Atypical haemolytic uraemic syndrome – European Clinical Trials Information Network https://clinicaltrials.eu Bridging Patients with Clinical Trials Fri, 12 Jun 2026 10:34:59 +0000 en-US hourly 1 https://wordpress.org/?v=7.0 https://clinicaltrials.eu/wp-content/uploads/2024/12/cropped-EU_icon-32x32.png Atypical haemolytic uraemic syndrome – European Clinical Trials Information Network https://clinicaltrials.eu 32 32 Study on the Effectiveness and Safety of Crovalimab for Children with Atypical Hemolytic Uremic Syndrome (aHUS) https://clinicaltrials.eu/trial/study-on-the-effectiveness-and-safety-of-crovalimab-for-children-with-atypical-hemolytic-uremic-syndrome-ahus/ Mon, 04 May 2026 07:56:00 +0000 https://clinicaltrials.eu/trial/study-on-the-effectiveness-and-safety-of-crovalimab-for-children-with-atypical-hemolytic-uremic-syndrome-ahus/ This clinical trial is focused on studying a rare disease called Atypical Hemolytic Uremic Syndrome (aHUS), which affects the blood and kidneys. The study is testing a treatment called Crovalimab, which is given as a solution for injection or infusion. The purpose of the study is to evaluate how effective and safe Crovalimab is for children who have not received any previous treatment that inhibits a part of the immune system known as the complement system.

Participants in the study will receive Crovalimab and will be monitored over a period of time to see how their condition responds to the treatment. The study will look at various health indicators, such as kidney function and blood parameters, to determine the treatment’s impact. The study will also monitor any side effects or reactions to the treatment, including those at the injection site or related to the immune system.

The study aims to gather information on how well Crovalimab works in managing aHUS and its potential benefits and risks. This information will help in understanding the treatment’s effectiveness and safety for children with this condition. The study is expected to continue until 2029, providing valuable insights into the management of aHUS in pediatric patients.

]]> Study of Eculizumab in Adults with Hypertensive Emergency-Related Hemolytic Uremic Syndrome Requiring Dialysis or with Severe Kidney Problems https://clinicaltrials.eu/trial/study-on-eculizumab-for-adults-with-hypertensive-emergency-associated-hemolytic-uremic-syndrome-and-severe-kidney-issues/ Wed, 29 Apr 2026 14:57:57 +0000 https://clinicaltrials.eu/trial/study-on-eculizumab-for-adults-with-hypertensive-emergency-associated-hemolytic-uremic-syndrome-and-severe-kidney-issues/ This study focuses on Hemolytic Uremic Syndrome associated with hypertensive emergency, a condition where severe high blood pressure causes damage to blood cells and kidneys. The study will evaluate a medication called eculizumab, which is given through intravenous infusion, in addition to standard blood pressure control treatment. This rare condition can lead to kidney failure requiring dialysis and may affect other organs including the brain, heart, and eyes.

The purpose of this research 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 last for approximately 13 weeks, during which participants will receive either eculizumab or standard treatment alone. The maximum daily dose of eculizumab will be 1200 mg, with a total maximum dose of 9600 mg over the treatment period.

Patients will be monitored for various health indicators, including kidney function, blood cell counts, and potential complications. The study will track how quickly patients recover from their condition and whether they still need dialysis after several months of treatment. Researchers will also examine how the body’s complement system (a part of the immune system) responds to the treatment and look for specific genetic variations that might affect treatment outcomes.

]]> Study on Switching from Anti-C5 Antibody Therapy to Iptacopan for Patients with Atypical Hemolytic Uremic Syndrome (aHUS) https://clinicaltrials.eu/trial/study-on-switching-from-anti-c5-antibody-therapy-to-iptacopan-for-patients-with-atypical-hemolytic-uremic-syndrome-ahus/ Wed, 29 Apr 2026 14:57:21 +0000 https://clinicaltrials.eu/trial/study-on-switching-from-anti-c5-antibody-therapy-to-iptacopan-for-patients-with-atypical-hemolytic-uremic-syndrome-ahus/ This clinical trial is focused on studying a rare disease called Atypical Hemolytic Uremic Syndrome (aHUS). This condition affects the blood and kidneys, leading to problems like low platelet counts, destruction of red blood cells, and kidney failure. The study will explore the effects of a treatment called iptacopan, which is taken as hard gelatin capsules. Participants in the study will switch from their current treatment, an anti-C5 antibody therapy, to iptacopan.

The purpose of the study is to evaluate the safety and effectiveness of iptacopan in preventing the symptoms of aHUS over a 12-month period. Participants will be monitored to see if they remain free from signs of a condition called Thrombotic Microangiopathy (TMA), which is a complication of aHUS that affects small blood vessels. The study will involve regular check-ups and blood tests to assess the health of the participants’ blood and kidneys.

Throughout the study, the health of participants will be closely observed, including any changes in blood and kidney function. The study will also track any side effects or serious health events that may occur. The goal is to determine if iptacopan can be a safe and effective alternative to the current anti-C5 antibody treatments for managing aHUS.

]]> Study on the Effects and Safety of Crovalimab for Adults and Adolescents with Atypical Hemolytic Uremic Syndrome (aHUS) https://clinicaltrials.eu/trial/study-on-the-effects-and-safety-of-crovalimab-for-adults-and-adolescents-with-atypical-hemolytic-uremic-syndrome-ahus/ Wed, 29 Apr 2026 14:56:40 +0000 https://clinicaltrials.eu/trial/study-on-the-effects-and-safety-of-crovalimab-for-adults-and-adolescents-with-atypical-hemolytic-uremic-syndrome-ahus/ This clinical trial is focused on studying atypical Hemolytic Uremic Syndrome (aHUS), a rare disease that affects the blood and kidneys. The study will evaluate a treatment called Crovalimab, which is administered as a solution for injection or infusion. The purpose of the study is to assess how effective and safe Crovalimab is for patients who have not previously received treatment that inhibits a part of the immune system known as the complement system.

Participants in the study will receive Crovalimab through injections under the skin or infusions into a vein. The study will monitor various health indicators, such as kidney function and blood health, to determine the treatment’s impact. The study will also track any side effects or reactions to the treatment, including changes in fatigue levels and any adverse events that may occur.

The trial aims to understand how well Crovalimab works in controlling the symptoms of aHUS and improving patients’ overall health. The study will gather information over a period of time to see how patients respond to the treatment and whether it helps maintain control over the disease. This research is important for developing new treatment options for people living with aHUS.

]]> Study on the Effectiveness and Safety of Iptacopan for Adults with Atypical Hemolytic Uremic Syndrome Not Previously Treated with Complement Inhibitors https://clinicaltrials.eu/trial/study-on-the-effectiveness-and-safety-of-iptacopan-for-adults-with-atypical-hemolytic-uremic-syndrome-not-previously-treated-with-complement-inhibitors/ Wed, 29 Apr 2026 14:27:51 +0000 https://clinicaltrials.eu/trial/study-on-the-effectiveness-and-safety-of-iptacopan-for-adults-with-atypical-hemolytic-uremic-syndrome-not-previously-treated-with-complement-inhibitors/ This clinical trial is focused on studying a condition called atypical hemolytic uremic syndrome (aHUS). aHUS is a rare disease that affects the blood and kidneys, leading to problems like low platelet counts, destruction of red blood cells, and kidney damage. The treatment being tested in this study is a medication called iptacopan, which is taken orally in the form of hard gelatin capsules. Iptacopan is also known by its code name, LNP023.

The purpose of the study is to evaluate the effectiveness and safety of iptacopan in adult patients with aHUS who have not previously received a type of treatment known as complement inhibitor therapy. Participants in the study will take iptacopan twice daily for a period of 26 weeks. During this time, researchers will monitor the participants to see if they achieve a complete response in their condition, which means improvement in blood and kidney function without the need for other treatments like plasma exchange or specific antibodies.

The study will also look at how quickly participants respond to the treatment, changes in blood parameters, kidney function, and overall health and well-being. Safety evaluations will be conducted to monitor any side effects or adverse events. The study aims to provide valuable information on the potential benefits of iptacopan for individuals with aHUS.

]]> Study on Ravulizumab for Children with Paroxysmal Nocturnal Hemoglobinuria or Atypical Hemolytic Uremic Syndrome https://clinicaltrials.eu/trial/study-on-ravulizumab-for-children-with-paroxysmal-nocturnal-hemoglobinuria-or-atypical-hemolytic-uremic-syndrome/ Wed, 29 Apr 2026 14:23:43 +0000 https://clinicaltrials.eu/trial/study-on-ravulizumab-for-children-with-paroxysmal-nocturnal-hemoglobinuria-or-atypical-hemolytic-uremic-syndrome/ This clinical trial is focused on studying two rare blood disorders: Paroxysmal Nocturnal Hemoglobinuria (PNH) and Atypical Hemolytic Uremic Syndrome (aHUS). These conditions can cause severe health issues, including the destruction of red blood cells, blood clots, and kidney problems. The study will use a treatment called Ravulizumab, which is a type of medication known as a monoclonal antibody. This medication is designed to help manage these disorders by targeting specific proteins in the body that contribute to the symptoms.

The purpose of the study is to understand how Ravulizumab works in children aged 2 to under 18 years who have either PNH or aHUS. Participants will receive the medication through an injection under the skin. The study will last for about a year, during which time the effects of the medication will be closely monitored. This includes looking at how the body processes the drug and how it affects the symptoms of the diseases. The study will also assess the safety of the medication and any side effects that may occur.

Throughout the study, participants will have regular check-ups to monitor their health and the effectiveness of the treatment. The study aims to provide valuable information that could improve the management of PNH and aHUS in children. By participating, researchers hope to gather data that could lead to better treatment options for these serious conditions. The study will also evaluate the use of a special device that helps deliver the medication, ensuring it is administered correctly and safely.

]]> Study on Long-Term Safety and Effects of Iptacopan for Patients with Atypical Hemolytic Uremic Syndrome (aHUS) https://clinicaltrials.eu/trial/study-on-long-term-safety-and-effects-of-iptacopan-for-patients-with-atypical-hemolytic-uremic-syndrome-ahus/ Wed, 29 Apr 2026 14:22:53 +0000 https://clinicaltrials.eu/trial/study-on-long-term-safety-and-effects-of-iptacopan-for-patients-with-atypical-hemolytic-uremic-syndrome-ahus/ This clinical trial is focused on studying a rare disease called Atypical Hemolytic Uremic Syndrome (aHUS). This condition affects the blood and kidneys, leading to problems like low platelet counts, destruction of red blood cells, and kidney failure. The treatment being tested in this study is a medication called Iptacopan, which is taken as hard gelatin capsules. The purpose of the study is to evaluate the long-term safety and tolerability of Iptacopan in individuals with aHUS who have already participated in a previous study involving this medication.

Participants in this study will continue taking Iptacopan to see how well it works over a longer period. The study will monitor the safety of the medication by checking for any side effects and changes in health, such as blood tests and heart monitoring. The study will also look at how well the medication helps manage the symptoms of aHUS, like maintaining healthy blood counts and kidney function, without the need for other treatments.

The study is designed to last several years, allowing researchers to gather comprehensive data on the effects of Iptacopan over time. Participants will be regularly assessed to ensure their safety and to track the effectiveness of the treatment in managing aHUS symptoms. This research aims to provide valuable information on the long-term use of Iptacopan for people living with aHUS.

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