Myasthenia gravis is an autoimmune disease; a disease that occurs when the immune system attacks the body's own tissues. In generalized myasthenia gravis (gMG), that attack interrupts the connection between nerves and muscles; called the ?neuromuscular junction.' This study is to study the safety, tolerability and efficacy of a study drug called Zilucoplan in adult participants with gMG.
Participation in this study will last approximately 16 weeks and will include approximately 7 study visits.
The purpose of this study is to find out more information about the study drug iloprost for the treatment of symptomatic Raynaud's phenomenon (RP) attacks in people with scleroderma. A Raynaud's attack is defined as one where you notice at least one color change of your finger(s) (blue, white, or red) associated with at least one symptom (pain, numbness, tingling, and/or discomfort of the finger[s]). Your participation in this study will last approximately 9 weeks and will include 8 visits to the study center and 1 phone call from the study staff.
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.
The purpose of this research study is to evaluate the value of educational information given on an iPad about the risks and benefits of lupus medications. The information is intended to encourage conversations between the patient and doctor about lupus treatments. This research will test the feasibility and effectiveness of using the iPad in lupus clinics nationwide. Participants will be given information about lupus treatments on an iPad during the clinic visit before seeing their doctor and will be interviewed about the feasibility of using the iPad during a regular clinic visit.
The study is using cemdisiran compared to placebo injections to determine the safety and efficay of cemdisiran in treating IgA Nephropathy. The study includes a screening period (up to 90 days), 8 month treating period, and 52 week Open Label extension period. Injections occur monthly during the treatment period. Patients will be randomized 2:1 to the cemdisiran or placebo arms.
Generalized Myasthenia gravis (gMG) is a rare neuromuscular inflamation disorder that involve all voluntary muscle groups. This study is to evaluate the safety and efficacy of ravulizumab for the treatment of patients with generalized myasthenia gravis (gMG).
There will be 3 periods in this study; a 4 week Screening Period, a 26-week Randomized-Controlled Period consisting of 8 study visits, and an Open-Label Extension Period consisting of 17 visits that will continue for up to 2 years. Meningococcal vaccination is required for all patients prior to intravenous administration of study drug. The overall study duration for an individual participant is estimated to take up to 2.5 years.
The purpose of this study is to evaluate how safe and effective intravenous eculizumab is in pediatric patients 6 to less than 18 years old with generalized Myasthenia gravis (gMG). Eculizumab is already approved for use in adult patients with gMG in the US, Europe and Japan, but currently has not been approved for use in pediatric patients.
The study's duration is approximately 4.7 years with 4 treatment periods consisting of a Screening Period (2 to 4 weeks), Primary Evaluation Treatment Period (26 weeks), Extension Period (up to an additional 208 weeks), and Follow-up Period (8 weeks). All patients who complete Week 26 of Study ECU-MG-303 will continue receiving eculizumab in the Extension Period of this study for up to an additional 208 weeks.
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 primary objective of this study is to evaluate the efficacy of lenabasum compared to placebo in participants with dermatomyositis (DM), and to evaluate the safety and tolerability of lenabasum in participants with Dermatomyositis (DM).
Autoimmune diseases such as DN result from the immune system becoming over-active and attacking parts of the body. This over-active immune response also causes chronic inflammation. The growth of scar tissue in muscle, skin and internal organs with chronic inflammation from DM makes them not work as well as they should. Lenabasum may help the body stop the chronic inflammation and stop scarring fro getting worse without preventing the normal response of the immune system.
Lenabasum is an investigational drug that will be taken orally twice a day. The study is divided into 2 separate parts, Part-A is the main study and if participants qualify they can volunteer to participate in the optional extension Part-B of the study. It will take about 14 months to complete Part-A of this research study. During this time, participants will make a total of 12 study visits. The optional extension Part-B of this study is expected to last a year consisting of approximately 8 visits.
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.