View clinical trials related to Scleroderma, Diffuse.
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.
The purpose of this study is to evaluate the efficacy and safety of treatment with subcutaneous anifrolumab versus placebo in adult participants with systemic sclerosis. The target population for this study includes patients who meet the 2013 American College of Rheumatology/European League Against Rheumatism (ACR/EULAR) classification for systemic sclerosis, either limited or diffuse cutaneous subsets, with a disease duration of less than 6 years from first non-Raynaud's phenomenon symptom.
The objective of this before-after non-randomized trial study is to evaluate the efficacy of ultrasound therapy in combination with manual therapy in the management of systemic sclerosis patients with IDU. The main questions it intends to answer are: • Is this combination of treatments effective in these patients in terms of improvement in hand functional ability, pain relief, injury healing, and quality of life? Participants will receive rehabilitation treatment consisting of a combination of manual therapy (McMennel joint manipulation, pumping, and connective tissue massage) and US water immersion. The researchers will compare the group of participants, called the treatment group, with a control group to see if: • Is the combined treatment of ultrasound therapy and manual therapy more effective in these patients than manual therapy alone?
This is a double-blind, randomized, placebo-controlled, PoC study to evaluate the efficacy, safety, and tolerability of efzofitimod in patients with SSc-ILD. The primary objective of the study is to evaluate the PoC for efficacy in a population with SSc-ILD. While improvement of ILD is the outcome of interest, the study will also evaluate changes in the skin. After initial screening (up to 4 weeks), approximately 25 eligible participants will be randomized 2:2:1 to 1 of 2 active (experimental) dose arms or placebo, administered every 4 weeks up to and including Week 20.
This study investigates the efficacy and safety of belimumab compared to placebo, in addition to standard therapy, for the treatment of participants with systemic sclerosis associated interstitial lung disease (SSc-ILD). The study will evaluate the effect of belimumab treatment on lung function as well as on extra-pulmonary disease manifestations, including skin thickening and general symptoms, such as fatigue, that impact quality of life (QoL).
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.
The purpose of this study is to establish the tolerability, preliminary efficacy, and pharmacokinetics of CC-97540 in participants with severe, refractory autoimmune diseases.
Leap Motion Based Gamefication Exercises in the Individuals With Systemic Sclerosis
The main purpose of the study is to evaluate the efficacy of vixarelimab compared with placebo on lung function in participants with idiopathic pulmonary fibrosis (IPF) and in participants with systemic sclerosis-associated interstitial lung disease (SSc-ILD). Participants who complete 52-weeks of treatment in the Double-blind Treatment (DBT) period can choose to enroll in the optional Open-label Extension (OLE) period to receive treatment with vixarelimab for another 52 weeks.