrombocyte responses to caplacizumab in a large cohort will reliably identify these different responders
Measured: Enrollment
Correlation of platelet responses to caplacizumab with patient outcome
It will be hypothesized the existence of different clusters of caplacizumab responders during the first weeks of therapy, when ADAMTS13 activity typically is still <10%. A detailed cluster analysis and description of thrombocyte responses to caplacizumab in a large cohort will reliably identify these different responders
Measured: Enrollment
Risk stratification of iTTP patients based on their platelet response to caplacizumab
Description: Predicting the individual thrombocyte response to caplacizumab improves risk stratification of iTTP patients after initiation of caplacizumab therapy. An early risk stratification allows an optimal timing of monitoring intervals during the first weeks after diagnosis, which are often critical
Measured: Enrollment
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted25 May 2023
- Study start30 Oct 2023
- Primary completion30 Mar 2025
- Study completion30 Mar 2025
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Acquired Thrombotic Thrombocytopenic PurpuraStudies of the same condition, open and under way.
- 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
- 223 participants (actual)
Sponsor and investigators
- Lead sponsor
- University of Cologne
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Prof. Dr. Paul Brinkkoetter, University of Cologne
- Investigators
- Lucas Kühne, MD, Principal investigator, Department II of Internal Medicine, University Hospital of Cologne
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05876221
- Sponsor's protocol ID
- 2020-11-17
- Acronym
- PREDICT2020
- Status last verified
- Sep 2025
- First submitted
- 16 May 2023
- Last updated
- 12 Sep 2025
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 12 Sep 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Platelet Response to Caplacizumab in the Treatment of Acquired Thrombotic Thrombocytopenic Purpura
- ClinicalTrials.gov ID
- NCT05876221
- Acronym / sponsor ID
- PREDICT2020 / 2020-11-17
- Keywords
- caplacizumab, platelet responses, aTTP, iTTP
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 223 participants (actual)
- Lead sponsor
- University of CologneUniversity, hospital or other
- Study start
- 30 Oct 2023 (actual)
- Primary completion
- 30 Mar 2025 (actual)
- Study completion
- 30 Mar 2025 (actual)
- First posted
- 25 May 2023 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The interpretation of platelet counts has to be revaluated in the light of caplacizumab. By effectively blocking platelet binding sites on VWF-multimers, the nanobody leads to a rapid normalization of the platelet count within 3 to 4 days. Most importantly, caplacizumab uncouples platelet counts from ADAMTS13 activity and thereby launches unprecedented thrombocyte dynamics, with potential pitfalls for over- and undertreatment.
A relevant number of patients responds to caplacizumab with a brisk increase in platelet count, followed by a marked dip of platelets (patient on the left). This may mislead treating physicians into re-intensifying therapy, with a respective risk for adverse side-effects and complications. Taken together, these observations call for reliable descriptions and the identification of predictive parameters to predict the platelet response upon administration of caplacizumab in a large patient cohort. Here, PREDICT-2020 is designed as a retrospective study to specifically address the following aspects:
- Identifying and describing clusters of platelet responses to caplacizumab
- Identifying potential pitfalls for treating physicians
- Predicting the individual thrombocyte response
- Correlating platelet responses with individual patient outcome
Study design
- Observational model
- Cohort
- Time perspective
- Retrospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| aTTP-Patients | – | Drug: CabliviPatients with immune Thrombotic Thrombocytopenic Purpura, who have been treated with caplacizumab (Cablivi®) |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
- Study population
- In this trial will be enclosed iTTP patients treated with caplacizumab from: * the German real-world cohort of iTTP patients treated with caplacizumab in the years 2018 to 2020 , and * the respective TITAN and HERCULES trial cohorts.
Key inclusion criteria (3 listed)
- Confirmed diagnosis of autoimmune thrombotic thrombocytopenic purpura
- Treatment with at least a single dose of caplacizumab, either i.v. or s.c.
- Male or female patients older than 18 years of age
Key exclusion criteria (1 listed)
- Hereditary thrombotic thrombocytopenic purpura
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| CabliviPatients with immune Thrombotic Thrombocytopenic Purpura, who have been treated with caplacizumab (Cablivi®) | Drug | aTTP-Patients |
Primary outcome
Reliable description and prediction of platelet responses to caplacizumab
Time frame: Enrollment
Reliable description and prediction of platelet responses to caplacizumab employing mathematic modelling algorithms
3 secondary outcomes
Determination of different clusters of platelet responses to caplacizumab
Time frame: Enrollment
Correlation of platelet responses to caplacizumab with patient outcome
Time frame: Enrollment
Risk stratification of iTTP patients based on their platelet response to caplacizumab
Time frame: Enrollment
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 12 Sep 2025.
Related trials for Acquired Thrombotic Thrombocytopenic Purpura
Enrolling by invitationObservational study
Comparative Autoantibody and Immunologic Cell Marker Study
Autoimmune Diseases, Lupus Erythematosus, Systemic
Active, not recruitingObservational study
Retrospective Analysis of the Efficiency of Caplacizumab in the Treatment of aTTP
Acquired Thrombotic Thrombocytopenic Purpura
RecruitingObservational study
Daratumumab in Immune-mediated Thrombotic Thrombocytopenic Purpura
Thrombotic Thrombocytopenic Purpura, Acquired
Recruiting
Association of TNFAIP3 With Immune-mediated TTP
Thrombotic Thrombocytopenic Purpura, Acquired
Active, not recruitingObservational study
Improvement of Immunologic and Molecular Techniques for the Diagnosis and Follow-up of Patients With Thrombotic Thrombocytopenic Purpura
Acquired Thrombotic Thrombocytopenic Purpura
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Lucas Kühne, MD principal investigator
Department II of Internal Medicine, University Hospital of Cologne
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Acquired Thrombotic Thrombocytopenic Purpura, run by University of Cologne at 1 site in Germany.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 3 inclusion and 1 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 12 Sep 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Germany. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Reliable description and prediction of platelet responses to caplacizumab (time frame: Enrollment). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 12 Sep 2025.