52 Clinical Trials for Various Conditions
This is an observational study to: * evaluate the on-treatment clinical performance of ravulizumab in relation to the pre-treatment time period (time period prior to exposure), * enhance knowledge regarding conventional MRI outcomes in people with NMOSD treated with ravulizumab, * identify factors suggestive of subclinical disease progression through conventional MRI sequences, * determine if treatment with ravulizumab, impacts longitudinal 3D conformational MRI measures at the dorsal medulla and other regions of the CNS, and * identify biomarkers (e.g., serum neurofilament light chain (sNfL), conventional and novel MRI markers, etc.) related to disease activity.
Neuromyelitis Optica
The purpose of this proof of concept study is to assess the acceptability and safety of MR-C-014 in persons with Neuromyelitis optica spectrum disorder (NMOSD) who have a gait deficit.
Neuromyelitis Optica Spectrum Disorder
The purpose of this study is to understand the effects of long-term treatment with inebilizumab on circulating levels of immunoglobulins, B-cell counts, and other safety measures, and to further monitor repletion of immunoglobins and B-cell counts in participants with NMOSD who discontinue treatment. The objectives include: 1. To establish the nadir in circulating immunoglobulins (Ig) during chronic treatment with inebilizumab and ascertain the time needed to ensure restoration of pre-treatment baseline serum levels of IgG and IgM after discontinuation of treatment 2. To characterize B-cell counts throughout treatment with inebilizumab and after discontinuation until repletion of Immunoglobulin (Ig levels) 3. To assess long-term safety of inebilizumab 4. To assess other long-term effects of inebilizumab
Neuromyelitis Optica Spectrum Disorder
The goal of this clinical trial is to evaluate the safety and efficacy of nabiximols, a cannabinoid spray, for the treatment of moderate to severe spasticity in adult patients with AQP4-IgG positive and antibody-negative NMOSD. The main question it aims to answer is whether treatment with nabiximols improves patient-reported spasticity ratings compared to treatment with a placebo. This trial will also answer whether nabiximols impact pain, spasm frequency, mood, walking ability, and sleep. Participants will be mailed the treatments and placebo treatments, and will be asked to complete study visits and questionnaires remotely. There is also an optional sub-study that involves in-person visits with ultrasound imaging and in-person neurologic exams.
NMO Spectrum Disorder, Spasticity, Muscle
This is an observational study to monitor female participants exposed to UPLIZNA during pregnancy. This study requires voluntary reporting of pregnancies in female participants with NMOSD exposed to UPLIZNA during pregnancy or within 6 months preceding conception. Pregnancy-related data, potential confounding factors and information related to pregnancy outcome will be collected. The schedule of office visits and all treatment regimens will be determined by the treating healthcare provider. Duration of the study is 10 years, at minimum.
Neuromyelitis Optica Spectrum Disorder, Pregnancy Related
A Phase 2, open-label, multicenter study to evaluate the pharmacokinetics (PK), pharmacodynamics (PD), and safety of inebilizumab in eligible pediatric participants 2 to \< 18 years of age with recently active neuromyelitis optica spectrum disorder (NMOSD) who are seropositive for autoantibodies against aquaporin-4 (AQP4-immunoglobulin \[Ig\]G).
Neuromyelitis Optica Spectrum Disorder
Objective of the trial is to describe the efficacy and safety of satralizumab in patients with aquaporin-4 (AQP4) antibody seropositive NMOSD, either treatment naive or inadequate responders to previous treatment with rituximab (RTX) (or its biosimilar)
Neuromyelitis Optica Spectrum Disorder, NMOSD
This study will primarily evaluate the pharmacokinetics of satralizumab in pediatric patients aged 2-11 years with anti-aquaporin-4 (AQP4) antibody seropositive neuromyelitis optica spectrum disorder (NMOSD). Efficacy, safety, tolerability, and pharmacodynamics will be evaluated in a descriptive manner, given the small number of patients who will be enrolled in this study.
Neuromyelitis Optica Spectrum Disorder, NMOSD
This is a Prospective, observational research study for patients with NMOSD under the care of a licensed neurologist. Approximately 800 patients and 35 clinical sites in North America will be recruited to participate with no defined upper limit for either target.
Neuromyelitis Optica Spectrum Disorder
This multicenter, single-arm, open-label study will evaluate the long-term safety and efficacy of satralizumab in participants with neuromyelitis optica spectrum disorder (NMOSD) who completed open-label extension (OLE) period of studies BN40898 and BN40900. Participants will receive satralizumab as monotherapy or in combination with one of the following background immunosuppressive treatments: azathioprine (AZA), mycophenolate mofetil (MMF), or oral corticosteroids.
Neuromyelitis Optica Spectrum Disorder
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).
Neuromyelitis Optica, Neuromyelitis Optica Spectrum Disorder
This study is designed to treat your disease with an autologous stem cell transplant using a regimen of immune suppressant drugs and chemotherapy to reset your immune system and to determine if your disease will go into long-term remission.
Neuromyelitis Optica, Devic's Disease, NMO Spectrum Disorder
Ublituximab (also known as LFB-R603) is a monoclonal antibody that specifically binds to the trans-membrane antigen CD20. The binding induces immune response that causes lysis of B cells. The rationale for using ublituximab in neuromyelitis optica (NMO) and neuromyelitis optica spectrum disorder (NMOSD) is based on the known roles of B cells, antibody production and plasma cells in the pathophysiology of NMO. NMO is characterized by the presence of an anti-Aquaporin-4 (AQP4) antibody, which can only be produced by differentiation of B cells to plasma cells. Because these anti-AQP4 antibodies may be pathogenic, B cells recognizing AQP4 may be directly involved in the disease process as well. B cells also play a role as potent antigen presenting cells in NMO. The strongest evidence of the importance of B cells in NMO comes from studies of B cell depletion, most commonly with anti-CD20 monoclonal antibody, rituximab (Rituxan®). Rituximab has been shown in five retrospective and two prospective studies to be effective in reducing NMO relapses up to 90% and achieving remission in up to 80% of patients solely by its action on CD20+ B cells, despite no change in plasma cell population and anti-AQP4 antibody titers. These human trials strongly suggest a critical role for B cells in the pathophysiology of human disease. While typically used in the prevention of disease, B-cell depletion may be beneficial in the treatment of an acute relapse as well. Emerging evidence indicates that peripheral B cells are activated during a relapse and plasmablast production of anti-AQP4 antibodies spikes. B cells are also found within acute lesions of the spinal cord and optic nerve suggesting roles both in the blood and in the central nervous system during a relapse.
Neuromyelitis Optica, Neuromyelitis Optica Spectrum Disorder
To compare the efficacy of inebilizumab (MEDI-551) versus placebo in reducing the risk of an neuromyelitis optica/neuromyelitis optica- spectrum disorders (NMO/NMOSD) attack in participants with NMO/NMOSD.
Neuromyelitis Optica and Neuromyelitis Optica Spectrum Disorders
The objectives of this study are to evaluate the efficacy, safety, pharmacodynamic, pharmacokinetic and immunogenic profiles of satralizumab in participants with NMO and NMOSD.
Neuromyelitis Optica (NMO), NMO Spectrum Disorder (NMOSD)
The objective of this study is to evaluate the efficacy, safety, pharmacodynamic, pharmacokinetic, and immunogenic profiles of satralizumab, compared with placebo, in addition to baseline immunosuppressive treatment in participants with NMO and NMOSD.
Neuromyelitis Optica (NMO), NMO Spectrum Disorder (NMOSD)
Neuromyelitis optica (NMO) is an inflammatory disease of the central nervous system that is associated with autoantibodies to aquaporin-4. Treatment options for prevention of clinical relapses of NMO include immunosuppressive medications. Plasma exchange (PLEX) is commonly used as a rescue therapy for NMO relapses but ongoing, regular PLEX procedures (maintenance PLEX) is sometimes used to prevent relapses. This observational registry will record feasibility, tolerability, safety, and preliminary efficacy data regarding maintenance PLEX for NMO.
Neuromyelitis Optica, Neuromyelitis Optica Spectrum Disorder, Recurrent Idiopathic Longitudinally Extensive Transverse Myelitis
The overall objective is to evaluate the tolerability/safety and preliminary efficacy of CINRYZE® (C1 esterase inhibitor \[human\]) as add-on therapy for treatment of acute optic neuritis and/or transverse myelitis in NMO and NMOSD. Primary Objective: To evaluate the safety and tolerability of 3-5 doses of 1000 - 2000 Units intravenous CINRYZE in NMO/NMOSD patients during an acute exacerbation. Secondary Objectives: * To determine the frequency of adverse events with CINRYZE in this patient population. * To determine the effect of CINRYZE on NMO clinical scores (Expanded Disability Status Scale and Low Contrast Visual Acuity). * To compare the change in MRI lesion size and extent following a course of CINRYZE.
Neuromyelitis Optica
The purpose of this study is to determine if the drug eculizumab reduces the attack rate and improves outcome in patients with neuromyelitis optica.
Neuromyelitis Optica, Devic's Disease
Neuromyelitis optica (NMO) is a severe demyelinating disease that selectively involves the optic nerves and the spinal cord but usually spares the brain. NMO is considered to have a B cell induced pathogenesis. Mitoxantrone (MITO, Novantrone®), a synthetic anthracenedione approved for worsening relapsing-remitting multiple sclerosis (MS) and secondary progressive MS, has been shown to primarily suppress the humoral response. We conducted a prospective 2-year study to evaluate the benefit of MITO in five relapsing NMO patients.
Neuromyelitis Optica, Myelitis, Transverse, Demyelinating Autoimmune Diseases, CNS, Autoimmune Diseases of the Nervous System
Neuromyelitis optica (NMO) is an autoimmune disease that affects the central nervous system. Patients have relapses (also known as attacks) which are often quite severe and leave them with significant disability. Without treatment, within 5 years 50% of NMO patients are blind in one or both eyes or require walking assistance (cane, walker or wheelchair). NMO has only been relatively recently described and is fairly rare. Most NMO patients' immune systems produce abnormal antibodies against aquaporin-4 (AQP4), which is found in certain cells in the central nervous system. When these AQP4 antibodies bind to AQP4, they trigger a cascade of events involving the immune system which eventually leads to damage to the nervous system. This ultimately leads to disability, some of which is permanent. Until now, treatments for NMO have been mostly focused on decreasing production of this AQP4 antibody. However, recent experiments in animal models of NMO have shown the importance of what happens inside the central nervous system after the antibody binds to the nervous system cell. Specifically, researchers have noted the importance of a specific cell type, eosinophils, in causing damage in NMO lesions. In a recent study, researchers showed they could prevent damage from NMO by blocking eosinophils using cetirizine, which is a popular over-the-counter allergy medicine. Cetirizine is already known to be safe and well-tolerated in the general population. In this study, the researchers plan to add cetirizine on to patients' current NMO treatment. The researchers aim to show that it is safe, well-tolerated, and that with cetirizine, NMO patients have less relapses and therefore less disability over the course of the year following initiation of treatment. The researchers also plan to study how cetirizine changes the immunological profile in NMO patients by examining blood and cerebrospinal fluid.
Neuromyelitis Optica
Neuromyelitis Optica (NMO) is a rare, devastating demyelinating disease of the central nervous system (CNS) that has different causes and treatments from the more common demyelinating disease multiple sclerosis (MS). Current NMO therapies are nonspecific and have varying and often suboptimal benefit. The investigators will evaluate whether use of alpha1-antitrypsin (A1AT, an FDA-approved medication for patients with congenital deficiency of A1AT associated with emphysema) can benefit acute attacks of NMO, improving patient disability and quality of life.
Neuromyelitis Optica
The goal of this research study is to investigate whether Rituximab is safe to use in patients suffering from NMO, or who are at high risk for developing NMO. It is thought that NMO is caused by the immune system reacting against the optic nerves and spinal cord. B cells are a part of the immune system that may contribute to the illness. Rituximab is an antibody that depletes B cells. Depletion of these B cells with Rituximab may induce remission of the disease. Because pathological and serological studies suggest that NMO appears to be, at least in part, a B-cell mediated disease Rituximab, is an attractive treatment candidate for this disease.
Neuromyelitis Optica
This study is designed to examine whether treating Devic's disease patients with high dose cyclophosphamide together with rabbit antithymocyte globulin (rATG)/rituximab (drugs which reduce the function of the immune system), followed by return of previously collected patient's stem cells will result in improvement in Devic's disease. Stem cells are undeveloped cells that have the capacity to grow into mature blood cells, which normally circulate in the blood stream. The purpose of the intense chemotherapy is to destroy the cells in patient's immune system, which may be causing his/her disease. The purpose of the stem cell infusion is to produce a normal immune system that will no longer attack patient's body. The purpose of study is to examine the safety and efficacy of this treatment. The drugs used in this study treatment are drugs for commonly used for immune suppression.
Devic's Disease
This project aims to study whether a structured wellness program intervention can improve quality of life among people living with Neuromyelitis Optica Spectrum Disorder (NMOSD). This pilot will test a multi-modal wellness program tailored to NMOSD patients that includes services from physical therapists, dietitians, social workers, nurse practitioners and cognitive therapists. The trial will be designed as a randomized controlled trial, randomizing patients to immediately starting the program as well as a 6 month delayed start. The intervention would leverage an existing clinical comprehensive MS wellness program at the Corinne Dickinson for Multiple Sclerosis (MS) at Mount Sinai Medical Center. The primary endpoint of this study would be evaluating quality of life, as assessed by MS-Quality of Life -54.
Neuromyelitis Optica Spectrum Disorder (NMOSD)
Long-term, multicenter, multinational, observational, registry of patients with AQP4+ NMOSD that is designed to collect data on clinical outcomes and safety in patients prescribed Alexion C5 inhibitor therapies (C5IT). The registry will also collect data on patient reported outcomes (PROs), quality of life (QoL), and targeted AQP4+ NMOSD therapies used to provide evidence on the real-world impact of ALXN-C5IT on patients with AQP4+ NMOSD.
Neuromyelitis Optica Spectrum Disorder, NMOSD, AQP4+ NMOSD
NMOSDCopilot is a digital tool developed for the self-assessment of Neuromyelitis Optica Spectrum Disorder symptoms that impact patients' functioning and quality of life. It has been co-designed with the help of patient advocacy groups, NMOSD patients and medical experts. It includes a smartphone-based application for patients, connected to a web portal developed for healthcare professionals (HCSPs). The patient application is composed of vision, walking, cognition, and dexterity e-active tests inspired by clinical standards, as well as e-questionnaires. The HCP web portal is a desktop-based software that allows HCPs to access the results generated via the patient application and facilitates remote monitoring of patients' symptoms. The objectives of this study are to validate the accuracy, reliability and reproducibility of the unsupervised at-home self-assessment of symptoms on the patient's smartphone versus the standard in-clinic testing, as well as to evaluate the safety of use of the tool, its usability, and satisfaction towards the patient application among NMOSD patients, and the HCP web dashboard among HCPs.
Neuromyelitis Optica
The primary purpose of this study is to evaluate the safety and efficacy of ravulizumab in pediatric participants with Neuromyelitis Optica Spectrum Disorder (NMOSD).
Neuromyelitis Optica Spectrum Disorder
The primary purpose of this study is to evaluate the efficacy and safety of ravulizumab for the treatment of adult participants with NMOSD.
Neuromyelitis Optica, Neuromyelitis Optica Spectrum Disorder
This study is being done to develop a database of pediatric patients in order to study the cause, early detection and best treatment for neuromyelitis optica spectrum disorder (NMOSD) in pediatric patients.
Neuromyelitis Optica, NMO Spectrum Disorder