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Clinical Trials for Thrombotic Microangiopathy

There are currently 4 ongoing clinical trials investigating treatments for thrombotic microangiopathy (TMA), a serious condition where small blood vessels become damaged and form blood clots. These trials are being conducted across multiple European countries and are testing different medications aimed at improving outcomes for patients who develop TMA after receiving a stem cell transplant. The trials include studies for both children and adults, using medications such as narsoplimab, pegcetacoplan, and ravulizumab.

Clinical trial locations

Study of Narsoplimab for Children with High-Risk Blood Vessel Damage After Stem Cell Transplant

This trial is investigating narsoplimab for children who develop a serious complication called thrombotic microangiopathy after receiving a stem cell transplant. This condition causes damage to small blood vessels, which can lead to problems such as low platelet counts and organ damage.

Who can participate: Children aged between 28 days and 18 years who have received a stem cell transplant from a donor and have been diagnosed with high-risk TMA. To be considered high-risk, patients must have a low platelet count (below 50,000 per microliter) or a significant drop in platelets, along with signs of red blood cell damage. Additional high-risk features include lasting TMA despite treatment changes, kidney problems, digestive system involvement confirmed by biopsy, nervous system complications, or certain blood test abnormalities.

Who cannot participate: The trial excludes patients who do not have TMA despite having undergone a stem cell transplant, those who are not considered high-risk for the condition, and those who do not meet the age requirements.

What the study aims to do: The main goal is to evaluate whether narsoplimab can improve survival rates and overall health in children with high-risk TMA following stem cell transplantation. Researchers will monitor participants for 100 days after diagnosis to assess survival rates and will continue to track other health outcomes over a longer period.

Investigational drug: Narsoplimab is a monoclonal antibody given through an intravenous infusion. It works by blocking a protein called MASP-2, which plays a role in the body’s immune response. By inhibiting this protein, narsoplimab aims to reduce inflammation and blood vessel damage in children with TMA.

Study of Pegcetacoplan (APL-2) in Patients with Transplant-associated Thrombotic Microangiopathy After Stem Cell Transplantation

This study focuses on adults who develop TMA after receiving a stem cell transplant. The condition affects small blood vessels and can damage various organs throughout the body.

Who can participate: Adults aged 18 years or older who have received a stem cell transplant from a related or unrelated donor using blood stem cells, bone marrow, or umbilical cord blood. Participants must have a confirmed diagnosis of transplant-associated TMA with specific signs including low platelet count, elevated lactate dehydrogenase levels, and at least two additional markers such as damaged red blood cells in blood tests, low hemoglobin requiring transfusions, protein in urine, elevated blood protein levels, or high blood pressure. The TMA must persist despite initial treatment, and patients must have elevated protein levels in their urine. Women of childbearing age must use approved birth control methods and have a negative pregnancy test.

Who cannot participate: The trial excludes children under 12 years old, patients who have received organ transplants other than stem cell transplants, those with active uncontrolled infections, anyone who received another experimental treatment within 30 days, pregnant or breastfeeding women, patients with severe liver or kidney problems requiring dialysis, those with active cancer requiring treatment, patients with severe heart conditions, anyone who had major surgery in the past 30 days, those allergic to the study medication, and patients with severe mental health conditions or who cannot follow study procedures.

What the study aims to do: The trial aims to understand how the body processes pegcetacoplan and to evaluate its safety in patients with transplant-associated TMA. Researchers will monitor various aspects of health, including blood tests and organ function, for 24 weeks. The study will assess whether the medication helps improve symptoms, including kidney function, blood cell counts, and other health measures. Success is measured by improvement in blood test results, organ function, and reduced need for blood transfusions.

Investigational drug: Pegcetacoplan is administered under the skin and works by targeting and inhibiting complement protein C3, which plays a crucial role in the immune system’s complement cascade. By blocking this protein, pegcetacoplan helps prevent excessive immune activation that leads to blood vessel damage and blood clot formation.

Study of Ravulizumab for Children with Thrombotic Microangiopathy After Stem Cell Transplant

This trial is testing ravulizumab for children who develop TMA after receiving a stem cell transplant. The condition can affect multiple organs including the kidneys, heart, lungs, nervous system, and digestive tract.

Who can participate: Children aged between 28 days and 18 years who have received a stem cell transplant within the past 12 months and have a confirmed diagnosis of TMA. The diagnosis must include new onset of low platelet count or resistance to platelet transfusion, along with at least one sign of red blood cell breakdown, protein in the urine, new onset of anemia, or high blood pressure. The TMA must persist for at least 72 hours after initial treatment. Children must weigh at least 5 kilograms and should be vaccinated against meningococcal infections if possible, with preventive antibiotics given according to institutional guidelines.

Who cannot participate: Patients who have had a stem cell transplant but do not have TMA, those outside the specified age range, patients unable to follow study procedures or attend required visits, those currently in another clinical trial, patients with certain medical conditions that could interfere with the study, pregnant or breastfeeding patients, and those with a history of allergic reactions to ravulizumab.

What the study aims to do: The trial aims to evaluate how effective ravulizumab is in treating TMA in children following stem cell transplantation. Researchers will monitor how quickly TMA responds to treatment, any changes in organ function, and overall survival rates. The study will track participants over a period of up to one year, assessing various health indicators and looking for any relapses of TMA.

Investigational drug: Ravulizumab is given through an intravenous infusion in addition to best supportive care. It is a monoclonal antibody that works by inhibiting part of the immune system called the complement system. By blocking specific proteins in the blood that cause inflammation and blood vessel damage, ravulizumab aims to reduce the complications associated with TMA.

Study on Ravulizumab for Patients with Thrombotic Microangiopathy After Stem Cell Transplant

This is a placebo-controlled trial studying ravulizumab in adults and adolescents who develop TMA after stem cell transplantation. The condition can lead to serious complications including low platelet counts, anemia, and organ damage.

Who can participate: Individuals aged 12 years or older who have received a stem cell transplant within the past 12 months. Participants must have a confirmed TMA diagnosis including new onset of low platelet count or lack of response to platelet transfusions, at least one sign of red blood cell breakdown, protein in urine, and new onset of anemia or high blood pressure. The TMA must persist despite initial treatment for at least 72 hours. Participants must weigh at least 30 kilograms and should be vaccinated against meningococcal infections if possible. Those under 18 should also receive vaccinations against Haemophilus influenzae type b and Streptococcus pneumoniae if feasible. All participants should receive preventive antibiotics for at least 2 weeks after meningococcal vaccination, or throughout treatment and for 8 months afterward if unable to receive the vaccine.

Who cannot participate: Patients who have had a stem cell transplant but do not have TMA, those outside the specified age range, and those not meeting other specific study requirements.

What the study aims to do: This trial compares ravulizumab to a placebo to determine how effective the medication is in treating TMA. Participants will receive either ravulizumab or placebo through regular infusions and will be monitored closely for several months. The study will assess whether ravulizumab can improve symptoms and prevent further complications. Researchers will track the response to treatment, changes in organ function, any TMA relapses, and overall survival at various time points including 100 days, 26 weeks, and 52 weeks.

Investigational drug: Ravulizumab is administered intravenously and works by targeting specific proteins in the immune system’s complement pathway. As a monoclonal antibody, it is designed to help reduce damage to blood vessels and improve blood flow by inhibiting inflammation and blood clotting processes. The study will compare outcomes between patients receiving ravulizumab and those receiving a placebo.

Summary

These four clinical trials represent important research efforts to find better treatments for thrombotic microangiopathy occurring after stem cell transplantation. The trials are being conducted across nine European countries, with particularly strong representation in Spain, France, Germany, and Italy, where multiple studies are taking place.

Two distinct investigational approaches are being tested: complement inhibitors and immunomodulatory therapies. Three of the four trials are investigating ravulizumab, a complement system inhibitor, while one trial each is testing narsoplimab and pegcetacoplan. This concentration of studies on complement inhibition reflects growing scientific understanding that the complement system plays a key role in TMA development.

The trials span different age groups, with two studies specifically focused on pediatric populations (children from 28 days to under 18 years) and two including adolescents and adults. This comprehensive approach ensures that treatments can be evaluated for effectiveness across all age groups affected by this serious complication.

All trials share common entry requirements, including the need for a recent stem cell transplant within the past 12 months and specific diagnostic criteria for TMA such as low platelet counts, signs of red blood cell damage, and evidence of organ involvement. An important safety requirement across all studies is vaccination against meningococcal infections, reflecting the mechanism of action of these complement-targeting therapies.

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