Myasthenia Gravis

Researchers are evaluating the effectiveness and safety of B007 in people with generalized myasthenia gravis, a condition that causes muscle weakness. This randomized, double-blind, placebo-controlled Phase II/III study aims to understand how well B007 works compared to a placebo in improving daily living activities affected by this condition. Participants will receive either a high or low dose of B007 or a matching placebo through subcutaneous injections on days 1 and 15. The study includes careful monitoring over approximately 16 to 24 weeks to assess changes in symptoms and quality of life, with a safety follow-up lasting about one year. During the trial, participants will be evaluated through various measures including the Myasthenia Gravis-Activities of Daily Living profile, quality of life questionnaires, and composite scores related to the condition. Researchers will also track any side effects or adverse events. The total participation time varies, with key assessments occurring around 16 to 24 weeks and safety monitored for about a year.

Researchers are studying a new feedback-enabled magnetic device designed to temporarily manage blepharoptosis, a condition where the upper eyelid droops. The study aims to evaluate how well the device improves eyelid height, symmetry of blinks, and reduces symptoms like visual field obstruction, blurred vision, and discomfort on the eyelid and surrounding skin. The safety and effectiveness of this device will be compared with outcomes from patients who undergo surgery for the same condition. The device includes near-infrared sensors, a magnetic actuator, an iron sheet for attaching to the affected eyelid, and a biosignal module. During the study, participants will use the device for sessions lasting about 120 minutes each. Researchers will measure the distance the eyelid lifts and assess muscle activity related to eyelid movement. Both subjective sensations and any side effects will be monitored to evaluate the device’s performance. Participants will have their eyelid measurements taken before and after using the device, along with assessments of visual field changes and muscle activity. These evaluations occur shortly after device use and include checking for any skin irritation or discomfort. The study also includes follow-up examinations comparing device use with surgical treatment outcomes at one and six months. The total participation time for each device use session is about two hours, with longer-term follow-up for surgical cases.

This research focuses on people who have previously been treated with KYV-101, an autologous CAR T cell therapy, to monitor long-term safety and persistence of the treatment. It aims to collect information about delayed side effects and ongoing presence of the gene-modified cells in participants who received at least one infusion of KYV-101 in earlier clinical trials sponsored by Kyverna Therapeutics. Participants in this observational study will continue to be followed for up to 15 years after their initial KYV-101 treatment. The study will track various health outcomes including treatment-related adverse events, new or returning malignancies, neurological and autoimmune conditions, blood disorders, infections, and specific laboratory tests related to the therapy. For some participants with certain conditions, additional measures like medication use and functional assessments will be monitored for shorter periods. Throughout the study, participants will undergo regular health evaluations, lab tests, and questionnaires to assess the long-term effects of KYV-101. Researchers will collect data on safety events and laboratory markers up to 15 years, with some specific tests monitored up to 5 years. The overall goal is to better understand the long-term impact and safety profile of the gene-modified therapy in people treated previously.

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.

This research aims to understand disease activity in patients with Congenital Myasthenic Syndromes (CMS) caused by mutations in the genes DOK7, MUSK, AGRN, or LRP4. It will evaluate symptoms and quality of life in these patients to gather important information about their condition. The study is observational and collects both retrospective and prospective data to better comprehend CMS. Participants will attend up to four study visits where clinical assessments will be performed. These assessments include evaluating changes in health status related to CMS and collecting data on diagnosis, healthcare use, and medications over a period of up to 12 months. Researchers will also monitor various scores related to muscle strength, daily living activities, fatigue, and quality of life. During the study, participants will undergo evaluations such as the Quantitative Myasthenia Gravis score, MG-ADL, PROMIS-GHS, PROMIS-DFL, EQ-5D-5L, 3TUG, and Neuro-QoL Fatigue score to measure their symptoms and well-being. The study aims to summarize these outcomes and track changes over a year. Participation involves attending scheduled visits for assessments and providing clinical information to help researchers understand CMS progression better.

This research aims to observe the long-term safety of efgartigimod treatment in adults with generalized myasthenia gravis (gMG). It compares patients who are starting or have recently started efgartigimod treatment with those who have not been exposed to efgartigimod and are receiving other standard treatments. The study is non-interventional and prospective, focusing on real-world safety outcomes over an extended period. Participants are divided into two groups: one receiving efgartigimod and another receiving other medications for myasthenia gravis. This study does not involve any new treatments being administered as part of the trial but monitors patients during their routine care. The study will continue for up to 10 years to gather long-term safety data. During the study, researchers will track the occurrence of serious infections and other safety concerns in both groups. Patients will be monitored through their regular clinical visits without additional interventions from the study. Participants may be followed for up to 10 years to assess safety outcomes associated with their treatments in everyday medical practice.

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 evaluating the safety and tolerability of ARGX-119 in adults with DOK7-Congenital Myasthenic Syndromes, a rare genetic condition affecting muscle function. The study also looks at how the body processes ARGX-119, the immune response to the treatment, and its potential impact on patients' symptoms and daily functioning. This Phase 1b trial is sponsored by argenx and aims to gather important early data about ARGX-119. Participants will be randomly assigned in a 4:1 ratio to receive either ARGX-119 or a placebo through intravenous infusions during a double-blinded treatment period. After this phase, they will enter a follow-up period. Those who complete follow-up may join an active-treatment period where they receive open-label ARGX-119. The entire study lasts about 38 months. Throughout the study, participants will undergo regular assessments including monitoring for adverse events, measuring changes in walking distance using the 6-minute walk test, and evaluations of muscle strength and daily living activities. Blood samples will be collected to study drug levels and immune reactions. Researchers will track various health scales and questionnaires to understand how the treatment affects fatigue, lung function, and quality of life over time.

Researchers are evaluating IM-101 in adults with generalized myasthenia gravis (gMG) and ocular myasthenia gravis (oMG), focusing on those with acetylcholine receptor (AChR) antibody-positive and AChR antibody-negative forms. The goal is to assess the safety, tolerability, how the drug moves through and affects the body, and the potential effectiveness of IM-101. This Phase 1b/2 trial is sponsored by ImmunAbs Inc. and includes multiple study parts to explore these aspects thoroughly. Participants will receive IM-101 or a placebo through intravenous infusions. In Part A, participants get a loading dose on Day 1 and Day 15, followed by a maintenance dose on Day 29, with doses escalating across cohorts. In Part B, dosing includes loading doses on Day 1 and Day 15 and maintenance doses on Days 29, 57, and 85. Some may receive additional doses depending on decisions by an independent data monitoring committee. The study uses randomized, double-blind assignments to compare different dose levels and placebo effects. During the study, participants will be monitored for side effects and safety up to about 99 days in Part A and 169 days in Part B. Researchers will assess the impact of treatment on daily living activities and specific myasthenia gravis severity scores at baseline and Week 16. Safety assessments include tracking adverse events, serious adverse events, and events leading to discontinuation. Participants will undergo regular evaluations to measure the pharmacokinetics and pharmacodynamics of IM-101, with follow-up visits scheduled according to the dosing timeline.

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.