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Study on the Safety and Effectiveness of Crovalimab for Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH)

Fast replyInvestigationalNo 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 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.

The research process

The trial runs in 5 steps – from screening to follow-up. Each step says what happens and what the team monitors.

  1. Step 1

    Initial visit and screening

    Upon joining the study, you will attend an initial visit where medical history and current health status are reviewed. This includes a physical examination and blood tests to ensure eligibility for the trial.

    Vaccinations against Neisseria meningitidis are required, following local guidelines. If not already vaccinated, this will be arranged during the screening process.

  2. Step 2

    Treatment initiation

    If eligible, you will begin treatment with crovalimab. This medication is administered either as a subcutaneous injection or an intravenous infusion, depending on the specific requirements of the study part you are enrolled in.

    The initial dose will be given under medical supervision to monitor for any immediate reactions.

  3. Step 3

    Regular treatment and monitoring

    You will receive regular doses of crovalimab over a period of 5 months. The frequency and dosage will be determined by the study protocol and your specific needs.

    Regular follow-up visits will be scheduled to monitor your response to the treatment and to check for any side effects. Blood tests and other assessments will be conducted during these visits.

  4. Step 4

    Open label extension (optional)

    If you complete the initial treatment phase and derive benefit from crovalimab, you may be eligible to continue in an open label extension phase. This allows for long-term monitoring and continued access to the medication.

    During this phase, regular assessments will continue to ensure ongoing safety and effectiveness of the treatment.

  5. Step 5

    End of study

    At the end of your participation in the study, a final visit will be conducted. This includes a comprehensive health assessment and discussion of your experience during the trial.

    You will receive guidance on any further medical care or follow-up that may be necessary after the study concludes.

Who can join the trial?

18 criteria

  • For Part 1 (Healthy Volunteers):
    • Must be a healthy male between 21 and 55 years old.
    • Must have received a vaccination against Neisseria meningitidis, which is a type of bacteria that can cause serious infections, covering serogroups B, A, C, W, and Y.
    • Must test negative for hepatitis B, hepatitis C, and HIV.
    • Must have been vaccinated against hepatitis B.
    • Must agree not to smoke during the in-clinic part of the study.
    • For Parts 2, 3, and 4 (Patients with PNH):
      • Must be a male or female between 18 and 75 years old.
      • Must have received a vaccination against Neisseria meningitidis according to local guidelines or standard care for those at increased risk.
      • Must have a stable dose of other therapies like immunosuppressants, corticosteroids, or iron supplements for at least 28 days before screening.
      • Women who can have children must have a negative pregnancy test.
      • For Part 2 and Part 4 Arm A (Untreated PNH Patients):
        • Hepatitis B patients can join if their liver function tests are less than 2 times the upper limit of normal and there is no liver function impairment.
        • Must not have been treated with any complement inhibitor or stopped treatment due to lack of efficacy based on a specific genetic mutation.
        • For Part 3 and Part 4 Arm B (PNH Patients Treated with Eculizumab):
          • Must test negative for hepatitis B, hepatitis C, and HIV, but those treated successfully for hepatitis C or B are eligible.
          • Must be adequately controlled in the opinion of the investigator.
          • Must receive regular infusions of eculizumab, a medication used to treat PNH.
          • For Open Label Extension (OLE) (PNH Patients):
            • Must have completed Parts 2, 3, and 4 of the study.
            • Must have benefited from treatment with crovalimab, according to the investigator.
            • Women who are not post-menopausal or have not had surgical sterilization must agree to use effective contraception or remain abstinent during treatment and for a specified period after the last dose.
            • Must maintain current vaccinations for Neisseria meningitidis serotypes A, C, W, Y, and B according to local guidelines or standard care.

Who cannot join the trial?

10 criteria

  • Patients with any other serious medical conditions that might affect their participation.
  • Patients who have had an allergic reaction to similar medications in the past.
  • Patients who are currently participating in another clinical trial.
  • Patients who are pregnant or breastfeeding.
  • Patients with a history of drug or alcohol abuse.
  • Patients who have received any other investigational drug within the last 30 days.
  • Patients with a history of certain blood disorders.
  • Patients who have had a major surgery within the last 3 months.
  • Patients with uncontrolled high blood pressure.
  • Patients with active infections that require treatment.
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Investigated drugs

Crovalimab is a medication being studied for its potential to help people with a condition called paroxysmal nocturnal hemoglobinuria (PNH). PNH is a rare blood disorder that can cause red blood cells to break down too early, leading to various health problems. Crovalimab works by targeting a part of the immune system known as the complement system, which is involved in this breakdown of red blood cells. The study aims to see if crovalimab is safe and how well it works in both healthy volunteers and patients with PNH. It also looks at how the body processes the medication and its effects over time. The goal is to find out if crovalimab can be a safe and effective treatment option for people with PNH.

What is already known about the treatment

Crovalimab – Crovalimab is administered through injections, typically under the skin, and is being studied for its safety and effectiveness in treating paroxysmal nocturnal hemoglobinuria (PNH), a rare blood disorder. Currently, it is in clinical trials, and researchers are evaluating its impact on patients who are either new to treatment or switching from other therapies. The main therapeutic use of crovalimab is to help manage PNH by reducing the breakdown of red blood cells. At the molecular level, crovalimab works by inhibiting a part of the immune system called the complement system, which is overactive in PNH patients. It is classified as a complement inhibitor, a type of medication that helps control immune system activity.

Investigated diseases

Paroxysmal nocturnal hemoglobinuria (PNH) – Paroxysmal nocturnal hemoglobinuria is a rare blood disorder characterized by the destruction of red blood cells. This destruction occurs due to a defect in the surface proteins of red blood cells, making them susceptible to attack by the body's immune system. The disease often leads to episodes of dark-colored urine, especially noticeable in the morning. Over time, the breakdown of red blood cells can cause anemia, leading to symptoms such as fatigue and weakness. Patients may also experience blood clots and impaired bone marrow function. The progression of PNH can vary, with some individuals experiencing mild symptoms and others facing more severe complications.
Trial detailsLast updated 2 Oct 2026
Age18+ yearsPhasePhase I/IITrial ID2023-506287-14-00Protocol codeBP39144Estimated enrolment32 patientsSponsorF. Hoffmann-La Roche AG

sourced from the EU Clinical Trials Register and site verification

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