View clinical trials related to Scleroderma, Systemic.
Filter by:The mechanical properties of healthy and SSc-diseased skin will be assessed by suction based measurements. The negative pressure needed to gain a certain tissue elevation, tissue elevation in response to a certain negative pressure as well as the time of retraction of tissue will be recorded and analyzed. Mesurments will be done with the new developed Aspiration Device_Nimble and with the CE-certified Cutometer MPA 580.
The purpose of this study is to determine whether a regimen of high-dose immunoablative therapy will demonstrate safety that is consistent or improved with other published regimens in SSc patients, while maintaining a treatment effect.
The overall objective is to propose a comprehensive analysis of the biological properties of the stromal vascular fraction evaluated in the SCLERADEC 2 clinical trial (n = 15 available) and preserved in the biological collection, compared to healthy donors (n = 10). This characterization will focus on the exploration of the phenotypic and functional characteristics of the main cellular subpopulations present in the stromal vascular fraction of scleroderma patients likely to be associated with a better regenerative vascular or anti-fibrotic activity of the cell therapy product. The main objective will be to validate whether the supposed mechanism of action of this innovative therapy, in relation to the representativity of the endothelial progenitors, carrying the vascular regeneration activity, is preserved in the sclerodermic context. A total of 30 subjects (20 systemic Scleroderma patients and 10 healthy donors) will be included.
Systemic sclerosis (SSc) is an uncommon chronic rheumatic disease with an unknown cause and unpredictable course. The inability, in addition to easy fatigability, starts a vicious circle that leads to a fast deterioration of physical conditions that cause a reduction of aerobic exercise capacity and, consequently, of health-related quality of life (HRQoL). Aerobic exercise has already been shown to be safe and effective in improving exercise capacity and HRQoL of patients with chronic cardiovascular and pulmonary diseases. However, few studies have evaluated the role of specific exercise programs on the muscular impairment in SSc. Nevertheless, the results obtained in preliminary reports are promising, and, for these reasons, the management of muscular impairment in SSc could include an appropriate rehabilitation program besides pharmaceutical and surgical treatments. The primary aim of this study will be to evaluate the effect of an individualized exercise program performed at home on aerobic capacity evaluated by 6 minutes walking test. Secondary aims will be to evaluate: 1- VO2max, measured by cardiopulmonary test; 2- the effect of the same program on the muscular strength of upper and lower limbs; 3- the efficacy of a self-administered stretching program for finger joint motion. Secondary aims will be also 1-to ascertain whether a comprehensive exercise program may affect, besides physical function, HRQoL; 2- and to evaluate the adherence during two periods of three months, one whit and one without supervision and reinforcement by a phone call. All the patients with a diagnosis of SSc, according to the criteria of American College of Rheumatology (ACR), who attended the Rheumatologic outpatient clinic of our institution will be evaluated in order to participate in the rehabilitation program. The pneumological examination and two days of screening and testing will take place at the outpatient's clinic of Respiratory Medicine and Sport of our institution.
Systemic Sclerosis (SSc) is an autoimmune connective tissue disease characterized by autoantibodies, fibrosis and microvascular injury and endothelial cell activation that results in vascular damage. Vascular injury induces both innate and acquired immune responses resulting in fibroblast activation and organ fibrosis. SSc may target multiple organs, including: skin, lungs, heart, vascularization, kidneys, the gastrointestinal tract and musculoskeletal structures. Mortality among scleroderma patients is significant, with a 3.5 standardized mortality ratio (SMR) in studies of prevalent cases. This mortality may be increased in the early years of the disease, reaching a SMR of 4 in a multinational inception cohort. In general, treatment strategies target involved organs as early as possible to avoid damage. Many treatment options are available for each manifestation, but evidence with respect to the order of treatment is scarce. Financial costs, the lack of proper outcome measures, difficulty to recruit patients as a rare disease, all prevent the development of new big clinical trials, oppositely to other common diseases such as stroke or cancer. The heterogeneous features of SSc may make trials challenging. The current guidelines available are the British guidelines (2017) , and the updated European League Against Rheumatism (EULAR) guidelines, published in 2017. Management guidelines have some gaps regarding second-line treatment, combinations and there are no proposed algorithms. With the pragmatic trials, the investigators intend to fill the gap between the complicated randomized clinical trials and the observational studies. Using the treatments that have already been proved useful in SSc, in an open-label randomized way and based on some refined expert-made algorithms, will allow the investigators to establish the order in how to use them. Patients will be offered to participate with the collection of their clinical data and, if they give their consent, they will be randomized according to the algorithms. There will be an optional part of the study consisting in the collection of blood samples and skin samples for future research.
This study was designed to test the hypothesis that, irrespective of the degree of interstiaI lung disease and/or pulmonary arterial hypertension, the combined measurement of lung diffusing capacity for nitric oxide and carbon monoxide, might be useful to provide a mechanistic interpretation of changes of diffusion subcomponents in systemic sclerosis (SSc).
This study is designed to treat systemic sclerosis (scleroderma) patients with an autologous stem cell transplant using a regimen of immune suppressant drugs and chemotherapy that is less toxic to your heart.
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
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).
Our study aims at defining the role of circulating microparticles in the physiopathology of two rare auto-immune diseases: systemic lupus erythematosus (SLE) and systemic scleroderma (SSc). Microparticles might have an prognostic and diagnostic interest as well as potential for the discovery of new therapeutic strategies.