Clinical Prognosis and Progression of Myasthenia Gravis Patients

NCT04101578 | Unknown FDA Drug

• 1 other identifier: 2017YFC0907705
• Study Type: observational
• Target: 2,000
• Locations: 1 country
• Sites: 1

Brief Summary

This study collects the clinical data of myasthenia gravis (MG) patients, assesses outcomes and adverse effects of different treatment regimens, and searches for risk factors of conversion to generalized MG.

Conditions

Myasthenia Gravis

Outcome Measures

Primary Outcomes (4)

• Conversion rates from ocular to generalized MG at the last visit and risk factors.

  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.

  Baseline, 48 months

• Change in Quantitative Myasthenia Gravis (QMG) Scores from Baseline to 48 months.

  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 (None=0, Mild=1, Moderate=2, Severe=3).

  Baseline, 12 months, 24 months, 36 months, 48 months

• Change in MG-specific Activities of Daily Living scale (MG-ADL).

  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.

  Baseline,3months, 6 months, 9 months, 12 months, 18 months, 24 months, 30months, 36 months, 42 months, 48 months

• The proportion of patients reaching minimal manifestations (MM) or better.

  Clinical statuses of patients are assessed and categorized according to Myasthenia Gravis Foundation of America (MGFA) postintervention status (PIS). MM or better includes Minimal Manifestation (MM), Pharmacologic Remission (PR) or Complete Remission (CR).

  48 months

Secondary Outcomes (2)

• Proportion of Patients with Treatment-related Adverse Experiences.

  3 months, 6 months, 12 months, 24 months, 36 months, 48 months

• Changes in titers of MG antibodies.

  Baseline, 12 months, 24 months, 36 months, 48 months

Study Arms (2)

Ocular MG

Patients with autoimmune MG whose symptoms restricted to extraocular muscles

Drug: Symptomatic Treatment, Steroids, Immunosuppressive Agents, Plasma Exchange(PE), Intravenous Immunoglobulin(IVIg)

Generalized MG

Patients not only suffer from extraocular muscles weakness but also from limb weakness, bulbar symptoms, or even respiratory failure

Drug: Symptomatic Treatment, Steroids, Immunosuppressive Agents, Plasma Exchange(PE), Intravenous Immunoglobulin(IVIg)

Interventions

Symptomatic Treatment, Steroids, Immunosuppressive Agents, Plasma Exchange(PE), Intravenous Immunoglobulin(IVIg) DRUG

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

Also known as: Pyridostigmine Bromide, Prednisone, Methylprednisolone, Azathioprine, Tacrolimus, Cyclosporin A, Cyclophosphamide, Mycophenolate Mofetil, Methotrexate

Generalized MG; Ocular MG

Eligibility Criteria

• Age: 15 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

Patients with Myasthenia Gravis

You may qualify if:

• Age \>14.
• Clinical Diagnosis of MG with supporting evidence:
• unequivocal clinical response to pyridostigmine
• positive antibody testing
• decrement \>10% in repetitive nerve stimulations study (RNS) .
• Willingness to sample collection, imaging study and other disease-related examinations and assessments.
• Patients with informed consent.

You may not qualify if:

• History of chronic degenerative, psychiatric, or neurologic disorder other than MG that can produce weakness or fatigue.
• Age ≤14 years.
• Severe anxiety, depression or schizophrenia.
• Cognitive impairment or mini-mental state examination (MMSE) score ≤24.
• Severe systemic illness with life-expectancy less than 4 years.
• Unwillingness to consent for collection of biological samples.
• Inability to provide informed consent.

Sponsors & Collaborators

• Da, Yuwei, M.D.: lead

Study Sites (1)

Xuan Wu Hospital, Capital Medical University

Beijing, Beijing Municipality, 100053, China

RECRUITING

Biospecimen

Retention: SAMPLES WITH DNA

Peripheral blood samples are collected annually.

MeSH Terms

Conditions

Myasthenia Gravis

Interventions

Steroids; Immunosuppressive Agents; Immunoglobulins, Intravenous; Pyridostigmine Bromide; Prednisone; Methylprednisolone; Azathioprine; Tacrolimus; Cyclosporine; Cyclophosphamide; Mycophenolic Acid; Methotrexate

Condition Hierarchy (Ancestors)

Paraneoplastic Syndromes, Nervous System; Nervous System Neoplasms; Neoplasms by Site; Neoplasms; Paraneoplastic Syndromes; Autoimmune Diseases of the Nervous System; Nervous System Diseases; Neurodegenerative Diseases; Neuromuscular Junction Diseases; Neuromuscular Diseases; Autoimmune Diseases; Immune System Diseases

Intervention Hierarchy (Ancestors)

Fused-Ring Compounds; Polycyclic Compounds; Immunologic Factors; Physiological Effects of Drugs; Pharmacologic Actions; Chemical Actions and Uses; Immunoglobulin G; Immunoglobulin Isotypes; Antibodies; Immunoglobulins; Immunoproteins; Blood Proteins; Proteins; Amino Acids, Peptides, and Proteins; Serum Globulins; Globulins; Pyridinium Compounds; Pyridines; Heterocyclic Compounds, 1-Ring; Heterocyclic Compounds; Pregnadienediols; Pregnadienes; Pregnanes; Prednisolone; Pregnadienetriols; Thionucleosides; Sulfur Compounds; Organic Chemicals; Mercaptopurine; Purines; Heterocyclic Compounds, 2-Ring; Heterocyclic Compounds, Fused-Ring; Nucleosides; Nucleic Acids, Nucleotides, and Nucleosides; Macrolides; Lactones; Cyclosporins; Peptides, Cyclic; Macrocyclic Compounds; Peptides; Phosphoramide Mustards; Nitrogen Mustard Compounds; Mustard Compounds; Hydrocarbons, Halogenated; Hydrocarbons; Phosphoramides; Organophosphorus Compounds; Caproates; Acids, Acyclic; Carboxylic Acids; Fatty Acids; Lipids; Aminopterin; Pterins; Pteridines

Study Officials

• yuwei Da, M.D.

  Xuan Wu Hospital, Capital Medical University

  STUDY CHAIR

Central Study Contacts

yuwei Da, M.D.

CONTACT

00-86-010-83198492; dayuwei1000@163.com

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: INDIV
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: September 16, 2019
• First Posted: September 24, 2019
• Study Start: February 8, 2017
• Primary Completion: June 1, 2023
• Study Completion: December 31, 2024
• Last Updated: August 23, 2021

Record last verified: 2021-08

Locations

CN (1)