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Cytomegalovirus Infections

Myasthenia Gravis

Asthenia

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Muscular Diseases

Neuromuscular Manifestations

Neurologic Manifestations

Syndrome

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Paraneoplastic Syndromes, Nervous System

Nervous System Neoplasms

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Autoimmune Diseases of the Nervous System

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Neuromuscular Diseases

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Cytomegalic Inclusion Disease

All Drugs and Chemicals

Vasodilator Agents

Antibodies

Neostigmine

Immunoglobulins

Acetylcholine

Immunologic Factors

Cholinesterase Inhibitors

Enzyme Inhibitors

Cholinergic Agents

Neurotransmitter Agents

underwent the following examinations: serum acetylcholine receptor antibody, erythrocyte deposition rate, hypersensitive C protein, neostigmine test, chest CT, low frequency repetitive nerve stimulation (orbicularis oculi muscle, levator palpebrae muscle, frontalis muscle), thyroid function and color ultrasound.

underwent the following examinations: serum acetylcholine receptor antibody, erythrocyte deposition rate, hypersensitive C protein, neostigmine test, chest CT, low frequency repetitive nerve stimulation (orbicularis oculi muscle, levator palpebrae muscle, frontalis muscle), thyroid function and ultrasound.

Serum acetylcholine receptor antibody, erythrocyte deposition rate, hypersensitive C protein, neostigmine test,thymus CT, low frequency repeated nerve stimulation

qing Zhou

Ocular myasthenia gravis (OMG) is an autoimmune neuromuscular disease mediated by pathogenic autoantibodies. Its etiology and pathogenesis are not completely clear. The occurrence and development of OMG are the result of the joint participation of genetic factors, environmental factors and immune factors. The role of infectious factors in the pathogenesis of autoimmune diseases is a hot topic in the international discussion.By analyzing the difference in the positive rate and titer of CMV antibodies between ocular myasthenia gravis and healthy people. By analyzing the correlation between the positive rate and titer of CMV antibodies and ocular myasthenia gravis clinical manifestations, acetylcholine receptor antibodies, ESR, hypersensitive C-reactive protein, complement C3, complement C4 and thymus status.To investigate the correlation between cytomegalovirus and ocular myasthenia gravis. It can provide new insights for further studies on the etiology and possible pathogenesis of ocular myasthenia gravis.

A total of 75 patients and 40 healthy volunteers are collected who are diagnosed with ocular myasthenia gravis in the First Affiliated Hospital of Jinan University from January 2019 to January 2022 . All subjects undergo eye examination and cytomegalovirus antibody detection. OMG patients undergo the following examinations: serum acetylcholine receptor antibody, erythrocyte deposition rate, hypersensitive C protein, neostigmine test, chest CT, low frequency repetitive nerve stimulation (orbicularis oculi, levator palpebrae superioris, frontalis muscle), thyroid function and color ultrasound. The difference of lgG positive rate ,lgM positive rate of CMV and antibody titers of lgG and lgM are analyzed between the two groups, and the relationship between CMV antibody and OMG clinical manifestations, acetylcholine receptor antibody, laboratory indicators and thymus status are analyzed.

Study on Correlation Between Cytomegalovirus and Ocular Myasthenia Gravis

CMV-lgG titer ≥14IU/ mL is positive, 12-14 IU/ mL is critical, and 0-12 IU/ mL is negative. CMV-lgM titer \> 22U/ mL is positive, 18-22U/mL is critical, and 0-18U/mL is negative.

Concentrations of cytomegalovirus lgG and lgM antibodies is collected for analysis.

XiaoYong Liu

Deputy chief physician

The outcome measure was time to Myasthenia gravis generalization. The explanatory variables were age at onset, sex, first-year anti-acetylcholinereceptor antibody positivity, repetiting nerve stimulation showing electromyogram decrement and steroid use. Kaplan-Meier survival estimations, descriptive and multivariate Cox model analyses were computed. A nomogram combining explanatory vraiables was used to establish a score to predict the probability of OMG generalization.

Use of nomogram for predictions of Ocular Mysathenia Gravis generalization

Secondary ocular myasthenia gravis (OMG) generalization represents a pejorative evolution and no validated generalization prevention strategy exists. The aim of this observational study was to determine the percentage of patients with OMG generalization and identify factors predictive of that pejorative evolution. Data from patients with OMG registered in the Fondation Hospital A. de Rothschild database between January 1990 and January 2017 were collected. Among the 183 patients registered in this database, 151 patients with available informations were analyzed.

Ocular Mysathenia Gravis Generalization

Use of Nomogram for Predictions of Ocular Mysathenia Gravis Generalization

Kaplan-meier survival estimations, descriptive and multivariate Cox model analyses were computed (age at onset, first year anti-acetylcholine receptor antibody positivity, electromyogram decrement, steroid use).

Fondation Ophtalmologique Adolphe de Rothschild

Dr Amélie Yavchitz

Anti-Inflammatory Agents

Antineoplastic Agents

Prednisone

Pyridostigmine Bromide

Glucocorticoids

Hormones

Hormone Antagonists

Antineoplastic Agents, Hormonal

Prednisone will be adjusted based on combined measures of tolerability and efficacy. Capsules will contain 10mg of prednisone.

Placebo dosages will be adjusted based on combined measures of tolerability and efficacy. Capsules will contain matching placebo.

Placebo

Michael Benatar, MBChB, DPhil

Gil Wolfe, MD

Donald Sanders, MD

The purpose of this study is to evaluate the efficacy and tolerability of prednisone in patients diagnosed with ocular myasthenia.

Funding Source - FDA OOPD

The purpose of this study is to learn two things about prednisone in patients with ocular myasthenia. The first thing we aim to learn is whether or not prednisone is effective in improving the symptoms of double vision and drooping eyes that are experienced by patients with ocular myasthenia. The second thing we aim to learn is whether we can find a dose of prednisone that is well tolerated and safe. The overall goal is to find out whether a dose of prednisone that is safe and well tolerated is also effective in improving the symptoms of ocular myasthenia.

After completing screening assessments to confirm eligibility, all participants will receive treatment with pyridostigmine. If a participant's symptoms do not resolve within the first month while being treated with pyridostigmine, they will be randomized to receive prednisone or placebo. The amount of study medication a participant receives will depend on how their symptoms respond to the medication and if they experience any side effects.

After four months, participants that continue to have symptoms of ocular myasthenia and do not have side effects will receive open label high dose prednisone. Participants that no longer have symptoms will taper their dose of study drug in a double-blind fashion.

Efficacy of Prednisone In the Treatment of Ocular Myasthenia

Efficacy of Prednisone In the Treatment of Ocular Myasthenia: The EPITOME' Study

Failure to achive sustatined minimal manifestation status by week 16

University of Miami

University of Rochester

Michael Benatar

MBChB, DPhil

Corticosteroid

Prednisone: Prednisone will be adjusted based on combined measures of tolerability and efficacy. Capsules will contain 10mg of prednisone.

Pyridostigmine: Prior to randomization, pyridostigmine dosage increments will be made as needed for myasthenic symptoms and as tolerated according to a pre-specified dosage titration schedule. Following randomization, dose will remain constant.

Matched, inactive substance

Placebo: Placebo dosages will be adjusted based on combined measures of tolerability and efficacy. Capsules will contain matching placebo.

Pyridostigmine: Prior to randomization, pyridostigmine dosage increments will be made as needed for myasthenic symptoms and as tolerated according to a pre-specified dosage titration schedule. Following randomization, dose will remain constant.

Total

Age, Categorical

Age, Continuous

Sex: Female, Male

Ethnicity (NIH/OMB)

Race (NIH/OMB)

Early termination leading to small numbers of subjects analyzed

Dr. Benatar

Treatment Failure

Mean Ocular QMG Changes from Baseline to Week 16 . 95% CI and p-values obtained from the analysis of covariance model to adjust for the baseline value of the outcome variable.

Change in Ocular Quantitative Myasthenia Score From Baseline to Week 16

Mean Change in quality of life as measured by the NEI-VFQ-25 at the end of Double- Blinded Treatment. 95% CI and p-values obtained from the analysis of covariance model to adjust for the baseline value of the outcome variable.

Change in Quality of Life as Measured by the NEI-VFQ-25 Measures

Mean Change in quality if life as measured by the MG-QOL-15 score at the end of Double- Blinded Treatment. 95% CI and p-values obtained from the analysis of covariance model to adjust for the baseline value of the outcome variable.

Change in Quality of Life as Measured by the MG-QOL-15 Score

Mean Change NEI-VFQ-25 10 item neuro-opthtalmological supplement score baseline to week 16. 95% CI and p-values obtained from the analysis of covariance model to adjust for the baseline value of the outcome variable.

Change in Quality of Life as Measured by the 10-Item Neuro-ophthalmological Supplement to the NEI-VFQ-25

Double Blinded: 16 Weeks

Overall Study: 36 Weeks

Anti-Infective Agents

Anticonvulsants

Antirheumatic Agents

Mycophenolic Acid

Tacrolimus

Bromides

Immunosuppressive Agents

Azathioprine

Calcineurin Inhibitors

Anti-Bacterial Agents

Antibiotics, Antitubercular

Antitubercular Agents

Antimetabolites

Newly-onset OMG patients who agreed to join the follow-up cohort

Treatment regimens are determined according to the physician's judgment and preferences of the patients.

Symptomatic Treatment, Steroids, Immunosuppressive Agents( azathioprine, tacrolimus, Mycophenolate Mofetil)

Jun Guo, M.D.

Jun Guo

This study collects the clinical data of new-onset ocular myasthenia gravis (OMG) patients, assesses outcomes and adverse effects of different treatment options, and evaluate risk factors of conversion to generalized MG(GMG).

This is a multicenter, observational cohort trial in the real-world clinical setting recruiting new-onset OMG patients from Neurology Departments of 7 hospitals in different regions of China. Clinical manifestations, laboratory test results, chest imaging and history of thymectomy are recorded. Treatment option are determined according to the physician's judgment and preferences of the patients. Patients are followed up prospectively on regular to assess the outcomes of treatments and monitor any side effects. Peripheral blood samples are collected annually. The investigators plan to recruit a final sample of 200 patients for analysis.

TheRapeutic Effect of Different immunosuppressAnts on Non-Thymoma Ocular Myasthenia Gravis: a Real-world Study

Ocular MG patients are followed up to determine the ratio of conversion to generalized disease at the end of follow-up. The clinical records will be retrospectively analyzed to search for risk factors of progressing.

Ocular MG patients are followed up to determine the ratio of patients that achieve the status of MGSTI 2 or better at each period piont of follow-up. The clinical records will be retrospectively analyzed to search for risk factors prolonged the regimens reduction.

Treatment-Related Adverse Events (AEs) are evaluated in patients receiving different treatment protocol

The improvement of clinic status of OMG patients was assessed by investigators according to QMG and ARS-MG score. The QMG is a 13-item scale which measures ocular, bulbar, limb function and respiratory function. The total score ranges from 0 (no myasthenic findings) to 39 (maximal myasthenic deficits) obtained by summing the responses to each individual item.The ARS-MG is a 8-item scale which has a more detailed measure to assess ocular function . The total score ranges from 0 (no myasthenic findings) to 60 (maximal myasthenic deficits) obtained by summing the responses to each individual item.

The improvement of clinic status of OMG patients was assessed by patients themselves according to MG-ADL score. The The MG-ADL is an 8-item scale to assess symptoms of myasthenia gravis patients obtained by summing the responses to each individual item (Grades: 0,1,2,3). The score ranges from 0 to 24.

MG antibodies are detected at enrollment and the titers of antibodies will be monitored annually.

RNS and SFEMG will be monitored on time to assess the clinic status.

Ocular MG patients are followed up to determine the relapse rate at the end of follow-up. The clinical records will be retrospectively analyzed to search for risk factors of recurrence.

Tang-Du Hospital

Associate Professor

Mark JW Larkin, PhD

An international, non-interventional, prospective, community recruited, longitudinal, lifestyle study which aims to collect Real-World Evidence (RWE) from people living with myasthenia gravis (MG) in order to map the disease's natural history, its burden on patients and their families, its treatment, as well as its medical, social and pharmacoeconomic impact.

Data was collected from people with MG using the MyRealWorld™ RWE platform , developed by the healthcare digital research company, Vitaccess. Participants downloaded a 'bring your own device' (BYOD) study app, MyRealWorld™ MG, onto their smartphones, which they used to enter data and complete surveys about their MG.

The Impact of Myasthenia Gravis in the Real World

Aggregated data may be available via application.

The EQ-5D-5L comprises two parts - the EQ-5D-5L descriptive system and the EQ Visual Analogue Scale (EQ VAS).

The descriptive system comprises 5 dimensions (mobility, self care, usual activities, pain/discomfort, and anxiety/depression), each with 5 levels (no problems, slight problems, moderate problems, severe problems, and extreme problems - i.e., higher scores represent worse health). The digits for 5 dimensions can be combined in a 5-digit number describing the respondent's health state.

The EQ VAS records the respondent's self-rated health on vertical, visual analogue scale with endpoints labelled 'the best health you can imagine' and 'the worst health you can imagine'. Higher scores represent better self-perceived health.

The MG-ADL is a myasthenia gravis-specific 8-item scale, which assesses the severity of the following symptoms: talking, chewing, swallowing, breathing, impairment of ability to brush teeth/comb hair, impairment of ability to rise from a chair, double vision, and eyelid droop. Each of the 8 items has 4 response options; 0 (Mild), 1 (Moderate), 2 (Mild) and 3 (Severe). The MG-ADL total score can be calculated by totalling all 8 items out of a maximum score of 24. A higher score indicates severe impairment.

The MG-QOL15r is a myasthenia gravis-specific health-related quality of life survey, which assesses the impact of myasthenia gravis over the past few weeks on the following domains: emotions, physical health, self-care, social life, and impact on role. Each of the 15 items has three response options: 0 (Not at all), 1 (Somewhat), and 2 (Very much). The MG-QoL15r total score can be calculated by totalling all 15 items out a maximum score of 30. A higher score indicates severe impairment.

The HADS is a frequently used self-rating scale developed to assess psychological distress in non-psychiatric patients. It consists of two subscales, Anxiety and Depression. The instrument comprises 14 items, 7 for the anxiety subscale and 7 for the depression subscale. Each item is scored on a 4-point response scale, scale wording changes for each item with each coding response corresponding with greater impairment or severity. Domain scores can be calculated by totalling all 7 items out of a maximum score of 21. High scores indicate greater severity of that impairment

The HUI3 is a generic 8-item survey, which provides descriptive evidence on multiple dimensions of health status, a score for each dimension of health, and a health-related quality of life score for overall health.Health dimensions include vision, hearing, speech, ambulation/mobility, pain, dexterity, emotion and cognition. Each dimension has five or six response options. It has recall periods of either 'usual health', the past week, two weeks, or four weeks. Scores on individual items are combined to given a combined health state which can then be converted to health utilities. Lower health utilities represent worse health.

This is a bespoke survey designed to understand the impact of COVID-19 on study participants. It includes questions about whether participants have had COVID, and the consequences if so, whether they have been vaccinated, and how the wider pandemic has affected their MG treatment and quality of life.

The SSQ is a single question that asks respondents what percentage of normal they feel with respect to their MG. The PASS is a single question that asks respondents whether they would consider their current disease state to be satisfactory if they had to stay in it for the next few months. The SSQ and PASS will be presented to participants at the same time, as a question pair.

The EQ-5D-3L is a three-level version of the EQ-5D-5L. It includes the same dimensions as the EQ-5D-5L, but each has only three levels: no problems, some problems, and extreme problems. The EQ VAS is unchanged from the EQ-5D-5L version.

The PROMIS sleep disturbance measure assesses self-reported frequency, severity, and duration of dyspnea (shortness of breath) over the last 7 days using either a 0-10 numeric rating scale or 0-4 rating scale to assess dyspnea characteristics.Each item is evaluated individually according to the numeric rating scale. No total score is calculated for this instrument. Higher scores on items indicate more severe symptoms.

The FACIT-Fatigue measure is a generic 13-item survey which assesses fatigue levels and impact over the past 7 days. The level of fatigue is measured on a 4-point Likert scale (4 = not at all fatigue to 0 = very much fatigued). Scores range from 0-52. The higher the score, the better the quality of life.

The PROMIS sleep disturbance measure assesses self-reported perceptions of sleep quality, sleep depth, and restoration associated with sleep over the last 7 days using a 5-point scale. Scores range from 6-30. Higher scores represent worse sleep disturbance.

Vitaccess Ltd

Rituximab

Antineoplastic Agents, Immunological

Immediate standardized treatment with corticosteroids. Standardized treatment in the experimental arm will start at 0.5mg/kg/d prednisone.

immediate treatment with corticosteroids

rituximab added if ocular symptoms reappear as corticosteroids are tapered off. Rituximab will be given at a dose of 500mg/6months for 12 months.

addition of rituximab if recurrence

Myasthenia is an autoimmune disease causing dysfunction of the neuromuscular junction, resulting in fluctuating and variable muscle weakness.

In the initial phase of the disease, 70% of patients present with ocular onset myasthenia (OMG), i.e. weakness limited to the oculomotor muscles. Generalization to skeletal, bulbar and axial muscles occurs in 20-40% of cases, with a higher frequency in the first and second years, respectively 46% and 60% of generalizations. This reflects the maturation of the autoimmune response in the early years of the disease, and represents a therapeutic window of opportunity to modify the course of the disease.

Generalization is a critical event, putting the patient at risk of admission to an intensive care unit and necessitating the use of long-term immunosuppressants.

There is currently no validated strategy for preventing generalization. On the one hand, a preventive role for corticosteroid therapy in ocular-onset myasthenia has been observed in some studies, but not confirmed by others. These contradictory results may be explained by the bias of retrospective observational studies and the use of different corticosteroid administration regimens.

On the other hand, recent data on the use of low-dose Rituximab in the early phase of the disease shows greater efficacy than later use, enabling prolonged remission of the disease with a very good tolerability profile.

We propose to compare in a randomized controlled trial the usual practice with a proactive strategy with a standardized corticosteroid regimen immediate at diagnosis.

Patients with ocular myasthenia are usually treated symptomatically with acetylcholinesterase inhibitors. The introduction of corticosteroids is delayed and limited to patients with persistent disabling diplopia or ptosis with occlusion. When corticosteroids are tapered off, ocular symptoms may recur. This level of corticosteroid dependence observed in patients treated for ocular myasthenia has not been specifically studied. In order to reduce the levels of corticosteroids administered and avoid recurrence of ocular symptoms and their delayed generalization, it is usually proposed to introduce another immunosuppressant.

The aim of this study is to evaluate the efficacy of a standardized proactive prevention strategy on the generalization of ocular onset myasthenias during the first 2 years. It will combine immediate treatment with corticosteroids at the time of diagnosis, with the addition of rituximab in the event of recurrence of ocular symptoms as corticosteroids are tapered off.

Immediate Corticosteroid Therapy and Rituximab to Prevent Generalization in Ocular Myasthenia: a PROBE Multicenter Open-label Randomized Controlled Trial.

Generalization is defined by a loss of 5 points or more on any Myasthenic Muscle Score (MMS) item, excluding "eyelid occlusion" and "extrinsic ocular musculature".

Progress towards generalization will be assessed by an expert physician, blinded to the intervention arm.

Study title	NCT ID	Conditions	Interventions	Eligibility	Organization
Study on Correlation Between Cytomegalovirus and Ocular Myasthenia Gravis	NCT05091177	Ocular Myasthen... Cytomegalovirus...	Serum acetylcho...	18 Years - 50 Years	First Affiliated Hospital of Jinan University
Ocular Mysathenia Gravis Generalization	NCT04667650	Ocular Myasthen...	Use of nomogram...	-	Fondation Ophtalmologique Adolphe de Rothschild
Efficacy of Prednisone In the Treatment of Ocular Myasthenia	NCT00995722	Ocular Myasthen...	Prednisone Placebo	18 Years -	University of Miami
Ocular Mysathenia Gravis Generalization	NCT04667650	Ocular Myasthen...	Use of nomogram...	-	Fondation Ophtalmologique Adolphe de Rothschild
TheRapeutic Effect of Different immunosuppressAnts on Non-Thymoma Ocular Myasthenia Gravis: a Real-world Study	NCT04182984	Ocular Myasthen...	Symptomatic Tre...	18 Years - 75 Years	Tang-Du Hospital
The Impact of Myasthenia Gravis in the Real World	NCT04176211	Myasthenia Grav...		18 Years -	Vitaccess Ltd
The Impact of Myasthenia Gravis in the Real World	NCT04176211	Myasthenia Grav...		18 Years -	Vitaccess Ltd
TheRapeutic Effect of Different immunosuppressAnts on Non-Thymoma Ocular Myasthenia Gravis: a Real-world Study	NCT04182984	Ocular Myasthen...	Symptomatic Tre...	18 Years - 75 Years	Tang-Du Hospital
Immediate Corticosteroid Therapy and Rituximab to Prevent Generalization in Ocular Myasthenia: a PROBE Multicenter Open-label Randomized Controlled Trial.	NCT06342544	Ocular Myasthen...	immediate treat... addition of rit...	18 Years -	Fondation Ophtalmologique Adolphe de Rothschild
Immediate Corticosteroid Therapy and Rituximab to Prevent Generalization in Ocular Myasthenia: a PROBE Multicenter Open-label Randomized Controlled Trial.	NCT06342544	Ocular Myasthen...	immediate treat... addition of rit...	18 Years -	Fondation Ophtalmologique Adolphe de Rothschild
Efficacy of Prednisone In the Treatment of Ocular Myasthenia	NCT00995722	Ocular Myasthen...	Prednisone Placebo	18 Years -	University of Miami

Spots Global Cancer Trial Database for ocular myasthenia gravis