Efficacy of a personalized caplacizumab regimen based on ADAMTS-13 activity monitoring in adult acquired thrombotic thrombocytopenic purpura: A phase II, multicenter non-inferiority single-arm study

Efficacy of a personalized caplacizumab regimen based on ADAMTS-13 activity monitoring in adult acquired thrombotic thrombocytopenic purpura: A phase II, multicenter non-inferiority single-arm study - CAPLAVIE

Status

Not yet recruiting

Phases

Phase 2

Study type

Interventional

Source

EU CTR

Registry ID

EUCTR2020-005288-30-FR

Enrollment

125

Registered

2020-11-06

Start date

2020-12-28

Completion date

Unknown

Last updated

2021-01-05

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

acquired thrombotic thrombocytopenic purpura MedDRA version: 20.0 Level: PT Classification code 10043648 Term: Thrombotic thrombocytopenic purpura System Organ Class: 10005329 - Blood and lymphatic system disorders

Interventions

Trade Name: CABLIVI Pharmaceutical Form: Powder and solvent for solution for injection

Eligibility

Sex/Gender

All

Inclusion criteria

Inclusion criteria: - 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; - 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: o Women of childbearing potential : ? Effective contraception according to WHO definition (estrogen-progestin or intrauterine device or tubal ligation) since at least 1 month and; ? Negative blood pregnancy test; o Women surgically sterile (absence of ovaries and/or uterus); o Postmenopausal women (non-medically induced amenorrhea for at least 12 months prior to the inclusion visit). Are the trial subjects under 18? no Number of subjects for this age range: F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 100 F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range 25

Exclusion criteria

Exclusion criteria: - Platelet count > 100 G/L; - Patients with a French score 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); - 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.

Design outcomes

Primary

Measure	Time frame
Main Objective: To evaluate the feasibility of a personalized caplacizumab regimen in acquired, immune-mediated Thrombotic Thrombocytopenic Purpura (aTTP) based on ADAMTS13 activity monitoring assessed by a composite criteria including mortality, refractoriness and/or exacerbation at 30 days post-Plasma Exchange (PE) treatment. ;Secondary Objective: To evaluate the feasibility of a personalized caplacizumab regimen based on ADAMTS13 activity monitoring on: - Response to treatment (platelet count recovery); - Durable remission achievement; - Mortality at D30 and D90 post-PE treatment; - Refractoriness at D30 post-PE treatment; - Exacerbation at D30 post-PE treatment; - Duration of plasma exchange (PE) treatment and the associated plasma volumes; - occurrence of neuro-cognitive sequelae at 30-day and 90-day post-PE treatment ; - Quality of life; To evaluate the cost of the strategy; To perform a safety analysis. ;Primary end point(s): The primary endpoint is the time up to the occurrence of a composite endpoint of death, refractoriness and/or exacerbation at 30 days post-PE treatment.;Timepoint(s) of evaluation of this end point: 30 days post-PE treatment.	—

Measure

Time frame

Main Objective: To evaluate the feasibility of a personalized caplacizumab regimen in acquired, immune-mediated Thrombotic Thrombocytopenic Purpura (aTTP) based on ADAMTS13 activity monitoring assessed by a composite criteria including mortality, refractoriness and/or exacerbation at 30 days post-Plasma Exchange (PE) treatment. ;Secondary Objective: To evaluate the feasibility of a personalized caplacizumab regimen based on ADAMTS13 activity monitoring on: - Response to treatment (platelet count recovery); - Durable remission achievement; - Mortality at D30 and D90 post-PE treatment; - Refractoriness at D30 post-PE treatment; - Exacerbation at D30 post-PE treatment; - Duration of plasma exchange (PE) treatment and the associated plasma volumes; - occurrence of neuro-cognitive sequelae at 30-day and 90-day post-PE treatment ; - Quality of life; To evaluate the cost of the strategy; To perform a safety analysis. ;Primary end point(s): The primary endpoint is the time up to the occurrence of a composite endpoint of death, refractoriness and/or exacerbation at 30 days post-PE treatment.;Timepoint(s) of evaluation of this end point: 30 days post-PE treatment.

—

Secondary

Measure	Time frame
Secondary end point(s): - Time to platelet count recovery (as defined by a platelet count = 150 G/L with a subsequent interruption of daily PE within 5 days); - Occurrence of durable remission achievement (platelet count = 150 G/L for = 30 consecutive days following PE interruption); - Time to durable remission achievement; - Occurrence of death within 30 days and 90 days post-PE treatment; - Occurrence of refractoriness at D30 post-PE treatment; - Occurrence of exacerbations at D30 post-PE treatment; - Duration of daily PE with the corresponding plasma volume; - Total number of PE and the corresponding plasma volume during the full study drug treatment period; - Cognitive assessment based on MMS score and neurological assessment based on Rankin score at baseline, D30 and D90 post-PE treatment; - Quality of life based on global post-traumatic score at baseline, D30 and D90 post-PE treatment; - Budget impact: cost of the management of patients treated with the regimen according to the study: each medical information department of each center participating in the study will send the information relating to the stay of each patient included in the study (diagnostic group for each patient). Supplements (resuscitation, intensive care, plasma exchange, etc.) and non-reimbursed molecules will be considered if this is the case in addition to hospitalization such as Rituximab front line and daily subcutaneous Caplacizumab; - Occurrence of bleeding events at D30 and D90 post-PE treatment; - Occurrence of AE and SAE during the study.;Timepoint(s) of evaluation of this end point: - Occurrence of death: within 30 days and 90 days post-PE treatment; - Occurrence of refractoriness: at D30 post-PE treatment; - Occurrence of exacerbations: at D30 post-PE treatment; - Cognitive assessment based on MMS score and neurological assessment based on Rankin score : at baseline, D30 and D90 post-PE treatment; - Quality of life based on global post-traumatic score : at baseline, D30 an	—

Measure

Time frame

Secondary end point(s): - Time to platelet count recovery (as defined by a platelet count = 150 G/L with a subsequent interruption of daily PE within 5 days); - Occurrence of durable remission achievement (platelet count = 150 G/L for = 30 consecutive days following PE interruption); - Time to durable remission achievement; - Occurrence of death within 30 days and 90 days post-PE treatment; - Occurrence of refractoriness at D30 post-PE treatment; - Occurrence of exacerbations at D30 post-PE treatment; - Duration of daily PE with the corresponding plasma volume; - Total number of PE and the corresponding plasma volume during the full study drug treatment period; - Cognitive assessment based on MMS score and neurological assessment based on Rankin score at baseline, D30 and D90 post-PE treatment; - Quality of life based on global post-traumatic score at baseline, D30 and D90 post-PE treatment; - Budget impact: cost of the management of patients treated with the regimen according to the study: each medical information department of each center participating in the study will send the information relating to the stay of each patient included in the study (diagnostic group for each patient). Supplements (resuscitation, intensive care, plasma exchange, etc.) and non-reimbursed molecules will be considered if this is the case in addition to hospitalization such as Rituximab front line and daily subcutaneous Caplacizumab; - Occurrence of bleeding events at D30 and D90 post-PE treatment; - Occurrence of AE and SAE during the study.;Timepoint(s) of evaluation of this end point: - Occurrence of death: within 30 days and 90 days post-PE treatment; - Occurrence of refractoriness: at D30 post-PE treatment; - Occurrence of exacerbations: at D30 post-PE treatment; - Cognitive assessment based on MMS score and neurological assessment based on Rankin score : at baseline, D30 and D90 post-PE treatment; - Quality of life based on global post-traumatic score : at baseline, D30 an

—

Countries

France

Contacts

Public ContactMarty

CHU de Rouen

Nell.Marty@chu-rouen.fr0033232886265

Outcome results

None listed

Source: EU CTR (via WHO ICTRP) · Data processed: Feb 4, 2026