Ongoing Clinical Trials for Idiopathic Inflammatory Myopathy
Currently, there are 6 ongoing clinical trials investigating new treatments for idiopathic inflammatory myopathy, a group of rare autoimmune diseases that cause chronic muscle inflammation and weakness. These studies are testing innovative therapies including immunomodulatory drugs and cell-based treatments across multiple European countries, offering hope for improved management of these challenging conditions.
Clinical trial locations
- Austria
- Belgium
- Bulgaria
- Cyprus
- Czechia
- Denmark
- France
- Germany
- Study on Long-term Safety and Efficacy of Efgartigimod in Adults with Active Idiopathic Inflammatory Myopathy Using a Drug Combination
- Study on the Safety of CNTY-101 and Aldesleukin for Patients with Active Autoimmune Diseases: Lupus, Scleroderma, and Myositis
- Study on the Safety of MB-CART19.1 for Patients with Active Systemic Lupus Erythematosus, Systemic Sclerosis, or Dermatomyositis/Polymyositis
- Study on the Effects and Safety of Efgartigimod PH20 SC for Adults with Active Idiopathic Inflammatory Myopathy
- Greece
- Hungary
- Study on Long-term Safety and Efficacy of Efgartigimod in Adults with Active Idiopathic Inflammatory Myopathy Using a Drug Combination
- Study on the Long-Term Safety of Dazukibart in Patients with Idiopathic Inflammatory Myopathies, Including Dermatomyositis and Polymyositis
- Study on the Effects and Safety of Efgartigimod PH20 SC for Adults with Active Idiopathic Inflammatory Myopathy
- Ireland
- Italy
- Lithuania
- Netherlands
- Study on Long-term Safety and Efficacy of Efgartigimod in Adults with Active Idiopathic Inflammatory Myopathy Using a Drug Combination
- Study of intravenous immunoglobulin combined with prednisone compared to prednisone alone in newly diagnosed patients with inflammatory myositis
- Study on the Effects and Safety of Efgartigimod PH20 SC for Adults with Active Idiopathic Inflammatory Myopathy
- Poland
- Portugal
- Slovakia
- Spain
- Sweden
Study on Long-term Safety and Efficacy of Efgartigimod in Adults with Active Idiopathic Inflammatory Myopathy Using a Drug Combination
This study is examining the long-term safety and effectiveness of efgartigimod PH20 SC, a medication given as an injection under the skin, for adults with active muscle inflammation. The main focus is understanding how well the treatment works over extended periods and how well patients tolerate it.
Who can participate: Adults who have completed a previous study called ARGX-113-2007 are eligible to join. Participants must be able to provide informed consent and agree to follow contraceptive measures as required. Women who can have children must have a negative pregnancy test before starting treatment.
Who cannot participate: People without active inflammatory muscle disease, those under 18 years of age, and individuals from vulnerable populations may not be eligible to participate.
What the study involves: The trial evaluates how the medication affects the use of other drugs like corticosteroids, aiming to see if it can help reduce the need for these medications over time. Participants will have regular check-ups to monitor their health, including vital signs, electrocardiogram tests, and laboratory measurements. The study will track improvements in muscle function, measure disease activity, and assess whether participants can achieve periods without disease activity or even remission. The research is expected to continue until September 2027.
The investigational drug: Efgartigimod PH20 SC is an immunomodulatory medication administered subcutaneously. It works by targeting and reducing levels of certain antibodies that contribute to the disease.
Study on the Long-Term Safety of Dazukibart in Patients with Idiopathic Inflammatory Myopathies, Including Dermatomyositis and Polymyositis
This clinical trial focuses on understanding the long-term safety of dazukibart, a monoclonal antibody medication designed to target proteins that may contribute to muscle inflammation. The study specifically includes people with dermatomyositis and polymyositis, which cause muscle weakness and, in some cases, skin rashes.
Who can participate: Both males and females with dermatomyositis or polymyositis who have completed a 52-week treatment period in a previous related study without stopping early are eligible. Participants must not be part of vulnerable populations.
Who cannot participate: People with other serious health conditions that could interfere with the study, recent infections requiring antibiotics, recent use of other investigational drugs, history of allergic reactions to similar medications, drug or alcohol abuse, pregnancy or breastfeeding, or inability to follow study procedures are excluded.
What the study involves: Participants will receive dazukibart through injections along with other medications including various corticosteroids and immunosuppressive drugs. The study lasts up to 52 weeks with regular assessments to monitor safety, changes in muscle strength, disease activity, and quality of life. The trial is estimated to end in November 2027.
The investigational drug: Dazukibart is an immunomodulatory agent given by injection that works by reducing inflammation at the molecular level.
Study on the Safety of CNTY-101 and Aldesleukin for Patients with Active Autoimmune Diseases: Lupus, Scleroderma, and Myositis
This trial tests a new cell therapy called CNTY-101 for treating autoimmune diseases including inflammatory muscle diseases. The treatment uses specially modified immune cells to help reduce harmful immune system activity.
Who can participate: People with inflammatory muscle diseases must show active muscle inflammation through biopsy or imaging, or have signs of lung disease related to the condition. They must test positive for specific antibodies related to muscle inflammation and, if experiencing muscle weakness, must meet certain test score criteria.
Who cannot participate: People with other autoimmune diseases not specified in the study, recent serious infections, current use of immunosuppressive drugs, recent cancer history (with certain exceptions), recent live vaccines, uncontrolled high blood pressure, significant heart problems, pregnancy or breastfeeding, substance abuse history, recent participation in other trials, or known allergies to study medications cannot participate.
What the study involves: After initial assessments confirming eligibility, participants receive CNTY-101 through an intravenous infusion directly into the bloodstream. They may also receive aldesleukin through a subcutaneous injection. Throughout the study, regular monitoring includes blood tests, imaging studies, and other assessments to track health and treatment effects.
The investigational drug: CNTY-101 uses engineered NK cells derived from stem cells to target and reduce B cells, which can attack the body’s own tissues in autoimmune diseases.
Study on the Safety of MB-CART19.1 for Patients with Active Systemic Lupus Erythematosus, Systemic Sclerosis, or Dermatomyositis/Polymyositis
This study investigates MB-CART19.1, a treatment using a patient’s own T-cells that have been modified to better target disease. The cells are changed using a special process to express a receptor that targets a protein called CD19.
Who can participate: Adults 18 years or older with dermatomyositis or polymyositis who meet specific classification criteria are eligible. They must have active muscle inflammation shown by biopsy or MRI, test positive for specific myositis antibodies, have muscle weakness meeting certain criteria, and have not responded well to or cannot tolerate standard treatments including glucocorticoids and at least two other medications.
Who cannot participate: The trial excludes people with dermatomyositis, polymyositis, systemic sclerosis, or systemic lupus erythematosus based on specific study design requirements.
What the study involves: After initial assessments and preparation including various tests, participants receive the modified T-cells through an intravenous infusion. Close monitoring follows, especially during the first four weeks, to watch for potential side effects including cytokine release syndrome and neurotoxicity. Regular follow-up visits continue through May 2026 to assess long-term safety and effectiveness.
The investigational drug: MB-CART19.1 is an anti-CD19 CAR T cell therapy, a form of immunotherapy that uses modified immune cells to target and eliminate B cells contributing to autoimmune conditions.
Study of intravenous immunoglobulin combined with prednisone compared to prednisone alone in newly diagnosed patients with inflammatory myositis
This study examines whether adding early intravenous immunoglobulin treatment to standard prednisone therapy helps newly diagnosed patients achieve better outcomes after 12 weeks. The medication being tested is Nanogam, given through intravenous infusion along with sodium chloride solution.
Who can participate: Adults aged 18 or older with a diagnosis of dermatomyositis, antisynthetase syndrome, immune mediated necrotizing myopathy, or overlap/non-specific myositis (including polymyositis) diagnosed less than 12 months ago are eligible. They must have at least 10% muscle weakness when tested plus abnormal results in two other muscle function tests, and be able to understand and sign informed consent.
Who cannot participate: People under 18 or over 75 years, pregnant or breastfeeding women, those who received immunoglobulins in the past 6 months, people with known allergies to immunoglobulin products, active cancer or recent cancer history, severe kidney or heart disease, active infections, history of blood clots, other autoimmune diseases requiring different treatment, recent use of experimental drugs, inability to provide consent, recent drug or alcohol abuse, certain mental conditions, or lack of reliable contraception are excluded.
What the study involves: This double-blind study means neither patients nor doctors know which treatment combination each participant receives. The treatment period lasts three months with multiple infusions. Monitoring includes muscle strength testing, quality of life questionnaires, physical activity measurements using a special device, whole body muscle MRI, and blood samples to measure various markers including antibody levels.
The investigational drugs: Intravenous immunoglobulin is a blood product containing pooled antibodies from thousands of donors that helps modulate the immune system and reduce inflammation. Prednisone is a corticosteroid that reduces inflammation and suppresses the immune system.
Study on the Effects and Safety of Efgartigimod PH20 SC for Adults with Active Idiopathic Inflammatory Myopathy
This trial investigates efgartigimod PH20 SC, given as a solution for injection, compared to placebo alongside standard immune-modulating treatments. The study evaluates how well the medication improves symptoms, its safety, and how well patients tolerate it.
Who can participate: Adults with a confirmed or likely diagnosis of inflammatory muscle disease including dermatomyositis, juvenile dermatomyositis, polymyositis (which may include antisynthetase syndrome), or immune-mediated necrotizing myopathy are eligible. They must have active disease shown by abnormal enzyme levels, recent electromyography showing active disease, active skin rash, recent muscle biopsy showing active inflammation, or recent MRI showing active inflammation. Participants must have muscle weakness and be receiving allowed treatments. Women who can become pregnant must have a negative pregnancy test before starting.
Who cannot participate: People with other active autoimmune diseases that might interfere, recent infections requiring antibiotics or antivirals, recent use of other investigational drugs, history of severe allergic reactions to study medications, pregnancy or breastfeeding, significant heart, liver, or kidney problems, recent cancer history (with certain exceptions), recent live vaccines, drug or alcohol abuse history, or inability to comply with study procedures are excluded.
What the study involves: After initial assessment and random assignment to receive either efgartigimod PH20 SC or placebo, participants receive subcutaneous injections alongside standard therapy. Regular monitoring assesses treatment efficacy and safety, including improvement scores, muscle strength, and overall disease activity. The study is estimated to conclude in August 2026.
The investigational drug: Efgartigimod PH20 SC is an immunomodulatory medication that works by targeting and reducing the activity of certain antibodies that contribute to muscle inflammation.
Summary
These six ongoing clinical trials represent significant advances in research for idiopathic inflammatory myopathy. A notable pattern emerges showing strong concentration of trials across multiple European countries, with Germany participating in four of the six trials, demonstrating particular commitment to research in this area. Many trials span numerous countries, reflecting the collaborative nature of European medical research and the relatively rare nature of these conditions requiring multi-center cooperation.
Two distinct therapeutic approaches are being investigated: immunomodulatory drugs that target specific immune system components, and innovative cell-based therapies using modified immune cells. Efgartigimod appears in two separate trials, suggesting significant interest in this particular medication for treating inflammatory muscle diseases. The studies vary in their specific focus, with some examining newly diagnosed patients while others concentrate on long-term safety in patients who have already received initial treatment.
Most trials require participants to have active disease and evidence of muscle inflammation confirmed through various methods including blood tests, imaging, or biopsies. Several studies specifically exclude patients with other serious health conditions or recent infections, reflecting the need for careful safety monitoring when testing immune-modulating treatments. The treatment periods range from 12 weeks to over a year, with some studies extending follow-up until 2026 or 2027 to gather comprehensive long-term safety data.
