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Not yet recruitingRare disease

Study on Rituximab, Tocilizumab, and Tofacitinib for Patients with Granulomatosis with Polyangiitis Not Responding to Standard Therapy

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 treatments for a condition called Granulomatosis with Polyangiitis (GPA), which is a rare disease that causes inflammation of blood vessels. The study is designed for patients who have not responded well to standard treatments, which typically include a combination of medications like glucocorticoids and cyclophosphamide or glucocorticoids and rituximab. The purpose of the study is to find the most effective treatment strategy for these patients.

The trial will explore three different treatment approaches. The first approach involves using rituximab together with another type of medication known as a conventional disease-modifying antirheumatic drug (cDMARD), which could be methotrexate, azathioprine, or mycophenolate mofetil, with a preference for methotrexate. The second approach uses tocilizumab, and the third approach involves tofacitinib. These treatments will be compared to see which is most effective in helping patients achieve remission, which means the disease is no longer active.

Participants in the study will receive one of these treatments for up to 52 weeks. The study will monitor how well each treatment works in reducing disease activity and helping patients feel better. The trial will also look at how patients and doctors assess the disease's activity and any side effects that may occur. The goal is to determine which treatment offers the best chance of remission for patients with GPA who have not responded to previous therapies.

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

    Enrollment

    Upon joining the study, eligibility is confirmed based on specific criteria, including age, diagnosis, and previous treatment response.

    Written informed consent is required to participate in the study.

  2. Step 2

    Initial assessment

    An initial assessment is conducted to evaluate the current state of the disease and establish a baseline for future comparisons.

    This includes a review of medical history and current symptoms.

  3. Step 3

    Treatment allocation

    Participants are assigned to one of three treatment strategies based on the study design.

    The options include: a combination of rituximab with a conventional disease-modifying antirheumatic drug (cDMARD) such as methotrexate, azathioprine, or mycophenolate mofetil; tocilizumab; or tofacitinib.

  4. Step 4

    Treatment administration

    The treatment is administered according to the assigned strategy.

    Rituximab is given intravenously, while methotrexate, azathioprine, and mycophenolate mofetil are taken orally.

    Tocilizumab is administered via subcutaneous injection, and tofacitinib is taken orally.

  5. Step 5

    Monitoring and follow-up

    Regular monitoring is conducted to assess the response to treatment and any side effects.

    Follow-up visits occur at specified intervals, including weeks 12, 24, and 52, to evaluate disease activity and patient-reported outcomes.

  6. Step 6

    Evaluation of outcomes

    The primary outcome is assessed at 12 weeks, focusing on the proportion of patients achieving a response or remission.

    Secondary outcomes include assessments at weeks 24 and 52, comparing disease activity and patient-reported outcomes over time.

Who can join the trial?

12 criteria

  • Patients must have been newly diagnosed or have a relapsing condition called granulomatosis with polyangiitis (GPA) according to specific criteria set in 2022.
  • Patients must be aged 18 years or older.
  • Patients must have active symptoms that are due to GPA.
  • Patients must have had an inadequate response to previous standard treatments, which include either:
    • A combination of glucocorticoids (a type of steroid) plus cyclophosphamide (a medication used to suppress the immune system).
    • AND/OR a combination of glucocorticoids plus rituximab (a medication that targets certain immune cells).
    • An inadequate response to treatment is defined as:
      • The disease getting worse despite 12 weeks of standard treatment.
      • Or not having at least a 50% reduction in disease activity after 12 weeks of treatment.
      • Or having ongoing active disease that requires continuing corticosteroids at a dose of more than 7.5 mg/day of prednisone (a type of steroid) after 12 weeks of treatment.
      • Patients must be on a stable dose of oral glucocorticoids of at least 7.5 mg/day of equivalent prednisone for the 4 weeks before joining the study. Short-term high doses of methylprednisolone (another steroid) are allowed if needed due to the severity of the condition before starting the study treatment.
      • If patients are currently treated with conventional disease-modifying anti-rheumatic drugs (cDMARD), they must be on a stable dose for 4 weeks before joining the study.
      • Patients must be able to understand the study requirements, provide written consent to participate, and agree to follow the study procedures, including attending required study visits.
      • Patients must have some form of social security or be entitled to it.

Who cannot join the trial?

5 criteria

  • Patients who have not had an inadequate response to standard treatments for their condition. This means if the usual treatments are working well for you, you cannot participate.
  • Patients who are not diagnosed with granulomatosis with polyangiitis (GPA). This study is only for those with this specific condition.
  • Patients who are not within the age range specified for the study. The study has specific age requirements that must be met.
  • Patients who are part of a vulnerable population. This means if you are in a group that needs special protection, you may not be eligible.
  • Patients who are not able to follow the study procedures or attend the required visits. This means if you cannot commit to the study schedule, you cannot participate.
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Investigated drugs

  • Rituximab

    This medication is used in combination with other drugs to help manage granulomatosis with polyangiitis. It works by targeting specific cells in the immune system to reduce inflammation and help control the disease.

  • Methotrexate

    Often used alongside rituximab, this medication helps to suppress the immune system and reduce inflammation. It is a conventional disease-modifying antirheumatic drug (cDMARD) that can help manage symptoms and prevent disease progression.

  • Azathioprine

    Another cDMARD that may be used with rituximab, azathioprine helps to lower the activity of the immune system, reducing inflammation and helping to control the disease.

  • Mycophenolate mofetil

    This medication can also be combined with rituximab. It works by suppressing the immune system to help reduce inflammation and manage the symptoms of granulomatosis with polyangiitis.

  • Tocilizumab

    This medication is used to help control inflammation by blocking a specific protein in the body that contributes to the immune response. It is considered as one of the potential therapies for patients who do not respond adequately to standard treatments.

  • Tofacitinib

    This medication works by interfering with the activity of certain enzymes in the immune system, helping to reduce inflammation and manage symptoms in patients with granulomatosis with polyangiitis.

What is already known about the treatment

  • Rituximab

    This medication is administered intravenously and is used in combination with other drugs to treat patients with granulomatosis with polyangiitis who have not responded adequately to standard therapies. Rituximab is well-established in medical literature as a treatment for certain autoimmune diseases and cancers. It works by targeting and depleting CD20-positive B cells, which are part of the immune system and play a role in inflammation. Rituximab is classified as a monoclonal antibody.

  • Tocilizumab

    Administered via injection, tocilizumab is used for patients with granulomatosis with polyangiitis who do not respond well to standard treatments. It is recognized in medical literature for its role in treating autoimmune conditions. Tocilizumab works by blocking the interleukin-6 receptor, which helps reduce inflammation. It is classified as an interleukin-6 receptor antagonist.

  • Tofacitinib

    This medication is taken orally and is being evaluated for patients with granulomatosis with polyangiitis who have not had success with standard care. Tofacitinib is known in medical literature for its use in treating certain autoimmune diseases. It functions by inhibiting Janus kinases, which are enzymes that play a role in the inflammatory process. Tofacitinib is classified as a Janus kinase inhibitor.

Investigated diseases

Granulomatosis with Polyangiitis – This is a rare disease characterized by inflammation of the blood vessels, which can lead to damage in various organs, particularly the respiratory tract and kidneys. The disease often begins with symptoms like sinusitis, nosebleeds, or a persistent cough. As it progresses, it can cause more severe symptoms such as kidney dysfunction, skin rashes, and joint pain. The inflammation is due to an abnormal immune response, which can result in the formation of granulomas, or small areas of inflammation. Over time, the disease can lead to significant organ damage if not managed properly. The progression and symptoms can vary widely among individuals, making it a complex condition to understand and manage.
Trial detailsLast updated 2 Oct 2026
Age18+ yearsPhasePhase IIITrial ID2024-516687-28-00Protocol codeAPHP200026Estimated enrolment42 patientsSponsorAssistance Publique Hopitaux De Paris

sourced from the EU Clinical Trials Register and site verification

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