Paroxysmal nocturnal haemoglobinuria – European Clinical Trials Information Network https://clinicaltrials.eu Bridging Patients with Clinical Trials Wed, 24 Jun 2026 04:15:06 +0000 en-US hourly 1 https://wordpress.org/?v=7.0 https://clinicaltrials.eu/wp-content/uploads/2024/12/cropped-EU_icon-32x32.png Paroxysmal nocturnal haemoglobinuria – European Clinical Trials Information Network https://clinicaltrials.eu 32 32 Study of Pozelimab and Cemdisiran in Adults with Paroxysmal Nocturnal Hemoglobinuria Not Responding Well to Current C5 Inhibitor Treatment https://clinicaltrials.eu/trial/study-of-pozelimab-and-cemdisiran-in-adults-with-paroxysmal-nocturnal-hemoglobinuria-not-responding-well-to-current-c5-inhibitor-treatment/ Wed, 29 Apr 2026 15:07:35 +0000 https://clinicaltrials.eu/trial/study-of-pozelimab-and-cemdisiran-in-adults-with-paroxysmal-nocturnal-hemoglobinuria-not-responding-well-to-current-c5-inhibitor-treatment/ This study focuses on patients with Paroxysmal Nocturnal Hemoglobinuria (PNH), a rare blood disorder where the immune system attacks and destroys red blood cells. The study will test a combination of two medications: Pozelimab and Cemdisiran. This treatment is specifically intended for patients whose current PNH treatment is not working effectively to control the breakdown of red blood cells.

The study will evaluate how well the combination of Pozelimab (also known as REGN3918) and Cemdisiran (also known as ALN-CC5) works in treating PNH. Both medications will be given as injections – Pozelimab can be administered into a vein or under the skin, while Cemdisiran is given as an injection under the skin. The treatment period will last for approximately 80 weeks.

During the study, researchers will monitor how effectively this combination therapy controls the destruction of red blood cells in patients. They will also track other important factors such as changes in blood tests, energy levels, and any side effects that may occur. The study aims to help determine if this new combination treatment could be beneficial for PNH patients who are not responding well to their current therapy.

]]> Study on the Safety and Effectiveness of Crovalimab for Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) https://clinicaltrials.eu/trial/study-on-the-safety-and-effectiveness-of-crovalimab-for-patients-with-paroxysmal-nocturnal-hemoglobinuria-pnh/ Wed, 29 Apr 2026 15:05:24 +0000 https://clinicaltrials.eu/trial/study-on-the-safety-and-effectiveness-of-crovalimab-for-patients-with-paroxysmal-nocturnal-hemoglobinuria-pnh/ This clinical trial is focused on studying a condition called Paroxysmal Nocturnal Hemoglobinuria (PNH). PNH is a rare blood disease that causes red blood cells to break down too early, leading to various health issues. The study is testing a treatment called Crovalimab, which is a type of medication known as a monoclonal antibody. Monoclonal antibodies are proteins made in a lab that can bind to substances in the body, such as harmful cells, and help the immune system destroy them. Crovalimab is designed to block a part of the immune system that is overactive in people with PNH.

The purpose of the study is to evaluate the safety and effectiveness of Crovalimab in both healthy volunteers and patients with PNH. The study will be conducted in several parts. Initially, healthy volunteers will receive a single dose of Crovalimab to assess its safety. Following this, patients with PNH who have not been treated with similar medications before, as well as those switching from another treatment, will receive multiple doses over a period of five months. The study will also include an open-label extension phase to assess the long-term safety of Crovalimab.

Participants in the study will receive Crovalimab through injections or infusions, which are methods of delivering medication directly into the body. The study will monitor how the body processes the medication and its effects on the disease. This includes measuring changes in blood markers and symptoms related to PNH, such as fatigue and quality of life. The study aims to provide valuable information on how Crovalimab can help manage PNH and improve the lives of those affected by this condition.

]]> Study on the Safety and Effects of Iptacopan in Children with Paroxysmal Nocturnal Hemoglobinuria (PNH) Aged 2 to 17 Years https://clinicaltrials.eu/trial/study-on-the-safety-and-effects-of-iptacopan-in-children-with-paroxysmal-nocturnal-hemoglobinuria-pnh-aged-2-to-17-years/ Wed, 29 Apr 2026 15:05:18 +0000 https://clinicaltrials.eu/trial/study-on-the-safety-and-effects-of-iptacopan-in-children-with-paroxysmal-nocturnal-hemoglobinuria-pnh-aged-2-to-17-years/ This clinical trial is focused on studying a rare blood disease called Paroxysmal Nocturnal Hemoglobinuria (PNH) in children and teenagers aged 2 to under 18 years. The study will use a medication known as iptacopan hydrochloride, which is taken as hard gelatin capsules. The purpose of the study is to understand how the body processes this medication and to check its safety and how well it is tolerated by the participants.

Participants in the study will take iptacopan hydrochloride for a period of 26 weeks. During this time, researchers will monitor the participants to ensure the medication is safe and to observe any side effects. The study will also look at how the medication affects the levels of hemoglobin, a protein in red blood cells that carries oxygen, and other important health markers. Some participants may be switching from another treatment, while others may be new to this type of medication.

The study will not involve any placebo treatments. Participants will receive the actual medication throughout the study. The goal is to gather information that could help improve treatment options for children and teenagers with Paroxysmal Nocturnal Hemoglobinuria in the future. The study is expected to continue until 2031, allowing researchers to collect comprehensive data over time.

]]> Study on Optimizing Ravulizumab Dosing for Adults with Paroxysmal Nocturnal Hemoglobinuria (PNH) https://clinicaltrials.eu/trial/study-on-optimizing-ravulizumab-dosing-for-adults-with-paroxysmal-nocturnal-hemoglobinuria-pnh/ Wed, 29 Apr 2026 15:03:07 +0000 https://clinicaltrials.eu/trial/study-on-optimizing-ravulizumab-dosing-for-adults-with-paroxysmal-nocturnal-hemoglobinuria-pnh/ This clinical trial is focused on studying a rare blood disease called Paroxysmal Nocturnal Hemoglobinuria (PNH). PNH is a condition where red blood cells break down too early, leading to symptoms like fatigue and abdominal pain. The study will use a medication called Ravulizumab, which is also known by its code name ALXN1210. Ravulizumab is given as an infusion, which means it is administered directly into the bloodstream through a vein.

The purpose of the study is to compare two different ways of giving Ravulizumab to adults with PNH. One group will receive the standard dose, while the other group will receive a personalized dose tailored to their needs. The study aims to see how effective the personalized dosing is compared to the standard dosing. Participants will be monitored for changes in their symptoms and overall health during the study.

Throughout the study, researchers will look at various factors, such as the level of a substance called LDH in the blood, which can indicate how well the treatment is working. They will also assess the quality of life of participants and how often they need blood transfusions. The study will help determine if personalized dosing of Ravulizumab can provide better outcomes for people with PNH.

]]> Study of Danicopan with Ravulizumab or Eculizumab for Children with Paroxysmal Nocturnal Hemoglobinuria and Extravascular Hemolysis https://clinicaltrials.eu/trial/study-of-danicopan-with-ravulizumab-or-eculizumab-for-children-with-paroxysmal-nocturnal-hemoglobinuria-and-extravascular-hemolysis/ Wed, 29 Apr 2026 15:02:45 +0000 https://clinicaltrials.eu/trial/study-of-danicopan-with-ravulizumab-or-eculizumab-for-children-with-paroxysmal-nocturnal-hemoglobinuria-and-extravascular-hemolysis/ This clinical trial is focused on studying a rare blood disease called Paroxysmal Nocturnal Hemoglobinuria (PNH). PNH is a condition where red blood cells break down too early, leading to various health issues. The study is testing a treatment involving a medication called Danicopan, which is used in combination with other treatments like Ravulizumab or Eculizumab. These medications are designed to help manage the symptoms of PNH, particularly in children who experience significant breakdown of red blood cells outside of blood vessels, known as extravascular hemolysis.

The purpose of the study is to evaluate how effective Danicopan is when added to the existing treatments of Ravulizumab or Eculizumab. Participants in the study will take Danicopan in the form of a film-coated tablet, which is taken orally. The study will monitor changes in the participants’ health over a period of time, specifically looking at improvements in their blood levels and overall well-being. The study will last for several weeks, and participants will have regular check-ups to assess their progress.

This trial is open-label, meaning both the participants and the researchers know which treatment is being administered. The study aims to provide valuable information on how well Danicopan works in helping children with PNH manage their condition more effectively. Participants will be closely monitored to ensure their safety and to gather data on the treatment’s impact on their health.

]]> Study on the Safety and Effectiveness of Pozelimab and Cemdisiran for Adults with Paroxysmal Nocturnal Hemoglobinuria Not Recently Treated with Complement Inhibitors https://clinicaltrials.eu/trial/study-on-the-safety-and-effectiveness-of-pozelimab-and-cemdisiran-for-adults-with-paroxysmal-nocturnal-hemoglobinuria-not-recently-treated-with-complement-inhibitors/ Wed, 29 Apr 2026 15:02:05 +0000 https://clinicaltrials.eu/trial/study-on-the-safety-and-effectiveness-of-pozelimab-and-cemdisiran-for-adults-with-paroxysmal-nocturnal-hemoglobinuria-not-recently-treated-with-complement-inhibitors/ This clinical trial is focused on studying a rare blood disease called Paroxysmal Nocturnal Hemoglobinuria (PNH). PNH is a condition where red blood cells break down too early, leading to various health issues. The study is testing a combination of two treatments, Pozelimab and Cemdisiran, to see how safe and effective they are for patients with PNH who have not recently received or have never received treatment with complement inhibitors, which are drugs that help prevent the breakdown of red blood cells.

The purpose of the study is to compare the effects of the combination therapy of Pozelimab and Cemdisiran with other treatments, such as Ravulizumab and Eculizumab, which are also used to treat PNH. The study will last for 26 weeks, during which participants will receive either the combination therapy or one of the other treatments. The study will monitor how well the treatments control the breakdown of red blood cells and whether they help avoid the need for blood transfusions.

Participants will be divided into two groups, known as cohorts. One group will receive the combination therapy and be compared to those receiving Ravulizumab, while the other group will be compared to those receiving Eculizumab. The study will assess the effects of the treatments by measuring levels of a substance called lactate dehydrogenase (LDH) in the blood, which indicates how much red blood cell breakdown is occurring. The study aims to provide valuable information on the safety and effectiveness of the new combination therapy for managing PNH.

]]> Long-term safety and effectiveness study of pozelimab and cemdisiran combination treatment in adults with paroxysmal nocturnal hemoglobinuria (PNH) https://clinicaltrials.eu/trial/study-on-long-term-safety-and-effectiveness-of-pozelimab-and-cemdisiran-for-adults-with-paroxysmal-nocturnal-hemoglobinuria-pnh/ Wed, 29 Apr 2026 15:02:05 +0000 https://clinicaltrials.eu/trial/study-on-long-term-safety-and-effectiveness-of-pozelimab-and-cemdisiran-for-adults-with-paroxysmal-nocturnal-hemoglobinuria-pnh/ This study focuses on patients with Paroxysmal Nocturnal Hemoglobinuria (PNH), a rare blood disorder where the immune system attacks and destroys red blood cells, causing them to break down prematurely. The study will evaluate a combination treatment using two medications: Pozelimab and Cemdisiran. Both medications are given as solutions for injection under the skin.

The main purpose of this research is to understand how safe and effective this combination therapy is when used for a long time in patients with PNH. The study will look at how well the treatment works by monitoring various aspects of the disease, including the levels of a substance called lactate dehydrogenase (LDH) in the blood, which indicates the extent of red blood cell destruction.

During the study, participants will receive both medications through subcutaneous injections. The treatment period will last for approximately 108 weeks, during which researchers will monitor the patients’ response to the treatment and any side effects that may occur. The study will also track other important measures such as the need for blood transfusions and changes in patients’ quality of life.

]]> Study of pegcetacoplan safety and effectiveness in adolescents aged 12-17 with paroxysmal nocturnal hemoglobinuria https://clinicaltrials.eu/trial/study-on-the-safety-and-effects-of-pegcetacoplan-in-adolescents-aged-12-17-with-paroxysmal-nocturnal-hemoglobinuria/ Wed, 29 Apr 2026 14:59:36 +0000 https://clinicaltrials.eu/trial/study-on-the-safety-and-effects-of-pegcetacoplan-in-adolescents-aged-12-17-with-paroxysmal-nocturnal-hemoglobinuria/ This study focuses on Paroxysmal Nocturnal Hemoglobinuria, a rare blood disorder where the immune system attacks and destroys red blood cells. The study will evaluate a medication called pegcetacoplan, given as a solution that is injected under the skin. This medicine works by helping to protect red blood cells from being destroyed by the body’s immune system.

The purpose of this research is to understand how the medication works in young patients aged 12-17 years and to determine if it is safe and effective for treating their condition. The study will look at how the medication affects various aspects of the blood, including hemoglobin levels (a protein that carries oxygen in red blood cells) and other markers that show how well the treatment is working.

Participants will receive treatment with pegcetacoplan for 16 weeks, during which they will need regular check-ups to monitor their health and how well the medication is working. The medication will be given through injections under the skin. Throughout the study, doctors will closely monitor the participants’ blood test results and any side effects that may occur. The total study duration may extend up to 52 weeks to gather long-term safety and effectiveness information.

]]> Study on the Long-Term Safety and Effects of OMS906 for Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) https://clinicaltrials.eu/trial/study-on-the-long-term-safety-and-effects-of-oms906-for-patients-with-paroxysmal-nocturnal-hemoglobinuria-pnh/ Wed, 29 Apr 2026 14:29:35 +0000 https://clinicaltrials.eu/trial/study-on-the-long-term-safety-and-effects-of-oms906-for-patients-with-paroxysmal-nocturnal-hemoglobinuria-pnh/ This clinical trial is focused on studying a rare blood disease called Paroxysmal Nocturnal Hemoglobinuria (PNH). PNH is a condition where red blood cells break down too early, leading to various health issues. The study will use a treatment known as OMS906, which is given as a solution for injection. The purpose of the study is to evaluate the long-term safety and how well patients tolerate this treatment when it is administered every eight weeks.

Participants in the study will receive OMS906 through an intravenous injection, which means it will be delivered directly into a vein. The study will last for a period of up to two years, during which the effects of the treatment will be closely monitored. This includes checking for any side effects and assessing the overall health of the participants. The study aims to understand how safe the treatment is over a long period and how well it works in managing the symptoms of PNH.

Throughout the study, participants will have regular check-ups to monitor their health, including tests to measure their blood levels and other important health indicators. The study will also look at how the treatment affects the need for blood transfusions and whether it helps maintain healthy blood levels. By the end of the study, researchers hope to gather valuable information about the long-term use of OMS906 in treating PNH.

]]> Study on Long-Term Safety and Tolerability of Iptacopan for Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) https://clinicaltrials.eu/trial/study-on-long-term-safety-and-tolerability-of-iptacopan-for-patients-with-paroxysmal-nocturnal-hemoglobinuria-pnh/ Wed, 29 Apr 2026 14:28:47 +0000 https://clinicaltrials.eu/trial/study-on-long-term-safety-and-tolerability-of-iptacopan-for-patients-with-paroxysmal-nocturnal-hemoglobinuria-pnh/ This clinical trial is focused on studying the long-term safety and tolerability of a medication called iptacopan in patients with a rare blood disorder known as Paroxysmal Nocturnal Hemoglobinuria (PNH). PNH is a condition where red blood cells break down too early, leading to various health issues. The medication being tested, iptacopan, is taken in the form of hard gelatin capsules and is designed to help manage this condition.

The purpose of the study is to evaluate how safe and well-tolerated iptacopan is when used over a long period. Participants in this study are individuals who have already completed previous phases of clinical trials with iptacopan. The study will monitor participants for any side effects or health changes while they continue taking the medication. This includes regular check-ups and assessments to ensure the medication is working as intended without causing harm.

Throughout the study, participants will be observed for any adverse events, which are unexpected health issues that may arise. The study will also track the stability of hemoglobin levels, which are important for carrying oxygen in the blood, and check for any need for blood transfusions. Additionally, the study will look for any occurrences of breakthrough hemolysis, which is when red blood cells break down too quickly, and any major vascular events, which are serious issues related to blood vessels. The study aims to provide valuable information on the long-term use of iptacopan for managing PNH.

]]> Study Comparing Crovalimab and Eculizumab for Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) Currently on Complement Inhibitors https://clinicaltrials.eu/trial/study-comparing-crovalimab-and-eculizumab-for-patients-with-paroxysmal-nocturnal-hemoglobinuria-pnh-currently-on-complement-inhibitors/ Wed, 29 Apr 2026 14:28:31 +0000 https://clinicaltrials.eu/trial/study-comparing-crovalimab-and-eculizumab-for-patients-with-paroxysmal-nocturnal-hemoglobinuria-pnh-currently-on-complement-inhibitors/ This clinical trial is focused on studying a rare blood disease called Paroxysmal Nocturnal Hemoglobinuria (PNH). PNH is a condition where red blood cells break down too early, leading to various health issues. The study is comparing two treatments: Crovalimab and Eculizumab. Both are medications that help manage PNH by inhibiting a part of the immune system known as the complement system, which is overactive in people with PNH.

The purpose of the study is to evaluate the safety and tolerability of Crovalimab compared to Eculizumab. Participants in the study will receive either Crovalimab or Eculizumab through injections or infusions. The study will monitor participants over a period to observe any side effects and how well the treatments work in managing the symptoms of PNH. Some participants may receive a placebo, which is a substance with no active medication, to help compare the effects of the actual treatments.

Throughout the study, participants will have regular check-ups to assess their health and the effectiveness of the treatment. This includes monitoring vital signs, blood tests, and any reactions to the medication. The study aims to provide valuable information on how these treatments can help people with PNH and improve their quality of life. The trial is expected to continue until 2030, allowing for comprehensive data collection and analysis.

]]> Study on the Effectiveness and Safety of Crovalimab vs. Eculizumab for Adults and Adolescents with Paroxysmal Nocturnal Hemoglobinuria (PNH) Not Treated with Complement Inhibitors https://clinicaltrials.eu/trial/study-on-the-effectiveness-and-safety-of-crovalimab-vs-eculizumab-for-adults-and-adolescents-with-paroxysmal-nocturnal-hemoglobinuria-pnh-not-treated-with-complement-inhibitors/ Wed, 29 Apr 2026 14:27:53 +0000 https://clinicaltrials.eu/trial/study-on-the-effectiveness-and-safety-of-crovalimab-vs-eculizumab-for-adults-and-adolescents-with-paroxysmal-nocturnal-hemoglobinuria-pnh-not-treated-with-complement-inhibitors/ This clinical trial is focused on studying a rare blood disease called Paroxysmal Nocturnal Hemoglobinuria (PNH). PNH is a condition where red blood cells break down too early, leading to various health issues. The study is comparing two treatments: Crovalimab and Eculizumab. Crovalimab is a solution that can be given either through an injection under the skin or directly into a vein, while Eculizumab is given through a vein. Both treatments are designed to help manage the symptoms of PNH by preventing the breakdown of red blood cells.

The purpose of this study is to evaluate how effective and safe Crovalimab is compared to Eculizumab in patients who have not been treated with similar medications before. Participants in the study will receive either Crovalimab or Eculizumab and will be monitored over a period of time to see how well the treatments work in preventing blood transfusions and controlling the breakdown of red blood cells. The study will also look at other factors such as the stability of hemoglobin levels, which is a protein in red blood cells that carries oxygen, and any side effects that may occur.

Throughout the study, researchers will collect information on how patients respond to the treatments, including any changes in fatigue levels, which is a common symptom of PNH. They will also monitor for any reactions at the injection site or other side effects. The study aims to provide valuable insights into the management of PNH and help determine the best treatment options for patients with this condition.

]]> Study on the Long-term Safety and Effectiveness of Danicopan with Ravulizumab or Eculizumab for Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) https://clinicaltrials.eu/trial/study-on-the-long-term-safety-and-effectiveness-of-danicopan-with-ravulizumab-or-eculizumab-for-patients-with-paroxysmal-nocturnal-hemoglobinuria-pnh/ Wed, 29 Apr 2026 14:27:43 +0000 https://clinicaltrials.eu/trial/study-on-the-long-term-safety-and-effectiveness-of-danicopan-with-ravulizumab-or-eculizumab-for-patients-with-paroxysmal-nocturnal-hemoglobinuria-pnh/ This clinical trial is focused on studying a rare blood disease called Paroxysmal Nocturnal Hemoglobinuria (PNH). PNH is a condition where red blood cells break down too early, leading to various health issues. The study is testing a treatment called danicopan, which is used as an additional therapy alongside another type of medication known as a Complement Component 5 Inhibitor (C5i). The C5i medications being used in this study include Ultomiris and Soliris. Danicopan is taken as a film-coated tablet, while Ultomiris and Soliris are given as solutions for infusion, which means they are administered directly into the bloodstream through a vein.

The purpose of this study is to understand the long-term safety of using danicopan as an add-on treatment for patients with PNH who have previously been treated with danicopan in another study. Participants will continue their treatment over a period of time, during which they will receive either danicopan with a C5i or a placebo. The study will monitor any side effects and changes in health, such as hemoglobin levels, which is a protein in red blood cells that carries oxygen, and other blood-related measurements.

Throughout the study, participants will have regular check-ups to track their health and any changes in their condition. The study aims to gather information on how well danicopan works when used with C5i medications and to ensure it is safe for long-term use. This research is important for improving treatment options for people living with PNH.

]]> Study on the Effectiveness and Safety of Iptacopan for Adults with Paroxysmal Nocturnal Hemoglobinuria (PNH) Switching from Anti-C5 Antibody Treatment https://clinicaltrials.eu/trial/study-on-the-effectiveness-and-safety-of-iptacopan-for-adults-with-paroxysmal-nocturnal-hemoglobinuria-pnh-switching-from-anti-c5-antibody-treatment/ Wed, 29 Apr 2026 14:26:53 +0000 https://clinicaltrials.eu/trial/study-on-the-effectiveness-and-safety-of-iptacopan-for-adults-with-paroxysmal-nocturnal-hemoglobinuria-pnh-switching-from-anti-c5-antibody-treatment/ This clinical trial is focused on studying a rare blood disease called Paroxysmal Nocturnal Hemoglobinuria (PNH). PNH is a condition where red blood cells break down too early, leading to various health issues. The trial will use a treatment called iptacopan, which is taken as hard gelatin capsules. Iptacopan is also known by its code name LNP023. The purpose of the study is to evaluate if iptacopan is as effective as the current standard treatment for PNH when patients switch to it.

Participants in the study will be adults who have been previously treated with an anti-C5 antibody, a type of medication used to manage PNH. The study will involve taking iptacopan orally twice a day. The trial will monitor changes in hemoglobin levels, which is a measure of the amount of red blood cells in the blood, over a period of time. This will help determine the effectiveness and safety of iptacopan for people with PNH.

The study is designed to last for a specific period, during which participants will be regularly assessed to track their response to the treatment. The goal is to see if iptacopan can maintain or improve hemoglobin levels in patients who switch from their current treatment. This trial is an important step in exploring new treatment options for individuals living with PNH.

]]> 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.

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