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Tailored drug regimen shows promise for rare blood disorder

NCT ID NCT04720261

Disease control Sponsor: University Hospital, Rouen Source: ClinicalTrials.gov ↗

What the study statuses mean

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Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early This study
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This study tested whether giving the drug caplacizumab based on regular blood tests (ADAMTS13 activity) could improve outcomes for adults with acquired thrombotic thrombocytopenic purpura (aTTP), a rare condition causing dangerous blood clots. The trial enrolled 58 adults and monitored for death, lack of response, or worsening disease within 30 days. The goal was to see if a personalized approach could reduce complications while using the drug more efficiently.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 2

Tests whether the treatment actually works, and watches for side effects, in a larger group.

Participants

58 people

The number who actually took part.

Started

Apr 2021

Finished

May 2023

Lead sponsor

Other sponsor

The registry's catch-all category, for sponsors it does not file as a company, a government agency, or a research network.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

18 to 100 years

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

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.

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Conditions

The condition(s) this trial relates to.

acquired thrombotic thrombocytopenic purpura

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Thrombotic thrombocytopenic purpura, acquired

Contacts and locations

Locations

  • Ah-Hp Hopital Saint Louis

    Paris, 75010, France

  • Ap-Hm Hopital de La Conception

    Marseille, 13005, France

  • Ap-Hp Hopital de Cochin

    Paris, 75014, France

  • Ap-Hp Hopital de Necker

    Paris, 75015, France

  • Ap-Hp Hopital de St Antoine

    Paris, 75571, France

  • CHU CAEN

    Caen, 14033, France

  • Ch Edouard Herriot

    Lyon, 69003, France

  • Ch Valenciennes

    Valenciennes, 59300, France

  • Chu Amiens

    Amiens, 80054, France

  • Chu Angers

    Angers, 49933, France

  • Chu Besancon

    Besançon, 25030, France

  • Chu Bordeaux

    Bordeaux, 33404, France

  • Chu Clermont-Ferrand

    Clermont-Ferrand, 63003, France

  • Chu Lille

    Lille, 59037, France

  • Chu Limoges

    Limoges, 87042, France

  • Chu Montpellier

    Montpellier, 34295, France

  • Chu Nancy

    Nancy, 54035, France

  • Chu Nantes

    Nantes, 44093, France

  • Chu Reims

    Reims, 51092, France

  • Chu Rennes

    Rennes, 35033, France

  • Chu Rouen

    Rouen, 76031, France

  • Chu Tours

    Tours, 37044, France

  • Fort de France

    Fort-de-France, 97261, Martinique

  • Reunion Sud

    Saint-Paul, 97448, France

More trials for these conditions

Other studies related to the condition(s) this trial covers.