Working…
ClinicalTrials.gov
ClinicalTrials.gov Menu

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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT04182984
Recruitment Status : Recruiting
First Posted : December 2, 2019
Last Update Posted : December 4, 2019
Sponsor:
Information provided by (Responsible Party):
Jun Guo, MD, Tang-Du Hospital

Brief Summary:
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).

Condition or disease Intervention/treatment
Ocular Myasthenia Gravis Drug: Symptomatic Treatment, Steroids, Immunosuppressive Agents( azathioprine, tacrolimus, Mycophenolate Mofetil)

Detailed Description:
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.

Layout table for study information
Study Type : Observational [Patient Registry]
Estimated Enrollment : 200 participants
Observational Model: Cohort
Time Perspective: Prospective
Target Follow-Up Duration: 3 Years
Official Title: TheRapeutic Effect of Different immunosuppressAnts on Non-Thymoma Ocular Myasthenia Gravis: a Real-world Study
Actual Study Start Date : November 4, 2019
Estimated Primary Completion Date : November 4, 2024
Estimated Study Completion Date : November 4, 2024


Group/Cohort Intervention/treatment
Patients with autoimmune ocular MG
Newly-onset OMG patients who agreed to join the follow-up cohort
Drug: Symptomatic Treatment, Steroids, Immunosuppressive Agents( azathioprine, tacrolimus, Mycophenolate Mofetil)
Treatment regimens are determined according to the physician's judgment and preferences of the patients.
Other Name: Pyridostigmine Bromide, Prednisone,Azathioprine, Tacrolimus, Mycophenolate Mofetil,




Primary Outcome Measures :
  1. Conversion rate from ocular to generalized MG at the last follow-up visit. [ Time Frame: 144 weeks ]
    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.

  2. Changes in the ratio of patients who achieve the level 2 of MG Status and Treatment Intensity (MGSTI) scale or better. [ Time Frame: Baseline, 12 weeks, 24 weeks, 36 weeks, 48 weeks, 60 weeks, 72 weeks, 96 weeks, 120 weeks, 144 weeks ]
    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.

  3. Changes in proportion of patients with treatment-related adverse events. [ Time Frame: Baseline, 12 weeks, 24 weeks, 36 weeks, 48 weeks, 60 weeks, 72 weeks, 96 weeks, 120 weeks, 144 weeks ]
    Treatment-Related Adverse Events (AEs) are evaluated in patients receiving different treatment protocol

  4. Changes in scores of Quantitative Myasthenia Gravis (QMG) scale and of Absolute and Relative Score of MG(ARS-MG) scales from Baseline. [ Time Frame: 12 weeks, 24 weeks, 36 weeks, 48 weeks, 60 weeks, 72 weeks, 96 weeks, 120 weeks, 144 weeks ]
    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.

  5. Changes in scores of MG-specific Activities of Daily Living (MG-ADL) scale from Baseline. [ Time Frame: 12 weeks, 24 weeks, 36 weeks, 48 weeks, 60 weeks, 72 weeks, 96 weeks, 120 weeks, 144 weeks ]
    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.


Secondary Outcome Measures :
  1. Changes in titers of MG antibodies. [ Time Frame: Baseline, 48 weeks, 96 weeks, 144 weeks ]
    MG antibodies are detected at enrollment and the titers of antibodies will be monitored annually.

  2. Improvement of repeated frequency electrical stimulation tests (RNS) and single fiber electromyography (SFEMG). [ Time Frame: 12 weeks, 24 weeks, 36 weeks, 48 weeks, 60 weeks, 72 weeks, 96 weeks, 120 weeks, 144 weeks ]
    RNS and SFEMG will be monitored on time to assess the clinic status.

  3. Relapse rate during follow-up period. [ Time Frame: Baseline, 144 weeks ]
    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.



Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


Layout table for eligibility information
Ages Eligible for Study:   18 Years to 75 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population
Patients with newly-onset ocular myasthenia gravis
Criteria

Inclusion Criteria:

  1. Age >18 years and<75 years;
  2. Clinical Diagnosis of MG with supporting evidence:

    Patients with ocular muscle type MG who have never received treatment meet one of the diagnoses (1) and (2)-(5): (1) The patient's symptoms involve the extraocular muscles, except for drooping eyelids and diplopia, no other clinical symptoms; (2) unequivocal clinical response to pyridostigmine; (3) positive acetylcholine receptor antibody or musk antibody; (4) decrement of more than 10% in repetitive nerve stimulations study (RNS); (5) the "trembling" of the single fiber electromyography (SFEMG) is broadened with or without blockade;

  3. Willingness to sample collection, imaging study and other disease-related examinations and assessments;
  4. The results of pregnancy tests for female subjects with fertility during the screening period should be negative and effective contraception was used by the patient and her spouse during the study period;
  5. Patients with informed consent;
  6. Predicted survival time is longer than 3 years.

Exclusion Criteria:

  1. History of chronic degenerative, psychiatric, or neurologic disorder other than MG that can produce weakness or fatigue;
  2. Patients who may have other diseases that may lead to eyelid drooping, peripheral muscle weakness or diplopia;
  3. Age ≤18 years or ≥75 years;
  4. Patients who have been taking glucocorticoids or associated immunosuppressants due to other immune system diseases;
  5. Patients who cannot use immunosuppressants due to other chronic diseases;
  6. Patients who are unable to cooperate with follow-up and self-assessment due to severe mental illness or cognitive impairment;
  7. Pregnant women, lactating women and patients with fertility plans during the trial;
  8. Patients who have suffered from severe infections or malignant tumors in the last 1 month and are unable to receive immunosuppressants treatments;
  9. Patients who are not willing to cooperate with repeated frequency electrical stimulation tests and chest CT examinations;
  10. Patients who are not willing to participate in this study;
  11. Patients who are unable to sign informed consent;
  12. Predicted survival time is shorter than 3 years;
  13. Patients who are not suitable to participated in the trial after researchers' assessment.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04182984


Contacts
Layout table for location contacts
Contact: Jun Guo, M.D. 86-29-8477 8844 guojun_81@163.com

Locations
Layout table for location information
China, Shaanxi
Tangdu Hospotal Recruiting
Xi'an, Shaanxi, China
Contact: Jun Guo, M.D.    86-29-8477 8844    guojun_81@163.com   
Sponsors and Collaborators
Tang-Du Hospital
Investigators
Layout table for investigator information
Study Chair: Jun Guo Tangdu Hospital, Air Force Military Medical University

Layout table for additonal information
Responsible Party: Jun Guo, MD, Associate Professor, Tang-Du Hospital
ClinicalTrials.gov Identifier: NCT04182984     History of Changes
Other Study ID Numbers: 2019TDSNOMG
First Posted: December 2, 2019    Key Record Dates
Last Update Posted: December 4, 2019
Last Verified: December 2019

Layout table for additional information
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Product Manufactured in and Exported from the U.S.: No
Keywords provided by Jun Guo, MD, Tang-Du Hospital:
Ocular myasthenia gravis
Additional relevant MeSH terms:
Layout table for MeSH terms
Muscle Weakness
Myasthenia Gravis
Muscular Diseases
Musculoskeletal Diseases
Neuromuscular Manifestations
Neurologic Manifestations
Nervous System Diseases
Pathologic Processes
Signs and Symptoms
Autoimmune Diseases of the Nervous System
Neuromuscular Junction Diseases
Neuromuscular Diseases
Autoimmune Diseases
Immune System Diseases
Mycophenolic Acid
Azathioprine
Tacrolimus
Immunosuppressive Agents
Pyridostigmine Bromide
Physiological Effects of Drugs
Antineoplastic Agents
Immunologic Factors
Calcineurin Inhibitors
Enzyme Inhibitors
Molecular Mechanisms of Pharmacological Action
Antibiotics, Antineoplastic
Antibiotics, Antitubercular
Antitubercular Agents
Anti-Bacterial Agents
Anti-Infective Agents