In short

There are currently 3 ongoing clinical trials investigating new treatments for thrombotic thrombocytopenic purpura, a rare blood disorder that causes small blood clots to form throughout the body. These trials are testing medications including TAK-755 and caplacizumab in several European countries, offering new hope for patients with both immune-mediated and congenital forms of this condition. (Also known as: iTTP, cTTP, Upshaw-Schulman Syndrome, hereditary thrombotic thrombocytopenic purpura)

Clinical trial locations

Study on the Safety of TAK-755, Rituximab, and Glucocorticoids for Patients with Immune-Mediated Thrombotic Thrombocytopenic Purpura (iTTP)

This trial is testing TAK-755, also called recombinant ADAMTS13, which is a protein therapy designed to help manage the immune-mediated form of the condition. The treatment is given directly into a vein and is used alongside other medications including rituximab, a type of antibody therapy, and glucocorticoids, which are steroid medications.

The main goal of this study is to assess the safety of TAK-755 when used with minimal or no plasma exchange, a procedure that is typically used to treat this condition by removing and replacing blood plasma. Researchers will monitor participants throughout the study to track any side effects, measure how quickly patients respond to treatment, and observe changes in specific blood markers.

Inclusion criteria: To join this study, you must be at least 18 years old and have a confirmed diagnosis of immune-mediated thrombotic thrombocytopenic purpura. This diagnosis is confirmed through signs of low platelet counts (below 100,000 per microliter) and broken red blood cells, shown by elevated levels of a substance called LDH in the blood or visible damaged cells in blood tests. You must agree to follow all study procedures. Women who can become pregnant must have a negative pregnancy test and use effective birth control during the study, and men who are sexually active must also use birth control.

Exclusion criteria: No specific exclusion criteria were listed for this trial.

Investigational drug: TAK-755 is a medication being studied for its ability to break down blood clots and improve blood flow in patients with immune-mediated thrombotic thrombocytopenic purpura. It works by replacing a missing enzyme that helps regulate blood clotting.

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

This study explores the effectiveness and safety of caplacizumab combined with immunosuppressive therapy for treating adults with immune-mediated thrombotic thrombocytopenic purpura. Unlike traditional treatment approaches, this trial does not use therapeutic plasma exchange as the first-line treatment.

The study aims to evaluate how well this combination therapy works in helping patients achieve remission, meaning their symptoms improve or disappear. Participants will receive caplacizumab either as an injection into a vein or under the skin. Throughout the study, researchers will monitor how quickly platelet counts return to normal and whether patients experience any relapses or worsening of their condition.

Inclusion criteria: Participants must have a clinical diagnosis of immune-mediated thrombotic thrombocytopenic purpura, including low platelet counts, a type of anemia where red blood cells break down, and relatively normal kidney function. The diagnosis must be confirmed by a specific ADAMTS13 test within 48 hours. Participants must also have a French TMA score of 1 or 2, which helps assess the severity of the condition. Women who can become pregnant must not be pregnant or breastfeeding and must agree to use acceptable birth control during treatment and for at least 2 months after the last dose. Men with partners who can become pregnant must also follow birth control guidance for the same period.

Exclusion criteria: You cannot participate if you are under 18 years old, have other serious health conditions that might interfere with the study, are pregnant or breastfeeding, have a history of allergic reactions to the study medication, are currently in another clinical trial, have conditions affecting your ability to understand or follow study instructions, have a history of drug or alcohol abuse that might interfere with the study, or have received certain recent treatments that might interfere with the study.

Investigational drugs: Caplacizumab works by preventing the formation of blood clots, helping to reduce complications associated with this condition. Immunosuppressive therapy involves medications that reduce the strength of the immune system, preventing it from attacking the body's own cells and causing symptoms.

Study on the Safety and Effectiveness of TAK-755 (Apadamtase Alfa) for Patients with Severe Congenital Thrombotic Thrombocytopenic Purpura (cTTP)

This trial focuses on the congenital form of thrombotic thrombocytopenic purpura, also known as Upshaw-Schulman Syndrome or hereditary thrombotic thrombocytopenic purpura. The study is testing TAK-755, a protein-based medication designed to help manage this inherited condition.

The purpose of this long-term study is to evaluate the safety and tolerability of TAK-755 in patients with severe congenital thrombotic thrombocytopenic purpura. Participants will receive the treatment either as a regular preventive measure or as needed when symptoms occur. The medication is given either under the skin or directly into a vein. The study will continue until 2027, allowing researchers to gather comprehensive data on the effectiveness and safety of TAK-755.

Inclusion criteria: Participants must have completed a previous TAK-755 Phase 3 study or be new to the treatment. You or your legal representative must sign a consent form if you are 18 or older, or an assent form if you are younger. You must be between 0 and 70 years old and have severe congenital ADAMTS-13 deficiency, meaning very low levels of this specific protein in your blood. You should not show severe signs of the condition, including having platelet counts above 100,000 per microliter. Patients aged 16 and older must have a Karnofsky score of 70% or higher (measuring ability to perform daily activities), while those younger than 16 must have a Lansky score of 80% or higher. Females who can have children must have a negative pregnancy test and agree to use effective birth control. Sexually active males must also use contraception during the study and for at least 16 days after the last dose. You must be negative for hepatitis C virus or have a stable condition if positive.

Exclusion criteria: You cannot participate if you have any other serious medical condition that might interfere with the study, are currently in another clinical trial, have had recent major surgery or are planning surgery during the study period, have a known allergy to the study medication or its ingredients, are pregnant or breastfeeding, have a history of drug or alcohol abuse, or are unable to comply with study procedures or follow-up visits.

Investigational drug: TAK-755 is designed to prevent and treat blood clots by replacing a missing enzyme in the blood that is crucial for breaking down large protein molecules that can cause clotting. The study monitors how well patients tolerate the medication over a long period and tracks any side effects.

Summary

These three ongoing clinical trials represent important research efforts to improve treatment options for both immune-mediated and congenital forms of thrombotic thrombocytopenic purpura. The trials are being conducted across multiple European countries, with Austria, Italy, and Spain participating in all three studies, demonstrating strong research interest in these nations.

A notable focus of the research is on TAK-755, which appears in two of the three trials and is being tested for both immune-mediated and congenital forms of the condition. This medication works by replacing the missing or deficient ADAMTS13 enzyme that is central to the disease process. The other trial explores caplacizumab combined with immunosuppressive therapy, offering an alternative approach that avoids traditional plasma exchange procedures.

These studies are particularly significant because they are testing treatments that may reduce or eliminate the need for plasma exchange, a procedure that has been the standard treatment but requires specialized facilities and can be burdensome for patients. The trials vary in their expected completion dates, with one concluding in late 2024, another in 2025, and the longest-term study extending until 2027, reflecting the need for both immediate answers and long-term safety data.

Thrombotic thrombocytopenic purpura Trials in Disease

Clinical trials for Thrombotic thrombocytopenic purpura

Thrombotic thrombocytopenic purpura+1

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.