View clinical trials related to Scleroderma, Systemic.
Filter by:The purpose of this study is to assess the value in terms of sensitivity, specificity and likelihood ratio of the stress echocardiography in the screening of pulmonary arterial hypertension in patients with systemic sclerosis and indirect signs of pulmonary arterial hypertension.
A phase IIa open-label single center pilot study to assess the safety and efficacy of Nilotinib in patients with Scleroderma.
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.
Raynaud's phenomenon (RP) associated with scleroderma is a difficult problem to treat. Many patients develop ischemic digital ulcers due to severe RP that causes considerable morbidity and adversely affects the quality of life. In an earlier study, we have observed marked improvement in RP attacks and rapid healing of digital ulcers following therapy with phosphodiesterase V inhibitor, Tadalafil. In the present multicentric study we aim to study the efficacy of Tadalafil in patients continuing to have RP attacks despite using at least one or more vasodilators.
Thirty subjects with systemic sclerosis and 30 age and sex matched controls without any known condition that should cause increased skin hardness in the fingers with undergo examination by manual palpation and durometer measured hardness of their digital tuft skin by 2 observers on 2 separate occasions. There will be 1 hour between individual observer's scorings. Observers will be blinded from the observer's scores and from their previous scores. Results will be tabulated and compared for manual scores versus durometer measurements, intra-observer scores by both methods and inter-observer scores by both methods.
Systemic sclerosis is a chronic autoimmune connective tissue disorder with no universally accepted disease modifying regimen. Recruiting patients for systemic sclerosis treatment studies is difficult due to the limited availability of such patients and furthermore the use of a placebo arm is often deemed unethical due to the poor survival of diffuse systemic sclerosis patients. Long-term controlled trials examining functional outcomes and survival from novel therapeutic agents for systemic sclerosis are often difficult to undertake because of costs, rarity of the disease and ethical issues with the use of a true placebo. Open label single center studies while inferior to multicenter placebo controlled studies, have helped establish the benefits of certain pharmaceutical agents in systemic sclerosis, and while not universally accepted as disease modifying agents, have been used with some success to treat systemic sclerosis. The hypothesis on which we are basing this study is that an endothelin receptor antagonist and disease modifying agent with antifibrotic properties will have additive influence on fibrosis, inhibit cellular and humoral hyperactivity and interfere with smooth muscle proliferation in the vessel wall. The combination of these two agents will also be the first regimen to address the heterogeneity of scleroderma manifestations including ILD, pulmonary arterial hypertension and skin manifestations
Systemic sclerosis-associated pulmonary arterial hypertension (SSc-PAH) is a serious, life-threatening manifestation of systemic sclerosis (SSc), an autoimmune disease of the connective tissue characterized by scarring (fibrosis) and atrophy of the skin, joints and tendons, skeletal muscles, and internal organs, and immunological disturbances. One-year survival for patients with SSc-PAH ranges from 50-81%. There is currently no cure for SSc-PAH and treatment is limited to vasodilator therapy used in all forms of PAH. In recent studies, immunotherapy was shown to be effective in treating SSc-interstitial lung disease, another serious, life-threatening manifestation of SSc. In addition, there are compelling pre-clinical data and anecdotal clinical reports that suggest modulation of the immune system may be an effective strategy for treating SSc-PAH. To test this approach, this trial will determine if rituximab, an immunotherapy, has a marked beneficial effect on clinical disease progression, with minimal toxicity, in patients with SSc-PAH when compared to placebo.
The purpose of this study is to determine the clinical characteristics and hemodynamic profiles that predict exercise induced pulmonary hypertension in 15 patients with systemic sclerosis. The study also aims to determine the effectiveness of Ambrisentan for subjects with exercise induced Pulmonary Arterial Hypertension (PAH) with scleroderma
The purpose of the study is to see how well reduced intensity conditioning followed by a stem cell transplant from a donor (allogeneic) works in treating patients with severe systemic sclerosis. In an allogeneic stem cell transplant procedure, stem cells are taken from a healthy donor and transplanted into the patient. Stem cells can be donated by a family member or an unrelated donor who is a complete tissue type match.
This will be a 36-week, randomized, double-blind, parallel group study comparing the effects of tadalafil monotherapy, ambrisentan monotherapy and combination therapy with tadalafil and ambrisentan in patients with PAH-SSc. Standard outcome measures such as six-minute walk distance (6MWD), NYHA classification, and hemodynamic measurements will be assessed, as well as novel functional measures of RV-PV function including the transthoracic echocardiogram parameter tricuspid annular plane systolic ejection (TAPSE), contrast-enhanced cardiac MRI and heart rate variability assessed by Holter monitoring. This design (excluding a placebo-placebo arm) was selected for ethical concerns and to provide optimal efficiency and active therapy to all study subjects. It also allows for comparisons between the two monotherapies and with combination therapy.