Study on Caplacizumab and Immunosuppressive Therapy for Adults with Immune-Mediated Thrombotic Thrombocytopenic Purpura

1 1 1 1

What is this study about?

This clinical trial is focused on studying a rare blood disorder called immune-mediated thrombotic thrombocytopenic purpura (iTTP). This condition involves the formation of small blood clots throughout the body, which can lead to a low platelet count and damage to organs. The study will explore the effectiveness and safety of a treatment using a medication called caplacizumab, in combination with immunosuppressive therapy. Immunosuppressive therapy is a treatment that helps reduce the activity of the immune system. The study will not use the usual first-line treatment known as therapeutic plasma exchange, which is a procedure that removes and replaces blood plasma.

The purpose of the study is to evaluate how well the combination of caplacizumab and immunosuppressive therapy works in treating adults with iTTP. Participants in the study will receive caplacizumab, which is given as an injection either into a vein (intravenous) or under the skin (subcutaneous). The study will monitor participants over a period of time to see if they achieve remission, which means the symptoms of iTTP improve or disappear without needing therapeutic plasma exchange. The study will also look at the occurrence of any side effects or adverse events during the treatment period.

Participants will be observed for their response to the treatment, including how quickly their platelet count returns to normal and whether they experience any relapses or worsening of their condition. The study aims to provide valuable information on the potential benefits of using caplacizumab and immunosuppressive therapy for treating iTTP, offering insights into alternative treatment options for this serious condition.

1 joining the study

Upon joining the study, a clinical diagnosis of immune-mediated thrombotic thrombocytopenic purpura (iTTP) is confirmed. This includes checking for low platelet count, specific blood cell damage, and normal kidney function.

The diagnosis is confirmed through a test called ADAMTS13 within 48 hours.

2 treatment initiation

The treatment involves the administration of caplacizumab, a medication provided as a 10 mg powder and solvent for solution for injection.

The medication is administered either intravenously (IV) or subcutaneously (SC), depending on the specific requirements of the treatment plan.

3 treatment duration

The treatment period is divided into two phases: the on-treatment period from day 1 to day 84, and the overall study period from day 1 to day 168.

During this time, the effectiveness of the treatment is monitored, focusing on achieving remission without the need for therapeutic plasma exchange (TPE).

4 monitoring and evaluation

Throughout the study, the response to treatment is evaluated by monitoring platelet count and other health indicators.

The occurrence of any adverse events, including serious ones, is recorded to ensure safety.

5 completion of study

The study is estimated to conclude by December 13, 2024.

Upon completion, the results will be analyzed to determine the proportion of participants achieving remission and other health outcomes.

Who Can Join the Study?

  • Participants must have a clinical diagnosis of immune mediated thrombotic thrombocytopenic purpura (iTTP). This includes having low platelet counts, a type of anemia where red blood cells are broken down, and relatively normal kidney function. The diagnosis should be confirmed by a specific test called ADAMTS13 within 48 hours (2 days).
  • Participants must have a clinical diagnosis of iTTP and a French TMA score of 1 or 2. This score helps assess the severity of the condition.
  • Female participants must not be pregnant or breastfeeding. They must either be women who cannot have children or agree to use an acceptable method of birth control during the treatment period and for at least 2 months after the last dose of the study drug.
  • Male participants with female partners who can have children must agree to follow the birth control guidance during the treatment period and for at least 2 months after the last dose of the study drug.

Who Cannot Join the Study?

  • Patients who are not adults cannot participate. This means you must be 18 years or older.
  • Patients who have other serious health conditions that might interfere with the study cannot participate.
  • Patients who are pregnant or breastfeeding cannot participate.
  • Patients who have a history of allergic reactions to the study medication or similar medications cannot participate.
  • Patients who are currently participating in another clinical trial cannot participate.
  • Patients who have a condition that affects their ability to understand or follow the study instructions cannot participate.
  • Patients who have a history of drug or alcohol abuse that might interfere with the study cannot participate.
  • Patients who have received certain treatments recently that might interfere with the study cannot participate.

Where you can join this trial?

Verified and Recommended Sites

No sites found in this category

Verified Sites

Site Name City Country Status
Medizinische Hochschule Hannover Hanover Germany
Hospital Universitario Y Politecnico La Fe Valencia Spain
Medical University Of Vienna Vienna Austria
Centre Hospitalier Universitaire De Lille Lille France
Universitaet Leipzig Leipzig Germany
Katholieke Universiteit te Leuven Leuven Belgium

Other Sites

Site Name City Country Status
Centro Ricerche Cliniche Di Verona S.r.l. Verona Italy
Institute Of Hematology And Blood Transfusion Prague Czechia
Hospital General Universitario Gregorio Maranon Madrid Spain
Azienda Unita Locale Socio Sanitaria N 8 Berica Vicenza Italy
Geniko Nosokomeio Thessalonikis George Papanikolaou Thessaloniki Greece
AORN San Giuseppe Moscati Avellino Avellino Italy
Evangelismos S.A. Athens Greece
Centre Hospitalier Universitaire Rouen Rouen France
Virgen del Rocío University Hospital Sevilla Spain
Fondazione IRCCS Ca Granda Ospedale Maggiore Policlinico Milan Italy
Hospital Clinic De Barcelona Barcelona Spain
Charite Universitaetsmedizin Berlin KöR Berlin Germany
Fakultni Nemocnice Brno Brno Czechia
Fakultni Nemocnice Hradec Kralove Novy Hradec Kralove Czechia
Meander Medical Center Amersfoort The Netherlands
IRCCS Ospedale Policlinico San Martino Genoa Italy
Hopital Beaujon Clichy France
Centre Hospitalier Universitaire Dinant Godinne Sainte-Elisabeth-UCL-Namur Namur Belgium
Ucjoyjenfmxbsumtwtkle Esjhk Afa Essen Germany
Urgxdgisbc Hiecspnm Chnvddt Cologne Germany
Aacclygtht Pesjcnkh Hmiktzwx Di Mjopdiqsw Marseille France
Etlsmkb Ukqkrhopifjn Moaxcjl Chjbvph Rzzurfdup (rzletnu Mce Rotterdam The Netherlands
Gmjyiv Uipsoruvin Ftkscvphi Frankfurt Germany

Want to learn more about this study or check if you can participate? Contact us.

Trial status

Country Status Recruitment Start
Austria Austria
Not recruiting
21.11.2022
Belgium Belgium
Not recruiting
21.11.2022
Czechia Czechia
Not recruiting
21.11.2022
France France
Not recruiting
21.11.2022
Germany Germany
Not recruiting
21.11.2022
Greece Greece
Not recruiting
21.11.2022
Italy Italy
Not recruiting
21.11.2022
Spain Spain
Not recruiting
21.11.2022
The Netherlands The Netherlands
Not recruiting
21.11.2022

Trial locations

Investigated drugs:

Caplacizumab is a medication used in this study to help treat a condition called immune-mediated thrombotic thrombocytopenic purpura (iTTP). It works by preventing the formation of blood clots, which can help reduce the risk of complications associated with this condition.

Immunosuppressive Therapy (IST) involves using medications that suppress or reduce the strength of the body’s immune system. In this trial, IST is used alongside caplacizumab to help manage iTTP by preventing the immune system from attacking the body’s own cells, which can lead to the symptoms of this condition.

Thrombotic Thrombocytopenic Purpura – This is a rare blood disorder characterized by the formation of small blood clots throughout the body. These clots can lead to a low platelet count, which is essential for normal blood clotting, and can cause bleeding issues. The disease often begins suddenly and can progress rapidly, affecting various organs due to restricted blood flow. Symptoms may include fatigue, fever, and neurological changes such as confusion or headaches. The condition is immune-mediated, meaning the body’s immune system mistakenly attacks its own cells, contributing to the disease’s progression. It requires careful management to prevent complications related to blood clotting and bleeding.

Trial ID:
2024-513262-19-00
Protocol code:
EFC16521
NCT ID:
NCT05468320
Trial Phase:
Therapeutic confirmatory (Phase III)

Other Trials to Consider

  • A study to evaluate the safety and effectiveness of urokinase, catalytic domain, fused with a single-chain antibody against von Willebrand factor in patients with immune-mediated thrombotic thrombocytopenic purpura.

    Not yet recruiting

    1 1
    France Germany Italy Spain
  • Study on the Safety and Effectiveness of TAK-755 (Apadamtase Alfa) for Patients with Severe Congenital Thrombotic Thrombocytopenic Purpura (cTTP)

    Not recruiting

    1 1 1
    Investigated diseases:
    Investigated drugs:
    Austria France Germany Italy Poland Spain