Skip to content
Clinical Trials – home

Rare diseases

Investigational molecules

Locations

Our offerNewsroom

Not recruiting

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

Verified siteRegistered drugNo placebo
Clinical Trials Concierge

Prefer not to search? Our Concierge searches the trials for you.

What is this trial about?

A plain-language summary of the goals, design and what participants do

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.

The research process

The trial runs in 6 steps – from screening to follow-up. Each step says what happens and what the team monitors.

  1. Step 1

    Initial assessment

    Upon joining the study, an initial assessment is conducted to confirm eligibility. This includes verifying a documented diagnosis of paroxysmal nocturnal hemoglobinuria (PNH) through specific blood tests.

    Additional criteria include a body weight of at least 40 kg, specific blood cell counts, and agreement to use contraception if applicable.

  2. Step 2

    Randomization

    Participants are randomly assigned to receive either crovalimab or eculizumab. This process ensures that the study results are unbiased.

  3. Step 3

    Treatment administration

    The treatment involves receiving the assigned medication. Crovalimab can be administered either intravenously (IV) or subcutaneously (SC), while eculizumab is administered intravenously.

    The frequency and dosage of the medication are determined by the study protocol and are monitored by the study team.

  4. Step 4

    Monitoring and assessments

    Regular monitoring is conducted to assess the effectiveness and safety of the treatment. This includes measuring blood levels of specific markers and evaluating the control of hemolysis.

    Participants are also monitored for any side effects or adverse reactions to the medication.

  5. Step 5

    Evaluation of outcomes

    The study evaluates several outcomes, including the ability to avoid blood transfusions and control hemolysis, as well as the impact on fatigue and other health indicators.

    The presence of any adverse events or reactions is also recorded and analyzed.

  6. Step 6

    Completion of study

    The study is expected to conclude by June 2028. At the end of the study, a final assessment is conducted to evaluate the overall effectiveness and safety of the treatments.

Who can join the trial?

6 criteria

  • Must have a body weight of at least 40 kg (about 88 pounds).
  • Must have a confirmed diagnosis of Paroxysmal Nocturnal Hemoglobinuria (PNH), which is a rare blood condition, confirmed by a special test called high sensitivity flow cytometry of white blood cells (WBCs).
  • Must have a level of LDH (Lactate Dehydrogenase, an enzyme found in the blood) that is at least twice the upper limit of normal (ULN) during screening.
  • Must have a platelet count of at least 30,000 per cubic millimeter (mm3) at screening, without needing a blood transfusion within 7 days before the test. Platelets are cells that help with blood clotting.
  • Must have an ANC (Absolute Neutrophil Count, a type of white blood cell) greater than 500 per microliter (micro L) at screening.
  • For female patients who can have children: Must agree to either not have sexual intercourse or use birth control methods to prevent pregnancy.

Who cannot join the trial?

4 criteria

  • Patients who have a different condition than Paroxysmal Nocturnal Hemoglobinuria (PNH) cannot participate. PNH is a rare blood disorder.
  • Patients who are not within the specified age range for the study cannot participate. The study includes certain age groups.
  • Patients who are not male or female cannot participate, as the study includes both genders.
  • Patients who are considered part of a vulnerable population may not be eligible. Vulnerable populations include groups that might need special protection.
Clinical Trials Concierge

Prefer not to search? Our Concierge searches the trials for you.

Tell us about your condition – we search every trial in Europe and connect you with the right site.

We usually reply within a few days

Verified sites

All sites with verified contact details – recruitment status may not be available; ask directly

Trial locations

Where you can join this trial

Countries are shaded by recruitment status. Click a recruiting country to ask about joining there.

Not recruiting
Not finding your country?

Not sure what to do next?

Joining a clinical trial can seem overwhelming. We guide you step by step, so you know exactly what to expect and how we support you through the process.

See the full process and FAQ

Investigated drugs

  • Crovalimab

    is a medication being studied for its effectiveness in treating patients with Paroxysmal Nocturnal Hemoglobinuria (PNH). It is designed to work by inhibiting a part of the immune system known as the complement system, which is overactive in PNH patients. This overactivity can lead to the destruction of red blood cells, so crovalimab aims to prevent this process and improve patient outcomes.

  • Eculizumab

    is another medication used in the study as a comparison to crovalimab. It is already approved for the treatment of PNH and works similarly by blocking the complement system to prevent the breakdown of red blood cells. The study aims to compare the effectiveness and safety of crovalimab against eculizumab in patients who have not been previously treated with complement inhibitors.

What is already known about the treatment

  • Crovalimab

    Crovalimab is administered via intravenous infusion, allowing it to enter the bloodstream directly. It is currently being studied in clinical trials for its effectiveness in treating Paroxysmal Nocturnal Hemoglobinuria (PNH), a rare blood disorder. The medication works by inhibiting a part of the immune system known as the complement system, which is overactive in PNH patients. Crovalimab is classified as a complement inhibitor, aiming to reduce the destruction of red blood cells and improve symptoms in affected individuals.

  • Eculizumab

    Eculizumab is also administered through intravenous infusion and is an established treatment for Paroxysmal Nocturnal Hemoglobinuria (PNH). It is widely recognized in medical literature as an effective therapy for this condition. Eculizumab functions by blocking the complement protein C5, preventing the breakdown of red blood cells. It belongs to the pharmacological class of complement inhibitors and is used to manage symptoms and improve quality of life in patients with PNH.

Investigated diseases

Paroxysmal Nocturnal Hemoglobinuria (PNH) – This is a rare blood disorder where red blood cells break down earlier than normal. It is caused by a mutation in the PIGA gene, leading to a deficiency of proteins that protect red blood cells from the immune system. As a result, patients may experience episodes of dark-colored urine, especially in the morning, due to the release of hemoglobin. Over time, this can lead to symptoms such as fatigue, shortness of breath, and an increased risk of blood clots. The disease can progress with varying severity, and symptoms may fluctuate over time. It primarily affects adults and can significantly impact quality of life.
Trial detailsLast updated 7 Oct 2026
Age18+ yearsPhasePhase IIITrial ID2023-506498-36-00Protocol codeBO42162Estimated enrolment209 patientsSponsorF. Hoffmann-La Roche AG

sourced from the EU Clinical Trials Register and site verification

Want to learn more about this trial or check if you can participate?

Clinical Trials Concierge

Prefer not to search? Our Concierge searches the trials for you.

Tell us about your condition – we search every trial in Europe and connect you with the right site.