Active clinical trials for "Scleroderma, Diffuse"

This study evaluates if dabigatran etexilate is safe for use in patients with Scleroderma and Interstitial Lung Disease. All patients will receive 75mg of dabigatran etexilate twice a day for 6 months.

Systemic sclerosis (SSc) is a rare multisystem connective-tissue disorder characterized by three major pathological hallmarks: widespread fibrosis, vasculopathy and immunological abnormalities. As all connective tissues can be affected, this condition has multiple effects on the orofacial region. Indeed, the latter is involved in approximately 80% of SSc patients. Oral manifestations have a major impact on quality of life and require specific treatments that should be performed as early as possible. Widening of the periodontal ligament space, that seems to be linked to an increased collagen synthesis, is one of the most common dental radiographic finding. However, this radiologic sign has been mostly studied on two-dimensional radiographs. The investigators have recently described in a patient suffering from SSc the existence of calcifications within the periodontal ligament space using Cone Beam Computed Tomography (CBCT) approach (Jung et al., Oral Surg Oral Med Oral Pathol Oral Radiol 2013). Such calcifications, that have never been observed before, could be part of the phenotypic spectrum of the disease, in particular when dystrophic calcinosis is associated. They could furthermore constitute a specific feature of SSc. However, this radiographic sign requires to be investigated in a largest number of patients. Several cytokines have been implicated in SSc pathogenesis. A recent study has revealed that elevated CXCL4 serum levels correlate with disease complications, suggesting that this molecule could be used as a prognostic biomarker. Increased IL-6 serum levels also correlate with SSc severity. Gingival crevicular fluid can be easily collected from the gingival crevice surrounding the teeth and constitute an indicator of local but also systemic inflammation. Analysis of gingival crevicular fluid cytokine profile could contribute to the identification of specific SSc biomarkers and allow a better comprehension of oral manifestations pathogenesis. The aim of this case-control study is to characterize precisely the oral manifestations associated with SSc within the National Referral Center for Rare Autoimmune Diseases (Strasbourg, France) patient cohort in order to identify specific radiological, clinical and/or biological signs. Some of them could be correlated to the severity or to the prognosis of the disease. To the investigators knowledge it is the first study using tridimensional CBCT approach.

Trial Design Patients with borderline PAH indicated by borderline mPAP values will be included in this single centre study. This clinical investigation is performed as a Proof-of-Concept (PoC) investigator initiated trial (IIT) using a prospective, randomized, double-blind, parallel group, placebo-controlled, phase IIA clinical study design. On their first visit their medical history will be obtained and physical examination will be conducted. Moreover, an electrocardiogram (ECG), laboratory testing (NT-proBNP, uric acid and other laboratory tests), echocardiography at rest and right heart catheterization will be carried out. If patients have been identified within the last 6 months before screening investigations by right heart catheterization, the measurements are considered valid as baseline investigations and will not be repeated. If patients fulfill the inclusion criteria and still suffer from borderline mPAP values they will be invited to join the study. The clinical investigations will begin within 28 days. The prospective study will comprise a 6 months study period (180 ±2 weeks) plus the screening phase up to 28 days and a follow-up phase of 30 ±7 days.

Systemic sclerosis is an autoimmune disease characterized by skin lesions and visceral responsible for significant morbidity. Microcirculatory disorders and tissue fibrosis are excessive severity of the disease. This condition can affect the hands with a major functional consequence severely impairing the quality of life of patients. Adipose tissue is used in plastic surgery for over a century for the filling of depressions in the skin. In addition to the volume effect, a trophic effect on the surrounding tissue was noted. It is shown that the stromal vascular fraction is responsible for this regenerative effect. In a previous study the investigators have demonstrated in a mouse model that the subcutaneous adipose tissue provides a trophic effect on SSc skin lesions by reducing the fibrosis of the dermis and providing a pro angiogenic. Objectives and means: This is a clinical study evaluating an innovative cell therapy procedure. The objective of this study was to evaluate the effects of injection of autologous stromal vascular fraction of adipose origin according to the system Celution ® (Cytori Therapeutics, Inc.., United Kingdom) in digital in patients with scleroderma cutaneous hands. Eleven patients with scleroderma with the hands will be included in the study. Due to the nature of the orphan disease, a longitudinal study be conducted, where each patient will have own control. The evaluation will be pre and post operative for a period of six months. This evaluation will be based on clinical criteria (trophic balance, functional) and laboratory (capillaroscopy, Doppler ultrasound of the arteries of the forearm, laser-Doppler tissue). Project schedule and implementation phases: The project will run over a period of twelve months. Patients will be followed for a period of six months. Analyzes clinical, paraclinical, and exploitation of results will be achieved over a period of six months. Expected Results: This study will validate the functional and trophic effects of reinjection of autologous stromal vascular fraction of adipose tissue issue on the fingers of patients with scleroderma. Conclusion: This innovative cell therapy could represent an alternative treatment for patients with scleroderma in check, intolerant or insufficiently relieved by medical treatment currently available in the scleroderma hand

Primary Objective: - To evaluate safety and tolerability of 8-week oral administration of SAR100842 in patients with diffuse cutaneous systemic sclerosis. Secondary Objectives: To evaluate the pharmacodynamic effect of SAR100842 in patients with systemic sclerosis as measured by disease related biomarkers and Lysophosphatidic acid (LPA) receptor signaling markers in blood and skin; To explore the effect of SAR100842 on skin thickness in patients with systemic sclerosis as measured by the modified Rodnan Skin Score (mRSS); To explore the effect of SAR100842 on quality of life as measured by the Scleroderma Modified Health Assessment Questionnaire (SHAQ); To document long term safety of SAR100842 during the extension part.

Interstitial lung disease (ILD) is characterised by inflammation and scarring of the lung and is the leading cause of death in patients with systemic sclerosis, and contributes significantly to morbidity and mortality in many other connective tissue diseases (CTDs) such as polymyositis/dermatomyositis and mixed connective tissue disease. When ILD is extensive and/or progressive, immunosuppressive medication is often required to stabilize lung disease and alleviate symptoms. Current standard care for CTD associated ILD is extrapolated from studies performed in individuals with systemic sclerosis and comprises low dose corticosteroids and intravenous cyclophosphamide followed by oral azathioprine. In some individuals even this intensive immunosuppression is insufficient to prevent deterioration, and in a significant minority of affected individuals this results in respiratory failure and death. Rituximab has recently been reported as an effective 'rescue therapy' for stabilizing and even improving ILD in this patient group. Based on observations gained from this experience, the investigators believe that rituximab is a potential important alternative to current best therapy for this patient group. This study has therefore been initiated to evaluate the efficacy of rituximab (compared with standard therapy) in patients with progressive CTD related ILD.

The primary objective of the study is to determine the activity of selexipag on Raynaud attack frequency in subjects with Raynaud's Phenomenon (RP) secondary to Systemic Sclerosis (SSc).

Early phases of systemic sclerosis is characterized by inflammatory and microvasculature alterations. Sildenafil citrate has been shown to have vasodilatory effects and to enhance vasculogenesis. The purpose of this study is to evaluate the effect of sildenafil citrate on hand blood flow of patients with systemic sclerosis, using Laser Doppler Imaging.

The purpose of this study is to determine if fresolimumab is safe in treating people with systemic sclerosis (scleroderma) and to investigate the effect of fresolimumab in the skin of these individuals.

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