Clinical Research Directory

A Study to Assess the Long-term Safety and Efficacy of a Subcutaneous Formulation of Efgartigimod in Adults With Active Idiopathic Inflammatory Myopathy

The main purpose of this study is to measure the long-term safety and tolerability of efgartigimod PH20 SC in adult participants with Idiopathic Inflammatory Myopathy (IIM) who previously participated in ARGX-113-2007. The study consists of a treatment period where participants will receive efgartigimod PH20 SC for up to 51 months. The treatment period will be followed by a treatment-free safety follow-up period of 56 days.

Teams Engaged in Accessible Mental Health Interventions for Lupus Erythematosus and Dermatomyositis Stress

The objectives of this study are to determine if the 'Teams Engaged in Accessible Mental Health Interventions for Lupus Erythematosus and Dermatomyositis Stress' (TEAM-LEADS) intervention is feasible and acceptable to adolescents and young adults with lupus and dermatomyositis and whether it can help reduce stress and promote cardiovascular health behaviors in these individuals.

Adult and Juvenile Myositis

This study will evaluate subjects with adult- and childhood-onset myositis to learn more about their cause and the immune system changes and medical problems associated with them. Myositis is an inflammatory muscle disease that can damage muscles and other organs, resulting in significant disability. Children or adults with polymyositis or dermatomyositis or a related condition may be evaluated under this study. Healthy children or adults will also be enrolled as "controls," for comparison of test results. All patients will undergo a complete history (including completing some questionnaires) and physical examination, review of medical records, and blood and urine tests. Patients may then choose to participate in an additional 1- to 5-day evaluation, which will include some or all of the following diagnostic, treatment or research procedures: 1. Standardized muscle strength testing, range of motion of joints and walking (gait) analysis by a physiotherapist; completion of a questionnaire regarding ability to perform daily tasks 2. Skin assessment, possibly including photographs of lesions and a skin biopsy (removal of a small skin sample under local anesthetic) 3. Magnetic resonance imaging (scans that use magnetic fields to visualize tissues) of leg muscles 4. Swallowing studies, including a physical examination and questionnaire on swallowing ability, studies of tongue strength, and ultrasound imaging during swallowing, and possibly, a modified barium swallow 5. Voice and speech assessment, possibly including computerized voice analysis and laryngoscopy-analysis of the larynx (voice box) using a small rigid scope with a camera placed in the mouth to view and record vocal cord function 6. Pulmonary function tests (measurement of air moved into and out of the lungs, using a breathing machine) to evaluate lung function and, possibly, chest X-ray 7. Electrocardiogram (measurement of the electrical activity of the heart) and, possibly, echocardiogram (ultrasound imaging of the heart) 8. Endocrine evaluation 9. Eye examination, in patients with vision loss or other eye symptoms 10. Nutrition assessment to evaluate muscle mass and muscle wasting, including tape measurements or bioelectric impedance testing, a painless procedure in which wires are attached to the extremities with a sticky paste. 11. Muscle ultrasound. 12. Electromyography (record of the electrical activity of muscles) 13. Muscle or skin biopsy (removal of a small piece of muscle tissue for microscopic examination) All patients may have only a one-time evaluation or may return for one follow-up evaluations (either the 1-day or 3- to 5-day evaluation) over a 1-year period. Healthy children will undergo a medical history and brief physical examination; blood and urine tests; speech and swallowing studies including questionnaires and physical examination, tongue strength, and ultrasound study; and bioelectric impedance testing. Children 8 to 18 years old may also have exercise testing.

Environmental Risk Factors for Myositis in Military Personnel

Background: * Myositis is a rare disease in which the body s immune cells attack the muscle tissue. It can cause muscle weakness, swelling, and pain. It can develop in people with no history of muscle problems. Environmental exposures may determine who develops myositis. Genes may also affect development of the disease. * Some people who serve in the military develop myositis. However, other military personnel do not. Researchers want to compare military personnel with and without myositis. They will look for common factors that might have led to the disease. Objectives: \- To study environmental risk factors for myositis in military personnel. Eligibility: * Military personnel who developed myositis during their period of service. * Healthy military personnel who do not have myositis or another autoimmune disease. Design: * Participants will have a physical exam and medical history. * Participants will fill out forms about environmental exposures, particularly while in the military. The questions will ask about past infections, vaccines and medications, and personal habits. They will also ask about participants occupations during military service and their deployments. * Participants will also provide blood samples for study. * No treatment will be provided as part of this study.

A Study to Understand How the Study Medicine Dazukibart Works in People With Idiopathic Inflammatory Myopathies

The purpose of this study is to understand how the study medicine, dazukibart, works in people with active idiopathic inflammatory myopathies (dermatomyositis \[DM\] or polymyositis \[PM\]). Idiopathic inflammatory myopathies are a group of disorders that show inflammation of the muscles used for movement. There are several types of idiopathic inflammatory myopathies, including DM and PM. DM and PM involve weakness of the muscles closest to the center of the body, such as the muscles of the hips, thighs, upper arms, and neck. People with these forms of idiopathic inflammatory myopathies may find it difficult to climb stairs, get up from a seated position, or lift items above their head. People with DM can also have a skin rash. These disorders negatively impact the quality of life and functioning of patients. In addition to the above, these disorders can affect how the lungs and heart work. This study is seeking participants who took part in a DM and PM study with dazukibart before. Some participants will receive study medicine, and some participants will not receive study medicine and only complete safety follow-up. The study medicine will be given as an intravenous (IV) infusion (directly into the veins). This takes about 1 hour, every 4 weeks, from Day 1 to Week 48 (about 12 months) of the study. This will be followed by a safety follow-up period that lasts about 4 months after the last infusion. Participants who receive study medicine will have about 18 study visits at the site over about 16 months. There will also be participants enrolled in this study who will not receive study medicine. Such participants will only take part in safety follow-up visits as they do not want to or are not eligible to receive dazukibart. These participants will not receive study medicine and will have up to 4 study visits at the site every 4 weeks to complete safety follow-up.

RESET-Myositis: An Open-Label Study to Evaluate the Safety and Efficacy of CABA-201 in Subjects With Active Idiopathic Inflammatory Myopathy or Juvenile Idiopathic Inflammatory Myopathy

RESET-Myositis: Open-Label Study to Evaluate the Safety and Efficacy of CABA-201 in Subjects with Active Idiopathic Inflammatory Myopathy or Juvenile Idiopathic Inflammatory Myopathy

A Study Evaluating the Effects of GLPG3667 Given as Oral Treatment for up to 24 Weeks in Adults With Dermatomyositis

The purpose of this study is to evaluate the efficacy, safety, tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) of orally administered GLPG3667 once daily for 24 weeks in adult participants with dermatomyositis (DM), followed by an open-label extension (OLE) period until Week 48.

Testing an Immunotherapy Anti-cancer Drug, Nivolumab, for Advanced Cancers in Patients With Autoimmune Disorders, AIM-NIVO

This phase Ib trial studies the side effects of nivolumab and to see how well it works alone and in combination with other treatments, such as ipilimumab, cabozantinib, platinum containing therapy, and fluoropyrimidine, in treating patients with autoimmune disorders and cancer that has spread from where it first started (primary site) to nearby tissue, lymph nodes, or distant parts of the body (advanced), to other places in the body (metastatic) or cannot removed by surgery (unresectable). Immunotherapy with monoclonal antibodies, such as nivolumab and ipilimumab, may help the body's immune system attack the cancer, and may interfere with the ability of tumor cells to grow and spread. Cabozantinib blocks certain proteins, which may help keep tumor cells from growing. It may also prevent the growth of new blood vessels that tumors need to grow. Cabozantinib is a type of tyrosine kinase inhibitor and a type of angiogenesis inhibitor. Chemotherapy drugs, such as platinum containing therapies and fluoropyrimidine, work in different ways to stop the growth of tumor cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. Giving nivolumab alone and in combination with other treatments, including ipilimumab, cabozantinib, platinum containing therapy, or fluoropyrimidine, may be safe, tolerable, and/or effective in treating patients with autoimmune disorders and advanced, metastatic, or unresectable cancer.

Environmental Risk Factors for the Anti-synthetase Syndrome

Background: * Like other complex diseases, autoimmune diseases are the result of numerous causes, including genetic and environmental factors. Some researchers believe that people who are susceptible to autoimmune disorders develop them when the body reacts to environmental or other factors by creating white blood cells that attack the body s own tissues, which then progresses to autoimmune diseases. These immune-triggered disorders can overlap with one another to some extent, but most autoimmune diseases have certain distinct triggers. * The autoimmune disorder myositis weakens the muscles and may cause other health problems. Environmental exposures associated with myositis include ultraviolet radiation, stressful life events and muscle overexertion, collagen implants, infections such as retroviruses and streptococci bacteria, and certain drugs and chemicals. Some individuals with myositis also produce proteins in the blood called autoantibodies that react with certain parts of the person s own cells, called synthetases, which are involved in making new proteins. A syndrome called the anti-synthetase syndrome, which includes myositis and lung disease, is associated with having the anti-synthetase autoantibodies. Researchers are interested in studying differences in environmental exposures in individuals with myositis. This study is being conducted to determine if persons with the anti-synthetase syndrome have had different environmental exposures before disease onset compared with other patients with myositis who do not have this syndrome and also compared with healthy volunteers. Objectives: \- To determine whether selected infectious and noninfectious environmental exposures are more common in individuals who have myositis with the anti-synthetase syndrome, compared with healthy volunteers. Eligibility: \- Individuals who have been diagnosed with myositis (with or without anti-synthetase autoantibodies), and healthy volunteers without autoimmune disorders. Design: * Participants will be screened with a full medical history and physical examination, and will provide blood, urine and house dust samples. * Participants will complete questionnaires about their medical history and the types of exposures they have had at work, at home, and elsewhere. Participants who have myositis will also be asked about certain infections, heavy exercise or physical exertion, sun exposure, tobacco and alcohol use, and stressful events prior to being diagnosed with the disease. Healthy volunteers will be asked about the same exposures before the date of diagnosis of disease of the myositis subject to which they have been matched. * Participants will receive a kit that contains instructions and a filter to be put onto their vacuum cleaner to collect house dust in the bedroom. This dust will be kept for possible future analyses of infectious or toxic agents based on the other results from the study. * Individuals with myositis will have other tests as clinically indicated, including lung function tests and imaging studies.

Biomarkers of Cancer-Associated Myositis

The project is about dermatomyositis (DM), an autoimmune disease characterized by inflammation in the skeletal muscles and skin. Patients with DM have an increased risk of cancer, with a cancer incidence between 5.5% and 42%. Cancers in these patients are one of the leading causes of death. Some myositis-specific autoantibodies have been discovered and some of them are associated with cancer development. However, DM patients negative for all the known autoantibodies can also develop cancer. The Investigator hypothesized that antibodies against plasma membrane antigens and soluble immune checkpoints can be responsible for the association between cancers and autoimmunity. Therefore, the present study aimed to identify novel antigens and immunological pathways in patients with DM with cancer versus patients with DM without cancer, to identify those patients who need cancer screening. The Investigators focus on soluble immune checkpoint molecules and autoantibodies directed against plasma membrane antigens.

Intralesional Injection of STS in Treatment of Calcinosis

The specific objective of this study is to perform a small, open-label study to assess the safety and efficacy of intralesional, subcutaneous injection of STS on calcinosis symptoms and lesion size in systemic sclerosis (SSc), mixed connective tissue disease (MCTD) and dermatomyositis (DM) patients. Injection will be guided by ultrasound, lesion size assessed by ultrasound, and symptom burden by patient-reported outcome measures.

Exploratory Clinical Study of Anti-CD19/BCMA Universal CAR-T Cell Injection for the Treatment of Refractory Autoimmune Diseases

A single arm, open-label pilot study is designed to determine the safety and effectiveness of anti-CD19/BCMA-UCAR-T cells in patients with autoimmune diseases. 36-72 patients are planned to be enrolled in the dose-escalation trial.

Emapalumab MDA5 Rapidly Progressive Interstitial Lung Disease (RP-ILD) Study

This is a proof of concept study to determine if Emapalumab appears effective for the treatment of anti-MDA5 antibody positive rapidly progressive interstitial lung disease (MDA5 RP-ILD). Emapalumab is a medication that is currently used for a severe problem with the immune system, called macrophage activation syndrome, and this disease shares some similar features with MDA5 RP-ILD.

Clinical Responsiveness of Dermatomyositis Using Cutaneous Dermatomyositis Disease Area and Severity Index (CDASI)

The Evaluation of Clinical Responsiveness Using the Cutaneous Dermatomyositis Disease Area and Severity Index (CDASI), established in 2008, is a one-site database study conducted at the University of Pennsylvania. The database has yielded valuable information and clinical insights into the pathophysiology, disease processes, including psychological responses, treatments and quality of life associated with dermatomyositis. The CDASI database incorporates the Cutaneous Dermatomyositis Disease Area and Severity Index), a validated outcome measure of disease responsiveness in patients, and other assessment tools, surveys and patient information to help validate the clinical course and quality of life of patients with dermatomyositis. The CDASI database has led to publication of comparison studies of CDASI and other clinical instruments and the effect of dermatomyositis on Quality of Life (QoL). The CDASI database is an ongoing resource that enables clinicians to evaluate the evolving clinical changes, treatment modalities and patient response to a challenging disease. Data will be analysed over a 5 years.

Add-on Intravenous Immunoglobulins in Early Myositis

In patients with myositis early immunomodulation by intensive treatment ("hit-early/hit-hard" principle) may induce faster reduction of disease activity and prevent chronic disability. Intravenous immunoglobulin (IVIg) in addition to standard treatment with glucocorticoids may be beneficial for this purpose: add-on IVIg improved symptoms in steroid-resistant myositis, and first-line monotherapy IVIg led to a fast and clinically relevant response in a pilot study in nearly 50% of patients with myositis.

Rheumatology Patient Registry and Biorepository

To facilitate clinical, basic science, and translational research projects involving the study of rheumatic diseases.

A Long-Term Follow-Up Study for Participants Previously Treated With KYV-101

The purpose of this long-term follow-up (LTFU) study is to collect delayed adverse events (AEs) and understand the persistence of KYV-101 (autologous CAR T cell product; gene-modified product), in participants who have been administered KYV-101 (gene-modified product; autologous CAR T cell product). This LTFU protocol will be open to any participant who received at least one infusion of KYV-101 in a previous Kyverna sponsored clinical trial or Investigator Initiated Trial (IIT).

RCT of Tocilizumab for Anti-MDA5+DM

The goal of this clinical trial is to learn if tocilizumab works to treat anti-MDA5+ dermatomyositis (anti-MDA5+DM) in adults. It will also learn about the safety of tocilizumab. The main questions it aims to answer are: Does tocilizumab improve patients' clinical symptoms? Does tocilizumab improve patients' respiratory failure? What medical problems do participants have when taking tocilizumab? Researchers will compare tocilizumab to a placebo (a look-alike substance that contains no drug) to see if tocilizumab works to treat patients with anti-MDA5+ DM. Participants will: Take tocilizumab or a placebo every two weeks for 2 months Visit the clinic once every 2 weeks for checkups and tests

MIHRA - Patient-Rooted Insights for Shaping Myositis Science (PRISMS)

Myositis diseases are each rare diseases. As in other rare diseases, people living with myositis diseases face physical and psychosocial challenges that may not be recognized in current research priorities. The PRISMS study is a global investigation that collects patient perspectives through (mostly online) methods of open-ended questions, community forums and survey to identify the most pressing research concerns as identified by patients. Findings will be analyzed to create a patient-voiced set of research priorities that can guide the direction of research and help inform funding decisions across myositis diseases. Potential participants can express interest via https://mihrafoundation.org/mihra-programs/mihra-patient-contact-registry/

Panniculitis in Dermatomyositis

Adipose tissue involvement is rare in dermatomyositis. The occurrence of partial or diffuse lipodystrophy is a rare but well-characterized manifestation, particularly in juvenile forms of dermatomyositis. Panniculitis, on the other hand, is exceptional and rarely described in the literature, mostly in the form of clinical cases. Panniculitis in dermatomyositis is exceptional. There are no validated diagnostic criteria or treatment recommendations. Treatment is based, by analogy, on the therapeutic strategy for lupus panniculitis and involves synthetic antimalarials combined with steroids and/or immunosuppressants. The aim of this study is to describe panniculitis in dermatomyositis in order to determine whether there are clinical or histological characteristics that distinguish it from other causes of panniculitis, particularly panniculitis associated with lupus erythematosus.

Severity Factors of Dermatomyositis in the Caribbean Population - DM-ANTILLES

Dermatomyositis is a rare chronic autoimmune and inflammatory disease that affects the skin and striated muscles. Its prognosis is linked to visceral involvement (lungs, heart, and oropharyngeal region) and to the possible presence of associated cancer. The implementation of the research will allow identification of incident cases of dermatomyositis in Guadeloupe and the characterization of the disease in the overseas population, in the absence of data in the literature.

Topical Ruxolitinib Cream for Refractory Cutaneous Dermatomyositis

This study will assess the safety and efficacy of topical ruxolitinib for treating the refractory cutaneous manifestations in patients with dermatomyositis. The investigators' hypothesis is that topical ruxolitinib will be both safe and effective for such patients.

CAR-T Cells in Systemic B Cell Mediated Autoimmune Disease

The investigational product is designed to effectively combat B cells in patients with autoimmune diseases. Autologous T cells enriched with CD4/CD8 are genetically engineered using a lentiviral vector to express chimeric antigen receptors (CARs) that target the CD19 antigen on the cell surface of B cells and their precursors. During treatment, patients undergo leukapheresis, lymophodepleting chemotherapy and administration of the expanded CD19-CAR-transduced T cells.

A Study to Investigate the Efficacy and Safety of Brepocitinib in Adults With Dermatomyositis

This is a phase 3, multicenter, randomized, placebo-controlled, double-blind study of treatment with brepocitinib (TYK2/JAK1 inhibitor) in adults with dermatomyositis (DM). The primary objective of this study is to assess the efficacy of two dose levels of brepocitinib in comparison to placebo, as measured by differences in the Total Improvement Score (TIS). After 52 weeks of double-blind treatment, participants have the option to continue therapy in a 52 week open-label extension phase where all participants will receive brepocitinib.