9 Clinical Trials for Various Conditions
This study will define the major genetic risk and protective factors for idiopathic inflammatory myopathies (IIM), a group of immune disorders affecting connective tissues such as muscles. It will also identify new environmental risk factors for IIM and identify immune responses in myositis and related diseases. There are many forms of IIMs, and the causes of these diseases are unknown. However, scientists suspect that they result when people with some genetic factors that predispose them-that is, put them at greater risk-are exposed to certain environmental triggers. Some of those triggers include food, drugs, biologics (such as a vaccine to prevent disease), medical devices and occupational exposures. Patients, including children under 18, who had a diagnosis of myositis, a related autoimmune disease, or a rheumatic disease, as well as their blood relatives, and control subjects who were in good health have already been recruited for this study. The evaluation consisted of one outpatient visit to the patient's doctor, who will obtain a medical history and conduct a physician examination. Patients spent 20 to 30 minutes to answer written questions. There was a blood collection of about 6 tablespoons. If there was a major change in patients' medical conditions, they were asked to return for a second outpatient evaluation to determine whether any of the blood tests or antibodies, which show an immune response, had changed. Blood samples collected will be used only for laboratory research studies. The samples have been identified by a code, and all other identifying information have been removed. During the study, researchers will explore possible environmental risk factors, including studies of infectious and non-infectious agents. They will analyze the blood for genetic markers and test for certain antibodies. Laboratory results will be evaluated as they relate to the signs, symptoms, and severity of patients' illnesses. That would help researchers to better understand patterns of the diseases and the outcomes for patients. This study will not have a direct benefit for patients. However, results from the study can be made available to patients' doctors for use in appropriate care. Also, it is hoped that information gained can help other people in the future.
Autoimmune/Connective Tissue Diseases, Idiopathic Inflammatory Myopathies, Polymyositis, Dermatomyositis
Juvenile dermatomyositis (JDM) is a connective tissue disease that causes skin rash and weak muscles in children. The purpose of this study is to measure the absorption of oral prednisolone and intravenous (IV) methylprednisolone and to determine levels of disease activity indicators in the blood. These levels will be compared to see if there are patterns specific to active and less active JDM.
Vasculitis, Hypersensitivity, Connective Tissue Diseases, Dermatomyositis, Vasculitis
This study of inflammatory muscle diseases-polymyositis and dermatomyositis and related disorders-will examine what causes these diseases and describe the clinical features (signs and symptoms) associated with them. Inflammation and degeneration of skeletal muscles in these disorders leads to weakness and muscle wasting. The skin, lungs and other organs may also be involved. Patients 16 years of age and older with polymyositis, dermatomyositis, or a related disorder may be eligible for this study. Participants will undergo a complete history and physical examination, including routine blood and urine tests. Additional procedures for diagnosis, treatment or research may include: 1. Blood sample for genetic studies. 2. Muscle biopsy-removal of a tissue sample for microscopic examination. Under local anesthetic, a 1/2- to 1-inch long incision is made in the thigh or upper arm, and a small piece of muscle is removed. 3. Electromyography-measurement of the electrical activity of a muscle. A needle is inserted through the skin into a muscle to record its electrical activity. 4. Magnetic resonance imaging-visualization of organs or tissues, using a magnetic field and radio waves. The patient lies on a table inside a narrow cylinder (the MRI scanner) with a strong magnetic field for the scanning. 5. Manual muscle strength testing by a physiotherapist. 6. Swallowing studies using ultrasound (imaging using sound waves) and X-rays (barium swallow) to evaluate swallowing and speaking abilities. 7. Questionnaires on swallowing ability and ability to perform daily living activities 8. Pulmonary function tests-measurement of movement of air in and out of the lungs. The patient breathes into a machine to evaluate lung function. 9. Chest X-rays to evaluate lung function. 10. Electrocardiogram and, if necessary, Holter monitoring (measurement of the electrical activity of the heart) and echocardiogram (ultrasound imaging of the heart) to evaluate heart function. 11. Apheresis-collection of white blood cells for research. Whole blood is collected through a needle placed in an arm vein. The blood circulates through a machine that separates it into its components. The white cells are removed and the rest of the blood is returned to the body through the same needle or through a second one placed in the other arm. 12. MR guided muscle biopsy-measurement of glycogen in muscle tissue using magnetic resonance imaging. Certain patients may undergo this experimental procedure to compare MRI findings with those of muscle biopsy. The affected muscles are identified using MRI and the biopsy incision is made. MRI is then used to guide the biopsy needle to the muscle and a small piece is removed. Patients who are eligible for experimental treatment studies will be offered the opportunity to join them. Others will be advised of treatment recommendations.
Dermatomyositis, Polymyositis
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.
Systemic Sclerosis (SSc), Dermatomyositis, Mixed Connective Tissue Disease (MCTD), Calcinosis
To facilitate clinical, basic science, and translational research projects involving the study of rheumatic diseases.
Rheumatic Diseases, Adult Onset Still Disease, Ankylosing Spondylitis, Psoriatic Arthritis, Reactive Arthritis, Antiphospholipid Syndrome, Systemic Lupus Erythematosus, Behcet Disease, Dermatomyositis, Polymyositis, Giant Cell Arteritis, Lyme Disease, Mixed Connective Tissue Disease, Polymyalgia Rheumatica, Rheumatoid Arthritis, Sarcoidosis, Systemic Sclerosis, Scleroderma, Sjogren's Syndrome, Undifferentiated Connective Tissue Diseases
Patient Power is a patient research network and database (registry) to collect prospective information about demographics, self-reported diagnoses and medications, and willingness to participate in research from participants with rheumatoid arthritis (RA), spondyloarthritis (SpA), other musculoskeletal conditions, chronic neurological conditions like migraine, chronic pulmonary conditions like Chronic Obstructive Pulmonary Disease (COPD), asthma, autoimmune dermatological conditions such as psoriasis, and other chronic inflammatory or immune-mediated conditions. In addition, since patients with chronic conditions often have other co-morbidities like cardiovascular health and obesity-related metabolic disorders, these conditions will also be included. Participants will provide information from their smartphones or personal computers. The information will be used by researchers and clinicians to help patients and their providers make better, more informed decisions about treatment of chronic conditions.
Rheumatoid Arthritis, Ankylosing Spondylitis, Fibromyalgia, Gout, Crohn Disease, Juvenile Idiopathic Arthritis, Lupus Erythematosus, Myositis, Osteoarthritis, Osteoporosis, Psoriasis, Psoriatic Arthritis, Scleroderma, Dermatomyositis, Inflammatory Bowel Diseases, Polymyositis, Axial Spondyloarthritis, Diffuse Idiopathic Skeletal Hyperostosis, Polymyalgia Rheumatica, Giant Cell Arteritis, Temporal Arteritis, Wegener, Relapsing Polychondritis, Undifferentiated Connective Tissue Disease, Spinal Cord Injuries, Alzheimer Disease, Amyotrophic Lateral Sclerosis, Ataxia, Bell Palsy, Brain Tumor, Cerebral Aneurysm, Epilepsy, Guillain-Barre Syndrome, Headache, Head Injury, Hydrocephalus, Lumbar Disc Disease, Meningitis, Multiple Sclerosis, Muscular Dystrophy, Neurocutaneous Syndromes, Parkinson Disease, Stroke, Cluster Headache, Tension-Type Headache, Chronic Obstructive Pulmonary Disease, Asthma, Lung Cancer, Cystic Fibrosis, Sleep Apnea, Eczema, Alopecia, Chronic Inflammation, Unstable Angina, Heart Attack, Heart Failure, Arrythmia, Valve Heart Disease, High Blood Pressure, Congenital Heart Disease, Peripheral Arterial Disease, Diabetes, Chronic Liver Disease, Obesity
This CARRA Registry study will create a foundational database for rheumatic diseases of childhood using a novel informatics infrastructure developed as part of the larger clinical project. The creation of a CARRA-wide informatics infrastructure will enable efficient, observational, disease-related data capture across all CARRA sites for pediatric rheumatic diseases. The CARRA Registry study will demonstrate the feasibility of expanding to more data intensive registries for observational studies, comparative effectiveness research, pharmaceutical clinical trials and translational research.
Juvenile Idiopathic Arthritis, Systemic Lupus Erythematosus, Mixed Connective Tissue Disease, Juvenile Ankylosing Spondylitis, Juvenile Dermatomyositis, Localized Scleroderma, Systemic Sclerosis, Vasculitis, Sarcoid, Fibromyalgia, Primary, Auto-inflammatory Disease, Idiopathic Uveitis Idiopathic
The first objective of this protocol is to assess the tolerability and safety of N-acetylcysteine (NAC) in patients with connective tissue disease related interstitial lung disease (CTD-ILD).
Interstitial Lung Disease, Connective Tissue Disease
OBJECTIVES: I. Determine the safety and long term complications of total body irradiation in combination with cyclophosphamide, anti-thymocyte globulin, and autologous CD34-selected peripheral blood stem cell (PBSC) transplantation in children with refractory autoimmune disorders. II. Determine the efficacy of this treatment regimen in these patients. III. Determine the reconstitution of immunity after autologous CD34-selected PBSC transplantation in these patients. IV. Determine engraftment of autologous CD34-selected PBSC in these patients.
Systemic Sclerosis, Systemic Lupus Erythematosus, Dermatomyositis, Juvenile Rheumatoid Arthritis, Autoimmune Diseases