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

Verified siteRegistered drugNo placebo
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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 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.

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 that the patient is eligible for treatment with ravulizumab according to relevant guidelines and is 16 years or older.

    The patient must provide written informed consent to participate in the trial.

  2. Step 2

    Treatment initiation

    The patient begins treatment with ravulizumab, administered as an infusion. The product used is Ultomiris 300 mg/30 mL concentrate for solution for infusion.

    The treatment involves either a standard dosing regimen or an individualized dosing regimen, depending on the study group assignment.

  3. Step 3

    Monitoring and evaluation

    Throughout the trial, the patient's response to treatment is monitored. This includes measuring the percentage of change in LDH (lactate dehydrogenase) levels compared to baseline levels.

    The concentration of ravulizumab in the blood is also measured to ensure it remains above 100 mg/L.

  4. Step 4

    Assessment of symptoms

    The occurrence of breakthrough hemolysis is assessed. This is defined as the appearance of new or worsening symptoms such as fatigue, hemoglobinuria, abdominal pain, shortness of breath, anemia, or other related symptoms.

    Quality of life is evaluated using the FACIT-Fatigue Scale, and patient preferences are recorded.

  5. Step 5

    Comparison of dosing strategies

    Differences between the standard and individualized dosing strategies are analyzed. This includes comparing the number of transfusions required and the cumulative dose of ravulizumab over 52 weeks.

    The number of infusions and appointments with the physician are also tracked as indicators of treatment costs.

  6. Step 6

    Completion of trial

    The trial is expected to conclude by December 16, 2027. At this point, all data collected will be analyzed to determine the effectiveness of the dosing strategies.

Who can join the trial?

3 criteria

  • The patient must be eligible for treatment with ravulizumab according to the relevant Dutch guidelines or already receiving ravulizumab treatment as per the label and Dutch guidelines. Ravulizumab is a medication used to treat certain blood disorders.
  • The patient must be willing to provide written informed consent. This means they agree to participate in the study after being informed about it.
  • The patient must be 16 years or older.

Who cannot join the trial?

3 criteria

  • Patients who do not have Paroxysmal Nocturnal Hemoglobinuria (PNH) cannot participate. PNH is a rare blood disorder where red blood cells break down too early.
  • Patients who are not adults cannot participate. This means individuals under 18 years old are excluded.
  • Patients who are part of a vulnerable population cannot participate. Vulnerable populations include groups like pregnant women or people who cannot make decisions for themselves.
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Investigated drugs

Ravulizumab is a medication used in the treatment of a condition called Paroxysmal Nocturnal Hemoglobinuria (PNH). This condition is a rare blood disorder that can cause severe symptoms due to the destruction of red blood cells. Ravulizumab works by blocking a part of the immune system that leads to the breakdown of these cells. The clinical trial aims to compare the effectiveness of personalized dosing of ravulizumab with the standard dosing in adult patients with PNH.

What is already known about the treatment

Ravulizumab – Ravulizumab is administered through an intravenous infusion, meaning it is delivered directly into the bloodstream via a vein. It is currently recognized in medical literature as a treatment for paroxysmal nocturnal hemoglobinuria (PNH), a rare blood disorder. The main therapeutic indication for ravulizumab is to reduce the destruction of red blood cells in patients with PNH. At the molecular level, ravulizumab works by inhibiting a part of the immune system known as the complement system, specifically targeting the C5 protein to prevent it from breaking down red blood cells. It is classified pharmacologically as a monoclonal antibody.

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 symptoms such as fatigue, abdominal pain, and dark urine, especially at night or in the morning. The breakdown of red blood cells can lead to anemia and other complications. Over time, the condition can cause blood clots and damage to organs due to reduced blood flow. The disease progresses with episodes of hemolysis, which can vary in frequency and severity among individuals.
Trial detailsLast updated 7 Oct 2026
Age18+ yearsPhasePhase IIITrial ID2024-519207-10-00Protocol code116326Estimated enrolment25 patientsSponsorStichting Radboud universitair medisch centrum

sourced from the EU Clinical Trials Register and site verification

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