Thrombotic Thrombocytopenic Purpura, AcquiredPhase 2CAPLAVIENCT04720261
Efficacy of a Personalized Caplacizumab Regimen Based on ADAMTS13 Activity Monitoring in Adult aTTP
A Phase 2 study testing Caplacizumab in people with Thrombotic Thrombocytopenic Purpura, Acquired, run by University Hospital, Rouen at 24 sites in 2 countries.
Trial summary
The aim of the study is to evaluate the efficacy of a personalized caplacizumab regimen based on ADAMTS13 activity monitoring in adult acquired thrombotic thrombocytopenic purpura (aTTP): This study is a phase II, prospective, multicenter non-inferiority single-arm study. Read more about this study
- Who is it for?
- Ages 18 to 100 years
- Conditions the study is for
- Thrombotic Thrombocytopenic Purpura, Acquired
- What is being tested?
- Caplacizumab
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 58 participants
- Where
- 24 sitesin 2 countries
- Study dates
- 26 Apr 2021to 16 May 2023
- Phase
- Phase 2
- 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 to 100 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Adult patients ≥ 18 years;
- Clinical diagnosis of aTTP based on standard clinical and laboratory criteria (French Score ≥ 2): i.e., thrombotic microangiopathy syndrome with platelet count ≤ 30 G/L and serum creatinine ≤ 200 μmol/L; severe ADAMTS13 deficiency is not a requirement for inclusion of patients with a French score of 2 [31];
- Patient having read and understood the information letter and signed the Informed Consent Form. If the patient is unable to express his consent, the consent will be signed by his representative ((1) the trusted person, or failing that, (2) a family member, or (3) a close relative of the person concerned). In this case, consent to continue the study will subsequently be requested from the patient (article L1122-1-1 of the CSP);
- Patient affiliated with, or beneficiary of a social security (national health insurance) plan;
- For women:
- Women of childbearing potential :
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- Effective contraception according to WHO definition (estrogen-progestin or intrauterine device or tubal ligation) since at least 1 month and;
- Negative blood pregnancy test;
- Women surgically sterile (absence of ovaries and/or uterus);
- Postmenopausal women (non-medically induced amenorrhea for at least 12 months prior to the inclusion visit).
Who cannot join
- Platelet count > 100 G/L;
- Patients with a French score < 2 (a serum creatinine level > 200 μmol/L +/- associated with a platelet count > 30 G/L), in order to exclude possible cases of atypical hemolytic uremic syndrome;
- Known other causes of cytopenias and/or organ failure including but not limited to: uncontrolled cancer, chemotherapy, transplant, drugs, HIV at AIDS stage;
- Pregnant women (positive result from a blood pregnancy test) or patients with an imminent project of pregnancy; breastfeeding women (due to lack of pharmacological data for caplacizumab during pregnancy and breastfeeding);
- Congenital TTP;
- Clinically significant active bleeding or high risk of bleeding (excluding thrombocytopenia);
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- Chronic treatment with anticoagulant that cannot be interrupted safely, including but not limited to: vitamin K antagonists, direct oral anticoagulant, low molecular weight heparin or heparin;
- Malignant hypertension;
- Contra-indication to CABLIVI 10 mg powder and solvent for solution for injection: hypersensitivity to caplacizumab or to any of the excipients;
- Contra-indication to PE treatment;
- Contra-indication to corticosteroid (= ((methyl)prednisone or (methyl)prednisolone)) or excipients;
- Contra-indication to rituximab or excipients and to its premedication;
- Person deprived of liberty by administrative or judicial decision or placed under judicial protection (guardianship or supervision);
- Participation in another drug interventional clinical trial within 30 days prior to inclusion and during the study.
Trial locations and contacts
24 sites in 2 countries.
24 sites in 2 countries
France23 sites
Chu Amiens
Amiens, 80054
Chu Angers
Angers, 49933
Chu Besancon
Besançon, 25030
Chu Bordeaux
Bordeaux, 33404
CHU CAEN
Caen, 14033
Chu Clermont-Ferrand
Clermont-Ferrand, 63003
Chu Lille
Lille, 59037
Chu Limoges
Limoges, 87042
Ch Edouard Herriot
Lyon, 69003
Ap-Hm Hopital de La Conception
Marseille, 13005
Chu Montpellier
Montpellier, 34295
Chu Nancy
Nancy, 54035
Chu Nantes
Nantes, 44093
Ah-Hp Hopital Saint Louis
Paris, 75010
Ap-Hp Hopital de Cochin
Paris, 75014
Ap-Hp Hopital de Necker
Paris, 75015
Ap-Hp Hopital de St Antoine
Paris, 75571
Chu Reims
Reims, 51092
Chu Rennes
Rennes, 35033
Chu Rouen
Rouen, 76031
Reunion Sud
Saint-Paul, 97448
Chu Tours
Tours, 37044
Ch Valenciennes
Valenciennes, 59300
About this study
Official title: Efficacy of a Personalized Caplacizumab Regimen Based on ADAMTS13 Activity Monitoring in Adult Acquired Thrombotic Thrombocytopenic Purpura: A Phase II, Multicenter Non-inferiority Single-arm Study.
The aim of the study is to evaluate the efficacy of a personalized caplacizumab regimen based on ADAMTS13 activity monitoring in adult acquired thrombotic thrombocytopenic purpura (aTTP): This study is a phase II, prospective, multicenter non-inferiority single-arm study.
Read the detailed description
ADAMTS13 activity will be evaluated on day 7 after the end of daily PE and every 7 days until ADAMTS13 activity ≥ 20% is reached. In case of persistent severe ADAMTS13 deficiency (≤ 20%), caplacizumab administration could be extended for a maximum of 58 days after the end of the daily PE period and should be accompanied by an adjusted immunosuppressive therapy as needed. This duration is in accordance with the HERCULES study protocol and its SmPC.
- Being tested
- Caplacizumab
- Medical subject headings
- Thrombotic thrombocytopenic purpura, acquired
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
CaplacizumabDrug
Analyse of ADAMTS13 activity in patients with aTTP treated with Caplacizumab in order to help adapting the treatment with caplacizumab in TTP patients
| Study group | Role | What they receive |
|---|---|---|
| Caplacizumab | Other | All patients in the study are aTTP and needs to be treated by caplacizumab. The duration of this treatment will be evaluated through the ADAMTS 13 activity. Receives: Drug: Caplacizumab |
What is being measured
The primary outcome is the main question the study is designed to answer.
To evaluate the feasibility of a personalized caplacizumab regimen in aTTP based on ADAMTS13 activity monitoring assessed by a composite criteria including mortality, refractoriness and/or exacerbation at 30 days post-PE treatment
composite endpoint defined by the occurrence of at least one the following events during the 30 days post-PE treatment: death, refractoriness or exacerbation.
Measured: 30 days post-PE treatment
Secondary outcomes (12)
Response to treatment (platelet count recovery)
to platelet count recovery (as defined by a platelet count ≥ 150 G/L with a subsequent interruption of daily PE within 5 days)
Measured: 30 days post-PE treatment
Durable remission achievement
Occurrence of durable remission achievement (platelet count ≥ 150 G/L for ≥ 30 consecutive days following PE interruption);
Measured: 90 days post-PE treatment
Mortality at D90 post-PE treatment
Occurrence of death within 90 days post-PE treatment
Measured: 90 days post-PE treatment
Refractoriness at D30 post-PE treatment
Occurrence of refractoriness at D30 post-PE treatment;
Measured: Day 30 post-PE treatment
Exacerbation at D30 post-PE treatment
Occurrence of exacerbations at D30 post-PE treatment
Measured: Day 30 post-PE treatment
Duration of plasma exchange (PE) treatment and the associated plasma volumes
Duration of daily PE with the corresponding plasma volume
Measured: 30 days
Duration of plasma exchange (PE) treatment and the associated plasma
Total number of PE and the corresponding plasma volume during the full study drug treatment period
Measured: 30 days
Occurrence of neurological sequelae treatment
Neurological assessment based on Rankin score
Measured: Day 90 post-PE treatment
Evaluate the Quality of life
Quality of life based on global post-traumatic score (PCL-S SCALE) at baseline, D90 post-PE treatment
Measured: Day 90 post-PE treatment
Evaluate the cost of the strategy
Costs of the patients' management (Direct hospital medical expenses, Suppléments, direct costs of home care, caplacizumab injections, rehospitalizations) of patients treated with the regimen according to the study
Measured: Day 90 post-PE treatment
To perform a safety analysis
Occurrence of AE and SAE during the study
Measured: 90 days post-PE treatment
Occurrence of cognitive sequelae treatment
Cognitive assessment based on MMS score
Measured: Day 90 post-PE treatment
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted22 Jan 2021
- Study start26 Apr 2021
- Primary completion16 May 2023
- Study completion16 May 2023
Why it stopped: Recommendations of the independent oversight committee.
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Thrombotic Thrombocytopenic Purpura, AcquiredStudies of the same condition, open and under way.
- More trials run by University Hospital, RouenThe 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 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Model description
- Experimental, prospective, non-comparative, multicentric national study
- Masking
- None (open label)
- Enrolment
- 58 participants (actual)
Sponsor and investigators
- Lead sponsor
- University Hospital, Rouen
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04720261
- Sponsor's protocol ID
- 2019/0408/HP
- Acronym
- CAPLAVIE
- Status last verified
- Feb 2026
- First submitted
- 12 Jan 2021
- Last updated
- 20 Feb 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 20 Feb 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Efficacy of a Personalized Caplacizumab Regimen Based on ADAMTS13 Activity Monitoring in Adult Acquired Thrombotic Thrombocytopenic Purpura: A Phase II, Multicenter Non-inferiority Single-arm Study.
- ClinicalTrials.gov ID
- NCT04720261
- Acronym / sponsor ID
- CAPLAVIE / 2019/0408/HP
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Recommendations of the independent oversight committee.
- Enrolment
- 58 participants (actual)
- Lead sponsor
- University Hospital, RouenUniversity, hospital or other
- Study start
- 26 Apr 2021 (actual)
- Primary completion
- 16 May 2023 (actual)
- Study completion
- 16 May 2023 (actual)
- First posted
- 22 Jan 2021 (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 aim of the study is to evaluate the efficacy of a personalized caplacizumab regimen based on ADAMTS13 activity monitoring in adult acquired thrombotic thrombocytopenic purpura (aTTP): This study is a phase II, prospective, multicenter non-inferiority single-arm study.
Detailed description
ADAMTS13 activity will be evaluated on day 7 after the end of daily PE and every 7 days until ADAMTS13 activity ≥ 20% is reached. In case of persistent severe ADAMTS13 deficiency (≤ 20%), caplacizumab administration could be extended for a maximum of 58 days after the end of the daily PE period and should be accompanied by an adjusted immunosuppressive therapy as needed. This duration is in accordance with the HERCULES study protocol and its SmPC.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
- Model description
- Experimental, prospective, non-comparative, multicentric national study
| Arm | Type | Receives |
|---|---|---|
| Caplacizumab | Other | Drug: CaplacizumabAll patients in the study are aTTP and needs to be treated by caplacizumab. The duration of this treatment will be evaluated through the ADAMTS 13 activity. |
Who may qualify
- Ages
- 18 to 100 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (10 listed)
- Adult patients ≥ 18 years;
- Clinical diagnosis of aTTP based on standard clinical and laboratory criteria (French Score ≥ 2): i.e., thrombotic microangiopathy syndrome with platelet count ≤ 30 G/L and serum creatinine ≤ 200 μmol/L; severe ADAMTS13 deficiency is not a requirement for inclusion of patients with a French score of 2 [31];
- Patient having read and understood the information letter and signed the Informed Consent Form. If the patient is unable to express his consent, the consent will be signed by his representative ((1) the trusted person, or failing that, (2) a family member, or (3) a close relative of the person concerned). In this case, consent to continue the study will subsequently be requested from the patient (article L1122-1-1 of the CSP);
- Patient affiliated with, or beneficiary of a social security (national health insurance) plan;
- For women:
Key exclusion criteria (14 listed)
- Platelet count > 100 G/L;
- Patients with a French score < 2 (a serum creatinine level > 200 μmol/L +/- associated with a platelet count > 30 G/L), in order to exclude possible cases of atypical hemolytic uremic syndrome;
- Known other causes of cytopenias and/or organ failure including but not limited to: uncontrolled cancer, chemotherapy, transplant, drugs, HIV at AIDS stage;
- Pregnant women (positive result from a blood pregnancy test) or patients with an imminent project of pregnancy; breastfeeding women (due to lack of pharmacological data for caplacizumab during pregnancy and breastfeeding);
- Congenital TTP;
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 |
|---|---|---|
| CaplacizumabAnalyse of ADAMTS13 activity in patients with aTTP treated with Caplacizumab in order to help adapting the treatment with caplacizumab in TTP patients | Drug | Caplacizumab |
Primary outcome
To evaluate the feasibility of a personalized caplacizumab regimen in aTTP based on ADAMTS13 activity monitoring assessed by a composite criteria including mortality, refractoriness and/or exacerbation at 30 days post-PE treatment
Time frame: 30 days post-PE treatment
composite endpoint defined by the occurrence of at least one the following events during the 30 days post-PE treatment: death, refractoriness or exacerbation.
12 secondary outcomes
Response to treatment (platelet count recovery)
Time frame: 30 days post-PE treatment
Durable remission achievement
Time frame: 90 days post-PE treatment
Mortality at D90 post-PE treatment
Time frame: 90 days post-PE treatment
Refractoriness at D30 post-PE treatment
Time frame: Day 30 post-PE treatment
Exacerbation at D30 post-PE treatment
Time frame: Day 30 post-PE treatment
Duration of plasma exchange (PE) treatment and the associated plasma volumes
Time frame: 30 days
Duration of plasma exchange (PE) treatment and the associated plasma
Time frame: 30 days
Occurrence of neurological sequelae treatment
Time frame: Day 90 post-PE treatment
Evaluate the Quality of life
Time frame: Day 90 post-PE treatment
Evaluate the cost of the strategy
Time frame: Day 90 post-PE treatment
To perform a safety analysis
Time frame: 90 days post-PE treatment
Occurrence of cognitive sequelae treatment
Time frame: Day 90 post-PE treatment
Results and safety
The study was stopped early (reason given: Recommendations of the independent oversight committee.). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 20 Feb 2026.
- Individual participant dataSharing planned: No
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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.
The record lists no contact. Use the official record to reach the sponsor.
24 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Caplacizumab in people with Thrombotic Thrombocytopenic Purpura, Acquired, run by University Hospital, Rouen at 24 sites in 2 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 10 inclusion and 14 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: stopped early, as of the sponsor's last update on 20 Feb 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 24 sites in France, Martinique. 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 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study was stopped early (reason given: Recommendations of the independent oversight committee.). Results are not available in the source record.
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?
To evaluate the feasibility of a personalized caplacizumab regimen in aTTP based on ADAMTS13 activity monitoring assessed by a composite criteria including mortality, refractoriness and/or exacerbation at 30 days post-PE treatment (time frame: 30 days post-PE treatment). 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 20 Feb 2026.