View clinical trials related to Systemic Sclerosis.
Filter by:Ectopic soft tissue calcifications or ossifications can complicate the course of numerous diseases; most of them are rare or very rare. Even if the clinical, radiological and pathological presentation of ectopic calcifications and ossifications are different, the same hypotheses are discussed considering their hypothetical pathophysiology. Indeed, high calcium phosphate product, local cellular lesions and abnormal transdifferentiation of mesenchymal cells are regularly evoked when pathophysiology of such calcifications or ossifications are discussed. Apart from several case reports that have not been confirmed so far, no medical treatments are available, leading to significant pain and impairment of quality of life for patients. Therefore, only surgical treatment can be proposed when the volume or the consequences of these calcifications/ossifications become too important. Sodium thiosulfate (STS) is currently used as a cyanide poisoning antagonist and a chemoprotectant against adverse effects of several chemotherapies such as Cisplatin. Numerous case reports and several studies have revealed the potential interest of STS in the treatment of uremic induced vascular or soft tissues calcifications. Recently, our group has developed an expertise in the use of STS for the treatment of ectopic soft tissue calcifications or ossifications. Considering these promising preliminary data, and their limits, we developed a strategy to treat soft tissue calcifications or ossifications based on a local administration of STS. The first results of this therapeutic strategy are highly promising and the local or systemic safety is satisfactory so far. These preliminary data also reported by others deserve to be confirmed in a prospective study. We propose in this project to conduct a prospective open controlled phase II trial in order to assess the efficacy and the safety of intralesional administration of STS for the treatment of calcifications secondary to dermatomyositis or systemic sclerosis and ectopic ossifications secondary to pseudo-hypoparathyroidism 1a type (PHP1A/iPPSD2) (inactivating parathyroid hormone / parathyroid-hormone-related peptid (PTH/PTHrP) signalling disorder).
Serum, synovial fluid and skin biopsies from patients will be collected to the biobank with rheumatoid diseases. These samples will later be used for clinical and basic research, following approval of each specific study by the IRB. The investigators intend to extract protein, DNA and RNA from each sample.
Pulmonary arterial hypertension (PAH) is a serious and often fatal complication of connective tissue diseases including systemic sclerosis and systemic lupus erythematosus. It has been reported primarily from European series that the prevalence of PAH in patients with connective tissue diseases particularly systemic sclerosis is as high as 15-20%. However, given the paucity of medical literature in the area from Chinese population, the prevalence of PAH amongst Chinese patients with these conditions is largely unknown. Even worse, PAH is often undiagnosed amongst patients with connective tissue diseases due to the lack of awareness and/or access to echocardiography, which is a non-invasive first-line screening tool for PAH. As a result, most patients at diagnosis of PAH are at a relatively late stage, rendering pharmacological treatment less effective. Here, the investigators propose a territory-wide pulmonary hypertension screening for patients with connective tissue disease in order to (1) detect pulmonary hypertension amongst patients with connective tissue disease through systematic screening, and (2) understand the prevalence of pulmonary hypertension in Chinese patients with connective tissue diseases.
A randomized controlled trial will be performed to confirm preliminary uncontrolled data indicating that regional adipose tissue grafting is effective in inducing digital ulcer healing in patients with systemic sclerosis. Systemic Sclerosis patients with digital ulcers will be randomized to be blindly treated with adipose tissue implantation or a sham procedure. Adipose tissue grafting will consist of injection at the base of the finger with digital ulcer of 0.5-1 ml of adipose tissue after centrifugation of fat aspirate. Sharm procedure will consist of false liposuction and local injection of saline solution. The primary end-point will be to compare the cumulative prevalence of healed digital ulcers in the two groups within the following 8 weeks.
Diffuse infiltrating pneumonia (DIP) is a severe complication of systemic sclerosis and one of the leading cause of death in this condition. The main objective of this study is to prospectively describe the evolution of DIP overtime and to find prognosis factors.
Pulmonary hypertension (PH) is a serious condition characterized by a mean pulmonary artery pressure >=25mmHg on right heart catheterization (RHC). Despite advances in PH care, outcomes are still sub-optimal and further research is required into the pathobiology of the disease and development of biomarkers that can guide clinical care. The investigators are establishing a biobank to collect samples (blood, urine, stool) from patients with pulmonary hypertension, patients at high risk for pulmonary hypertension, healthy controls, and patients undergoing right heart catheterization. Specimens will be stored for future investigations.
The MANUS Trial aims to examine the safety, feasibility and potential efficacy of intramuscularly injected allogeneic mesenchymal stromal cells as treatment for digital ulcers of systemic sclerosis.
Systemic sclerosis is an autoimmune connective tissue disease with undefined etiology and characterized by progressive fibrosis of the skin and major organs. Dry eyes and / or buccal syndrome is commonly reported in patients with systemic sclerosis. Goujerot-Sjogren syndrome is a chronic autoimmune disorder that is characterized by dryness of the eyes (xerophthalmia) and / or mouth (xerostomia). It may be primary or secondary to another connective tissue disease (such as lupus, rheumatoid arthritis or other). Several criteria have been validated to classify the SS but require a labial salivary gland biopsy, invasive act which complications can sometimes be reported (hematoma, lip sensory defect). Several scores based on the evaluation of the ultrasound homogeneity of the salivary glands were developed but no studies have evaluated ultrasound abnormalities of salivary glands in patients with systemic sclerosis.
This is a 12 week double-blind randomized placebo controlled trial in which 30 patients with very early SSc, fulfilling the Very Early Diagnosis Of Systemic Sclerosis (VEDOSS) criteria (9) will be randomized in a 2:1 fashion to receive intravenous methylprednisolone or placebo. Three-day treatment courses are given at week 0, week 4 and week 8. The final assessment is at week 12, and patients will be followed up to one year after baseline
Systemic sclerosis is a systemic connective tissue disease with physical and mental disturbances. Based on a pilot study the feasibility and effectiveness of a novel, self-developed concept of vocal intervention in Systemic Sclerosis under vocal pedagogical guidance and music therapy is assessed.