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Ongoing Clinical Trials for Atypical Haemolytic Uraemic Syndrome

This article provides information about 7 ongoing clinical trials investigating new treatments for atypical haemolytic uraemic syndrome (aHUS), a rare condition affecting blood cells and kidneys. The trials are testing medications such as eculizumab, iptacopan, ravulizumab, and crovalimab across multiple European countries including France, Italy, Germany, Spain, Belgium, Poland, and others.

Clinical trial locations

Study of Eculizumab in Adults with Hypertensive Emergency-Related Hemolytic Uremic Syndrome Requiring Dialysis or with Severe Kidney Problems

This trial is investigating the use of eculizumab for a specific form of the condition that occurs alongside severe high blood pressure, known as hypertensive emergency. The study is being conducted in France and focuses on adults who have developed severe kidney problems requiring dialysis or showing very high levels of creatinine in their blood.

Main inclusion criteria: Participants must be between 18 and 65 years old and have been hospitalized within the last 10 days with acute kidney failure requiring dialysis or high blood creatinine levels, alongside blood cell breakdown showing anemia, low platelets, and signs of blood cell destruction. They must also have severe high blood pressure with readings above 180/110 mmHg and evidence of organ damage affecting the brain, heart, or eyes. Women who can become pregnant must use effective birth control during the study and for 5 months afterward.

Main exclusion criteria: The trial excludes people under 18 or over 65 years of age, those who are pregnant or breastfeeding, and individuals with previous organ transplants or active cancer. People with severe heart disease, active infections including meningitis, known allergies to eculizumab, or participation in other clinical trials within the last 30 days cannot join. Those with uncontrolled high blood pressure, severe liver disease, active autoimmune conditions, history of substance abuse within the past year, or life expectancy less than 6 months are also excluded.

Focus of the trial: The study aims to determine whether early treatment with eculizumab, when added to standard blood pressure control, can help prevent the need for ongoing dialysis in patients with severe kidney problems. Participants will be randomly assigned to receive either eculizumab plus standard treatment or standard treatment alone for approximately 13 weeks. Researchers will monitor kidney function, blood cell counts, and potential complications, tracking how quickly patients recover and whether they still require dialysis after several months.

Investigational drug: Eculizumab is a specialized biological medication given through intravenous infusion that blocks part of the immune system called the complement cascade. It works by binding to and inhibiting the C5 complement protein, thereby preventing excessive complement activation that can damage blood vessels and organs, particularly the kidneys. The maximum daily dose is 1200 mg, with a total maximum dose of 9600 mg over the treatment period.

Study on Long-Term Safety and Effects of Iptacopan for Patients with Atypical Hemolytic Uremic Syndrome (aHUS)

This study in Czechia focuses on the long-term safety and effectiveness of iptacopan for patients who have already participated in a previous study involving this medication and are continuing to benefit from it. The trial is designed to gather comprehensive data over several years, continuing until April 2029.

Main inclusion criteria: Participants must have completed the full treatment period of a previous study for aHUS and still be receiving iptacopan treatment, which the study doctor considers beneficial. They must be willing and able to follow the study’s schedule and activities. Vaccinations against Neisseria meningitidis, Streptococcus pneumoniae, and Haemophilus influenzae must be up to date, including any necessary booster shots according to local health guidelines. Both males and females can participate.

Main exclusion criteria: People with any other serious medical conditions that might interfere with the study cannot participate. Pregnant or breastfeeding women, those who participated in another clinical trial within the last 30 days, and individuals with a history of allergic reactions to the study medication or similar drugs are excluded. The trial also excludes those unable to follow study procedures, people with a history of drug or alcohol abuse, certain infections, those who have received medications that might interfere with the study, people with a history of cancer (except some types of skin cancer), and those with uncontrolled high blood pressure.

Focus of the trial: The study monitors the long-term safety of iptacopan by checking for side effects and changes in health, including blood tests and heart monitoring. It also evaluates how well the medication helps manage symptoms, such as maintaining healthy blood counts and kidney function without the need for other treatments. The study assesses the absence of thrombotic microangiopathy manifestations without using anti-C5 antibody therapy.

Investigational drug: Iptacopan is administered orally in the form of hard gelatin capsules. It works by targeting a specific part of the immune system thought to play a role in causing the problems associated with aHUS, helping to manage symptoms and make it easier for patients to live with this condition over the long term.

Study on Switching from Anti-C5 Antibody Therapy to Iptacopan for Patients with Atypical Hemolytic Uremic Syndrome (aHUS)

This trial, conducted in Italy, Germany, Spain, and France, explores the effects of switching from current anti-C5 antibody treatment (such as eculizumab or ravulizumab) to iptacopan. The study will evaluate the safety and effectiveness of iptacopan over a 12-month period.

Main inclusion criteria: Participants must be at least 18 years old and willing to attend all scheduled study visits. They should have a confirmed diagnosis of aHUS with other similar conditions ruled out. Participants must currently be on anti-C5 antibody treatment for at least 3 months before the study starts and show improvement from this treatment for at least 3 months, demonstrated by a platelet count of at least 150 x 10⁹/L, normal LDH levels, and stable or improving kidney function. Vaccinations against certain infections must be completed at least 2 weeks before starting the new treatment, or if treatment needs to start sooner, antibiotics must be taken for at least 2 weeks after vaccination.

Main exclusion criteria: The trial excludes people with any other serious medical conditions that could interfere with the study, those currently participating in another clinical trial, and individuals who have had recent major surgery or are planning surgery during the study period. Pregnant or breastfeeding women, people with a history of drug or alcohol abuse, those with allergies to the study medication or its ingredients, and anyone unable to follow study procedures or attend required visits cannot participate.

Focus of the trial: The study monitors participants to see if they remain free from signs of thrombotic microangiopathy over the 12-month treatment period. Regular check-ups and blood tests assess blood and kidney health, including changes in platelet count, LDH, hemoglobin, and kidney function. Safety evaluations track any adverse events and monitor the requirement for dialysis.

Investigational drug: Iptacopan is taken orally in the form of hard gelatin capsules. It works by inhibiting a specific protein in the complement system, part of the immune system, thereby reducing inflammation and preventing blood vessel damage. The goal is to see if iptacopan can effectively prevent symptoms of thrombotic microangiopathy over the 12-month period.

Study on Ravulizumab for Children with Paroxysmal Nocturnal Hemoglobinuria or Atypical Hemolytic Uremic Syndrome

This study in Italy and Spain focuses on children aged 2 to under 18 years with either paroxysmal nocturnal hemoglobinuria or aHUS. The trial uses ravulizumab, a monoclonal antibody medication, to understand how it works in pediatric patients.

Main inclusion criteria: Participants must be between 2 and 17 years old at the time of consent and weigh at least 10 kg. A legal guardian must give written consent, and the participant must provide written assent if required. Vaccinations against meningococcal infection from specific groups must be current within the last 3 years or received at least 2 weeks before starting the study. Vaccinations for Streptococcus pneumoniae and Haemophilus influenzae type b must be completed according to local guidelines. For aHUS patients not previously treated with complement inhibitors, they must show signs of thrombotic microangiopathy, including low platelet count, evidence of red blood cell breakdown, and kidney injury. Those previously treated must show clinical evidence of response to treatment with stable parameters at screening.

Main exclusion criteria: Children not diagnosed with the specified conditions, those not within the specified age range, or unable to follow study procedures cannot participate. Those with other medical conditions that might interfere with the study, currently participating in another clinical trial, with a history of allergic reactions to the study medication, who are pregnant or breastfeeding, or have received certain medications recently are also excluded.

Focus of the trial: The study, lasting about a year, closely monitors how the body processes the drug and how it affects disease symptoms. Researchers assess the medication’s safety and any side effects that may occur. Regular check-ups monitor participants’ health and treatment effectiveness. The study also evaluates the use of a special device that helps deliver the medication correctly and safely.

Investigational drug: Ravulizumab is administered through subcutaneous injection. It works by blocking a part of the immune system that can attack the body’s own cells, helping to prevent the destruction of red blood cells and reducing the risk of kidney damage. The dosage and frequency are determined by the study protocol and adjusted based on the participant’s response and condition.

Study on the Effectiveness and Safety of Crovalimab for Children with Atypical Hemolytic Uremic Syndrome (aHUS)

This trial, conducted in Belgium, Poland, Italy, Hungary, France, and Spain, tests crovalimab in pediatric patients who have not received any previous treatment that inhibits the complement system. The study aims to gather information on the treatment’s effectiveness and safety, continuing until 2029.

Main inclusion criteria: Patients must have been vaccinated against Neisseria meningitidis within the last 3 years before starting treatment, following local guidelines for patients with complement deficiency. Female patients who can have children must agree to either abstain from heterosexual intercourse or use birth control during the study. For treatment-naive patients, the first signs of thrombotic microangiopathy must have appeared within 28 days before the first dose of crovalimab. For patients switching from other treatments, there must be clinical evidence of response to eculizumab or ravulizumab with stable parameters at screening.

Main exclusion criteria: Children who have previously received treatment with a complement inhibitor cannot participate. Those with any other medical condition that study doctors believe would make it unsafe, unable to follow study procedures or attend required visits, pregnant or breastfeeding, participating in another clinical trial, with a history of severe allergic reactions to study medications, or certain infections cannot join.

Focus of the trial: The study monitors various health indicators, such as kidney function and blood parameters, over a 24-week treatment period to determine the treatment’s impact. Researchers track changes in dialysis requirements, kidney function, and blood tests. The study also monitors any side effects, including injection site reactions and infections, and assesses the presence of anti-drug antibodies. The goal is to measure the proportion of patients achieving a complete response to treatment and maintaining control through Week 25.

Investigational drug: Crovalimab is administered as a solution for injection or infusion, given either under the skin or into a vein. It works by inhibiting the complement system, a part of the immune system that, when overactive, can lead to the symptoms of aHUS. The medication aims to prevent blood vessel damage and reduce disease symptoms.

Study on the Effectiveness and Safety of Iptacopan for Adults with Atypical Hemolytic Uremic Syndrome Not Previously Treated with Complement Inhibitors

This study in Slovakia, Austria, Greece, Slovenia, and Czechia evaluates iptacopan in adult patients who have not previously received complement inhibitor therapy. Participants will take the medication orally twice daily for 26 weeks.

Main inclusion criteria: Participants must be 18 years or older with signs of active thrombotic microangiopathy, including a low platelet count (less than 150,000 per microliter), high levels of LDH (at least 1.5 times the normal upper limit), low hemoglobin levels, and high serum creatinine levels indicating worsening kidney function during the screening period. Vaccinations against Neisseria meningitidis and Streptococcus pneumoniae must be completed at least 2 weeks before starting treatment, or if treatment starts earlier, antibiotics must be given. For patients with a kidney transplant, they must have a known history of aHUS before the transplant or meet specific criteria if no history is available.

Main exclusion criteria: The trial excludes people with a history of severe allergic reactions to the study medication, those currently participating in another clinical trial, individuals with a history of drug or alcohol abuse within the past year, pregnant or breastfeeding women, and anyone with other medical conditions that study doctors believe would make participation unsafe. Those who have received certain medications that might interfere with treatment, people with a history of certain types of cancer, severe liver or kidney disease, recent major surgery, or uncontrolled high blood pressure cannot participate.

Focus of the trial: Researchers monitor participants over 26 weeks to see if they achieve a complete response, which means improvement in blood and kidney function without the need for other treatments like plasma exchange or specific antibodies. The study evaluates how quickly participants respond to treatment, changes in blood parameters, kidney function, and overall health and well-being. Safety evaluations monitor any side effects or adverse events.

Investigational drug: Iptacopan is taken orally in the form of hard gelatin capsules twice daily. It works by inhibiting a component of the complement system, part of the immune system that can contribute to the disease process. The medication aims to help manage symptoms by reducing abnormal blood clotting and kidney damage associated with the condition.

Study on the Effects and Safety of Crovalimab for Adults and Adolescents with Atypical Hemolytic Uremic Syndrome (aHUS)

This trial, conducted in Poland, Germany, Italy, Belgium, France, Spain, and Hungary, assesses crovalimab in patients who have not previously received complement inhibitor treatment. The study is expected to conclude by December 2029.

Main inclusion criteria: Patients must have been vaccinated against Neisseria meningitidis within the last 3 years, following local guidelines for patients with complement deficiency. They must have adequate liver function, with liver enzyme levels no more than 3 times the upper limit of normal at screening, and no clinical signs of severe liver damage. Female patients who can have children must agree to either abstain from heterosexual intercourse or use contraception. For treatment-naive patients, the first signs of thrombotic microangiopathy must have appeared within 28 days before the first dose. For patients switching from other treatments, there must be clinical evidence of response with stable parameters.

Main exclusion criteria: People who have previously received treatment with a complement inhibitor cannot participate. Those not within the specified age range, with any medical condition that study doctors believe would make participation unsafe, unable to follow study procedures or attend required visits, pregnant or breastfeeding, or participating in another clinical trial at the same time are excluded.

Focus of the trial: The study monitors various health parameters throughout treatment, including the requirement for dialysis, changes in kidney function, blood parameters, and fatigue levels in adults. Researchers track the incidence and severity of any adverse events, including injection site reactions and infections. The primary goal is to determine the proportion of patients achieving a complete response to treatment, monitoring platelet counts, LDH levels, and serum creatinine levels. Secondary assessments include changes in chronic kidney disease stage, vital signs, and the presence of anti-drug antibodies.

Investigational drug: Crovalimab is administered through intravenous infusion, delivering the medication directly into the bloodstream. It works by inhibiting the complement system, aiming to prevent the immune system from attacking the body’s own cells. The medication is designed to manage the symptoms of aHUS by preventing blood vessel damage and reducing inflammation.

Summary

The 7 ongoing clinical trials for atypical haemolytic uraemic syndrome reflect the continuing research efforts to develop and evaluate treatment options for this rare condition. Several key observations emerge from this collection of trials.

The trials are distributed across multiple European countries, with notable concentration in France, Italy, Spain, and Germany. Belgium, Poland, and Hungary also host multiple studies, while Austria, Greece, Slovakia, Slovenia, and Czechia each conduct one trial. This geographic distribution ensures diverse patient populations and access to treatment research across Europe.

Four different investigational drugs are being studied: iptacopan, crovalimab, ravulizumab, and eculizumab. Iptacopan appears in three trials, examining its use in different patient populations including long-term safety, switching from other treatments, and first-line therapy. Crovalimab is featured in three trials focusing on both pediatric and adult/adolescent patients. These medications all work by targeting the complement system, which plays a central role in the disease process.

The trials address different patient needs, including treatment-naive patients who have never received complement inhibitors, patients switching from existing treatments, pediatric populations, and long-term safety monitoring. One unique trial focuses specifically on patients with hypertensive emergency-associated symptoms, demonstrating the recognition of different clinical presentations of the condition.

Most trials include comprehensive monitoring of kidney function, blood parameters, and safety outcomes. Several studies emphasize the importance of vaccination against certain bacterial infections before starting treatment, reflecting the known infection risks associated with complement inhibition. The trials typically span multiple months to years, allowing researchers to gather substantial data on both short-term and long-term effects of these treatments.

Ongoing Clinical Trials on Atypical haemolytic uraemic syndrome

  • Study of Eculizumab in Adults with Hypertensive Emergency-Related Hemolytic Uremic Syndrome Requiring Dialysis or with Severe Kidney Problems

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    3 1 1 1
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    Investigated drugs:
    France

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