Clinical trial NCT04101578: Clinical Prognosis and Progression of Myasthenia Gravis Patients
Official title: Prospective Observational Trial to Evaluate Clinical Prognosis and the Risk Factors for Progression for Myasthenia Gravis Patients
Study type: OBSERVATIONAL
Overall status: UNKNOWN
Conditions: Myasthenia Gravis
Lead sponsor: Da, Yuwei, M.D.
Sponsor class: INDIV
First posted: 2019-09-24
Enrollment: 2000
Sex: ALL
Minimum age (years): 15
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Patients with Myasthenia Gravis

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.

Detailed description: This is a multicenter, observational cohort trial in the real-world clinical setting recruiting MG patients from Neurology Departments of 6 hospitals in different regions of China. Clinical manifestations, laboratory test results, chest imaging and history of thymectomy are recorded. Patients will be classified by clinical manifestation as well as antibody status, and treatment regimens 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. Patients' clinical records are uploaded to an online database. The investigators plan to recruit a final sample of 2000 patients for analysis.

Eligibility criteria: Inclusion Criteria:

1. Age \>14.
2. Clinical Diagnosis of MG with supporting evidence:

   1. unequivocal clinical response to pyridostigmine
   2. positive antibody testing
   3. decrement \>10% in repetitive nerve stimulations study (RNS) .
3. Willingness to sample collection, imaging study and other disease-related examinations and assessments.
4. Patients with informed consent.

Exclusion Criteria:

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

Primary outcomes:
measure: Conversion rates from ocular to generalized MG at the last visit and risk factors.
timeFrame: Baseline, 48 months
measure: Change in Quantitative Myasthenia Gravis (QMG) Scores from Baseline to 48 months.
timeFrame: Baseline, 12 months, 24 months, 36 months, 48 months
measure: Change in MG-specific Activities of Daily Living scale (MG-ADL).
timeFrame: Baseline,3months, 6 months, 9 months, 12 months, 18 months, 24 months, 30months, 36 months, 42 months, 48 months
measure: The proportion of patients reaching minimal manifestations (MM) or better.
timeFrame: 48 months
Secondary outcomes:
measure: Proportion of Patients with Treatment-related Adverse Experiences.
timeFrame: 3 months, 6 months, 12 months, 24 months, 36 months, 48 months
measure: Changes in titers of MG antibodies.
timeFrame: Baseline, 12 months, 24 months, 36 months, 48 months
Other outcomes:
Locations:
city: Beijing
country: China
facility: Xuan Wu Hospital, Capital Medical University
state: Beijing
Study officials:
affiliation: Xuan Wu Hospital, Capital Medical University
name: yuwei Da, M.D.
role: STUDY_CHAIR
References: