Active clinical trials for "Scleroderma, Systemic"

Pulmonary arterial hypertension (PAH) is a condition characterized by an increased pulmonary vascular resistance that can lead to right heart failure and death. Several diseases are known etiologies of PAH including scleroderma and cirrhosis. The presence of PAH in the context of systemic sclerosis or cirrhosis has a dramatic impact on prognosis and survival of the connective tissue or liver disease. Despite advances in the diagnosis of PAH, echocardiography remains a necessary test for screening PAH in patients with scleroderma or cirrhosis. However, echocardiography is less than ideal for diagnosing PAH and predicting treatment response. Thus, there is a pressing need to identify methodologies that can accurately and non-invasively recognize the presence of PAH in patients with scleroderma and cirrhosis. Hypothesis: To measure endothelial function and exhaled gases in patients with scleroderma and cirrhosis. To assess whether they correlate with the presence or the development of PAH. The degree of local (forearm) capillary vasodilation during treprostinil iontophoresis identifies patients who will develop PAH and in those already diagnosed PAH predicts response to PAH-specific therapies.

The public health impact of this study will be the application of the greater understanding of the oral health status, oral health related behaviors, and quality of life of patients with systemic sclerosis (scleroderma or SSc). It is hoped that risks and protective factors can be identified to be associated with the oral health status of adults with systemic sclerosis,leading to the development of interventions for improved oral health in this population,and providing the basis for a larger study of oral health problems of adults with systemic sclerosis.

The study serves the identification of early forms of pulmonary arterial hypertension (PAH) in connective tissue disease and the hemodynamic follow-up of the investigated patients. The basic hypothesis is that PAH may start with a remodeling of small pulmonary arteries, which leads to a stiffening of the vessels, indicated by the inability to vasodilatation and thus a disproportional increase in pulmonary pressure during exercise. Recent studies have shown that a proportion of such patients may develop manifest PAH within a few years. The early identification of these patients and the understanding of the natural course of the disease may improve prognosis. The aim of the present study is to investigate hemodynamic and clinical changes in patients with connective tissue disease in a time interval of 3-5 years with a focus on the development of pulmonary hypertension.

Drug of investigation: Mycophenolate mofetil (MMF), given orally as a tablet twice daily. Dosage of drug: This study recruits patients who have been prescribed a steady dose of MMF in the range between 1000 and 3000 mg daily by their physician. Design: This is an open-label PK study. Disease studied: Systemic sclerosis (SSC, scleroderma). Variables assessed: Estimated AUC0-12 for MMF. Gastrointestinal manifestations of SSc. Concomitant medication. Study population: Inclusion criteria: Diagnosis of SSC fulfilling the 2013 classification criteria for this disease. Participant should have been prescribed a stable dose of MMF tablets, taken twice daily, for at least 3 months prior to the study. Exclusion criteria: Failure to comply with study protocol. Limited access to repeated venous puncture. Recipient of organ transplant. Pulmonary arterial hypertension. Number of participants: The study aims at the inclusion of 35 subjects. Primary objective: To investigate the PK of orally ingested MMF in SSC. Secondary objectives: To investigate how SSC manifested in the gastrointestinal (GI) tract may alter the PK of MMF. To investigate how the PK of MMF in SSc is altered by medications often used in SSC, i.e. proton pump inhibitors (PPI), NSAID and calcium channel blockers.

Systemic sclerosis (SSc) is a generalized disorder of connective tissue, arterioles and microvessels, characterized by the occurrence of fibrosis and vascular obliteration phenomena. The alterations in lung microvessels are found in pulmonary involvement of scleroderma, which are the most serious complications of the disease. In pulmonary emphysema, there are also changes in pulmonary microvasculature, which are involved in the onset and development of the disease. The confocal endomicroscopy is an endoscopic technique which can be performed during a bronchoscopy. This technique makes it possible to observe in real time the most distal pulmonary elements at the microscopic scale. After injection of fluorescein, then the technique of observing the pulmonary microvasculature, in vivo and in situ. The characterization of microvascular lesions in these two pathologies could improve understanding of their mechanisms and ultimately improve the early management of patients.

Systemic Sclerosis (SSc) is an inflammatory chronic disease that can lead to structural damage and handicap. The SSc physiopathology is multifactorial, including genetic and environmental risk factors. The identification of environmental factors implication is crucial to understand the SSc mechanism, and improves the diagnosis and the treatment of the disease.

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.

The purpose of this study is to assess whether skin biopsy specimens from patients with diffuse cutaneous systemic sclerosis (dcSSc) can be used as biomarkers (measures of activity and type) of disease to predict response to various experimental treatments. There are various experimental treatments being used in the treatment of slceroderma, but there is no way to predict response to any given therapy. The investigators will use DNA microarray to analyze the changes in gene expression in skin biopsies in response to various treatments. Our hypothesis is that the investigators will see changes in gene expression in response to various treatments that will give us insight into the cause of scleroderma. The investigators predict that they will be able to use this information to predict which experimental treatments will be beneficial to individual patients

Small bowel involvement is still recognized to be associated with great morbidity and mortality in SSc patients, leading particularly to malabsorption and intestinal pseudo-obstruction. Intestinal disorders directly related to SSc have, in fact, been reported to be one of the most common causes of death. In a previous prospective study, we have demonstrated the high prevalence of small intestinal involvement in SSc patients, using upper intestinal manometry; in turn, 88% of our SSc patients had upper intestinal motor disturbances. However, to date, no authors have yet analyzed the course of upper intestinal motor dysfunction in SSc. The aims of this study were therefore to assess the 5-year course of small bowel motor disorders, using manometry in patients with systemic sclerosis (SSc), and to investigate for an association between upper intestinal motor dysfunction outcome and other clinical manifestations of SSc.

To conduct cross-sectional and longitudinal studies of patients with idiopathic pulmonary fibrosis (IPF) and patients with progressive systemic sclerosis (PSS), with and without associated lung disease.