View clinical trials related to Scleroderma, Systemic.
Filter by:On December 27th 2020, the implementation of SARS-CoV-2 vaccination in France first became available amongst all immunosuppressed patients at very high risk of severe Coronavirus Disease (COVID-19) infection, specifically for Hematopoietic Stem Cell Transplant recipients or other fragile immunosuppressed patients. Systemic sclerosis (SSc) is an autoimmune disease characterized by dysregulation of the immune system, vascular damage and progressive fibrosis of the skin and internal organs (heart, lung and kidney) which may lead to severe comorbidities and SSc-related mortality. In severe rapidly progressive SSc, we and others demonstrated that autologous hematopoietic stem cell transplantation (AHSCT) is the only treatment so far allowing improvement in overall and event free survival up to 7 years after AHSCT. As soon as vaccines were available on January 1st, 2021 and following the French Haute Autorité de Santé (HAS) and Société Francophone de Greffe de Moelle et de Thérapie Cellulaire (SFGM-TC) good clinical practices (GCP) guidelines, the ad hoc COVID19 vaccinations were proposed to all SSc patients in France, including those previously treated by AHSCT and followed at Maladies Auto Immunes et Thérapie Cellulaire (MATHEC) Center of Reference for stem cell therapy at St-Louis hospital. In July 2021, the Autoimmune Diseases Working Party (ADWP) and the European society for Blood and Marrow Transplantation (EBMT) guidelines recommended vaccination against SARS-CoV2 as early as 3 months after HSCT for autoimmune diseases. Only one Israeli study so far has analyzed the efficacy of SARS-CoV-2 vaccination after autologous HSCT (AHSCT) in SSc patients.. We therefore designed the present case control study retrospective study to assess the acceptance and effectiveness of this vaccination program in SSc patients treated by AHSCT as compared to other fragile SSc individuals. The Primary objective is to evaluate the effectiveness of implementing the anti-SARS-CoV2 vaccination program according to the French GCP for COVID-19 prophylaxis in SSc fragile patients, in terms of risk of asymptomatic or symptomatic COVID-19 infection, among SSc patients treated by AHSCT compared to 1:1 matched non transplant SSc controls.
Mesenchymal stromal cells (MSC) are multipotent cells which carry immunomodulatory, pro-angiogenic and anti-fibrotic properties, that can target Systemic Sclerosis (SSc) pathogenesis and its clinical manifestations. The increasing use of MSC, harvested from bone marrow (MSC(M)), adipose tissue (MSC(AT)), or umbilical cord (MSC(UC)) in a variety of indications, provides consistent evidence supporting their safety in humans. The efficacy of MSC(M) intravenous (IV) injection for treating acute graft versus host disease led to their marketing approval in 2012 and MSC(AT) (Alofisel) were approved for severe Crohn's fistula in 2018. MSC represent a promising therapeutic approach for SSc. We previously a) showed disease-specific abnormalities in MSC(M) from SSc patients, providing strong rationale to use allogeneic MSC to treat SSc patients, b) completed the first phase I/II dose escalation trial using allogenic MSC(M) infusion in 20 severe SSc patients (ClinicalTrials.gov: NCT02213705, PHRC AOM 11-250) with no safety issues, significant improvement in skin fibrosis at 3 to 6 months after infusion which appeared lower thereafter, thereby supporting the need for repeated infusions. In vitro, experimental and clinical studies suggest that MSC properties vary according to their tissue of origin/source. We demonstrated that compared to MSC(M), MSC(AT) are easier to harvest and display higher proliferative capability before entering senescence, higher genetic stability, and superior immunosuppressive properties. The objective of the present research is the successful production of allogeneic MSC(AT) derived from selected healthy donors, with adequate phenotypic criteria according to the International Society for Cell & Gene Therapy. Considering the above rationale, these MSC(AT) will subsequently be used in a Phase I/II randomized clinical trial testing allogeneic MSC(AT) systemic infusion for treatment of severe systemic sclerosis.
Investigation of the effects of ESWT and PNF exercises added to ESWT on calcinosis in Ssc patients. Calcinosis cutis is a common, difficult-to-treat manifestation of systemic sclerosis associated with high morbidity. The aim of this study is to investigate the efficacy of ESWT therapy for calcinosis cutis in Ssc patients. The effects on grip strength, sleep, function and quality of life will be investigated.
Leap Motion Based Gamefication Exercises in the Individuals With Systemic Sclerosis
Scleroderma (SSc) is a rare crippling chronic disease associated with damage of the blood vessels and hardening of connective tissue. It has quite a number of complications including ulcers to the fingers and toes (digital ulcers). Digital ulcers (DU) are a frequent challenge in patients with SSc (PwSSc), as they approximately affect more than half of these patients. Care received by PwSSc DU is varied. Patients with three or more ulcers are considered to have severe disease therefore these patients usually receive expensive treatments and referred to specialist SSc clinics, while those with less ulcers may not receive the same treatments, and only see their general practitioner or local rheumatologist or left to their own means. Resulting in patients developing their own DU managing practices which may be good and some not. Currently, no research has fully identified the needs of PwSSc DU and self-care interventions to support them are lacking. The extent to which DU support needs are met by non-specialist health professionals is unknown. There is lack of literature in co-designing interventions with patients in SSc DU. Developing interventions and pathways for managing DU with patients and healthcare professionals (HCP) will enhance DU care and lessen the burden for the affected patients, a qualitative study is required. Aim The aim of this research is to co-design self-care opportunities and develop improvements in DU care. Objectives 1. to explore how patients with SSc DU are currently managed 2. to understand how they currently manage their ulcers and their healthcare needs. 3. to collaboratively co-design self-care opportunities and improvements in care. Study Methods Experience-based co-design (EBCD) qualitative methodology will be used to conduct the study. 10 HCP and 12-15 PwSSc DU will be invited to participate in consultation observations. Followed by individuals interviews for the HCP and PwSSc DU care and perceptions on unmet needs. The results will inform collaborative co-designing and development of interventions with patients and HCP. The same participants will also be invited to participate in three workshops involving designing, discussion, refinement and finalisation of the interventions. The interventions developed will be ready to be tested or being evaluated once they have been put in place..
The goal of this pilot study is to assess the feasibility of a larger study on the efficacy of mycophenolate mofetil in people diagnosed with systemic sclerosis with mild lung involvement. Participants will be recruited over 12 months at 3 academic centers and assigned randomly to receive either mycophenolate mofetil or placebo, a look-alike substance that contains no active drug, for 96 weeks.
The purpose of the study is to determine the feasibility of a non-contact custom splint fabrication method for patients with chronic diseases suffering from hypersensitive skin or compromised skin integrity. Custom splinting by occupational therapists involves molding low-temperature thermoplastic material directly on patients' skin; however, skin sensitivity is a contraindication for splint fabrication. The study aims to recruit 10 male or female patients with either a diagnosis of scleroderma (SSc) or arthritis. A scan of the patient's hand and a 3D printer will be used to create a precise model of a patient's hand on which a custom splint will be fabricated. By taking this approach, traditional splinting is substituted by avoiding direct contact with the material on the surface of the patient's upper extremity. This technique creates therapeutic opportunities for underserved patients by expanding splinting options for patients with scleroderma and arthritis, and addressing the challenges associated with managing chronic diseases.
Systemic sclerosis is a rare autoimmune disorder characterized by microangiopathy, activation of the immune system, and sclerosis of tissues including the skin. Facial involvement is frequent and disabling. It causes significant functional and aesthetic discomfort, and a major deterioration in quality of life. It results in a loss of suppleness of the skin and subcutaneous tissues, dysfunction of the temporomandibular joint, peribuccal rhagades, microstomia, and dry mouth causing difficulties in mouth opening, feeding, dental care, and weight loss. Facial involvement in systemic sclerosis can be assessed using the Mouth Handicap in Systemic Sclerosis (MHISS) score, a validated patient questionnaire assessing the functional and aesthetic consequences of systemic sclerosis on the face. Although common and disabling, facial involvement is underestimated and poorly managed. Immunosuppressive and/or anti-fibrosis drugs are not very effective. Facial rehabilitation could significantly improve the mouth handicap but facial rehabilitation is not currently performed in standard care in systemic sclerosis patients. The aim of the study is to evaluate the efficacy of a personalized rehabilitation program vs standard care in facial involvement of systemic sclerosis patients.
The primary objective of this study is to assess the safety and efficacy of the Celution Device in the processing of an autologous graft consisting of adipose derived regenerative cells (ADRCs) in the treatment of hand dysfunction due to scleroderma.
1.Assessment of slit2 level in SLE patients and its correlation with disease activity. 2-Assessment of slit2 level in SSC patients and its correlation with disease activity