Thrombotic Thrombocytopenic PurpuraPhase 3MAYARINCT05468320
Caplacizumab and Immunosuppressive Therapy Without Firstline Therapeutic Plasma Exchange in Adults With Immune-mediated Thrombotic Thrombocytopenic Purpura
A Phase 3 study testing Caplacizumab, Corticosteroids and anti-CD20 antibody in people with Thrombotic Thrombocytopenic Purpura, run by Sanofi at 27 sites in 11 countries.
Trial summary
This is a single group, treatment, Phase 3, open-label, single-arm study to evaluate the efficacy and safety of caplacizumab and immunosuppressive therapy (IST) without firstline therapeutic plasma exchange (TPE) with primary endpoint of remission in male and female participants aged 18 to 80 years with… Read more about this study
- Who is it for?
- Ages 18 to 80 years
- Conditions the study is for
- Thrombotic Thrombocytopenic Purpura
- What is being tested?
- Caplacizumab, Corticosteroids and anti-CD20 antibody
- Type of treatment
- Pill or oral medicine, Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 51 participants
- Where
- 27 sitesin 11 countries
- Study dates
- 21 Nov 2022to 26 Dec 2024
- Phase
- Phase 3
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
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 to 80 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Participants with a clinical diagnosis of iTTP (initial or recurrent), which includes thrombocytopenia, microangiopathic hemolytic anemia (eg, presence of schistocytes in peripheral blood smear) and relatively preserved renal function. The iTTP diagnosis should be confirmed by ADAMTS13 testing within 48 hours (2 days).
- Participants with a clinical diagnosis of iTTP and a French TMA score of 1 or 2.
- A female participant is eligible to participate if she is not pregnant or breastfeeding, and one of the following conditions applies:
- Is a woman of nonchildbearing potential (WONCBP), OR
- Is a woman of childbearing potential (WOCBP) and agrees to use an acceptable contraceptive method during the overall treatment period and for at least 2 months after the last study drug administration.
- Male participants with female partners of childbearing potential must agree to follow the contraceptive guidance as per protocol during the overall treatment period and for at least 2 months after last study drug administration.
Who cannot join
- Platelet count ≥100 x 10^9/L. Serum creatinine level >2.26 mg/dL (200 µmol/L) in case platelet count is >30 x 10^9/L (to exclude possible cases of atypical HUS).
- Known other causes of thrombocytopenia including but not limited to:
- Clinical evidence of enteric infection with E. coli 0157 or related organism.
- Atypical HUS.
- Hematopoietic stem cell, bone marrow or solid organ transplantation-associated thrombotic microangiopathy.
- Known or suspected sepsis.
Show all 17Show fewer
- Diagnosis of disseminated intravascular coagulation. Congenital TTP (known at the time of study entry). Clinically significant active bleeding or known co-morbidities associated with high risk of bleeding (excluding thrombocytopenia).
- Inherited or acquired coagulation disorders. Malignant arterial hypertension. Participants requiring or expected to require invasive procedures immediately (eg, stroke requiring thrombolytic therapy, those who need mechanical ventilation, etc.).
- Those presenting with severe neurological or cardiac disease. Clinical condition other than that associated with TTP, with life expectancy <6 months, such as end-stage malignancy.
- Known chronic treatment with anticoagulants and anti-platelet drugs that cannot be stopped (interrupted) safely, including but not limited to:
- vitamin K antagonists.
- direct-acting oral anticoagulants.
- heparin or low molecular weight heparin (LMWH).
- non-steroidal anti-inflammatory molecules other than acetyl salicylic acid. Participants who were previously enrolled in this clinical study (study EFC16521).
- Participants who received an investigational drug, or device, other than caplacizumab, within 30 days of anticipated IMP administration or 5 half-lives of the previous investigational drug, whichever is longer.
- Positive result on COVID test.
- The above information is not intended to contain all considerations relevant to a potential participation in a clinical trial.
Trial locations and contacts
27 sites in 11 countries.
27 sites in 11 countries
Italy5 sites
Investigational Site Number : 3800003
Avellino, Campania, 83100
Investigational Site Number : 3800006
Genova, 16132
Investigational Site Number : 3800001
Milan, Lombardy, 20122
Investigational Site Number : 3800005
Verona, 37134
Investigational Site Number : 3800004
Vicenza, 36100
United States4 sites
Johns Hopkins University- Site Number : 8400007
Baltimore, Maryland, 21287
University of Alabama- Site Number : 8400011
Birmingham, Alabama, 35233
The Ohio State University Comprehensive Cancer Center - Site Number : 8400001
Columbus, Ohio, 43210
Duke University Medical Center Site Number : 8400022
Durham, North Carolina, 27710-4000
France4 sites
Investigational Site Number : 2500002
Bois-Guillaume, 76230
Investigational Site Number : 2500005
Lille, 59037
Investigational Site Number : 2500003
Paris, 75010
Investigational Site Number : 2500001
Paris, 75012
Germany4 sites
Investigational Site Number : 2760006
Berlin, 10117
Investigational Site Number : 2760002
Cologne, 50937
Investigational Site Number : 2760001
Frankfurt am Main, 60590
Investigational Site Number : 2760004
Hanover, 30625
Czechia2 sites
Investigational Site Number : 2030001
Brno, 62500
Investigational Site Number : 2030003
Prague, 128 00
Japan2 sites
Investigational Site Number : 3920001
Iruma-gun, Saitama, 350-0495
Investigational Site Number : 3920003
Kurashiki-shi, Okayama-ken, 710-8602
United Kingdom2 sites
Investigational Site Number : 8260002
Liverpool, L7 8XP
Investigational Site Number : 8260001
London, London, City of, NW1 2PG
Netherlands1 site
Investigational Site Number : 5280002
Amersfoort, 3818 TZ
Spain1 site
Investigational Site Number : 7240001
Madrid / Madrid, Madrid, Comunidad de, 28007
About this study
Official title: An Open-label, Single-arm, Multicenter Study to Evaluate the Efficacy and Safety of Caplacizumab and Immunosuppressive Therapy Without Firstline Therapeutic Plasma Exchange in Adults With Immune-mediated Thrombotic Thrombocytopenic Purpura
This is a single group, treatment, Phase 3, open-label, single-arm study to evaluate the efficacy and safety of caplacizumab and immunosuppressive therapy (IST) without firstline therapeutic plasma exchange (TPE) with primary endpoint of remission in male and female participants aged 18 to 80 years with immune-mediated thrombotic thrombocytopenic purpura (iTTP).
The anticipated study duration per participant without a recurrence while on therapy is maximum 24 weeks (ie, approximately 1 day for screening + maximum 12 weeks of treatment for the presenting episode + 12 weeks of follow-up). Participants will have daily assessments during hospitalization and weekly visits for assessments during ongoing treatment with caplacizumab and IST. There will be 3 outpatient visits for assessments during the follow-up period. There will be two additional follow-up visits for participants who do not have ADAMTS13 activity levels of ≥50% at the time of caplacizumab discontinuation.
Read the detailed description
The anticipated study duration per participant with the presenting episode therefore is a maximum of about 24 weeks (ie, 1 day of screening + maximum 12 weeks of treatment for the presenting episode + 12 weeks of follow-up).
- Conditions
- Thrombotic Thrombocytopenic Purpura
- Being tested
- Caplacizumab, Corticosteroids, anti-CD20 antibody
- 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.
CaplacizumabDrug
Also called Cablivi®
Lyophilized powder for solution for injection.
CorticosteroidsDrug
Also called Prednisone® /Prednisolone®
Solution for injection or Tablet
anti-CD20 antibodyBiological
Also called rituximab or biosimilar
Solution for injection
| Study group | Role | What they receive |
|---|---|---|
| Caplacizumab & immunosuppressive therapy without 1st-line TPE | Experimental | All participants will receive open label caplacizumab daily and immunosuppressive therapy (corticosteroid +/-anti-CD20 therapy antibody [rituximab or biosimilar]) without first line TPE Receives: Drug: Caplacizumab; Drug: Corticosteroids; Biological: anti-CD20 antibody |
What is being measured
The primary outcome is the main question the study is designed to answer.
Percentage of Participants Who Achieved Remission Without Requirement of Therapeutic Plasma Exchange During Overall Study Period
Remission was defined as sustained clinical response with either no TPE and no anti- von Willebrand factor (vWF) therapy for >=30 days (clinical remission) or with attainment of a disintegrin and metalloproteinase with a thrombospondin type 1 motif13 (ADAMTS13) activity level >=50% (complete ADAMTS13 remission), whichever occurred first. Clinical response was defined as sustained platelet count >=150 × 10^9/liter (L) and lactate dehydrogenase (LDH) <1.5 × upper limit of normal (ULN) and no clinical evidence of new or progressive ischemic organ injury for at least 2 consecutive visits.
Measured: From first dose of study treatment (Day 1) up to end of follow-up (up to approximately 24 weeks)
Secondary outcomes (13)
Percentage of Participants Who Achieved Remission During Overall Study Period
Remission was defined as sustained clinical response with either no TPE and no anti- vWF therapy for >=30 days (clinical remission) or with ADAMTS13 activity level >=50% (complete ADAMTS13 remission), whichever occurred first. Clinical response was defined as sustained platelet count >=150 × 10^9/L and LDH <1.5 × ULN and no clinical evidence of new or progressive ischemic organ injury for at least 2 consecutive visits.
Measured: From first dose of study treatment (Day 1) up to end of follow-up (up to approximately 24 weeks)
Percentage of Participants Who Required Therapeutic Plasma Exchange During On-Treatment Period
TPE was a procedure in which blood of the participant was passed through a medical device which separated out plasma from other components of blood and the participant's plasma was removed and replaced with a replacement solution such as colloid solution (example, albumin and/or plasma) or a combination of crystalloid/colloid solution. TPE replenishes the ADAMTS13 enzyme and removes anti-ADAMTS13 antibodies, and ultra-large von Willebrand factor multimers gradually from the circulation.
Measured: From first dose of study treatment (Day 1) up to last dose of study treatment, approximately 12 weeks
Number of Participants With Treatment-Emergent Adverse Events (TEAEs), Treatment-Emergent Serious Adverse Events (TESAEs), and Treatment-Emergent Adverse Events of Special Interest (TEAESI)
An AE was any untoward medical occurrence in a participant or clinical study participant, temporally associated with the use of study treatment, whether or not considered related to the study treatment. TEAEs were defined as the AEs that developed, worsened or became serious during the treatment-emergent period. SAE: Any untoward medical occurrence that at any dose: resulted in death, was life-threatening, required inpatient hospitalization or prolongation of existing hospitalization, resulted in persistent or significant disability/incapacity, or was a congenital anomaly/birth defect, or any other situation where medical or scientific judgment of investigator were exercised. An AESI is an AE (serious or nonserious) of scientific and medical concern specific to the Sponsor's product or program, for which ongoing monitoring and immediate notification by the Investigator to the Sponsor was required.
Measured: From first dose of study treatment (Day 1) up to last dose of study treatment + 28 days (approximately 16 weeks)
Percentage of Participants Who Achieved Clinical Response During On-Treatment Period
Clinical response was defined as sustained platelet count >=150 × 10^9/L and LDH <1.5 × ULN and no clinical evidence of new or progressive ischemic organ injury for at least 2 consecutive visits.
Measured: From first dose of study treatment (Day 1) up to last dose of study treatment, approximately 12 weeks
Percentage of Participants Who Achieved Clinical Response During Overall Study Period
Clinical response was defined as sustained platelet count >=150 × 10^9/L and LDH <1.5 × ULN and no clinical evidence of new or progressive ischemic organ injury for at least 2 consecutive visits.
Measured: From first dose of study treatment (Day 1) up to end of follow-up (up to approximately 24 weeks)
Time to Platelet Count Response
Time to platelet count response was defined as time from start of study treatment to initial platelet count >=150 × 10^9/L that was sustained for >=2 days.
Measured: From first dose of study treatment (Day 1) up to end of follow-up (up to approximately 24 weeks)
Percentage of Participants Refractory to Therapy During On-Treatment Period
Refractory to therapy was defined as lack of sustained platelet count increment (over 2 consecutive days) or platelet counts <50 × 10^9/L and persistently elevated LDH (>1.5 × ULN) despite 5 days of treatment during the on-treatment period.
Measured: From first dose of study treatment (Day 1) up to last dose of study treatment, approximately 12 weeks
Percentage of Participants With Immune-mediated Thrombotic Thrombocytopenic Purpura (iTTP)-Related Death During On-Treatment Period
Percentage of participants with iTTP-related death during on-treatment period are reported.
Measured: From first dose of study treatment (Day 1) up to last dose of study treatment, approximately 12 weeks
Percentage of Participants With Immune-mediated Thrombotic Thrombocytopenic Purpura-Related Death During Overall Study Period
Percentage of participants with iTTP-related death during overall study period are reported.
Measured: From first dose of study treatment (Day 1) up to end of follow-up (up to approximately 24 weeks)
Percentage of Participants With a Clinical Exacerbation of Immune-Mediated Thrombotic Thrombocytopenic Purpura During On-Treatment Period
Clinical exacerbation was defined as after a clinical response and before a clinical remission, platelet count decreased to <150 × 10^9/L (with other causes of thrombocytopenia excluded), with or without clinical evidence of new or progressive ischemic organ injury, within 30 days of stopping TPE or anti-vWF therapy.
Measured: From first dose of study treatment (Day 1) up to last dose of study treatment, approximately 12 weeks
Percentage of Participants With a Clinical Exacerbation of Immune-Mediated Thrombotic Thrombocytopenic Purpura During Overall Study Period
Clinical exacerbation was defined as after a clinical response and before a clinical remission, platelet count decreased to <150 × 10^9/L (with other causes of thrombocytopenia excluded), with or without clinical evidence of new or progressive ischemic organ injury, within 30 days of stopping TPE or anti-vWF therapy.
Measured: From first dose of study treatment (Day 1) up to end of follow-up (up to approximately 24 weeks)
Percentage of Participants With a Clinical Relapse of Immune-Mediated Thrombotic Thrombocytopenic Purpura During On-Treatment Period
Clinical relapse was defined as after a clinical remission, platelet count decreased to <150 × 10^9/L (with other causes of thrombocytopenia ruled out), with or without clinical evidence of new ischemic organ injury. A clinical relapse had to be confirmed by documentation of severe ADAMTS13 deficiency (<10%).
Measured: From first dose of study treatment (Day 1) up to last dose of study treatment, approximately 12 weeks
Percentage of Participants With a Clinical Relapse of Immune-Mediated Thrombotic Thrombocytopenic Purpura During Overall Study Period
Clinical relapse was defined as after a clinical remission, platelet count decreased to <150 × 10^9/L (with other causes of thrombocytopenia ruled out), with or without clinical evidence of new ischemic organ injury. A clinical relapse had to be confirmed by documentation of severe ADAMTS13 deficiency (<10%).
Measured: From first dose of study treatment (Day 1) up to end of follow-up (up to approximately 24 weeks)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted21 Jul 2022
- Study start21 Nov 2022
- Primary completion26 Dec 2024
- Study completion26 Dec 2024
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Thrombotic Thrombocytopenic PurpuraStudies of the same condition, open and under way.
- More trials run by SanofiThe 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
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 51 participants (actual)
Sponsor and investigators
- Lead sponsor
- Sanofi
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Clinical Sciences & Operations, Study director, Sanofi
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05468320
- Sponsor's protocol ID
- EFC16521
- Other IDs
- U1111-1244-0426, 2024-513262-19, 2022-001177-31
- Acronym
- MAYARI
- Status last verified
- Dec 2025
- First submitted
- 18 Jul 2022
- Last updated
- 30 Dec 2025
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Yes: Qualified researchers may request access to patient level data and related study documents including the clinical study report, study protocol with any amendments, blank case report form, statistical analysis plan, and dataset specifications. Patient level data will be anonymized, and study documents will be redacted to protect the privacy of trial participants. Further details on Sanofi's data sharing criteria, eligible studies, and process for requesting access can be found at: https://vivli.org
Publications
Volker LA, Brinkkotter PT. Tailored treatment of acute immune-mediated thrombotic thrombocytopenic purpura. Hematology Am Soc Hematol Educ Program. 2025 Dec 5;2025(1):614-620. doi: 10.1182/hematology.2025000757.
PubMed 41348038
Study at a glance
From the registry record, last updated by the sponsor on 30 Dec 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Open-label, Single-arm, Multicenter Study to Evaluate the Efficacy and Safety of Caplacizumab and Immunosuppressive Therapy Without Firstline Therapeutic Plasma Exchange in Adults With Immune-mediated Thrombotic Thrombocytopenic Purpura
- ClinicalTrials.gov ID
- NCT05468320
- Acronym / sponsor ID
- MAYARI / EFC16521
- Conditions
- Thrombotic Thrombocytopenic Purpura
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 51 participants (actual)
- Lead sponsor
- SanofiIndustry (pharma, biotech, devices)
- Study start
- 21 Nov 2022 (actual)
- Primary completion
- 26 Dec 2024 (actual)
- Study completion
- 26 Dec 2024 (actual)
- First posted
- 21 Jul 2022 (actual)
- Results
- Posted 30 Dec 2025
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This is a single group, treatment, Phase 3, open-label, single-arm study to evaluate the efficacy and safety of caplacizumab and immunosuppressive therapy (IST) without firstline therapeutic plasma exchange (TPE) with primary endpoint of remission in male and female participants aged 18 to 80 years with immune-mediated thrombotic thrombocytopenic purpura (iTTP).
The anticipated study duration per participant without a recurrence while on therapy is maximum 24 weeks (ie, approximately 1 day for screening + maximum 12 weeks of treatment for the presenting episode + 12 weeks of follow-up). Participants will have daily assessments during hospitalization and weekly visits for assessments during ongoing treatment with caplacizumab and IST. There will be 3 outpatient visits for assessments during the follow-up period. There will be two additional follow-up visits for participants who do not have ADAMTS13 activity levels of ≥50% at the time of caplacizumab discontinuation.
Detailed description
The anticipated study duration per participant with the presenting episode therefore is a maximum of about 24 weeks (ie, 1 day of screening + maximum 12 weeks of treatment for the presenting episode + 12 weeks of follow-up).
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Caplacizumab & immunosuppressive therapy without 1st-line TPE | Experimental | Drug: Caplacizumab, Drug: Corticosteroids, Biological: anti-CD20 antibodyAll participants will receive open label caplacizumab daily and immunosuppressive therapy (corticosteroid +/-anti-CD20 therapy antibody [rituximab or biosimilar]) without first line TPE |
Who may qualify
- Ages
- 18 to 80 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (6 listed)
- Participants with a clinical diagnosis of iTTP (initial or recurrent), which includes thrombocytopenia, microangiopathic hemolytic anemia (eg, presence of schistocytes in peripheral blood smear) and relatively preserved renal function. The iTTP diagnosis should be confirmed by ADAMTS13 testing within 48 hours (2 days).
- Participants with a clinical diagnosis of iTTP and a French TMA score of 1 or 2.
- A female participant is eligible to participate if she is not pregnant or breastfeeding, and one of the following conditions applies:
- Is a woman of nonchildbearing potential (WONCBP), OR
- Is a woman of childbearing potential (WOCBP) and agrees to use an acceptable contraceptive method during the overall treatment period and for at least 2 months after the last study drug administration.
Key exclusion criteria (17 listed)
- Platelet count ≥100 x 10^9/L. Serum creatinine level >2.26 mg/dL (200 µmol/L) in case platelet count is >30 x 10^9/L (to exclude possible cases of atypical HUS).
- Clinical evidence of enteric infection with E. coli 0157 or related organism.
- Atypical HUS.
- Hematopoietic stem cell, bone marrow or solid organ transplantation-associated thrombotic microangiopathy.
- Known or suspected sepsis.
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 |
|---|---|---|
| CaplacizumabAlso: Cablivi®Lyophilized powder for solution for injection. | Drug | Caplacizumab & immunosuppressive therapy without 1st-line TPE |
| CorticosteroidsAlso: Prednisone® /Prednisolone®Solution for injection or Tablet | Drug | Caplacizumab & immunosuppressive therapy without 1st-line TPE |
| anti-CD20 antibodyAlso: rituximab or biosimilarSolution for injection | Biological | Caplacizumab & immunosuppressive therapy without 1st-line TPE |
Primary outcome
Percentage of Participants Who Achieved Remission Without Requirement of Therapeutic Plasma Exchange During Overall Study Period
Time frame: From first dose of study treatment (Day 1) up to end of follow-up (up to approximately 24 weeks)
Remission was defined as sustained clinical response with either no TPE and no anti- von Willebrand factor (vWF) therapy for >=30 days (clinical remission) or with attainment of a disintegrin and metalloproteinase with a thrombospondin type 1 motif13 (ADAMTS13) activity level >=50% (complete ADAMTS13 remission), whichever occurred first. Clinical response was defined as sustained platelet count >=150 × 10^9/liter (L) and lactate dehydrogenase (LDH) <1.5 × upper limit of normal (ULN) and no clinical evidence of new or progressive ischemic organ injury for at least 2 consecutive visits.
13 secondary outcomes
Percentage of Participants Who Achieved Remission During Overall Study Period
Time frame: From first dose of study treatment (Day 1) up to end of follow-up (up to approximately 24 weeks)
Percentage of Participants Who Required Therapeutic Plasma Exchange During On-Treatment Period
Time frame: From first dose of study treatment (Day 1) up to last dose of study treatment, approximately 12 weeks
Number of Participants With Treatment-Emergent Adverse Events (TEAEs), Treatment-Emergent Serious Adverse Events (TESAEs), and Treatment-Emergent Adverse Events of Special Interest (TEAESI)
Time frame: From first dose of study treatment (Day 1) up to last dose of study treatment + 28 days (approximately 16 weeks)
Percentage of Participants Who Achieved Clinical Response During On-Treatment Period
Time frame: From first dose of study treatment (Day 1) up to last dose of study treatment, approximately 12 weeks
Percentage of Participants Who Achieved Clinical Response During Overall Study Period
Time frame: From first dose of study treatment (Day 1) up to end of follow-up (up to approximately 24 weeks)
Time to Platelet Count Response
Time frame: From first dose of study treatment (Day 1) up to end of follow-up (up to approximately 24 weeks)
Percentage of Participants Refractory to Therapy During On-Treatment Period
Time frame: From first dose of study treatment (Day 1) up to last dose of study treatment, approximately 12 weeks
Percentage of Participants With Immune-mediated Thrombotic Thrombocytopenic Purpura (iTTP)-Related Death During On-Treatment Period
Time frame: From first dose of study treatment (Day 1) up to last dose of study treatment, approximately 12 weeks
Percentage of Participants With Immune-mediated Thrombotic Thrombocytopenic Purpura-Related Death During Overall Study Period
Time frame: From first dose of study treatment (Day 1) up to end of follow-up (up to approximately 24 weeks)
Percentage of Participants With a Clinical Exacerbation of Immune-Mediated Thrombotic Thrombocytopenic Purpura During On-Treatment Period
Time frame: From first dose of study treatment (Day 1) up to last dose of study treatment, approximately 12 weeks
Percentage of Participants With a Clinical Exacerbation of Immune-Mediated Thrombotic Thrombocytopenic Purpura During Overall Study Period
Time frame: From first dose of study treatment (Day 1) up to end of follow-up (up to approximately 24 weeks)
Percentage of Participants With a Clinical Relapse of Immune-Mediated Thrombotic Thrombocytopenic Purpura During On-Treatment Period
Time frame: From first dose of study treatment (Day 1) up to last dose of study treatment, approximately 12 weeks
Percentage of Participants With a Clinical Relapse of Immune-Mediated Thrombotic Thrombocytopenic Purpura During Overall Study Period
Time frame: From first dose of study treatment (Day 1) up to end of follow-up (up to approximately 24 weeks)
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 30 Dec 2025.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 41348038Volker LA, Brinkkotter PT. Tailored treatment of acute immune-mediated thrombotic thrombocytopenic purpura. Hematology Am Soc Hematol Educ Program. 2025 Dec 5;2025(1):614-620. doi: 10.1182/hematology.2025000757.
- EFC16521 Plain Language Results SummaryLink provided by the sponsor.
- Individual participant dataSharing planned: Yes. Qualified researchers may request access to patient level data and related study documents including the clinical study report, study protocol with any amendments, blank case report form, statistical analysis plan, and dataset specifications. Patient level data will be anonymized, and study documents will be redacted to protect the privacy of trial participants. Further details on Sanofi's data sharing criteria, eligible studies, and process for requesting access can be found at: https://vivli.org
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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.
Clinical Sciences & Operations study director
Sanofi
27 sites in 11 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Caplacizumab, Corticosteroids and anti-CD20 antibody in people with Thrombotic Thrombocytopenic Purpura, run by Sanofi at 27 sites in 11 countries.
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 6 inclusion and 17 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 30 Dec 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 27 sites in Italy, United States, France, Germany, Czechia and 6 more countries. 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.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 30 Dec 2025). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
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?
Percentage of Participants Who Achieved Remission Without Requirement of Therapeutic Plasma Exchange During Overall Study Period (time frame: From first dose of study treatment (Day 1) up to end of follow-up (up to approximately 24 weeks)). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT05468320, which also lists 1 linked publication.
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 30 Dec 2025.