Ongoing Clinical Trials for Granulomatosis with Polyangiitis
There are currently 4 ongoing clinical trials investigating new treatments for granulomatosis with polyangiitis, a rare autoimmune disease that causes inflammation of blood vessels. These studies are testing medications including iptacopan, imlifidase, rituximab combinations, and obinutuzumab across several European countries. (Also known as: Wegener’s granulomatosis, GPA, ANCA-associated vasculitis)
Clinical trial locations
- Austria
- Belgium
- Czechia
- Denmark
- France
- Study of iptacopan effectiveness in achieving remission in patients with active ANCA-associated vasculitis
- Study on Obinutuzumab for Patients with Relapsing Granulomatosis with Polyangiitis
- Study on Rituximab, Tocilizumab, and Tofacitinib for Patients with Granulomatosis with Polyangiitis Not Responding to Standard Therapy
- Germany
- Hungary
- Netherlands
- Spain
Study of iptacopan effectiveness in achieving remission in patients with active ANCA-associated vasculitis
This trial is testing a new oral medication called iptacopan for patients with active ANCA-associated vasculitis. The medication comes in hard gelatin capsules and works by targeting a specific part of the immune system called the complement pathway to reduce blood vessel inflammation.
Who can participate: Adults aged 18 and older with either newly diagnosed or relapsing granulomatosis with polyangiitis or microscopic polyangiitis. Participants must have active disease as measured by the Birmingham Vasculitis Activity Score, showing either at least one major symptom, three minor symptoms, or two kidney-related symptoms. They must also test positive for specific antibodies called anti-PR3 or anti-MPO, either during screening or previously documented.
Who cannot participate: People with severe allergic reactions to medications, pregnant or breastfeeding women, those with active infections including hepatitis B, hepatitis C, or HIV, severe liver or kidney disease requiring dialysis, recent cancer within the past five years, uncontrolled high blood pressure, or substance abuse within the past year are excluded. Patients who had major surgery within eight weeks or participated in other trials within 30 days also cannot join.
What the study involves: Participants will receive either iptacopan or a placebo along with standard treatments including rituximab and glucocorticoids. The study lasts 48 weeks with regular monitoring of disease activity, kidney function, and blood and urine tests. Doctors will assess whether patients achieve complete remission at 24 weeks and whether this remission is sustained through 48 weeks.
Study on Imlifidase for Patients with Severe ANCA-Associated Vasculitis and Lung Bleeding
This trial focuses on patients with severe ANCA-associated vasculitis who are experiencing life-threatening bleeding in the lungs, a condition called pulmonary hemorrhage. The medication being tested is imlifidase, given as an intravenous infusion directly into the bloodstream.
Who can participate: Patients must have a new or previous diagnosis of ANCA-associated vasculitis including granulomatosis with polyangiitis or microscopic polyangiitis, with an ANCA blood test level of 50 or higher measured within 14 days before joining. They must have confirmed lung bleeding shown on chest X-ray or CT scan with evidence such as coughing up blood, unexplained anemia, or acute respiratory distress syndrome. Female participants must be post-menopausal with no menstrual periods for at least 12 months.
Who cannot participate: Patients with other serious medical conditions, those who are pregnant or breastfeeding, recent participation in another trial within 30 days, known allergy to the study medication, drug or alcohol abuse within the past year, severe liver or kidney disease, active infections requiring treatment, or cancer within the last five years except certain skin cancers.
What the study involves: After providing informed consent and initial assessment including blood tests and lung imaging, participants receive imlifidase through intravenous infusion. The study monitors how quickly ANCA antibody levels decrease within 24 hours and tracks lung and kidney function, along with any side effects throughout the trial period.
Study on Rituximab, Tocilizumab, and Tofacitinib for Patients with Granulomatosis with Polyangiitis Not Responding to Standard Therapy
This study is designed for patients with granulomatosis with polyangiitis who have not responded well to standard treatments. The trial compares three different treatment approaches to find the most effective strategy for achieving remission.
Who can participate: Adults aged 18 or older with newly diagnosed or relapsing granulomatosis with polyangiitis who have active symptoms and have not responded adequately to standard treatments. An inadequate response means the disease worsened despite 12 weeks of treatment, less than 50% reduction in disease activity after 12 weeks, or ongoing active disease requiring more than 7.5 mg/day of prednisone after 12 weeks. Patients must be on a stable dose of oral glucocorticoids for four weeks before joining and have social security coverage.
Who cannot participate: Patients who have responded well to standard treatments, those not diagnosed with granulomatosis with polyangiitis, individuals outside the specified age range, vulnerable populations needing special protection, and those unable to follow study procedures or attend required visits.
What the study involves: Participants receive one of three treatment strategies for up to 52 weeks: rituximab combined with a conventional disease-modifying drug such as methotrexate, azathioprine, or mycophenolate mofetil; tocilizumab given via subcutaneous injection; or oral tofacitinib. The study monitors disease activity with regular assessments at weeks 12, 24, and 52 to evaluate which treatment most effectively reduces disease activity and helps patients achieve remission.
Study on Obinutuzumab for Patients with Relapsing Granulomatosis with Polyangiitis
This trial investigates obinutuzumab for patients whose granulomatosis with polyangiitis has relapsed after previous treatment. The medication targets specific immune system cells involved in causing blood vessel inflammation.
Who can participate: Adults aged 18 or older with relapsing granulomatosis with polyangiitis that is positive for PR3-ANCA antibodies. Participants must have active disease with a Birmingham Vasculitis Activity Score of 3 or higher and be within the first 21 days after starting or increasing glucocorticoids at 1 mg per kilogram of body weight per day or less. They must have health insurance or similar social security benefits.
Who cannot participate: Patients without PR3-ANCA positive granulomatosis with polyangiitis, those not experiencing a relapse, individuals unable to stop corticoid medications after completing a prednisone taper, patients outside the specified age range, and those considered part of vulnerable populations.
What the study involves: After initial health assessment and blood tests, participants receive obinutuzumab as an intravenous infusion. The study lasts approximately 12 months with key assessments at 6 months and 12 months. The primary goal is achieving a disease activity score of zero, elimination of PR3-ANCA antibodies, and successful discontinuation of corticosteroids after gradual dose reduction. Regular monitoring tracks treatment response, any infections or infusion reactions, and overall disease activity.
Summary
The four ongoing clinical trials for granulomatosis with polyangiitis reflect a diverse research effort across Europe, with France hosting three trials and Germany hosting two. The studies demonstrate different therapeutic approaches, from novel complement pathway inhibitors like iptacopan to established immune-modifying drugs being tested in new combinations.
A notable pattern is the focus on patients with difficult-to-treat disease, including those not responding to standard therapy and those with severe complications such as lung bleeding. The trials collectively test six different investigational drugs: iptacopan, imlifidase, rituximab, tocilizumab, tofacitinib, and obinutuzumab, representing various mechanisms of immune system modulation.
Most trials share similar age requirements, requiring participants to be 18 years or older, and consistently exclude pregnant or breastfeeding women, people with active infections, and those with recent cancer history. The study durations range from approximately 12 months to 52 weeks, with regular monitoring of disease activity and safety outcomes throughout.
