Active clinical trials for "Sclerosis"

Dalfampridine is a new medication that was FDA approved in 2010 to improve walking speed in people with Multiple Sclerosis (MS). People with MS walk slowly in part because MS damages the myelin insulation around nerves which slows conduction of messages from the brain to the leg muscles. Dalfampridine works by improving conduction in nerves with damaged myelin. Recent research indicates that imbalance in MS is in large part caused by poor conduction by the nerves that transmit information about the position of the legs to the brain. It is therefore likely that, by improving nerve conduction, dalfampridine will also improve imbalance in people with MS. Dalfampridine will be administered in this study by the same route (oral), dosage (10mg), and frequency (every 12 hours) approved by the FDA to improve walking speed in people with MS. The proposed pilot study will examine the effects of dalfampridine on imbalance in 24 subjects with Multiple Sclerosis (MS) and imbalance. This small pilot study will help to show if dalfampridine improves imbalance in MS and will guide the design and implementation of a larger full scale study to definitively determine if dalfampridine improves balance and prevents falls in people with MS.

This is a 2 stage exploratory study with a 3-month observational phase on the natural course, followed by a 12-month, open-label, non-comparative, single-arm, phase II pilot study on the efficacy, safety and tolerability of a low-protein diet (LPD) in 8 adult patients with Bethlem myopathy (BM) and Ullrich congenital muscular dystrophy (UCMD). Objective of this trial is to test the effect of a normocaloric LPD to reactivate autophagy in BM/UCMD patients. The primary end point of the study will be the change in muscle biopsy of Beclin 1, a marker of autophagy, at 1 year of LPD treatment when compared to baseline. The rationale rests on our discoveries that (i) mitochondrial dysfunction mediated by inappropriate opening of the PTP plays a key role in collagen VI myopathies; (ii) defective autophagy with impaired removal of defective mitochondria amplifies the defect; and (iii) reactivation of autophagy with a low-protein diet or treatment with cyclosporine A, the mitochondrial PTP inhibitor, cured Co6a1-/- mice, hinting at a common target among all beneficial treatments - namely autophagy. Specific aims of this project are to (i) study the modifications of clinical, nutritional and laboratory parameters in a cohort of patients with BM/UCMD during a 3-month observational period before starting the LPD treatment; (ii) assess the effect of a normocaloric LPD in correcting defective autophagy in muscle of patients; (iii) test if new non-invasive biomarkers of activation of autophagy examined in the blood are mirroring the effect of LPD in the muscle biopsy; (iv) assess the clinical efficacy and safety of the LPD with an innovative combination of complementary measures of the nutritional status in patients. The anticipated output is defining and validating a therapeutic nutritional approach in autophagy upregulation for BM/UCMD.

This is a one-year multi-centre, double blind, placebo controlled, randomized trial investigating oral vitamin D3 (Colecalciferol) as an add-on treatment to interferon-beta-1b for Multiple Sclerosis (MS). Not less than one month after initiation of therapy with interferon beta 1b, MS patients will be randomised to once weekly treatment with peroral colecalciferol capsules (Dekristol®, Swiss-Caps, Switzerland) containing 0.5 mg of vitamin D3, or to once weekly peroral treatment with matching placebo. The hypothesis is that vitamin D suppresses clinical and MRI activity of MS.

The purpose of this study is to determine whether cyclic oral methylprednisolone once every month has an effect on the intrathecal inflammation in patients suffering from progressive multiple sclerosis.

The primary objective of this study is to evaluate the long-term safety and tolerability of peginterferon beta-1a (BIIB017) in participants originally treated in Study 105MS301 (NCT00906399) who continue peginterferon beta-1a treatment. The secondary objective of this study is to describe long-term multiple sclerosis (MS) outcomes in participants originally treated in Study 105MS301 (NCT00906399) who continue peginterferon beta-1a treatment.

Objective: The primary goal of this study is to investigate the mechanism of action (MOA) of CD25-blocking therapies in high inflammatory multiple sclerosis (HI-MS). The secondary goal of this study is to assess long-term safety and efficacy of CD25-blocking therapies in HI-MS. Study population: Two cohorts of patients will be enrolled: Long-term daclizumab therapy cohort: Up to 15 daclizumab-treated patients with relapsing-remitting (RR-MS) or secondary-progressive MS (SP-MS) previously classified as HI-MS based on MRI/clinical criteria, who have been treated with IV daclizumab for a minimum of 1 year and responded to this therapy with significant (>70%) decrease in contrast-enhancing lesions (CEL) or stabilization/improvement of disease activity (>60% decrease in MS relapses and stable or improved EDSS disability score). New treatment cohort: Up to 15 HI-MS patients (RR- or SP-MS) with inadequate therapeutic response to first-line, FDA-approved immunomodulatory therapies for MS or who cannot, for any reason, be treated with first-line, FDA-approved immunomodulatory therapies for MS. Design: This is an open label, Phase I trial of 150 mg of daclizumab high yield process (DAC HYP) administered subcutaneously (SC) every 4 weeks for a total of 3 years. Outcome measures: Because the main goal of this study is to investigate the MOA of CD25-blocking therapies in MS, the primary outcomes are mechanistic immunological studies performed on clinical samples (peripheral blood mononuclear cells (PBMC), cerebrospinal fluid (CSF) cells and skin biopsies) derived from DAC HYP-treated patients. The secondary outcome measure is long-term safety and tolerability of subcutaneous DAC HYP in HI-MS patients.

This is a single center, multispecialty, randomized double blind placebo control feasibility clinical trial. The purpose is to evaluate the safety, feasibility and efficacy of percutaneous transluminal angioplasty in treating extracranial venous obstructive lesions, and its influence on the clinical outcomes of Multiple Sclerosis (MS) patients that have been found to have chronic cerebrospinal venous insufficiency (CCSVI).

Systemic sclerosis-associated pulmonary arterial hypertension (SSc-PAH) is a serious, life-threatening manifestation of systemic sclerosis (SSc), an autoimmune disease of the connective tissue characterized by scarring (fibrosis) and atrophy of the skin, joints and tendons, skeletal muscles, and internal organs, and immunological disturbances. One-year survival for patients with SSc-PAH ranges from 50-81%. There is currently no cure for SSc-PAH and treatment is limited to vasodilator therapy used in all forms of PAH. In recent studies, immunotherapy was shown to be effective in treating SSc-interstitial lung disease, another serious, life-threatening manifestation of SSc. In addition, there are compelling pre-clinical data and anecdotal clinical reports that suggest modulation of the immune system may be an effective strategy for treating SSc-PAH. To test this approach, this trial will determine if rituximab, an immunotherapy, has a marked beneficial effect on clinical disease progression, with minimal toxicity, in patients with SSc-PAH when compared to placebo.

The objective of this study is to evaluate the safety and efficacy of ONO-4641 in patients with relapsing-remitting multiple sclerosis over a 26-week treatment period.

This study is an extension to the study AC-058B201 and will investigate the long-term safety, tolerability and efficacy of ponesimod in patients with relapsing-remitting multiple sclerosis.