Active clinical trials for "Scleroderma, Systemic"

The Scleroderma Cyclophosphamide Or Transplant (SCOT) Trial is a Phase II/III interventional trial comparing two treatments for early, severe scleroderma. These two interventions are high dose immunosuppressive therapy followed by autologous stem cell transplantation and monthly high dose pulse cyclophosphamide (the later for 12 doses). While standard of care might be considered the optimal control arm for a trial such as this one, no such standard of care is available for the population of scleroderma patients defined by the eligibility criteria for this trial. The rheumatologists on the protocol team believe that the SCOT cyclophosphamide regimen represents the best control arm for this study. However, given concerns over use of a treatment arm as a control that has not been established as a standard of care, this registry was established. The registry will be a prospective, observational study of subjects with severe systemic sclerosis (SSc) who are eligible to participate in the Scleroderma Cyclophosphamide or Transplantation (SCOT) Study but are denied insurance coverage or decline to participate prior to randomization. Subjects will be accrued over the same period as the SCOT study. Subjects will follow the course of treatment prescribed by their treating physician with no interference from the registry. The primary purpose of this study is to document the disease course and outcome in a group of participants who are eligible for the SCOT study, but declined to participate, in order to determine whether their outcome is better, worse, or no different than those who participate in the treatment phase of the trial.

The prognosis of rheumatic diseases has improved considerably with development of therapy. However, infections are considered the most important cause of morbidity and mortality in this group of patients. One of the ways to prevent such complications is vaccination. In 2009, a new pandemic strain of influenza virus (A/H1N1/2009) has emerged raising major concerns for public health. Patients under immunosuppressive therapy have indication for immunization against influenza virus H1N1. There are, however, concerns about possibility of reactivation of autoimmune diseases, determine adverse events and insufficient immunogenicity in these patients. The lack of studies evaluating the efficacy and safety of the vaccine against influenza A(H1N1)/2009 in these rheumatic patients led to the development of this research. The objectives of this study are to evaluate the humoral response and safety of the vaccine virus A(H1N1)/2009 in immunosuppressed patients with rheumatic diseases compared to healthy controls. We have recruited 400 patients with rheumatoid arthritis, 350 with spondyloarthritis, 1000 with systemic lupus erythematosus (SLE), 150 with dermatomyositis (DM), 100 with mixed connective tissue disease, 150 with systemic vasculitis, 250 with systemic sclerosis (SSc) , 100 with Sjögren's syndrome, 100 with antiphospholipid syndrome, 100 patients with juvenile idiopathic arthritis, 80 with juvenile SLE, and 80 with juvenile DM, followed at our Rheumatology Outpatient Division and Unit Pediatric Rheumatology Children's Institute, HC-FMUSP. The control group was recruited were 200 healthy employees of ICHC-FMUSP. Informed consent was obtained from all participants and the study was approved by the Local Ethical Committee. All subjects were vaccinated against influenza virus A/(H1N1)/2009 (vaccine approved and supplied by Instituto Butantan-São Paulo). Blood samples was collected to measure levels of antibodies inhibiting hemagglutination by influenza virus A (H1N1)/2009 immediately prior to vaccination and 21 to 28 days after vaccination., Participants fulfilled a questionnaire on the immediate side effects of the vaccine. All patients with rheumatoid arthritis, spondyloarthritis, SLE, DM, systemic vasculitis, juvenile idiopathic arthritis, juvenile SLE, and DM were assessed before and 21 days after vaccination for clinical, laboratory parameters of disease activity as well as treatment. Continuous variables will be compared by t-test to evaluate differences between patients with rheumatic diseases versus healthy controls. Differences between categorical variables will be evaluated using the chi-square or Fisher exact test. Statistical significance was set at p<0.05.

Systemic Sclerosis (SSc) is an auto-immune systemic disease characterized by vascular damage, cutaneous and visceral fibrosis and a dysimmune condition. Therapies in this disease remain insufficient and the complications resulting from organs involvement lead to strong morbi-mortality.The dermic infiltrate of the patients includes a strong proportion of Tcells. T cells and Natural Killer (NK) cells are potentially involved in the vascular damage of the SSc. However mechanisms at the onset of this endothelial cytotoxicity and its impact on the capacities of regeneration of the endothelial tissue remain poorly understood. Fractalkine is at the same time an endothelial membrane-bound adhesion molecule and a chemokine which is able to bind CX3CR1 expressed by the immune populations. The purpose of the project is to define the role displayed by cytotoxic, circulating immune populations of SSc patients in endothelial cytotoxicity as well as the role of the axis Fractalkine / CX3CR1 in mediating the interactions between the immune cytotoxic cells and the endothelium.

The aim is to find the presence of anti vitamin D antibodies in scleroderma patients and compare with control. A second goal is anti vitamin D levels in serum of scleroderma patients in relation to the clinical manifestations of the disease.

New therapeutic approaches of MS are emerging, targeting different actors of the immune system. Some of them target a specific population of white blood cells: B lymphocytes composed of different subpopulations. The subsets of B cells express different functional properties that control the immune response, but these regulation mechanisms have yet to be clearly described. Some subpopulations could amplify inflammation through IL-6 production for example, whereas some ones contribute to its regulation through the production of IL-10. Using samples collected in a large cohort of individuals with risk of MS and treatment-naive patients in the early onset of the disease, the investigators aim to develop a 2 year follow-up study of the different blood B cells subset distribution and their functional properties in terms of pro- and anti-inflammatory cytokine production in MS. This approach can identify new biomarkers for monitoring of MS patients and lead to better define the indication use of depletive B cell drugs and not to counteract the regulatory action of these cells.

Vitamin D has been considered to possess anti-inflammatory and antimicrobial activity which may be a link for the known interaction of Systemic Sclerosis (SSc) and coronary heart disease (CHD). This study investigated the association between serum vitamin D levels and SSc and periodontitis in patients with SSc, CP and with CHD. Furthermore, the objective was to determine if periodontitis and CHD had an impact on serum vitamin D levels.

The safety and efficacy of vaccination against influenza in patients with scleroderma is not clear. The objective of this study is to assess its safety and efficacy in 50 patients with scleroderma in comparison with 30 controls

This study will examine the possible relationship between silicone implants or injections and the connective tissue diseases scleroderma and myositis. It will explore whether certain factors in the blood or the immune system or other factors are involved in the development of these diseases following silicone implantation or injection. Men and women 18 years of age and older who meet the following criteria may be eligible for this study: Group 1-Patients who have had silicone implants or injections and who later developed scleroderma or myositis Group 2-Patients with scleroderma or myositis who have not had silicone implants or injections Group 3-Healthy volunteers who have had silicone implants or injections and did not develop symptoms or other medical features of connective tissue disorders. Participants will have a thorough history and physical examination, blood and urine tests, chest X-ray and lung function tests. In addition, patients will complete a questionnaire about their procedure (including information such as the types of implanted devices and injections, reason for the procedure, post-operative complications, other illnesses or medical conditions present before and after the procedure, etc.).

Researchers are testing a web-based peer-led program to help manage energy and symptoms in people who have scleroderma. Resilience-based, Energy Management to Enhance Wellbeing (RENEW) was created by researchers, doctors, and patients with scleroderma. The goal is to help people with scleroderma feel better.

This is a pragmatic study in which will compare a detailed treat-to-target (T2T) treatment algorithm to standard care for SSc SIBO at multiple sites around the world. The treatment algorithm was developed from the results of a survey of SIBO treatment preferences of rheumatologists and gastroenterologists. Although the drugs in the algorithm are already used in SSc, there is no uniform way of doing this and assessing the patient response. A very standardized protocol was created with details of how to use the medications, the duration of use and the timing of different drugs. In addition, symptoms of SIBO will be dectected by having patients complete a validated screening questionnaire, the global symptomatic score (GSS), online every 3 months for the duration of the study. A score > 5 is very strongly related to bacterial overgrowth. In other studies, about 40% of unselected patients score at this level. This same questionnaire will be used in the T2T doctors' offices to decide if response is adequate and will also be used to assess outcome in the algorithm group versus standard care group. The primary outcome is the change in symptoms based on the total GSS. Secondary outcomes will include examination of all GSS subscales. HRQoL will be assessed by the social scale of the newly developed UCLA SSc GIT 2.0 questionnaire, which has become the standard GI questionnaire in SSc trials. RN. # 00296313