Clinical Prognosis and Progression of Myasthenia Gravis Patients

Myasthenia Gravis Clinical Trial

Official title:

Prospective Observational Trial to Evaluate Clinical Prognosis and the Risk Factors for Progression for Myasthenia Gravis Patients

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT04101578
• Other study ID #: 2017YFC0907705
• Status: Recruiting
• Start date: February 8, 2017
• Est. completion date: December 31, 2024

Study information

• Verified date: August 2021
• Source: Da, Yuwei, M.D.
• Contact: yuwei Da, M.D.
• Phone: 00-86-010-83198492
• Email: dayuwei1000[@]163.com
• Is FDA regulated: No
• Study type: Observational

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 2000
• Est. completion date: December 31, 2024
• Est. primary completion date: June 1, 2023
• Accepts healthy volunteers: No
• Gender: All
• Age group: 15 Years and older

Eligibility

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.

Related Conditions & MeSH terms

• Muscle Weakness
• Myasthenia Gravis

Intervention

Drug:
• Symptomatic Treatment, Steroids, Immunosuppressive Agents, Plasma Exchange(PE), Intravenous Immunoglobulin(IVIg)
Treatment regimens are determined according to the physician's judgment and preferences of the patients.

Locations

Country	Name	City	State
China	Xuan Wu Hospital, Capital Medical University	Beijing	Beijing

Sponsors (1)

Lead Sponsor	Collaborator
Da, Yuwei, M.D.

Country where clinical trial is conducted

China, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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
Primary	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
Primary	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
Primary	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	Proportion of Patients with Treatment-related Adverse Experiences.	Treatment-Related Adverse Events (AEs) are evaluated in patients of different regimens.	3 months, 6 months, 12 months, 24 months, 36 months, 48 months
Secondary	Changes in titers of MG antibodies.	MG antibodies are detected at enrollment and the titers of antibodies will be monitored annually.	Baseline, 12 months, 24 months, 36 months, 48 months