Hematopoietic Stem Cell Transplantation for Treatment of Multiple Sclerosis in Sweden
Multiple SclerosisMultiple Sclerosis1 moreThis is an observational cohort study with retrospective analysis of prospectively collected data. The study cohort is constituted of all patients with relapsing-remitting multiple sclerosis (RRMS) treated with autologous stem cell transplantation (AHSCT) in Sweden from 2004 when the first AHSCT was performed until 31 December 2019. The study aims to describe the effectiveness, safety and patient reported outcomes of AHSCT for MS through real world data. Treatment related mortality will be analyzed from start of mobilization until the end of the study. For other adverse events the data collection will end 3 months post-transplantation. A statistical subgroup comparison of efficacy and safety between the conditioning regimens BEAM-ATG and Cy-ATG will be included within the study.
Safety and Effectiveness of Cinnomer® (Glatiramer Acetate) in Multiple Sclerosis (MS) Treatment...
Relapsing Multiple SclerosisThis trial was an obsevational phase IV prospective multicenter study designed to evaluate the safety and effectiveness of Cinnomer® in patients with MS in Iran. The primary objective of this study was safety assessment of Cinnomer® Secondary objectives were: Effectiveness assessment of Cinnomer® Assessment of the patients' QoL Evaluation of the patients' depression status
Investigation of Novel Immunological Biomarkers by Mass Cytometry in Patients With Early Multiple...
Multiple SclerosisThe objective of CISCO is therefore to identify prognostic biomarkers of MS activity in early-stage patients.
Lysyl Oxidase, Semaphorin 7a and Semaphorin 3a in Patients With Systemic Sclerosis
Systemic SclerosisThe aim of our study is to find a biomarker for fibrosis or vasculopathy in systemic sclerosis. We will evaluate a possible correlation between semaphorin 7a, semaphorin 3a and lysyl oxidase and fibrosis (lung and skin) or vasculopathy in patients with systemic sclerosis. The results obtained may help us diagnose these complications of systemic sclerosis and hopefully even monitor patient treatment.
Pilot Study to Assess Disease Stability in a Natalizumab to Dimethyl Fumarate Crossover Design
Relapsing Remitting Multiple SclerosisThis study will observe participants with relapsing remitting multiple sclerosis who are switching therapies from natalizumab to dimethyl fumarate to determine disease stability.
Difference in Efficacy of Natalizumab Versus Fingolimod for the Treatment of Multiple Sclerosis...
Multiple SclerosisUnder the escalation treatment strategy when a patient displays breakthrough disease parameters under first line therapy, MS physicians are allowed by the EMEA to switch for Natalizumab (NTZ) or fingolimod (FGL). NTZ and FGL efficacy have been demonstrated by randomized therapeutic trial. As both treatments have been tested versus placebo a common way to compare them is to look at their respective annualized relapse risk ratio decrease. Roughly NTZ decrease by 70% and FGL by 50%. Nevertheless it is a terrible comparison since the placebo group had different behaviour in the 2 trials and the patients demographic features at baseline are also different. Therefore, it is right now totally impossible to compare these 2 drugs with a decent methodology. Only a head-to-head comparison could do it. Unfortunately this head-to-head comparison is not available and will not probably be done under the drug companies initiative. During the time of this study, we will perform a phase IV, observational, prospective head-to-head comparison of NTZ versus FGL efficacy in 600 patients. Our primary end point will be disease free patients after 1 year of treatment. Further, this trial will allow us to collect new biological samples, useful for a validation our project main aim. Further these new samples will be obtained from 3 European countries, which is a must if we want to generalize our conclusion obtained from a French cohort. Cooperation at the European level is thus essential for the implementation of this project .
Development of a Talk Therapy Protocol to Help Patients Make Treatment Decisions in MS
Relapsing-remitting Multiple SclerosisBetween 15-20% of MS patients decide not to initiate disease modifying therapies after being diagnosed with MS. For this study, we will develop a telephone-based talk therapy intervention and then conduct a randomized controlled trial. Patients will be assigned to either 5 weekly 20 minute telephone sessions of psychotherapy or a brief education control condition. We hypothesize that patients undergoing phone therapy will be more likely to indicate they are interested in initiating disease modifying medications than patients given brief education and treatment as usual.
Optical Coherence Tomography in Multiple Sclerosis Patients
Multiple SclerosisOptic NeuritisTo evaluate the ability of different spectral domain optical coherence tomography (OCT) devices, as well as different acquisition and analysis packages, to detect disease progression in patients with multiple sclerosis with and without a history of optic neuritis
Early Biomarkers of Autism in Infants With Tuberous Sclerosis Complex (TSC)
Tuberous Sclerosis ComplexThe investigators are enrolling 3-12 month old infants with a diagnosis of tuberous sclerosis complex (TSC) for a new study on early markers of autism. The study is looking for early signs for autism in a population (TSC) where autism is common. The goal of this project is to use behavioral testing, MRI and EEG techniques to identify children at risk for developing autism starting at 3 months of age and continuing until 36 months of age. Throughout the study, the investigators will recommend Early Intervention services for any child who shows early signs of autism.
Diaphragm Pacing System (DPS) In Participants With Amyotrophic Lateral Sclerosis (ALS)
Amyotrophic Lateral Sclerosis (ALS)The study is being conducted to determine if DPS treatment for people with ALS and hypoventilation is associated with improved survival or diaphragm function. The primary objective of the study is to conduct a multi-center, randomized controlled clinical trial comparing standard of care (control) to diaphragm stimulator treatment with the NeuRx® Diaphragm Pacing System™ (DPS) with respect to survival. The secondary objective of the study is to conduct a multi-center, randomized controlled clinical trial to compare standard of care treatment (control) to DPS in ALS subjects with hypoventilation.