KD025 is an investigational medication undergoing testing to determine if it may be effective in the treatment of diffuse systemic sclerosis (skin thickening on more than just the hands). KD025 has previously been tested in graft-versus-host disease, idiopathic pulmonary fibrosis, and psoriasis. It has shown preliminary effectiveness and safety in the treatment of these conditions. This study will randomly assign subjects to one of three treatment groups, 20mg of KD025 twice per day, 20mg of KD025 once per day, and placebo. The study will measure the improvement, stabilization or worsening of your symptoms, such as changes in your fatigue and pain levels, lung function, skin thickness and other patient reported outcomes. The study treatment period will last 1 year. The drug may help mitigate symptoms of systemic sclerosis and thus may be helpful with the disease in study. The population to be enrolled in this study will involve patients diagnosed with systemic sclerosis, diffuse subset, 18 years of age or older.
This research study aims to determine a less invasive way to assess heart function by taking measurements of the heart while subjects are performing an exercise cardiac MRI. Subjects will undergo two exercise phases and MRI measurements will be taken after each exercise phase. These measurements will be compared to available clinical data (including demographic, hemodynamic, radiologic, and functional) and future outcome data.
This is a pilot research study to determine the feasibility of gentle yoga and breathing exercises for symptom management in patients with scleroderma. Participants will have a first study visit that includes completing demographic and quality-of-life surveys, learning the gentle yoga and breathing exercises, and providing two saliva samples before and after doing the exercises. After this visit, participants will do the gentle yoga and breathing exercises twice daily at home (morning and evening) by following a video hyperlink. Participants will make an entry in their Yoga Practice Diaries each time they practice at home. On the 12th Week, participants will return to the clinic for a final study visit that includes doing the gentle yoga and breathing exercises, providing saliva samples, and completing a satisfaction survey. There will be a total of 2 clinic visits (on Weeks 1 and 12). The study coordinator will telephone participants weekly during the 10 weeks that they do the gentle yoga and breathing exercises at home to answer questions and help solve any problems. Each clinic visit will take about 2 hours for this research study, in addition to any routine clinical examinations. Each home yoga and breathing practice will take 1 hour. The total study duration is 12 Weeks (3 months). Participants will receive compensation for their travel in the form of prepaid Visa gift cards.
Scleroderma (systemic sclerosis) is a chronic autoimmune disease, characterized by dysregulation of immune cells in the blood and subsequent fibrosis and vascular dysfunction, associated with significant mortality and morbidity, disproportionately affecting women and African Americans, and without satisfactory treatments. Monocytes, a type of blood immune cells, are critically involved, but the mechanisms responsible for their deregulation in scleroderma remain largely unknown. The goal of this project is to understand how the regulation of monocytes differs between scleroderma and healthy individuals. Volunteers will be asked to provide a blood sample, for which modest compensation will be provided. This is not a drug study.
The goal of this study is to develop an early systemic sclerosis (SSc) registry in the United States (US). A registry is a group of patients that are observed over time. This is a non-interventional study, meaning that they are no study specific medications to take or procedures to undergo. The specific aims include ongoing assessment of the natural history of early SSc patients by capturing and analyzing clinical data, patient reported outcomes, and laboratory data. This is a multi-center study with sites spread across the U.S. This study is funded by the Scleroderma Research Foundation.
The purpose of the study is to generate a bio bank of specimens for research. We will tissue that would otherwise be discarded from clinical or surgical procedure and information from medical records. We will also collect discarded blood, urines and sputum. Collecting samples will help to better understanding the mechanisms of cardiovascular diseases, identify biomarkers for early diagnosis and to predict safety and efficacy of new therapies.
Patients with systemic sclerosis (SSc) related pulmonary arterial hypertension (SScPAH) have a worse prognosis than those with idiopathic PAH. We have recently discovered that heart cells in SScPAH do not contract or squeeze as well as in other forms of pulmonary hypertension. However, the mechanism leading to this dysfunction is not understood. To better study this and in hopes of developing a future therapy, we plan to collect tissue samples via a heart biopsy at the time of a clinically indicated heart catheterization.
This study will assess how 18 months of oral mycophenolate will compare to 18 months of mycophenolate plus pirfenidone, in the treatment of Systemic Sclerosis related Interstitial Lung Disease. Tolerability and toxicity will also be assessed, during this study.
This research is designed to test whether combining pirfenidone and mycophenolate will result in a more rapid and possibly greater improvement in lung function than occurs when mycophenolate is used alone. While both of these drugs have been approved by the U.S. Food and Drug Administration (FDA) to treat other medical conditions, neither drug has been FDA-approved for the treatment of scleroderma related lung disease. This research is being funded by the drug company, Genentech.
Often considered as related diseases, systemic lupus erythematosus (SLE) and systemic sclerosis (SSc) are severe autoimmune disorders characterized, among other, by dysregulation of immune cells in the blood. The roles of different immune cells in SLE and SSc remain unclear. It is of increasing importance to characterize specific immune cells and define their impact on autoimmune disease, which may lead to new therapies. The goal of this study is to identify blood immune cells associated with SLE and SSc.
This study is being conducted in order to test the safety of Brentuximab Vedotin in subjects with Diffuse Cutaneous Systemic Sclerosis. The study involves a Screening visit, a Baseline visit and 12 study visits occurring every 3 to 4 weeks, with enrollment lasting approximately 52 weeks, during which enrolled subjects will receive either the study compound or placebo.