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Ongoing Clinical Trials for Paroxysmal Nocturnal Haemoglobinuria

Paroxysmal nocturnal haemoglobinuria is a rare blood disorder where the immune system attacks and destroys red blood cells. There are currently 14 clinical trials exploring new treatment options across multiple European countries, testing various medications including complement inhibitors and combination therapies to improve disease management and patient quality of life.

Clinical trial locations

Long-term safety and effectiveness study of pozelimab and cemdisiran combination treatment in adults with paroxysmal nocturnal hemoglobinuria (PNH)

This study focuses on the long-term safety and effectiveness of a combination therapy using two medications: pozelimab and cemdisiran. Both are administered as subcutaneous injections. The trial is designed for adults with PNH who have previously participated in a related study and received this combination treatment without permanently stopping it.

Inclusion criteria: Participants must have completed the previous parent study for PNH treatment without permanently discontinuing the medication. They must be willing to attend clinic visits and receive required meningococcal vaccinations. For patients with a specific genetic variation called C5 polymorphism, documented confirmation of this variation is required along with confirmed PNH diagnosis through high-sensitivity flow cytometry. These patients must also have active disease with at least one PNH-related symptom and lactate dehydrogenase levels at least two times higher than normal. The study includes adults and adolescents aged 12 years and older, both males and females.

Exclusion criteria: Patients with a history of bone marrow failure syndrome, active or chronic bacterial infections, viral hepatitis, or HIV infection cannot participate. Those with a history of major organ transplantation, active cancer or cancer within the past five years, severe kidney disease requiring dialysis, or severe liver disease are excluded. Patients with blood clotting disorders, severe heart conditions, or uncontrolled high blood pressure are also ineligible. Recent participation in another clinical trial within 30 days, pregnancy or breastfeeding, known allergic reactions to similar medications, and mental conditions affecting informed consent are additional exclusion factors.

Focus: The study evaluates the long-term safety and effectiveness of combining pozelimab and cemdisiran. Participants undergo regular monitoring of blood levels, including lactate dehydrogenase which indicates red blood cell destruction, and hemoglobin levels. Safety assessments include monitoring for side effects, meningococcal vaccinations as required, and reporting any unusual symptoms. Quality of life evaluations assess fatigue levels and physical function. The study continues until May 2029 with regular clinic visits throughout the period.

Investigational drugs: Pozelimab is a monoclonal antibody that targets specific proteins in the immune system involved in red blood cell destruction. Cemdisiran reduces the activity of certain proteins contributing to red blood cell breakdown. The combination aims to provide better disease control than either medication alone.

Study of Danicopan with Ravulizumab or Eculizumab for Children with Paroxysmal Nocturnal Hemoglobinuria and Extravascular Hemolysis

This clinical trial evaluates danicopan as an additional treatment for children with PNH who experience significant breakdown of red blood cells outside blood vessels, known as extravascular hemolysis. The medication is used in combination with either ravulizumab or eculizumab. Danicopan is taken as a film-coated tablet orally.

Inclusion criteria: Participants must be between 12 and under 18 years old at the time of consent and weigh more than 25 kg. They must have a confirmed diagnosis of PNH with anemia showing hemoglobin levels of 10.5 g/dL or lower and an elevated reticulocyte count of 100 x 109/L or more. Participants must have been treated with either ravulizumab or eculizumab for at least 12 weeks with a stable dose. Required vaccinations include meningococcal infection from specific serogroups, Haemophilus influenzae type b, and Streptococcus pneumoniae. Female participants of childbearing potential must use effective contraception and have negative pregnancy tests. A legal guardian must be available to assist with study visits and maintaining health records, and participants must have access to emergency medical care.

Exclusion criteria: Patients with any other serious health condition that might interfere with the study are excluded. Those currently participating in another clinical trial, who have had recent major surgery or are planning surgery during the study, or who have known allergies to study medications cannot participate. Other exclusions include inability to comply with study procedures, pregnancy or breastfeeding, history of drug or alcohol abuse, infections requiring antibiotic treatment, history of cancer except certain skin cancers, and conditions affecting the immune system.

Focus: The study involves using danicopan as an additional treatment alongside ravulizumab or eculizumab. Regular monitoring occurs to assess effectiveness through changes in hemoglobin levels, reticulocyte counts, and quality of life scores. Evaluations take place at weeks 12 and 24, monitoring the need for blood transfusions and changes in fatigue levels. The study is expected to continue until February 2028.

Investigational drugs: Danicopan is being studied as an add-on treatment to help improve blood levels. Ravulizumab and eculizumab are medications that prevent red blood cell breakdown by blocking part of the immune system that attacks these cells.

Study of pegcetacoplan safety and effectiveness in adolescents aged 12-17 with paroxysmal nocturnal hemoglobinuria

This study evaluates the medication pegcetacoplan in adolescents aged 12-17 years with PNH. The medication is administered as a solution injected under the skin and works by helping to protect red blood cells from destruction by the immune system. The study assesses how the medication affects various blood parameters and determines its safety and effectiveness in this younger age group.

Inclusion criteria: Participants must be between 12 and 17 years old, weighing at least 20 kilograms with a body mass index below the 95th percentile for their age. They must have confirmed PNH diagnosis through special blood testing showing specific cell populations above 10 percent. Participants can be treatment-naive with low blood hemoglobin and high LDH levels, or currently on stable complement inhibitor treatment with low blood hemoglobin. Blood requirements include platelet count above 75,000 per cubic millimeter and neutrophil count above 1,000 per cubic millimeter. Participants must be willing to self-administer medication or have a caregiver who can. They must be vaccinated against specific bacteria or agree to receive vaccines within 14 days. Females of childbearing potential must have negative pregnancy tests and use approved birth control. Sexually mature males must use birth control and not donate sperm during the study and for 90 days after. Participants must maintain stable doses of current medications for specified periods and provide written informed consent.

Exclusion criteria: Exclusions include current or recent pregnancy or breastfeeding within three months, history of severe allergic reactions to medications, active uncontrolled bacterial infections, and severe liver problems. Other exclusions include major surgery in the past three months, participation in other clinical trials within 30 days, active cancer or cancer treatment, severe heart conditions or uncontrolled high blood pressure, history of blood clotting disorders, inability to follow study procedures, use of certain medications that might interact with the study drug, severe kidney disease, mental conditions affecting informed consent ability, and drug or alcohol abuse within the past year.

Focus: The treatment begins with pegcetacoplan at a dose of 1,080 mg given subcutaneously. Participants or caregivers learn to give injections. Required vaccinations are administered within 14 days if not already vaccinated. The first 16 weeks involve regular blood tests monitoring hemoglobin levels, LDH levels, and red blood cell counts. Treatment continues for up to 52 weeks with ongoing monitoring including complement system activity, quality of life measurements, and number of blood transfusions needed. Final evaluation occurs after the full 52-week period.

Investigational drugs: Pegcetacoplan is a medication designed to regulate part of the body’s complement system, helping protect red blood cells from destruction in patients with PNH.

Study on Optimizing Ravulizumab Dosing for Adults with Paroxysmal Nocturnal Hemoglobinuria (PNH)

This clinical trial compares two different ways of administering ravulizumab to adults with PNH. One group receives the standard dose while the other receives a personalized dose tailored to individual needs. The study aims to determine if personalized dosing provides better outcomes.

Inclusion criteria: Patients must be eligible for treatment with ravulizumab according to relevant Dutch guidelines or already receiving ravulizumab treatment. They must be willing to provide written informed consent and be 16 years or older.

Exclusion criteria: Patients without PNH, those who are not adults, and those from vulnerable populations cannot participate.

Focus: Upon joining, an initial assessment confirms eligibility. Treatment begins with ravulizumab administered as an infusion, using either a standard or individualized dosing regimen. Throughout the trial, response to treatment is monitored by measuring changes in lactate dehydrogenase levels and ensuring ravulizumab blood concentrations remain above 100 mg/L. The study assesses breakthrough hemolysis occurrence, defined as new or worsening symptoms like fatigue, dark urine, abdominal pain, shortness of breath, or anemia. Quality of life is evaluated using the FACIT-Fatigue Scale. Differences between dosing strategies are analyzed, including number of transfusions required, cumulative dose over 52 weeks, number of infusions, and physician appointments. The trial is expected to conclude by December 16, 2027.

Investigational drugs: Ravulizumab is a medication used to prevent the breakdown of red blood cells by blocking part of the immune system that leads to cell destruction.

Study on the Safety and Effectiveness of Pozelimab and Cemdisiran for Adults with Paroxysmal Nocturnal Hemoglobinuria Not Recently Treated with Complement Inhibitors

This study tests a combination of two treatments, pozelimab and cemdisiran, to evaluate their safety and effectiveness for patients with PNH who have not recently received or have never received treatment with complement inhibitors. The study compares the combination therapy effects with other treatments such as ravulizumab and eculizumab.

Inclusion criteria: Participants must have a confirmed PNH diagnosis through high-sensitivity flow cytometry. They must have active PNH with one or more related signs or symptoms and lactate dehydrogenase levels at least twice the upper limit of normal. Participants must be willing to attend clinic or remote visits, follow study procedures, and complete all required meningococcal vaccinations. Both males and females within the specified age range can participate.

Exclusion criteria: Patients who have recently received complement inhibitor treatment cannot participate. Those who are not treatment-naive for complement inhibitors are excluded.

Focus: The initial visit confirms eligibility through tests including high-sensitivity flow cytometry. Participants are randomly assigned to receive either the pozelimab and cemdisiran combination or ravulizumab/eculizumab. If assigned to the combination group, participants receive medications as solutions for injection with frequency and dosage explained by the study team. If in the ravulizumab or eculizumab group, medications are given as solutions for infusion. Throughout the 26-week treatment period, regular clinic visits monitor health and treatment effects. Blood tests measure lactate dehydrogenase levels to assess impact on hemolysis. Follow-up visits after treatment completion ensure well-being and gather long-term effects data.

Investigational drugs: Pozelimab and cemdisiran are medications being studied for their ability to control the overactive part of the immune system causing red blood cell breakdown. Ravulizumab and eculizumab are existing treatments used as comparisons.

Study on the Safety and Effects of Iptacopan in Children with Paroxysmal Nocturnal Hemoglobinuria (PNH) Aged 2 to 17 Years

This clinical trial focuses on understanding how the body processes iptacopan hydrochloride in children and teenagers aged 2 to under 18 years with PNH. The medication is taken as hard gelatin capsules and the study checks its safety and tolerability.

Inclusion criteria: Participants must be between 2 and 17 years old, with a confirmed PNH diagnosis through high-sensitivity flow cytometry. They must have a clone size of granulocytes/monocytes of 10 percent or more. Cohort 1 participants must weigh at least 35 kg. Those already on anti-C5 therapy must have been on a stable dose for at least six months. Treatment-naive participants must have hemoglobin levels less than 10 g/dL and lactate dehydrogenase levels more than 1.5 times the upper limit of normal. Participants must be vaccinated against Neisseria meningitidis and Streptococcus pneumoniae at least two weeks before starting treatment. Vaccination against Haemophilus influenzae is recommended at least two weeks before treatment.

Exclusion criteria: Patients with a history of allergic reactions to the study medication, those taking other medications that might interfere with the study drug, those with severe liver or kidney disease, pregnant or breastfeeding women, those with a history of certain types of cancer, recent participants in another clinical trial within 30 days, those with uncontrolled high blood pressure, and those with active infections requiring treatment cannot participate.

Focus: Upon joining, participants are informed about the trial’s purpose to assess iptacopan hydrochloride safety and tolerability. Participants must be vaccinated against certain infections at least two weeks before starting medication. Iptacopan hydrochloride is taken as hard gelatin capsules by mouth with dosage determined by the study team. The treatment period lasts 26 weeks with regular monitoring for side effects and health changes. Safety evaluations include monitoring adverse effects, laboratory parameters, vital signs, and cardiovascular health. Final evaluation occurs at the end of 26 weeks to assess overall medication impact.

Investigational drugs: Iptacopan is a medication being studied for its safety and body processing in young patients with PNH over a 26-week period.

Study Comparing Crovalimab and Eculizumab for Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) Currently on Complement Inhibitors

This study compares two treatments: crovalimab and eculizumab. Both are medications that help manage PNH by inhibiting the complement system. The study evaluates the safety and tolerability of crovalimab compared to eculizumab.

Inclusion criteria: Participants must weigh at least 40 kg with a confirmed PNH diagnosis through high sensitivity flow cytometry. Platelet count must be at least 30,000 per cubic millimeter without needing transfusion within seven days before testing. For Arm A and B patients, participants must be 18 years or older and have been treated with eculizumab for at least 24 weeks with lactate dehydrogenase levels no more than 1.5 times the upper limit of normal. Arm C participants must be under 18 years old and currently treated with eculizumab, or currently treated with ravulizumab, or treated with higher-than-approved eculizumab doses, or have a known C5 polymorphism with poorly controlled hemolysis. In France and Czech Republic, patients under 18 years are not eligible.

Exclusion criteria: Patients with severe allergic reactions history to study medications, those who received another investigational drug within 30 days, those with active infections requiring antibiotics, those with certain cancer types history, pregnant or breastfeeding women, those with drug or alcohol abuse history, those with certain heart conditions, those who had major surgery within three months, those with uncontrolled high blood pressure, and those with severe liver or kidney disease are excluded.

Focus: Initial assessment confirms eligibility including documented PNH diagnosis, body weight verification, platelet count and LDH level checking. Participants are randomly assigned to study groups. Arm A and B patients must be at least 18 years old with at least 24 weeks of eculizumab treatment. Arm C includes those under 18 years or with specific treatment histories. Participants receive either crovalimab or eculizumab with dosage depending on treatment group. Regular monitoring assesses safety and effectiveness including adverse events, vital signs, and laboratory results. Evaluations measure crovalimab or eculizumab blood concentration, anti-drug antibodies presence, and disease-related biomarker changes. Effectiveness is assessed by measuring hemolysis parameter changes, LDH levels, and hemoglobin stabilization over 25 weeks. Study completion includes questionnaires assessing treatment satisfaction and preference. The study concludes by January 31, 2030.

Investigational drugs: Crovalimab is being studied for treating PNH by inhibiting certain blood proteins causing red blood cell damage. Eculizumab is used as a comparison.

Study on Long-Term Safety and Tolerability of Iptacopan for Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH)

This clinical trial studies the long-term safety and tolerability of iptacopan in PNH patients. The medication is taken as hard gelatin capsules. Participants are individuals who have already completed previous phases of clinical trials with iptacopan.

Inclusion criteria: Participants must be 18 years or older with a PNH diagnosis. They must have completed the treatment extension period of previous Phase II or any Phase III clinical studies with iptacopan. All necessary vaccinations against Neisseria meningitidis, Streptococcus pneumoniae, and Haemophilus influenzae must be received. The study doctor must believe the participant may benefit from continuing iptacopan treatment with stability on this treatment for at least three months.

Exclusion criteria: Participants who have not completed the treatment extension period of previous Phase II and any Phase III clinical studies with iptacopan cannot participate.

Focus: Participation begins after completing previous Phase II or Phase III studies with iptacopan. Eligibility requires PNH diagnosis and clinical stability on iptacopan for at least three months with up-to-date vaccinations. Participants receive iptacopan as hard gelatin capsules taken orally. Regular safety evaluations monitor adverse events with periodic laboratory tests and vital sign checks. The study maintains hemoglobin levels and aims to avoid blood transfusions. Yearly follow-up intervals assess blood transfusion absence and breakthrough hemolysis occurrences. Monitoring for Major Adverse Vascular Events is included. The study ends by February 7, 2028, with final evaluations at study period end.

Investigational drugs: Iptacopan is a medication being studied for its long-term safety and tolerability in PNH patients who have completed earlier study phases.

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

This clinical trial studies ravulizumab, a monoclonal antibody medication, in children aged 2 to under 18 years with either PNH or atypical Hemolytic Uremic Syndrome. Participants receive medication through an injection under the skin. The study lasts about a year monitoring medication effects.

Inclusion criteria: Participants must be between 2 and 17 years old, either male or female, weighing at least 10 kg. A legal guardian must provide written consent with participant assent if required. Participants must be vaccinated against meningococcal infection, Streptococcus pneumoniae, and Haemophilus influenzae type b according to local guidelines. For PNH participants, confirmed diagnosis through specific blood tests is required. Treatment-naive participants must have shown PNH-related symptoms in the last three months and meet specific LDH test criteria. For aHUS participants, treatment-naive individuals must show Thrombotic Microangiopathy signs. Those previously treated with eculizumab or ravulizumab must have confirmed aHUS diagnosis with stable TMA parameters. Participants who have been treated with eculizumab or ravulizumab must have been on treatment for at least 90 days with no missed doses in the last two months. Female participants who can have children and male participants must follow specific contraception guidelines.

Exclusion criteria: Patients not diagnosed with PNH or aHUS, those outside the specified age range, those unable to follow study procedures, those with other medical conditions interfering with the study, those currently participating in another clinical trial, those with allergic reaction history to study medication, pregnant or breastfeeding women, and those who received certain medications recently cannot participate.

Focus: Upon joining, participants are informed about trial purpose, procedures, and potential risks and benefits. Consent is obtained from legal guardian with participant assent if applicable. Initial assessment confirms eligibility through medical history review and tests. Participants receive ravulizumab through subcutaneous injection with dosage determined by study protocol. Regular visits monitor health and treatment response including blood tests and assessments. Follow-up assessments evaluate treatment effectiveness and safety at specified intervals including Day 1, Day 15, Day 71, and through Week 52. Final assessment at study period end gathers comprehensive health status data and treatment impact.

Investigational drugs: Ravulizumab is a medication used to help treat children with certain blood disorders by blocking part of the immune system that can attack the body’s own cells.

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 study compares crovalimab and eculizumab in patients who have not been treated with similar medications before. Crovalimab can be given through injection under the skin or into a vein, while eculizumab is given through a vein.

Inclusion criteria: Participants must have body weight of at least 40 kg with confirmed PNH diagnosis through high sensitivity flow cytometry. Lactate dehydrogenase levels must be at least twice the upper limit of normal during screening. Platelet count must be at least 30,000 per cubic millimeter without transfusion within seven days before testing. Absolute Neutrophil Count must be greater than 500 per microliter. Female patients who can have children must agree to use birth control or remain abstinent.

Exclusion criteria: Patients with conditions other than PNH, those outside specified age range, those from vulnerable populations, and certain other medical conditions cannot participate.

Focus: Initial assessment confirms eligibility through PNH diagnosis verification, body weight check, blood cell count measurement, and contraception agreement if applicable. Participants are randomly assigned to receive either crovalimab or eculizumab unbiased. Treatment involves assigned medication with frequency and dosage determined by study protocol. Crovalimab can be administered intravenously or subcutaneously while eculizumab is administered intravenously. Regular monitoring assesses effectiveness and safety measuring blood levels of specific markers and hemolysis control evaluation. Participants are monitored for side effects or adverse reactions. Study evaluates outcomes including blood transfusion avoidance ability, hemolysis control, and fatigue impact. Adverse events or reactions presence is recorded and analyzed. Study concludes by June 2028 with final assessment evaluating overall treatments effectiveness and safety.

Investigational drugs: Crovalimab works by inhibiting the complement system to prevent red blood cell destruction. Eculizumab is used as a comparison.

Summary

The 14 ongoing clinical trials for paroxysmal nocturnal haemoglobinuria demonstrate significant research activity across Europe, with particular concentration in countries such as Italy, Spain, Germany, and France. The trials explore multiple therapeutic approaches, including complement inhibitors like crovalimab, eculizumab, and ravulizumab, as well as newer agents like iptacopan, pozelimab, cemdisiran, danicopan, pegcetacoplan, and OMS906.

A notable feature of the current research landscape is the focus on combination therapies, such as pozelimab with cemdisiran, and danicopan added to existing complement inhibitor treatments. Several studies specifically address pediatric and adolescent populations, including trials evaluating medications like iptacopan, danicopan, pegcetacoplan, and ravulizumab in children as young as 2 years old. This represents an important effort to establish safe and effective treatments for younger patients.

Many trials focus on long-term safety and effectiveness, with some extending for up to two years or more, reflecting the chronic nature of the condition and the need for sustained treatment monitoring. Several studies compare personalized dosing strategies with standard regimens, aiming to optimize treatment outcomes while minimizing potential side effects.

The geographic distribution of trials provides access to investigational treatments across multiple European countries, though availability varies by trial. Patients interested in participating should consult with their healthcare providers to determine eligibility and identify appropriate trials in their region.

Ongoing Clinical Trials on Haemoglobinuria

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