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Breakthrough drug combo may replace plasma exchange for rare blood disease

NCT ID NCT05468320

Disease control Sponsor: Sanofi 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 This study
The trial has finished. Results may not be published yet.
Stopped early
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This status has not been confirmed recently, so it may be out of date.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 a drug called caplacizumab, combined with immune-suppressing medicines, could treat a rare blood clotting disorder called immune-mediated thrombotic thrombocytopenic purpura (iTTP) without the usual plasma exchange procedure. Fifty-one adults aged 18 to 80 took part. The goal was to see if this drug-only approach could bring the disease under control and avoid the need for plasma exchange.

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 3

Large-scale testing in a bigger group. Usually the last step before a treatment can be approved.

Participants

51 people

The number who actually took part.

Started

Nov 2022

Finished

Dec 2024

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

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

Ages

18 to 80 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: Participants with a clinical diagnosis of iTTP (initial or recurrent), which includes thrombocytopenia, microangiopathic hemolytic anemia (eg, presence of schistocytes in peripheral blood smear) and relatively preserved renal function. The iTTP diagnosis should be confirmed by ADAMTS13 testing within 48 hours (2 days). Participants with a clinical diagnosis of iTTP and a French TMA score of 1 or 2. A female participant is eligible to participate if she is not pregnant or breastfeeding, and one of the following conditions applies: * Is a woman of nonchildbearing potential (WONCBP), OR * Is a woman of childbearing potential (WOCBP) and agrees to use an acceptable contraceptive method during the overall treatment period and for at least 2 months after the last study drug administration. Male participants with female partners of childbearing potential must agree to follow the contraceptive guidance as per protocol during the overall treatment period and for at least 2 months after last study drug administration. Exclusion Criteria: Platelet count ≥100 x 10\^9/L. Serum creatinine level \>2.26 mg/dL (200 µmol/L) in case platelet count is \>30 x 10\^9/L (to exclude possible cases of atypical HUS). Known other causes of thrombocytopenia including but not limited to: * Clinical evidence of enteric infection with E. coli 0157 or related organism. * Atypical HUS. * Hematopoietic stem cell, bone marrow or solid organ transplantation-associated thrombotic microangiopathy. * Known or suspected sepsis. * Diagnosis of disseminated intravascular coagulation. Congenital TTP (known at the time of study entry). Clinically significant active bleeding or known co-morbidities associated with high risk of bleeding (excluding thrombocytopenia). Inherited or acquired coagulation disorders. Malignant arterial hypertension. Participants requiring or expected to require invasive procedures immediately (eg, stroke requiring thrombolytic therapy, those who need mechanical ventilation, etc.). Those presenting with severe neurological or cardiac disease. Clinical condition other than that associated with TTP, with life expectancy \<6 months, such as end-stage malignancy. Known chronic treatment with anticoagulants and anti-platelet drugs that cannot be stopped (interrupted) safely, including but not limited to: * vitamin K antagonists. * direct-acting oral anticoagulants. * heparin or low molecular weight heparin (LMWH). * non-steroidal anti-inflammatory molecules other than acetyl salicylic acid. Participants who were previously enrolled in this clinical study (study EFC16521). Participants who received an investigational drug, or device, other than caplacizumab, within 30 days of anticipated IMP administration or 5 half-lives of the previous investigational drug, whichever is longer. Positive result on COVID test. The above information is not intended to contain all considerations relevant to a potential participation in a clinical trial.

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Conditions

The condition(s) this trial relates to.

acquired thrombotic thrombocytopenic purpura thrombotic thrombocytopenic purpura

As listed by the trial registrant

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

Thrombotic thrombocytopenic purpura

Contacts and locations

Locations

  • Duke University Medical Center Site Number : 8400022

    Durham, North Carolina, 27710-4000, United States

  • Investigational Site Number : 0560003

    Yvoir, 5530, Belgium

  • Investigational Site Number : 1240001

    Toronto, Ontario, M5B 1W8, Canada

  • Investigational Site Number : 2030001

    Brno, 62500, Czechia

  • Investigational Site Number : 2030003

    Prague, 128 00, Czechia

  • Investigational Site Number : 2500001

    Paris, 75012, France

  • Investigational Site Number : 2500002

    Bois-Guillaume, 76230, France

  • Investigational Site Number : 2500003

    Paris, 75010, France

  • Investigational Site Number : 2500005

    Lille, 59037, France

  • Investigational Site Number : 2760001

    Frankfurt am Main, 60590, Germany

  • Investigational Site Number : 2760002

    Cologne, 50937, Germany

  • Investigational Site Number : 2760004

    Hanover, 30625, Germany

  • Investigational Site Number : 2760006

    Berlin, 10117, Germany

  • Investigational Site Number : 3800001

    Milan, Lombardy, 20122, Italy

  • Investigational Site Number : 3800003

    Avellino, Campania, 83100, Italy

  • Investigational Site Number : 3800004

    Vicenza, 36100, Italy

  • Investigational Site Number : 3800005

    Verona, 37134, Italy

  • Investigational Site Number : 3800006

    Genova, 16132, Italy

  • Investigational Site Number : 3920001

    Iruma-gun, Saitama, 350-0495, Japan

  • Investigational Site Number : 3920003

    Kurashiki-shi, Okayama-ken, 710-8602, Japan

  • Investigational Site Number : 5280002

    Amersfoort, 3818 TZ, Netherlands

  • Investigational Site Number : 7240001

    Madrid / Madrid, Madrid, Comunidad de, 28007, Spain

  • Investigational Site Number : 8260001

    London, London, City of, NW1 2PG, United Kingdom

  • Investigational Site Number : 8260002

    Liverpool, L7 8XP, United Kingdom

  • Johns Hopkins University- Site Number : 8400007

    Baltimore, Maryland, 21287, United States

  • The Ohio State University Comprehensive Cancer Center - Site Number : 8400001

    Columbus, Ohio, 43210, United States

  • University of Alabama- Site Number : 8400011

    Birmingham, Alabama, 35233, United States

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