Ongoing Clinical Trials for Immune-Mediated Myositis
Two clinical trials are currently investigating new treatment approaches for immune-mediated myositis, a group of rare autoimmune conditions where the body’s immune system attacks its own muscles, causing inflammation and weakness. These studies are testing innovative therapies including cell-based treatments and oral medications that may help patients maintain stable disease control without relying on traditional intravenous treatments.
Clinical trial locations
- Austria
- France
- Germany
- Italy
- Netherlands
- Spain
Study on the Effects of Rapcabtagene Autoleucel and Drug Combination for Patients with Severe Refractory Inflammatory Muscle Diseases
This trial is investigating a new treatment approach for patients with severe inflammatory muscle diseases that have not responded to standard treatments. The study focuses on rapcabtagene autoleucel, an innovative cell therapy that uses the patient’s own immune cells, which are modified in a laboratory to better fight the disease.
Who can participate: The trial is open to men and women between 18 and 65 years old who have been diagnosed with myositis according to specific medical guidelines from 2017. Participants must test positive for myositis-specific antibodies, which are special proteins in the blood that help diagnose the condition. You should have active disease that is currently causing symptoms and must have tried high-dose glucocorticoids without success, as well as two other treatments that did not work. The trial requires that participants have severe myositis confirmed by a special medical committee.
Who cannot participate: The study excludes patients who have conditions other than severe refractory idiopathic inflammatory myopathies, those not within the specified age range, individuals unable to give informed consent or understand the study requirements, and members of vulnerable populations that the study is not designed to include.
Treatment approach: The main treatment being tested is rapcabtagene autoleucel, given as a single infusion directly into the bloodstream. Before this main treatment, participants receive rituximab and fludarabine phosphate through intravenous infusion to prepare the body. After the main treatment, supportive medications including mycophenolic acid and tacrolimus taken orally, as well as tocilizumab given intravenously, help maintain the treatment effects and manage the immune response. The study team will closely monitor participants throughout the year-long trial to assess improvements in their condition and check for any side effects.
Study goal: The primary aim is to determine whether rapcabtagene autoleucel can provide better results compared to other treatments selected by doctors. The study will measure improvements using the Total Improvement Score by week 52 and evaluate the safety and effectiveness of this novel cell therapy approach for severe inflammatory muscle diseases.
Study on Upadacitinib for Patients with Idiopathic Inflammatory Myopathies After Stopping IVIG
This clinical trial is evaluating whether patients with inflammatory muscle diseases can maintain stable disease control using an oral medication called upadacitinib after stopping their regular intravenous immunoglobulin treatments. The study covers several types of inflammatory muscle diseases including polymyositis, dermatomyositis, antisynthetase syndrome, overlap myositis, and immune-mediated necrotizing myopathy.
Who can participate: Eligible participants are adults between 18 and 65 years old who have been diagnosed with one of the specified inflammatory muscle diseases. You must have been receiving intravenous immunoglobulin at a stable dose for at least 12 weeks before joining the study and have had stable disease activity for at least 3 months according to your doctor’s judgment. Participants should also be on a stable regimen of background treatments such as immunosuppressive drugs, antimalarials, or low-dose corticosteroids for at least 12 weeks. Female participants must agree to use effective birth control methods unless they are unable to have children due to age or surgical procedures.
Who cannot participate: The study excludes patients who do not meet the age requirements or those who are part of vulnerable populations requiring special protection. Individuals who do not have the specific muscle diseases being studied or who cannot meet other eligibility criteria are also excluded.
Treatment approach: Participants are randomly assigned to receive either upadacitinib 30 mg or a placebo in a double-blind design, meaning neither participants nor researchers know who receives the actual medication. The treatment is taken orally once daily in the form of a prolonged-release tablet that slowly releases the medication over time. Participants continue their regular background treatments while taking the study medication for up to 16 weeks, with continued monitoring through week 20.
Study goal: The primary objective is to assess whether patients can maintain stable disease activity without needing intravenous immunoglobulins by week 16. Upadacitinib works by inhibiting Janus kinase enzymes, which play a role in the inflammatory process, thereby reducing inflammation and immune response. The study will also evaluate muscle strength, quality of life, time to symptom worsening, and any side effects experienced during the 20-week trial period.
Summary
The two ongoing clinical trials for immune-mediated myositis represent different approaches to managing these challenging conditions. One study focuses on patients with severe, treatment-resistant disease using an innovative cell therapy approach with rapcabtagene autoleucel, conducted across multiple European countries including Germany, Spain, France, Netherlands, and Italy. The other trial, taking place in Austria, investigates whether patients with more stable disease can reduce their reliance on intravenous immunoglobulin treatments by using the oral medication upadacitinib.
Both studies reflect the ongoing search for more effective and convenient treatment options for patients living with inflammatory muscle diseases. The cell therapy trial targets those who have exhausted other treatment options, while the upadacitinib study aims to improve quality of life by potentially replacing regular intravenous infusions with a once-daily oral medication. These trials offer hope for advancing the understanding and treatment of these rare autoimmune conditions.
