Ongoing Clinical Trials for Atypical Haemolytic Uraemic Syndrome
There are currently 7 clinical trials investigating new treatments for atypical haemolytic uraemic syndrome (aHUS), a rare condition affecting blood cells and kidneys. These studies are testing medications called complement inhibitors across multiple European countries, evaluating their effectiveness and safety in both adults and children. The trials include studies on iptacopan, crovalimab, ravulizumab, and eculizumab for patients who are either starting treatment for the first time or switching from existing therapies.
Clinical trial locations
- Austria
- Belgium
- Czechia
- France
- Study of Eculizumab in Adults with Hypertensive Emergency-Related Hemolytic Uremic Syndrome Requiring Dialysis or with Severe Kidney Problems
- Study on Switching from Anti-C5 Antibody Therapy to Iptacopan for Patients with Atypical Hemolytic Uremic Syndrome (aHUS)
- Study on the Effectiveness and Safety of Crovalimab for Children with Atypical Hemolytic Uremic Syndrome (aHUS)
- Study on the Effects and Safety of Crovalimab for Adults and Adolescents with Atypical Hemolytic Uremic Syndrome (aHUS)
- Germany
- Greece
- Hungary
- Italy
- Study on Switching from Anti-C5 Antibody Therapy to Iptacopan for Patients with Atypical Hemolytic Uremic Syndrome (aHUS)
- Study on Ravulizumab for Children with Paroxysmal Nocturnal Hemoglobinuria or Atypical Hemolytic Uremic Syndrome
- Study on the Effectiveness and Safety of Crovalimab for Children with Atypical Hemolytic Uremic Syndrome (aHUS)
- Study on the Effects and Safety of Crovalimab for Adults and Adolescents with Atypical Hemolytic Uremic Syndrome (aHUS)
- Poland
- Slovakia
- Slovenia
- Spain
- Study on Switching from Anti-C5 Antibody Therapy to Iptacopan for Patients with Atypical Hemolytic Uremic Syndrome (aHUS)
- Study on Ravulizumab for Children with Paroxysmal Nocturnal Hemoglobinuria or Atypical Hemolytic Uremic Syndrome
- Study on the Effectiveness and Safety of Crovalimab for Children with Atypical Hemolytic Uremic Syndrome (aHUS)
- Study on the Effects and Safety of Crovalimab for Adults and Adolescents with Atypical Hemolytic Uremic Syndrome (aHUS)
Study of Eculizumab in Adults with Hypertensive Emergency-Related Hemolytic Uremic Syndrome Requiring Dialysis or with Severe Kidney Problems
This trial focuses on a specific form of the condition that occurs when severe high blood pressure causes damage to blood cells and kidneys. The study is testing eculizumab, a medication given through intravenous infusion, in addition to standard blood pressure control treatment.
Who can participate: Adults aged 18 to 65 who have been hospitalized within the last 10 days with acute kidney failure requiring dialysis or high blood creatinine levels, blood cell breakdown showing anemia and low platelets, severe high blood pressure (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 after the last treatment.
Who cannot participate: People under 18 or over 65 years old, those who are pregnant or breastfeeding, anyone with previous organ transplantation, active cancer, severe heart disease, active infections including meningitis, known allergies to eculizumab, uncontrolled high blood pressure, severe liver disease, active autoimmune conditions, history of recent drug or alcohol abuse, or life expectancy less than 6 months.
Main goal: The purpose is to determine if early treatment with eculizumab can help prevent the need for ongoing dialysis in patients with severe kidney problems. The study will track how quickly patients recover and whether they still need dialysis after several months of treatment. Researchers will also examine how the body’s complement system responds to the treatment and look for specific genetic variations that might affect outcomes.
Study medication: Eculizumab works by blocking part of the body’s immune system called the complement system. The maximum daily dose is 1200 mg, with a total maximum dose of 9600 mg over the approximately 13-week treatment period.
Study on Long-Term Safety and Effects of Iptacopan for Patients with Atypical Hemolytic Uremic Syndrome (aHUS)
This clinical trial evaluates the long-term safety and effectiveness of iptacopan in individuals who have already participated in a previous study involving this medication. The treatment is taken as hard gelatin capsules.
Who can participate: Patients who have signed informed consent, completed a previous phase 3 study involving iptacopan and are currently benefiting from the treatment, have up-to-date vaccinations against Neisseria meningitidis, Streptococcus pneumoniae, and Haemophilus influenzae according to local health guidelines. Both males and females can participate.
Who cannot participate: Patients with other serious medical conditions that might interfere with the study, those who are pregnant or breastfeeding, anyone who participated in another clinical trial within the last 30 days, people with a history of allergic reactions to the study medication or similar drugs, those unable to follow study procedures, anyone with a history of drug or alcohol abuse, people with certain infections that could affect study results, those who have received medications that might interfere with the study, people with a history of cancer (except some types of skin cancer), or those with uncontrolled high blood pressure.
Main goal: The study monitors the long-term safety of iptacopan by checking for any side effects and changes in health through blood tests and heart monitoring. It also looks at how well the medication helps manage symptoms, such as maintaining healthy blood counts and kidney function, without the need for other treatments. The study is designed to last several years, until April 15, 2029.
Study medication: Iptacopan is taken orally as a pill. It works by targeting a specific part of the immune system thought to play a role in causing the blood and kidney problems associated with the condition.
Study on Switching from Anti-C5 Antibody Therapy to Iptacopan for Patients with Atypical Hemolytic Uremic Syndrome (aHUS)
This trial explores the effects of switching from current anti-C5 antibody therapy (such as eculizumab or ravulizumab) to iptacopan, which is taken as hard gelatin capsules.
Who can participate: Adults aged 18 or older who have been diagnosed with the condition (with other similar conditions ruled out), currently on anti-C5 antibody treatment for at least 3 months before the study starts, showing improvement from the treatment for at least 3 months with a platelet count of at least 150 x 10⁹/L, normal LDH levels, and stable or improving kidney function. Participants must be vaccinated against Neisseria meningitidis, Streptococcus pneumoniae, and Haemophilus influenzae at least 2 weeks before starting the new treatment.
Who cannot participate: Patients with other serious medical conditions that could interfere with the study, those currently participating in another clinical trial, anyone who has had recent major surgery or is planning surgery during the study, 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.
Main goal: The study evaluates the safety and effectiveness of iptacopan in preventing symptoms over a 12-month period. Participants will be monitored to see if they remain free from signs of thrombotic microangiopathy (TMA), a complication that affects small blood vessels. Regular check-ups and blood tests will assess blood and kidney health.
Study medication: Iptacopan is administered orally as tablets. It works by inhibiting a specific protein in the complement system, which is part of the immune system, thereby reducing inflammation and preventing blood vessel damage.
Study on Ravulizumab for Children with Paroxysmal Nocturnal Hemoglobinuria or Atypical Hemolytic Uremic Syndrome
This clinical trial is studying ravulizumab in children aged 2 to under 18 years who have either paroxysmal nocturnal hemoglobinuria (PNH) or atypical hemolytic uremic syndrome. The medication is administered through an injection under the skin.
Who can participate: Children between 2 and 17 years old weighing at least 10 kg (about 22 pounds), with a legal guardian who can give written consent. Participants must be vaccinated against meningococcal infection (groups A, C, Y, W135, and B) within the last 3 years or at least 2 weeks before starting the study, and vaccinated for Streptococcus pneumoniae and Haemophilus influenzae type b according to local guidelines. For those with the condition, specific diagnostic criteria and laboratory test requirements must be met.
Who cannot participate: Patients not diagnosed with PNH or aHUS, those not within the specified age range, anyone unable to follow study procedures, those with other medical conditions that might interfere with the study, anyone currently participating in another clinical trial, people with a history of allergic reactions to the study medication, pregnant or breastfeeding patients, and those who have received certain medications recently that might interfere with the study.
Main goal: The study will last for about a year, during which the effects of the medication will be closely monitored. This includes looking at how the body processes the drug and how it affects disease symptoms. The study will also assess safety and any side effects, with regular check-ups to monitor health and treatment effectiveness.
Study medication: Ravulizumab is a monoclonal antibody that 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.
Study on the Effectiveness and Safety of Crovalimab for Children with Atypical Hemolytic Uremic Syndrome (aHUS)
This trial tests crovalimab, given as a solution for injection or infusion, in pediatric patients who have not received any previous treatment that inhibits the complement system.
Who can participate: Patients must have been vaccinated against Neisseria meningitidis within the last 3 years before starting study treatment, following local guidelines for patients with complement deficiency. Female patients who can have children must agree to either not have heterosexual intercourse or use birth control during the study. For those new to treatment, the first signs of TMA must have appeared within 28 days before the first dose of crovalimab.
Who cannot participate: Patients who have previously received treatment with a complement inhibitor, those not within the specified age range for the study, anyone with medical conditions that would make participation unsafe, people unable to follow study procedures or attend required visits, and pregnant or breastfeeding patients.
Main goal: The study evaluates how effective and safe crovalimab is for children with the condition. It will monitor various health indicators, such as kidney function and blood parameters, over a treatment period of 24 weeks. The study will also track any side effects or reactions to the treatment, including injection site reactions and infections, and assess the presence of anti-drug antibodies.
Study medication: Crovalimab works by blocking a part of the immune system called the complement system, which is thought to play a role in the condition’s development. It is administered through subcutaneous (under the skin) and intravenous (into a vein) routes.
Study on the Effectiveness and Safety of Iptacopan for Adults with Atypical Hemolytic Uremic Syndrome Not Previously Treated with Complement Inhibitors
This clinical trial evaluates iptacopan, taken orally in the form of hard gelatin capsules, in adult patients who have not previously received complement inhibitor therapy.
Who can participate: Adults aged 18 years or older with signs of active thrombotic microangiopathy (TMA), 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. Participants must be vaccinated against Neisseria meningitidis, Streptococcus pneumoniae, and Haemophilus influenzae before starting study treatment.
Who cannot participate: Patients with a history of severe allergic reactions to the study medication, those currently participating in another clinical trial, people with a history of drug or alcohol abuse within the past year, pregnant or breastfeeding women, anyone with other medical conditions that study doctors believe would make participation unsafe, those who have received certain medications that might interfere with study treatment, people with a history of certain types of cancer, those with severe liver or kidney disease, anyone who has had recent major surgery, or people with uncontrolled high blood pressure.
Main goal: The purpose is to evaluate the effectiveness and safety of iptacopan over a period of 26 weeks. Participants will take the medication twice daily. Researchers will monitor whether participants achieve a complete response in their condition, meaning improvement in blood and kidney function without the need for other treatments like plasma exchange. The study will also look at how quickly participants respond to treatment, changes in blood parameters, kidney function, and overall health and well-being.
Study medication: Iptacopan is taken orally as tablets, twice daily. It works by inhibiting a component of the complement system, which is part of the immune system that can contribute to the disease process.
Study on the Effects and Safety of Crovalimab for Adults and Adolescents with Atypical Hemolytic Uremic Syndrome (aHUS)
This trial assesses crovalimab, administered as a solution for injection or infusion, for patients who have not previously received complement inhibitor treatment.
Who can participate: Patients must have been vaccinated against Neisseria meningitidis within the last 3 years, have adequate liver function (AST and ALT levels no more than 3 times the upper limit of normal with no clinical signs of cirrhosis), and female patients of childbearing potential must agree to abstinence or contraception. For new patients, the first signs of TMA must have appeared within 28 days before the first dose. For patients switching from other treatments, there must be clinical evidence of response shown by appropriate platelet counts, LDH levels, and stable or improving kidney function.
Who cannot participate: Patients who have previously received treatment with a complement inhibitor, those not within the specified age range, anyone with medical conditions that would make participation unsafe, people unable to follow study procedures or attend required visits, and pregnant or breastfeeding patients.
Main goal: The study evaluates how effective and safe crovalimab is for patients in controlling disease symptoms and improving overall health. Various health parameters are monitored, including the requirement for dialysis, changes in kidney function, blood parameters, and fatigue levels in adults. The study also tracks adverse events, including reactions at the injection site, infusion-related reactions, and infections. The primary goal is to determine the proportion of patients achieving a complete response to treatment.
Study medication: Crovalimab works by inhibiting the complement system. It is administered either subcutaneously or intravenously, depending on the specific requirements of the study. The study is estimated to conclude by December 7, 2029.
Summary
The seven clinical trials for atypical haemolytic uraemic syndrome span multiple European countries, with the highest concentration of studies in France, Italy, and Spain. Three main medications are being investigated: iptacopan (an oral medication tested in three trials), crovalimab (tested in three trials via injection or infusion), and eculizumab and ravulizumab (each tested in one trial). The trials address different patient populations, including adults who have never received complement inhibitor treatment, patients currently on anti-C5 antibody therapy who may benefit from switching to newer options, and pediatric patients aged 2 to 17 years. A notable focus across all trials is the requirement for vaccination against specific bacterial infections, particularly Neisseria meningitidis, before starting treatment, reflecting the increased infection risk associated with complement inhibition. Several studies are designed for long-term follow-up, extending until 2027-2029, which will provide valuable data on the sustained effectiveness and safety of these treatments. The research encompasses both immediate treatment responses and long-term outcomes, including kidney function preservation and the potential to reduce or eliminate the need for dialysis.