Immunosuppression and caplacizumab with plasma infusion instead of plasma exchange for immune TTP

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

The study will treat adults presenting with clinical immune-mediated TTP using a regimen of immunosuppressive drugs, caplacizumab, and controlled plasma infusions while omitting therapeutic plasma exchange. The investigators previously reported that adding caplacizumab to immunosuppression and PEX improved outcomes and reduced the number and volume of exchanges in 90 patients. Because PEX is invasive and associated with catheter- and plasma-related complications, a PEX-free regimen could lessen procedural risk and the burden of care. The protocol leverages shorter courses of plasma infusion together with caplacizumab to limit fluid overload and will be conducted at multiple French university hospitals.

Why it matters

Who can join: the full rules

Inclusion Criteria:

* Adult patient ≥ 18 years;
* Clinical diagnosis of iTTP 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; it is not necessary to have the laboratory result confirming the severe ADAMTS13 deficiency for inclusion of patient \[32\] (For patient with previous TTPflare, French score can be \< 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:

  * 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 before plasma treatment;
* Patients with a French score \< 2 (a serum creatinine level \> 200 μmol/L and/or with a platelet count \> 30 G/L), in order to exclude possible cases of hemolytic uremic syndrome (except for patient with previous TTPflare, French score can be \< 2);
* Other known causes of cytopenias and/or organ failure including but not limited to: uncontrolled cancer, chemotherapy, transplant, drugs, HIV at AIDS stage;
* Patients with a severe neurological disorder i.e. seizure, coma, focal deficiency, trouble of consciousness;
* 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);
* Weight \> 100KG;
* 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 Plasma 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.

Where it's running

• Chu Amiens
  Amiens, France Recruiting
• Chu Angers
  Angers, France Recruiting
• Chru Besanon
  Besançon, France Recruiting
• Chu Bobigny
  Bobigny, France Recruiting
• Hopital Jean Verdie
  Bondy, France Recruiting
• Chu Bordeaux
  Bordeaux, France Recruiting

Study contacts

• Study coordinator: Ygal Benhamou, Pr
• Email: ygal.benhamou@chu-rouen.fr
• Phone: 02 32 88 90 03

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Other studies for the same condition or treatment. Recruiting studies are listed first.

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How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.