View clinical trials related to Sclerosis.
Filter by: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.
Nine Multiple Sclerosis (MS) patients suffering an acute relapse from the outpatient or inpatient settings will be consented to be followed prospectively for three months post relapse, in an effort to identify markers of incomplete relapse recovery. Factor VIII-related labs will be drawn for three months without influencing standard of care treatment decisions. During this time, patients will be followed with clinical and diagnostic assessments in addition to blood tests including: Expanded Disability Status Scale (EDSS), Multiple Sclerosis Functional Composite (MSFC), recovery surveys, and MRIs of the brain, cervical spine, and thoracic spine with and without contrast. Clinical, imaging, and Factor VIII-related lab data individually or in aggregate will be correlated with relapse presence, severity, and extent of recovery following standard treatment interventions.
This study takes amyotrophic lateral sclerosis (ALS) patients as the main research object. Through collecting genetics, imaging and clinical symptoms for Exploratory research, we will construct the gene spectrum of ALS in China, explore unknown pathogenic genes, explore the characteristic image characteristics of ALS, and establish the iPSCs library of ALS, providing resources and basis for the research of pathogenesis and treatment targets of ALS.
This clinical trial is a phase 1 study in which investigations with the weakly radioactive substance [18F]-RoSMA-18-d6 are being carried out for the first time. This radiolabeled substance will be used to study a specific protein in the brain and spinal cord of patients with ALS and healthy individuals. This particular protein, the cannabinoid type 2 receptor, is thought to play a role in the disease process of ALS. Furthermore, it is assumed that this protein is found more frequently in the brain and spinal cord of patients with ALS compared to healthy individuals. The following questions will be answered by this clinical trial. 1. Is this protein found, as suspected, increased in the brain and spinal cord of ALS patients compared to healthy individuals ? 2. Does the amount of this protein change during the course of the disease? 3. Are there any correlations between the observed changes in the amount of protein and the assessment of the course of the disease?
Preclinical and clinical data strongly suggest that administration of salbutamol in ALS patients may improve walking capacity related to motor fatigue by enhancing neuromuscular transmission. Salbutamol may exert a neuroprotective effect and slow down the progression of clinical signs and symptoms. The main objective of the study is to test the efficacy of salbutamol on walking capacity in ALS patients and the secondary objective is to measure the target engagement of salbutamol on the neuromuscular junction (NMJ) at EMG (decrement of repetitive nerve stimulation in three nerves/muscle couples), as well as safety and tolerability. The exploratory objectives are to study the effect of salbutamol on fatigue scales, muscle strength, respiratory function, motor unit count, muscle and spinal MRI parameters and blood biomarkers
Multiple Sclerosis (MS), causing damage to myelin sheath and axons; It is a chronic disease with diffuse demyelinating lesion of the Central Nervous System (CNS) that often affects young adults, progresses with attacks and remissions, and may bring about functional limitation, disability or decrease in quality of life. The fact that Multiple Sclerosis is chronic and irreversible, the uncertainty and destructiveness of the disease process affect individuals physically over time, but it can also cause many negative symptoms from a mental perspective. Studies have shown that MS disease; anxiety, depression, loss of life purpose, intense hopelessness and suicide. Life purpose has been defined as struggling to achieve one's goals and creating meaning against existential neurosis. Having a life purpose increases the subjective well-being, life satisfaction and hope level of individuals. According to the Turkish Language Association, hope, which is defined as "the feeling of trust arising from hope" and which indicates the feeling of having positive expectations for the future, positively affects mental health by giving people the feeling that they can cope with negative experiences that they may encounter in the future. Hopelessness, which is the opposite of hope, is a feeling that causes mental problems such as depression and suicide as well as negatively affecting the mental health of the individual and is a part of these clinical pictures. Setting a life purpose has positive effects on hope. While a purposeful life increases the level of hope in people, it reduces hopelessness and causes the person to live a more meaningful life. The decrease or loss of hope and the purpose of life can cause significant problems for people such as depression, addiction or suicide may occur in people who have lost their life purpose and hope. Positive psychotherapy (PPT), one of the psychosocial-based intervention methods, is a therapy method with a humanistic approach, the theoretical foundations of which were established by Pesesschkian in 1970. There are three basic principles of therapy: hope, balance and consultation. In Positive Psychotherapy, the symptoms and ailments in the person; It is positively reinterpreted, emphasizing real talents. Sharing the function of the existing symptom with the client increases the client's acceptance and hope for himself and his situation, which in turn activates the hope principle.
The goal of this clinical investigation is to evaluate the effectiveness of home use of a lightweight robotic lower limb exoskeleton as a walking aid device on quality of life in patients with multiple sclerosis with gait disorders. Participants will wear an exoskeleton (Keeogo) for 8 weeks at home during the experimental phase. This phase is compared to an 8-week control phase at home with advice on regular physical activity adapted to their abilities.
In multiple sclerosis (MS) brains, inflammation induces specific abnormalities of synaptic transmission, collectively called inflammatory synaptopathy. Such synaptopathy consists in unbalanced glutamatergic and GABAergic transmission and in remarkable changes in synaptic plasticity, causing excitotoxic neurodegeneration and impairing the clinical compensation of the ongoing brain damage, thereby exacerbating the clinical manifestation of the disease. In progressive MS (PMS), synaptopathy is characterized by pathological potentatiation of glutamate-mediated synaptic up-scaling (Centonze et al., 2008; Rossi et al., 2013) and loss of long-term synaptic potentiation [LTP (Weiss et al., 2014)], both caused by proinflammatory molecules (released by microglia, astroglia, and infiltrating T and B lymphocytes) (Malenka et al., 2004; Di Filippo et al., 2017; Stampanoni Bassi et al., 2019). The combination of increased up-scaling and decreased LTP has a significant impact on the clinical manifestations of PMS, often presenting with signs and symptoms indicating length-dependent degeneration of neurons of the corticospinal tract. Altered LTP expression impairs brain ability to compensate ongoing neuronal loss (Stampanoni Bassi et al., 2020), and pathological TNF-mediated up-scaling may directly promote excitotoxic damage and neurodegeneration (Rossi et al., 2014). In addition, up-scaling and LTP are mutually exclusive at a given synapse through a mechanism of synaptic occlusion (i.e., pre-existing up-scaling saturates and prevents subsequent LTP expression), further promoting neurodegeneration by preventing the pro-survival effect of LTP, the induction of which activates intracellular anti-apoptotic pathways (Bartlett & Wang, 2013). It follows that a neuromodulation approach that can chronically (over several months) dampen up-scaling expression in the primary motor cortex (M1) of PMS patients could be beneficial by preventing excitotoxic neurodegenerative damage triggered by up-scaling itself (Centonze et al. 2008, Rossi et al. 2014), and also by promoting LTP induction and LTP-dependent functional compensation of deficits, thereby reducing the speed of the neurodegeneration process through increased LTP-dependent neuronal survival and preservation of dendritic spines (Ksiazek-Winiarek et al., 2015). Our study aims to test whether transcranial static magnetic field stimulation (tSMS) could represent such a therapeutic approach, as recently proposed in patients with amyotrophic lateral sclerosis (ALS) (Di Lazzaro et al, 2021). Forty (40) ambulatory patients with PMS, presenting with the ascending myelopathy phenotype of the disease, will be recruited at the MS Center of the Unit of Neurology of the IRCCS Neuromed in Pozzilli (IS). In this randomized, sham-controlled, double-blind, within-subjects, cross-over study (allocation ratio 1:1), we will test the ability of repeated sessions of tSMS applied bilaterally over the M1 to safely reduce disability progression in patients with PMS. Patients will be randomly assigned to either real or sham tSMS. Each patient will participate in two experimental phases (real or sham stimulation). Each patient will self-administer tSMS over right and left M1, two session per day, 60 minutes each. The order will be randomly established and counterbalanced across participants. Both investigators and participants will be blinded to stimulation parameters. In the "real stimulation" phase, tSMS will be applied for 120 minutes each day, at home, for 12 consecutive months. In the "sham stimulation" phase, sham tSMS will be delivered with non-magnetic metal cylinders, with the same size, weight and appearance of the magnets. Clinical evaluations, including the Multiple Sclerosis Functional Composite measure (MSFC) will be performed before, during and after each experimental phase ("real" and "sham"). In addition, blood levels of neurofilaments, excitability and plasticity of M1, and MRI measures of cortical thickness will be measured before, during and after each stimulation phase.
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 primary objective of the study is to estimate the incidence rate of serious adverse events (SAEs), including but not limited to malignancies and serious and opportunistic infections, among participants with MS treated with Vumerity, Tecfidera, other selected disease modifying therapies (DMTs [teriflunomide, beta interferons, or glatiramer acetate]), or Vumerity after switching from Tecfidera. The secondary objective of the study is to compare the incidence rate of SAEs, including but not limited to malignancies and serious and opportunistic infections, among MS participants treated with Vumerity, Tecfidera, and Vumerity after switching from Tecfidera with the incidence rate of MS participants treated with other selected DMTs (teriflunomide, beta-interferons, or glatiramer acetate), if the sample size allows.