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Efficacy of a Personalized Caplacizumab Regimen Based on ADAMTS13 Activity Monitoring in Adult aTTP (CAPLAVIE)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT04720261
Recruitment Status : Unknown
Verified January 2021 by University Hospital, Rouen.
Recruitment status was:  Not yet recruiting
First Posted : January 22, 2021
Last Update Posted : January 22, 2021
Information provided by (Responsible Party):
University Hospital, Rouen

Brief 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.

Condition or disease Intervention/treatment Phase
Thrombotic Thrombocytopenic Purpura, Acquired Drug: Caplacizumab Phase 2

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.

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 125 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Intervention Model Description: Experimental, prospective, non-comparative, multicentric national study
Masking: None (Open Label)
Primary Purpose: Treatment
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.
Estimated Study Start Date : May 1, 2021
Estimated Primary Completion Date : October 1, 2023
Estimated Study Completion Date : October 1, 2023

Arm Intervention/treatment
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.
Drug: Caplacizumab
Analyse of ADAMTS13 activity in patients with aTTP treated with Caplacizumab in order to help adapting the treatment with caplacizumab in TTP patients

Primary Outcome Measures :
  1. 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.

Secondary Outcome Measures :
  1. Response to treatment (platelet count recovery) [ Time Frame: 30 days post-PE treatment ]
    to platelet count recovery (as defined by a platelet count ≥ 150 G/L with a subsequent interruption of daily PE within 5 days)

  2. Durable remission achievement [ Time Frame: 90 days post-PE treatment ]
    Occurrence of durable remission achievement (platelet count ≥ 150 G/L for ≥ 30 consecutive days following PE interruption);

  3. Mortality at D90 post-PE treatment [ Time Frame: 90 days post-PE treatment ]
    Occurrence of death within 90 days post-PE treatment

  4. Refractoriness at D30 post-PE treatment [ Time Frame: Day 30 post-PE treatment ]
    Occurrence of refractoriness at D30 post-PE treatment;

  5. Exacerbation at D30 post-PE treatment [ Time Frame: Day 30 post-PE treatment ]
    Occurrence of exacerbations at D30 post-PE treatment

  6. Duration of plasma exchange (PE) treatment and the associated plasma volumes [ Time Frame: 30 days ]
    Duration of daily PE with the corresponding plasma volume

  7. Duration of plasma exchange (PE) treatment and the associated plasma [ Time Frame: 30 days ]
    Total number of PE and the corresponding plasma volume during the full study drug treatment period

  8. Occurrence of neurological sequelae treatment [ Time Frame: Day 90 post-PE treatment ]
    Neurological assessment based on Rankin score

  9. Evaluate the Quality of life [ Time Frame: Day 90 post-PE treatment ]
    Quality of life based on global post-traumatic score (PCL-S SCALE) at baseline, D90 post-PE treatment

  10. Evaluate the cost of the strategy [ Time Frame: Day 90 post-PE treatment ]
    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

  11. To perform a safety analysis [ Time Frame: 90 days post-PE treatment ]
    Occurrence of AE and SAE during the study

  12. Occurrence of cognitive sequelae treatment [ Time Frame: Day 90 post-PE treatment ]
    Cognitive assessment based on MMS score

Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.

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Ages Eligible for Study:   18 Years to 100 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

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 [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 :

      • 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).

Exclusion Criteria:

  • 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);
  • 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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT04720261

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Contact: Ygal BENHAMOU 0232889274

Sponsors and Collaborators
University Hospital, Rouen
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Responsible Party: University Hospital, Rouen Identifier: NCT04720261    
Other Study ID Numbers: 2019/0408/HP
First Posted: January 22, 2021    Key Record Dates
Last Update Posted: January 22, 2021
Last Verified: January 2021
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Product Manufactured in and Exported from the U.S.: No
Additional relevant MeSH terms:
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Purpura, Thrombocytopenic
Purpura, Thrombotic Thrombocytopenic
Blood Coagulation Disorders
Hematologic Diseases
Pathologic Processes
Skin Manifestations
Thrombotic Microangiopathies
Blood Platelet Disorders
Immune System Diseases