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Paroxysmal nocturnal haemoglobinuria – Trials in Disease

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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. Currently, 14 clinical trials are investigating various treatments across multiple European countries, offering hope for improved management of this challenging condition. These studies are testing both new medications and combination therapies to help patients better control their symptoms and improve their quality of life.

Clinical trial locations

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

This study evaluates the long-term safety and effectiveness of a combination treatment using two medications: pozelimab and cemdisiran. Both medications are given as injections under the skin and work together to prevent the immune system from attacking red blood cells.

Main inclusion criteria: Participants must have completed a previous study with these medications without permanently stopping treatment. They must be willing to attend regular clinic visits and receive required vaccinations against certain bacterial infections, particularly meningococcal vaccines. The study is open to adults and adolescents aged 12 years and older, both male and female.

Main exclusion criteria: People with a history of bone marrow failure, active bacterial or viral infections including HIV or hepatitis, major organ transplants, or cancer within the past 5 years cannot participate. Those with severe kidney disease requiring dialysis, severe liver disease, or blood clotting disorders are also excluded, as are pregnant or breastfeeding women.

Trial focus: The study monitors how well the combination treatment controls the disease over approximately 108 weeks. Researchers track lactate dehydrogenase levels, which indicate red blood cell destruction, hemoglobin levels, and the need for blood transfusions. Quality of life and fatigue levels are also assessed throughout the study.

Investigational drugs: Pozelimab blocks specific proteins in the immune system involved in destroying red blood cells, while cemdisiran reduces the activity of proteins that contribute to this breakdown. Together, they aim 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 trial investigates adding danicopan to existing treatments with ravulizumab or eculizumab for children experiencing significant red blood cell breakdown outside of blood vessels. The study uses danicopan as a film-coated tablet taken by mouth.

Main inclusion criteria: Children between 12 and under 18 years old weighing more than 25 kg can participate. They must have confirmed diagnosis and anemia with hemoglobin levels of 10.5 g/dL or lower and high reticulocyte counts. Participants must have been treated with ravulizumab or eculizumab for at least 12 weeks before joining and have up-to-date vaccinations against meningococcal infection, Haemophilus influenzae type b, and Streptococcus pneumoniae. Female participants must not be pregnant and must use effective contraception.

Main exclusion criteria: Children with other serious health conditions that might interfere with the study, those currently in another trial, or those who recently had major surgery cannot participate. Those with known allergies to study medications, inability to follow study procedures, pregnant or breastfeeding participants, and those with a history of drug or alcohol abuse are excluded.

Trial focus: The primary goal is to assess changes in hemoglobin levels from the study start to week 12. The study monitors blood parameters, side effects, and quality of life throughout the treatment period, which extends to week 24.

Investigational drugs: Danicopan is being tested as an add-on treatment to see if it improves blood levels when combined with ravulizumab or eculizumab. Both ravulizumab and eculizumab work by blocking part of the immune system to prevent red blood cell destruction.

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

This study evaluates pegcetacoplan, a medication given as injections under the skin, specifically in adolescent patients. The medication helps protect red blood cells from being destroyed by the immune system.

Main inclusion criteria: Adolescents between 12 and 17 years old weighing at least 20 kilograms with a confirmed diagnosis can participate. They must have either never received complement inhibitor treatment with low hemoglobin and high LDH levels, or be on stable complement inhibitor treatment with low hemoglobin. Required blood counts include platelets above 75,000 per cubic millimeter and neutrophils above 1,000 per cubic millimeter. Participants must be vaccinated against specific bacteria or agree to receive vaccines within 14 days. Female participants must have negative pregnancy tests and use birth control during the study.

Main exclusion criteria: Current or recent pregnancy or breastfeeding, severe allergic reactions to medications, active infections, severe liver or kidney problems, major surgery in the past 3 months, participation in other trials within 30 days, active cancer or cancer treatment, severe heart conditions, uncontrolled high blood pressure, and blood clotting disorders all exclude potential participants.

Trial focus: The study monitors hemoglobin levels, LDH levels, red blood cell counts, and overall health over 16 weeks, with potential extension up to 52 weeks. Quality of life measurements and blood transfusion needs are also tracked.

Investigational drugs: Pegcetacoplan targets and regulates part of the complement system to protect red blood cells from destruction, potentially improving anemia and reducing the need for blood transfusions.

Study on Optimizing Ravulizumab Dosing for Adults with Paroxysmal Nocturnal Hemoglobinuria

This study compares two different ways of administering ravulizumab: standard dosing versus personalized dosing tailored to individual needs. The medication is given as an infusion directly into the bloodstream.

Main inclusion criteria: Patients must be eligible for ravulizumab treatment according to Dutch guidelines or already receiving it per label and guidelines. Participants must be 16 years or older and willing to provide written informed consent.

Main exclusion criteria: Those without a confirmed diagnosis, individuals under 18 years old, and members of vulnerable populations cannot participate.

Trial focus: The study evaluates the effectiveness of personalized dosing compared to standard dosing by monitoring LDH levels and ensuring ravulizumab concentrations remain above 100 mg/L. It assesses breakthrough hemolysis, quality of life using the FACIT-Fatigue Scale, and tracks the number of transfusions and infusions needed over 52 weeks.

Investigational drugs: Ravulizumab blocks part of the immune system that leads to red blood cell breakdown, helping to manage symptoms and prevent complications.

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

This 26-week study tests the combination of pozelimab and cemdisiran in patients who have not recently received or have never received complement inhibitor treatment. The study compares this combination with ravulizumab and eculizumab.

Main inclusion criteria: Participants must have a confirmed diagnosis through high-sensitivity flow cytometry with active symptoms and LDH levels at least twice the upper limit of normal at screening. They must be willing to attend clinic visits and complete all required meningococcal vaccinations. The study is open to both male and female participants within the specified age range.

Main exclusion criteria: Those who have recently received complement inhibitor treatment or are not treatment-naive for complement inhibitors cannot participate.

Trial focus: The study measures LDH levels to assess treatment effectiveness in controlling red blood cell breakdown. It monitors the ability to avoid blood transfusions and tracks overall disease control throughout the 26-week period.

Investigational drugs: Pozelimab blocks the overactive complement system by targeting specific proteins, while cemdisiran reduces production of proteins involved in red blood cell destruction. Ravulizumab and eculizumab serve as comparison treatments, both working by blocking the complement system to prevent red blood cell breakdown.

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

This study evaluates iptacopan hydrochloride, taken as hard gelatin capsules, in children and teenagers over a 26-week period. The study focuses on understanding how the body processes this medication and monitoring its safety.

Main inclusion criteria: Children aged 2 to 17 years with confirmed diagnosis through high-sensitivity flow cytometry showing at least 10% clone size can participate. Those in Cohort 1 must weigh at least 35 kg. Participants on anti-C5 therapy must have been on a stable dose for at least 6 months. Those not previously treated must have hemoglobin less than 10 g/dL and LDH levels more than 1.5 times the upper limit of normal. Required vaccinations against Neisseria meningitidis, Streptococcus pneumoniae, and Haemophilus influenzae must be completed at least 2 weeks before starting treatment.

Main exclusion criteria: History of allergic reactions to the medication, use of medications that might interfere with the study drug, severe liver or kidney disease, pregnancy or breastfeeding, history of certain cancers, participation in another trial within 30 days, and uncontrolled high blood pressure all exclude potential participants.

Trial focus: The study assesses safety and tolerability while monitoring hemoglobin levels and other blood-related measurements. Regular health checks track any side effects throughout the 26-week treatment period.

Investigational drugs: Iptacopan works by blocking a protein that contributes to red blood cell destruction, helping to control the immune system’s complement pathway to prevent damage to blood cells.

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

This study compares two treatments for patients already receiving complement inhibitors. Crovalimab can be administered intravenously or subcutaneously, while eculizumab is given intravenously.

Main inclusion criteria: Participants must weigh at least 40 kg with confirmed diagnosis through high sensitivity flow cytometry and platelet count of at least 30,000 per cubic millimeter without recent transfusion. For Arms A and B, participants must be 18 years or older with at least 24 weeks of eculizumab treatment and LDH levels no more than 1.5 times the upper limit of normal. Arm C includes those under 18 years old on eculizumab, those on ravulizumab, or those with specific genetic variations. Note that in France and the Czech Republic, patients under 18 are not eligible.

Main exclusion criteria: History of severe allergic reactions to study medications, receipt of another investigational drug within 30 days, active infections requiring antibiotics, certain types of cancer history, pregnancy or breastfeeding, drug or alcohol abuse history, certain heart conditions, major surgery within 3 months, uncontrolled high blood pressure, and severe liver or kidney disease all exclude participants.

Trial focus: The study evaluates safety and tolerability by monitoring adverse events, vital signs, electrocardiograms, and laboratory tests. Effectiveness is measured by changes in hemolysis parameters, LDH levels, and hemoglobin stabilization over 25 weeks. The study continues until January 2030.

Investigational drugs: Both crovalimab and eculizumab work by blocking specific proteins in the blood to prevent red blood cell destruction. The study compares their safety and effectiveness in patients already receiving similar treatments.

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

This extension study evaluates the long-term safety and tolerability of iptacopan in patients who have already completed previous phases of clinical trials. The medication is taken as hard gelatin capsules.

Main inclusion criteria: Participants must be 18 years or older with a confirmed diagnosis and must have completed the treatment extension period of previous Phase II or any Phase III studies with iptacopan. They must have received all necessary vaccinations against Neisseria meningitidis, Streptococcus pneumoniae, and Haemophilus influenzae. The study doctor must believe the participant may benefit from continuing treatment and has been stable on iptacopan for at least 3 months.

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

Trial focus: The study monitors long-term safety through regular evaluations, laboratory tests, and vital sign checks. It tracks hemoglobin stability, absence of blood transfusions, occurrences of breakthrough hemolysis, and major vascular events through yearly follow-up intervals. The study is estimated to end by February 2028.

Investigational drugs: Iptacopan inhibits a specific protein in the complement system to prevent red blood cell destruction, providing ongoing disease management.

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

This study evaluates ravulizumab administered through subcutaneous injection in children aged 2 to under 18 years over approximately one year. The study focuses on understanding how children’s bodies process the medication and its effects on symptoms.

Main inclusion criteria: Children aged 2 to 17 years weighing at least 10 kg can participate. They must have a legal guardian or representative who can provide written consent, and the participant must provide assent if required. Required vaccinations against meningococcal infection, Streptococcus pneumoniae, and Haemophilus influenzae type b must be completed. For participants with the condition, specific diagnostic criteria and laboratory test requirements must be met. Those previously treated with eculizumab or ravulizumab must have been on treatment for at least 90 days with no missed doses in the last 2 months. Female participants who can have children and male participants must agree to follow specific contraception guidelines.

Main exclusion criteria: Those not diagnosed with the specified conditions, individuals not within the age range, inability to follow study procedures, other interfering medical conditions, current participation in another trial, history of allergic reactions to the medication, pregnancy or breastfeeding, and recent receipt of certain medications all exclude potential participants.

Trial focus: The study monitors how the medication moves through the body and its effects on disease symptoms. Regular assessments track medication response, side effects, and overall health throughout the study period.

Investigational drugs: Ravulizumab blocks part of the immune system that attacks blood cells, helping to prevent red blood cell destruction and reduce kidney damage risk.

Study on the Effectiveness and Safety of Crovalimab vs. Eculizumab for Adults and Adolescents with Paroxysmal Nocturnal Hemoglobinuria Not Treated with Complement Inhibitors

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

Main inclusion criteria: Participants must weigh at least 40 kg with confirmed diagnosis through high sensitivity flow cytometry. LDH 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 7 days before testing. Absolute neutrophil count must be greater than 500 per microliter at screening. Female participants who can have children must agree to use birth control methods to prevent pregnancy.

Main exclusion criteria: Those with conditions other than the specified disorder, individuals not within the specified age range, non-male or non-female participants, and members of vulnerable populations cannot participate.

Trial focus: The study evaluates effectiveness by measuring the ability to avoid blood transfusions and control red blood cell breakdown. It monitors LDH levels, hemoglobin stability, fatigue levels, and quality of life. Safety assessments track any side effects or reactions throughout the study, which concludes by June 2028.

Investigational drugs: Crovalimab works by inhibiting the overactive complement system in patients. The study compares its effectiveness and safety against eculizumab, an already approved treatment that works similarly by blocking the complement system.

Study on the Effectiveness and Safety of Iptacopan for Adults with Paroxysmal Nocturnal Hemoglobinuria Switching from Anti-C5 Antibody Treatment

This study evaluates whether iptacopan is as effective as current standard treatment when patients switch to it. The medication is taken as hard gelatin capsules twice daily by mouth.

Main inclusion criteria: Adults 18 years or older with confirmed diagnosis who have been on stable anti-C5 antibody treatment (eculizumab or intravenous ravulizumab) for at least 6 months can participate. Average hemoglobin level should be 10 g/dL or higher based on tests over the past 6 months, confirmed by two additional tests during screening. Participants must be vaccinated against Neisseria meningitidis and S. pneumoniae at least 2 weeks before starting treatment, with preventive antibiotics if treatment starts earlier. Vaccination against Haemophilus influenzae is recommended. Participants should be able to communicate well with the study doctor and understand study requirements.

Main exclusion criteria: Those without the specified diagnosis, individuals not within the age range, those not in included clinical trial groups, and members of vulnerable populations cannot participate.

Trial focus: The trial monitors hemoglobin level changes from before starting iptacopan to Day 126 and Day 168 to assess effectiveness and safety. The study concludes by March 2025.

Investigational drugs: Iptacopan inhibits a specific protein in the complement system to prevent red blood cell destruction, aiming to maintain disease control in patients switching from anti-C5 antibody therapy.

Summary

The 14 ongoing clinical trials for paroxysmal nocturnal haemoglobinuria reflect a significant concentration of research efforts across Europe, with particularly strong representation in Germany, Italy, France, Spain, and the Netherlands. A notable feature is the focus on multiple complement inhibitors and combination therapies, suggesting the field is moving toward more personalized treatment approaches.

Several trials are investigating newer medications such as iptacopan, crovalimab, pozelimab, and cemdisiran, alongside established treatments like eculizumab and ravulizumab. Many studies emphasize long-term safety and effectiveness, with some extending beyond 52 weeks to gather comprehensive data on medication tolerance and disease control.

An encouraging development is the inclusion of pediatric populations in several trials, addressing the important need for age-appropriate treatment options for children and adolescents. Studies are also exploring different administration methods, including subcutaneous injections that may offer greater convenience compared to intravenous infusions.

The research landscape demonstrates a commitment to improving quality of life through better symptom control, reduced need for blood transfusions, and minimization of breakthrough hemolysis events. These trials collectively represent hope for patients seeking more effective and tolerable treatment options for this challenging blood disorder.

Ongoing Clinical Trials on Paroxysmal nocturnal haemoglobinuria

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

    Recruiting

    3 1 1
    Investigated diseases:
    Investigated drugs:
    France Germany Italy The Netherlands Spain

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

    Recruiting

    3 1 1 1
    Investigated diseases:
    Investigated drugs:
    The Netherlands

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

    Recruiting

    3 1 1
    Investigated diseases:
    Investigated drugs:
    Greece Hungary Italy Poland Romania Spain

  • Study of Pozelimab and Cemdisiran in Adults with Paroxysmal Nocturnal Hemoglobinuria Not Responding Well to Current C5 Inhibitor Treatment

    Not yet recruiting

    3 1 1
    Investigated diseases:
    Investigated drugs:
    Italy Poland Spain

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

    Not recruiting

    3 1 1 1
    Investigated diseases:
    Investigated drugs:
    Greece Hungary Italy Poland Romania Spain

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

    Not recruiting

    3 1 1
    Investigated diseases:
    Investigated drugs:
    Czechia France Germany Italy Lithuania The Netherlands +1

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

    Not recruiting

    3 1 1 1
    Investigated diseases:
    Investigated drugs:
    Belgium Czechia Estonia France Germany Greece +7

  • Study on the Effectiveness and Safety of Crovalimab vs. Eculizumab for Adults and Adolescents with Paroxysmal Nocturnal Hemoglobinuria (PNH) Not Treated with Complement Inhibitors

    Not recruiting

    3 1 1 1
    Investigated diseases:
    Investigated drugs:
    France Germany Lithuania The Netherlands Poland Portugal +3

  • Study on the Effectiveness and Safety of Iptacopan for Adults with Paroxysmal Nocturnal Hemoglobinuria (PNH) Switching from Anti-C5 Antibody Treatment

    Not recruiting

    3 1 1
    Investigated diseases:
    Investigated drugs:
    France Germany Italy Spain

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