Guillain-Barre Syndrome

Researchers are investigating the effects of a multidomain lifestyle intervention to slow cognitive decline and dementia risk in elderly residents aged 60 to 80 in Zhejiang Province, China. The study builds on findings from Finland, where a similar approach combining physical activity, nutrition, cognitive training, social activities, and vascular risk management showed promise in healthy older adults at risk of cognitive decline. This trial aims to explore how such an intervention may change brain structure and function as well as cognitive abilities in an Asian population. Participants are randomly assigned to either a structured multidomain intervention group or a self-guided control group. The intervention group receives tailored nutritional guidance, cognitive training, physical exercises, and vascular risk monitoring, all adapted to Chinese cultural norms. The control group receives regular health education campaigns and basic health monitoring every 6 to 12 months. This trial runs for two years with ongoing assessments to evaluate the impact of these lifestyle changes. During the study, participants undergo various evaluations including cognitive tests measuring global and domain-specific cognition, neuroimaging scans like MRI and fMRI, laboratory blood tests, and physical performance measures such as grip strength and gait speed. Questionnaires assess quality of life, depression, sleep quality, and daily activities. Researchers also monitor cardiovascular health, dementia onset, and falls. This comprehensive monitoring will help determine the intervention's effects on brain health and daily functioning over the 24-month period.

Researchers are studying a surgically implanted functional electrical stimulation (FES) system designed to improve stability of the trunk and hips in people with spinal cord injuries, paralysis, tetraplegia, or paraplegia. This system uses small electric currents to make muscles contract, aiming to help users sit more steadily, breathe better, reach farther, push a wheelchair, or roll in bed. The study is conducted by the VA Office of Research and Development and focuses on how this neuroprosthesis affects posture and mobility. The study involves one surgical procedure to implant electrodes into the trunk and hip muscles. These electrodes connect to an 8-channel stimulator implanted in the abdomen. After surgery, participants spend two to six weeks at home with limited activity to allow healing. Following this, they undergo exercise and training to begin using the neuroprosthesis functionally. Participants will be assessed in the lab for strength, balance, and functional abilities both with and without the system, while the implanted device’s technical performance is also monitored. Participants will be involved for up to 36 months, during which researchers will measure the effects of trunk stimulation on posture control, breathing, pressure distribution while seated, reach ability, stability, and personal mobility. They will also work on designing a simple position controller. The study includes post-operative training and follow-up visits to track progress, device function, and safety over time.

Researchers are evaluating anitocabtagene autoleucel (anito-cel), a BCMA-directed CAR-T cell therapy, in adults with generalized myasthenia gravis (GMG) classified as MGFA Grade 2 to 4a. This Phase 1 open-label, multi-center study aims to assess the safety, tolerability, and preliminary efficacy of anito-cel in patients who require immunosuppressive therapy as determined by their neurologist. The study focuses on identifying dose-limiting toxicities (DLTs), the maximum tolerated dose (MTD), and selecting the recommended Phase 2 dose (RP2D) for treatment. The study includes sequential phases: screening, enrollment with leukapheresis to collect cells, pretreatment with a standard lymphodepletion chemotherapy regimen for 5 days, and a single intravenous infusion of anito-cel cells. Optional bridging therapy may be given while the CAR-T cells are prepared. Following infusion, participants will be closely monitored for safety and efficacy outcomes over time. Participants will undergo safety monitoring for at least 28 days after infusion, with safety data collected throughout the study duration of up to 24 months. Clinical effects will be evaluated using measures including the Myasthenia Gravis Activities of Daily Living (MG ADL) score, Quantitative Myasthenia Gravis (QMG) score, and Myasthenia Gravis Composite (MGC) scale. Additionally, blood tests will monitor autoantibody levels and pharmacokinetics of the therapy. This comprehensive follow-up ensures careful assessment of treatment impact and participant health.

Researchers are studying the safety and effectiveness of a single dose of nexiguran ziclumeran (NTLA-2001) compared to a placebo in people with hereditary transthyretin amyloidosis with polyneuropathy (ATTRv-PN). This phase 3, multinational, randomized, double-blind, placebo-controlled trial involves about 60 participants with this nerve disease and genetic condition affecting the peripheral nervous system. Participants will be randomly assigned to receive a single intravenous infusion of either nexiguran ziclumeran 55 mg or a placebo of normal saline. To give everyone a chance to receive the study drug, participants may switch to the other treatment group at either 12 or 18 months, depending on specific study criteria. The study is designed to compare the effects of the drug and placebo over time. During the study, participants will be monitored for nerve function using the Modified Neuropathy Impairment Score +7 (mNIS+7) over 18 months and blood levels of serum transthyretin at 29 days and 18 months. Quality of life, body mass index, and other health measures will also be evaluated. The study includes careful safety monitoring and will last up to 18 months with ongoing assessments to track participants' nerve health and overall well-being.

Researchers are conducting a multicenter, prospective observational study to understand the natural history of Charcot-Marie-Tooth Disease, Type 4J (CMT4J), a rare neurodegenerative and neuromuscular condition confirmed by specific genetic testing. The study will enroll 20 participants of any age who have a molecularly confirmed diagnosis of CMT4J. The purpose is to collect detailed health information and disease progression data over time, without providing any investigational treatments. Participants will follow a uniform protocol with visits scheduled every 12 months plus a 4-week window, for up to 2 years. At each visit, researchers will collect demographic and medical history data, perform physical and neurological exams, standard lab tests, and use several disease outcome measures. Additional assessments may include neuropsychological tests, nerve conduction studies, muscle MRI, pulmonary function tests, and scoliosis x-rays. Early termination visits are planned if participants leave before completing the study. Throughout the study, participants will undergo thorough evaluations to monitor their condition and collect data relevant to CMT4J progression. This includes clinical exams, imaging, lab tests, and questionnaires. Researchers will use this information to study disease patterns and outcomes over the 2-year follow-up period. No investigational products are administered, and visits may be unscheduled if necessary with approval. Participants are expected to comply with study procedures and travel requirements.

This observational study focuses on patients who have developed nervous system complications related to radiotherapy, often used in treating head and neck tumors and cancers of the central nervous system. Researchers aim to understand the clinical signs, treatment effects, disease progression, and prognosis of these complications, which can range from mild changes to severe neurological damage such as brain necrosis and nerve injury. The study is conducted with the involvement of Sun Yat-Sen Memorial Hospital of Sun Yat-Sen University. Participants receive optimized clinical treatment while being observed over time. Regular evaluations include blood, urine, stool, and cerebrospinal fluid tests alongside imaging techniques like MRI, CT, and ultrasound. Additional examinations such as hearing tests, eye exams, and neurological function scales are performed to assess the impact of radiotherapy on the nervous system. Researchers also collect biological samples to study genes, proteins, and metabolites involved in these complications, including analysis of gut microbiota interactions. During the study, participants undergo various assessments to monitor neurological function, psychiatric conditions, cognitive status, quality of life, pain, and drug side effects using multiple validated scales. Blood and stool samples are regularly collected, and cerebrospinal fluid or biopsy samples may be taken if necessary. The primary outcome measure is overall survival, tracked from the start of the study up to 20 years or until death. The study involves ongoing follow-up and monitoring to gather comprehensive data on the condition and treatment outcomes.

Researchers are studying how nerve test results change in people with urothelial carcinoma who are treated with enfortumab vedotin. This observational study focuses on assessing nerve function and patient-reported neuropathy in individuals with metastatic or muscle-invasive urothelial carcinoma. The study is led by Memorial Sloan Kettering Cancer Center and aims to understand chemotherapy-induced peripheral neuropathy linked to this treatment. Participants will undergo non-invasive nerve conduction studies to objectively measure nerve function, especially in sensory fibers affected by neuropathy. They will also provide self-reported information about their neuropathy symptoms. The study includes individuals planned to start systemic therapy with enfortumab vedotin, with or without pembrolizumab. During the study, nerve conduction tests and questionnaires about neuropathy will be collected from baseline up to 9 to 15 weeks. Researchers will monitor changes in nerve function and neuropathy severity over this period. Participants must be able to communicate in English and have a performance status allowing study participation. The study does not involve therapeutic interventions but focuses on observation and assessment of nerve health during treatment.

Researchers are evaluating the safety and effectiveness of NT 201, a type of botulinum toxin A, compared with a placebo in adults who have moderate to severe platysma prominence, which refers to noticeable bands in the neck area. This Phase 3 study aims to understand how well NT 201 works and how safe it is for these participants. The study is sponsored by Merz Aesthetics GmbH and will be conducted in Europe. Participants will be randomly assigned to receive either a single injection of NT 201 or a matching placebo injection in the main study period (MP) on Day 1. After this period, those who received NT 201 can enter an open-label extension period (OLEX) where they will receive another injection of NT 201 on Day 1 of that phase. The study involves a double-blind design, meaning neither the participants nor the researchers know who receives the active drug or placebo during the main period. During the study, participants will be assessed using the Merz Aesthetics Platysma Scale - Dynamic (MAPS-D) to measure improvement in platysma prominence at various time points, including Week 2 and up to Week 17 in the main period. Both investigator and participant assessments will be used to evaluate the degree of improvement. Satisfaction questionnaires and global aesthetic improvement scales will also be used to measure participant satisfaction and aesthetic changes. Safety and efficacy will be closely monitored throughout the study, which lasts through the main and extension periods.

Researchers are evaluating apitegromab in children younger than 2 years old who have 5q autosomal recessive Spinal Muscular Atrophy (SMA) and delayed motor milestones linked to SMA or a Children's Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP-INTEND) score below 55. This Phase 2, double-blind study aims to assess how the drug acts in the body (pharmacokinetics and pharmacodynamics), its effects on motor function, and its safety and tolerability. Participants will receive apitegromab through intravenous (IV) infusion every 4 weeks during a 48-week treatment period. All enrolled children must have been treated with an approved SMN1-targeted therapy like onasemnogene abeparvovec-xioi or be currently receiving an approved SMN2-targeted therapy such as nusinersen or risdiplam. The study compares low and high doses of apitegromab alongside these standard SMN therapies. During the study, researchers will monitor apitegromab levels in the body, its biological effects, and motor function improvements over 48 to 52 weeks. Safety and tolerability will also be assessed throughout this time. The trial involves regular assessments including motor function tests and safety evaluations to track progress and any adverse effects. Participation lasts approximately one year, with detailed follow-up to understand the treatment's impact.

Researchers are studying tanruprubart, also known as ANX005, in people aged 12 to 85 who have Guillain-Barré Syndrome (GBS). This open-label Phase 3 study aims to understand how the drug moves through and affects the body, as well as its early effectiveness and safety. The study is conducted in the United States, Canada, and Europe and is sponsored by Annexon, Inc. Participants will receive a single intravenous infusion of tanruprubart at a dose of 30 mg/kg on the first day of the study. There are no other treatment groups or placebo arms; all participants get the study drug. The trial focuses on monitoring drug levels and biological effects up to 15 days after infusion. During the study, researchers will measure how much tanruprubart is in the blood over time and the time to reach the highest concentration. They will also assess changes in a protein involved in the immune response and muscle strength scores up to one week after treatment. The total participation period includes assessments through day 15, with safety and efficacy monitored throughout.