51 Clinical Trials for Various Conditions
This purpose of this study is research the usefulness of MRI with PET/CT imaging for measuring brain inflammation and its relation to Multiple Sclerosis (MS).
Feraheme (ferumoxytol) is FDA-approved for iron supplementation and is composed of iron oxide nanoparticles classified among the ultra-small superparamagnetic iron oxides (USPIO). In this project we hypothesize that Feraheme could become a sensitive and specific marker of active inflammation in multiple sclerosis. We will explore this hypothesis taking advantage of ultra high field strength (7T) MRI to further increase the effectiveness of the contrast agent Feraheme at revealing inflammatory activity.
To establish a large, longitudinal collection of high quality samples and data from subjects with MS, selected other demyelinating diseases (Transverse Myelitis (TM), Neuromyelitis Optica (NMO) or Devic's, Acute Disseminated Encephalomyelitis (ADEM), and Optic Neuritis (ON)), and related and unrelated unaffected controls. Samples and data will be available as a shared resource to scientists researching the causes, sub-types, and biomarkers of MS and related demyelinating diseases.
The overall objective is to obtain an assessment of the pharmacokinetics of \[18F\]3F4AP in healthy volunteers and subjects with demyelinating diseases such as mild cognitive impairment (MCI), Alzheimer's Disease (AD), Multiple Sclerosis (MS), Spinal Cord Injury (SCI) and Spinal radiculopathy (SR).
The clinical trial is intended to assess for clinical evidence of Clemastine Fumarate as a myelin repair therapy in patients with acute inflammatory injury-causing demyelination as measured by multi-parametric MRI assessments. No reparative therapies exist for the treatment of acute demyelinating lesions. Clemastine fumarate was identified along with a series of other antimuscarinic medications as a potential remyelinating agent using the micropillar screen (BIMA) developed at the University of California, San Francisco (UCSF). Following in vivo validation, an FDA IND exemption was granted to investigate clemastine for the treatment of multiple sclerosis in the context of chronic optic neuropathy. That pilot study was recently completed and is the first randomized control trial documenting efficacy for a putative remyelinating agent for the treatment of MS. The preselected primary efficacy endpoint (visual evoked potential) was met and a strong trend to benefit was seen for the principal secondary endpoint assessing function (low contrast visual acuity). That trial number was 13-11577. This study seeks to follow up on that study and examine clemastine fumarate's protective and reparative effects in the context of acute demyelinating brain lesions as imaged by multi-parametric MRI assessments. The investigators will be assessing the effects of clemastine fumarate as a remyelinating therapy and assessing its effect on MRI metrics of lesions found in patients with a confirmed diagnosis of acute inflammatory injury-causing demyelination. In addition to using conventional multi-parametric MRI assessments, this study will also evaluate a new MRI technique called Ultrashort Echo Time (UTE) MRI to assess the effects of clemastine fumarate as a remyelinating therapy of acute lesions found in patients with a confirmed diagnosis of acute inflammatory injury-causing demyelination and compare it to the other assessments.
The goal of this observational study is to longitudinally investigating subjects with inaugural acute optic neuritis (ON). The main questions it aims to answer are: * Does the time to corticosteroid treatment affect the visual outcome at 6 months in subjects with acute multiple sclerosis (MS)-, aquaporin 4-IgG positive (AQP4-IgG+) and myelin-oligodendrocyte-glycoprotein-IgG positive (MOG-IgG+) ON? * How differ clinical, structural, and laboratory biomarkers in subjects with acute ON, including clinical isolated syndrome (CIS), MS-ON, AQP4-IgG+ON, MOG-IgG+ON and seronegative non-MS-ON? Participants will undergo * clinical examination, including clinical history, neurovisual and neurological tests * serum and cerebrospinal fluid examination * optical coherence tomography (OCT) * magnetic resonance imaging (MRI) * assessment of depression, pain, quality of life through validated questionnaires Researchers will compare subjects with MS-ON, AQP4-IgG+ON, MOG-IgG+ON and other ON (CIS, seronegative non-MS-ON) to detect diagnostic and predictive markers for the disease course.
The participant will receive weekly intramuscular treatment with AVONEX® (interferon beta 1-a) and a one-time high dose intravenous methotrexate with Leucovorin rescue, along with the standard solumedrol treatment before beginning AVONEX® treatment.
Transcranial Magnetic Stimulation (TMS) is a non-invasive technique to gather information about brain function. It is very useful when studying the areas of the brain related to motor activity (motor cortex, corticospinal tract, spinal cord and nerve roots). The procedure is conducted by transmitting a magnetic signal into the brain to stimulate an area of the body. Electrodes (small pieces of metal taped to areas of the body) are used in order to measure electrical activity. A magnetic signal is sent from a metal instrument held close to the patient's head, to an area of the brain responsible for motor activity of a certain area of the body. The electrodes pick up and record the electrical activity in the muscles. This study will employ the use of TMS to diagnose neurological disorders that affect the motor cortex or the corticospinal tract. Normal subjects are sometimes studied to investigate normal activity of the nervous system and to train doctors in clinical neurophysiology and electrodiagnostic medicine at the National Institutes of Health (NIH).
Chronic Inflammatory Demylinating Polyneuropathy (CIDP) is an autoimmune condition affecting the nervous system. Researchers believe the immune system begins attacking the cells covering nerves called myelin. The destruction of myelin causes muscle weakness, loss of sensation, abnormal levels of protein in the fluid surrounding the brain (CSF), and slowing of the nervous system. The disease progresses slowly and disables patients suffering from it. CIDP is treated with steroids, plasmapheresis, and immunosuppressive drugs. Many patients initially respond to these treatments, but develop resistance to the therapy or experience side effects causing the treatments to be stopped. Researchers believe that intravenous immunoglobulin (IVIg) may provide patients with CIDP a safer and more effective alternative to standard therapies for the disease. IVIg is a drug that has been used successfully to treat other immune-related diseases of the nervous system. However, because IVIg is so expensive, researchers believe it should first be proven effective on a small group of patients. The study will take 60 patients with CIDP and divide them into two groups. Group one will receive 2 injections of IVIg once a month for three months. Group two will receive 2 injections of placebo "inactive injection of sterile water" once a month for three months. Following the three months of treatment, group one will begin taking the placebo and group two will begin taking IVIg for an additional 3 months. The drug will be considered effective if patients receiving it experience a significant improvement (\>25%) in muscle strength.
This is an open label, Phase 1b, multiple ascending dose, and dose-expansion study of IDP-023 administered in combination with interleukin-2 (IL-2) and ocrelizumab to evaluate the safety, tolerability, and biologic activity on autoreactive immune cells in patients with refractory progressive multiple sclerosis.
A study to quantify changes in motor performance of epidural stimulation in progressive multiple sclerosis (MS) patients over the course of 12 rehabilitation sessions.
Over the past 10 years, the rates of multiple sclerosis (MS) have nearly doubled in the United States. This chronic, neuroinflammatory, and neurodegenerative disease is most often diagnosed between the ages of 20-40. Cognitive impairment effects up to 70% of people with MS (PwMS) and has a detrimental impact on mental health, social connections, and employment. Further, up to 50% of PwMS also struggle with depression. Numerous cognitive rehabilitation programs are available to address cognitive impairment, but few interventions have simultaneous effects on cognition and emotional well-being. Music interventions have potential to fill this gap. Brain imaging studies on music and emotion show that music can modulate activity in the brains structures that are known to be crucially involved in emotion. Further, music engages areas of the brain that are involved with paying attention, making predictions, and updating events in our memory. The purpose of this study is to determine the feasibility of an online musical training intervention (MTI) for PwMS and explore the potential effect on cognition, psychosocial, and functional well-being compared to an active control group (music listening (ML)). The specific aims are to: 1) determine the feasibility and acceptability of delivering the MTI virtually over three months to PwMS; 2) evaluate the effect of the MTI on cognitive functioning (processing speed, working memory, cognitive flexibility, response inhibition), psychosocial (anxiety, depression, stress, quality of life, self-efficacy) and functional (insomnia) well-being compared to ML; and 3) (exploratory aim) to utilize non-invasive neuroimaging to determine if pre-intervention brain activity predicts post-intervention cognitive functioning.
The primary goal of this study is to provide additional data regarding B and T-cell mediated responses to COVID-19 vaccines in MS patients treated with OCR and to determine which clinical and paraclinical variables correlating with vaccine immunogenicity. B-cell mediated humoral responses and adaptive T-cell mediated cellular responses were measured in patients treated with OCR who received any of the available SARS-CoV-2 vaccines, 3-4 weeks after completion of vaccination.
The overall objective is to obtain an initial assessment of the value of using \[18F\]3F4AP for imaging demyelinating diseases such as traumatic brain injury (TBI), neurodegenerative diseases such as mild cognitive impairment (MCI) and Alzheimer's Disease (AD): * Aim 1) Assess the safety of \[18F\]3F4AP in healthy volunteers and subjects with traumatic brain injury (TBI) and neurocognitive impaired subjects (AD/MCI). Hypothesis 1: Administration of \[18F\]3F4AP will result in no changes in vitals or other adverse events. * Aim 2) Assess the radiation doses to the main organs in healthy volunteers. Hypothesis 2: the radiation doses to each organ will be comparable in all subjects and within the acceptable limits. * Aim 3) Assess the pharmacokinetics of a bolus infusion of \[18F\]3F4AP in humans including healthy volunteers and patients. Hypothesis 3: the pharmacokinetics of \[18F\]3F4AP at the whole brain level will be similar in controls, TBI and AD/MCI subjects. The kinetics in demyelinated lesions will be slower than in healthy areas. * Aim 4) Correlate MR images with \[18F\]3F4AP PET images. Hypothesis 4A: all the lesions seen on the MRI will show increased signal (VT or SUV) on the PET images. Hypothesis 4B: some of the lesions on the MRI will show increased signal (VT or SUV) on the PET but not all. * Aim 5) Correlate \[18F\]3F4AP PET signal with neuropsychological testing in people with TBI and AD/MCI. Hypothesis 5A: increased PET signal (VT or SUV) will correlate with impaired Mini Mental State Examination (MMSE).
This is a clinical trial to determine the feasibility of a stationary aerobic cycling intervention and explore if aerobic exercise independently promotes remyelination in people with multiple sclerosis (MS).
The investigators are measuring the effectiveness of Ocrevus™ in helping patients with hand or arm weakness, especially if posed by a more advanced MS patient than those included in the clinical trials.
The purpose of this study is evaluate the natural course of disease progression related to gross motor function in children with metachromatic leukodystrophy (MLD).
This is a non-blinded, non-randomized dose escalation study of intravenous FBX-101 in which subjects will receive a single infusion of an adeno-associated virus gene therapy product, after more than 21 days of the HSCT (UCBT preferred HSCT source). Data from previously transplanted patients with infantile and late infantile Krabbe disease will be used as a comparator group.
This is a Phase 1/2 randomized, blinded, dose-escalation study to evaluate the safety and efficacy of intrathecal (IT) administration of SBT101, a recombinant adeno-associated virus serotype 9 (AAV9) containing a functional copy of the human adenosine triphosphate (ATP)-binding cassette transporter subfamily D member 1 (ABCD1; hABCD1) gene, in adult patients with adrenomyeloneuropathy (AMN) aged 18-65 years. Patients will receive a single dose of SBT101 via IT route (or an imitation procedure) and will be followed for safety and efficacy for 2 years. Patients receiving SBT101 will be followed for an additional 3 years (5 total) for Safety. Patients receiving an imitation procedure will be offered the opportunity to receive SBT101 after 2 years, as data indicate.
The purpose of this study is to assess the safety of metformin for treatment of progressive multiple sclerosis
The course of AMN-related disabilities over time is poorly or incompletely understood due to a limited number of patients and lack of treatments. This study will help obtain a better understanding of the progression of disease with AMN and facilitate efficient clinical development of future interventional medications.
The investigators have previously shown that youth with MS are very inactive, and that vigorous physical activity is associated with higher levels of well-being and lower MS disease activity in youth. Yet, no effective physical activity interventions have been developed for youth with MS to date. The investigators have taken input from youth with MS to create a Smartphone-based app (the ATOMIC - Active Teens with Multiple Sclerosis - App) that provides tailored physical activity information and coaching, provides tools to increase social connectedness, and promotes physical activity. This proposed research will therefore address the problem of inactivity in youth with MS by studying an intervention to increase physical activity.
The primary purpose of this study is to evaluate the efficacy and safety of ravulizumab for the treatment of adult participants with NMOSD.
The objective of this study is to evaluate the safety and efficacy of eculizumab in pediatric participants (aged 2 to \< 18 years) with relapsing neuromyelitis optica spectrum disorder (NMOSD).
Purpose of study is to determine safety and efficacy of use of autologous Adipose-Derived cellular Stromal Vascular Fraction (AD-cSVF) suspended in Normal Saline and delivered via intravascular system of quality of life and alteration of documented Advanced Muscular Sclerosis (MS). It is believed that the heterogeneous cell population which includes multipotent stem/stromal cells plus non-multipotent cellular elements are capable of immune modulation/inflammatory modulation properties. Exam of disease progression and quality of life changes will be evaluated by sophisticated mathematical non-biased MRI analysis.
The investigators propose to conduct a randomized 6-month intervention study comparing cognitive functioning in individuals with relapsing-remitting multiple sclerosis assigned to either a physical activity or an active water-intake control group. Individuals will complete pre-, mid- and post-assessments of cognitive, physical, and behavioral functioning.
Purpose of study is to determine safety and efficacy of use of autologous Adipose-Derived cellular Stromal Vascular Fraction (AD-cSVF) suspended in Normal Saline and delivered via intravascular system of quality of life and alteration of documented Muscular Sclerosis (MS) and related neurodegenerative patients. It is believed that the heterogeneous cell population which includes multipotent stem/stromal cells are capable of immune modulation/inflammatory modulation properties. Exam of disease progression and quality of life changes will be evaluated.
The aim of this study is to learn more about the following treatment options in adults with CIDP: * Subcutaneous self-infusion with HyQvia. * Intravenous infusion with Gammagard/Kiovig. Gammagard and Kiovig are the brand names for the same immunoglobulin compound. The study is in two parts. In Part 1, participants receive either HyQvia or a placebo subcutaneously. In Part 2 (only for participants who have a CIPD relapse during Part 1), participants will receive Gammagard Liquid/Kiovig intravenously. US participants will receive Gamunex-C. The first SC infusion will be given in the study clinic. The remaining SC infusions may be given in the study clinic or the participant's home. This will be decided by the study doctor and whether the participant or their caregiver can do the self-infusion.
People with Multiple Sclerosis (MS) often have pain and/or fatigue. Unfortunately, available treatments provide inadequate relief for the majority of these individuals. There remains an urgent need for additional treatment options for MS-related symptoms. The purpose of this study is to see if alternative treatments that involve self-hypnosis training, neurofeedback training and/or mindfulness meditation training, or a combination of some of these treatments can help decrease pain and fatigue in people with MS. A subject must have a diagnosis of MS, have chronic pain and/or fatigue, and be at least 18 years old to participate, among other criteria.
The purpose of this study is to determine whether eculizumab long-term use is safe and effective in patients with relapsing NMO.