Clinical trial NCT06700616: A Study in Patients With Myasthenia Gravis in China
Official title: A Multicenter, Prospective Cohort Study in Patients With Myasthenia Gravis in China
Study type: OBSERVATIONAL
Overall status: RECRUITING
Conditions: Myasthenia Gravis
Keywords: Myasthenia Gravis
Generalized Myasthenia Gravis
Refractory Myasthenia Gravis
Prospective Study
Observational Study
Cohort Study
Patient Registry
Chinese
Lead sponsor: AstraZeneca
Sponsor class: INDUSTRY
First posted: 2024-11-22
Enrollment: 1200
Sex: ALL
Minimum age (years): 0
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: This study will recruit patients diagnosed with MG by a physician. The diagnosis of MG patients newly diagnosed prior to enrollment should be based on the clinical characteristics of MG and at least one of the following criteria: (a) Positive serology tests for acetylcholine receptor antibody (AChR Ab), muscle-specific kinase antibody (MuSK Ab), or lipoprotein-related protein 4 (LRP4 Ab); (b) Results of electrophysiological studies (repetitive stimulation, single-fiber electromyography, or both) related to synaptic transmission disorder at the neuromuscular junction; (c) Positive pharmacological test results (such as neostigmine methylsulfate test). Previously diagnosed patients must have a documented MG diagnosis accepted by clinical physician.

Brief summary: This study is a multi-center, prospective cohort study designed to characterize current clinical practice, clinical and patient-reported outcomes (PROs), disease prognosis, treatment patterns and healthcare resource utilization for Chinese patients with myasthenia gravis (MG). This study will enroll patients with MG as diagnosed by physician. Approximately 1,200 MG patients are intended to be recruited from approximately 40 sites across majority of regions in China. The clinical and PROs included MGFA class, MGFA PIS, MG-ADL (Activities of Daily Living), QMG (Quantitative MG score), MG QOL-15R, EQ-5D etc. All MG patients enrolled will be followed up every 6 months until end of 2027.


Eligibility criteria: Inclusion Criteria:

This study will enroll patients of all ages who are diagnosed with MG of all MGFA classifications by physicians.

1. Patients must have the following data to be enrolled in this study:

   * MGFA classification
   * MG-ADL score
2. MG patients or their legal guardians/representatives are able to sign the Informed Consent Form (ICF).

Exclusion Criteria:

1. Patients aged ≥18 years with ocular myasthenia gravis (MGFA class I) lasting for more than 2 years;
2. Patients who are currently participating in an interventional clinical trial cannot be enrolled in this study.

Primary outcomes:
measure: Clinical outcome
timeFrame: Every 6 months up to 3 years
measure: Treatment method
timeFrame: Every 6 months up to 3 years
measure: Patient-reported outcome
timeFrame: Every 6 months up to 3 years
measure: Clinical outcome
timeFrame: Every 6 months up to 3 years
measure: Clinical outcome
timeFrame: Every 6 months up to 3 years
measure: Clinical outcome
timeFrame: Every 6 months up to 3 years
measure: Clinical outcome
timeFrame: Every 6 months up to 3 years
measure: Patient-reported outcome
timeFrame: Every 6 months up to 3 years
measure: Patient-reported outcome
timeFrame: Every 6 months up to 3 years
measure: Treatment duration
timeFrame: Every 6 months up to 3 years
measure: Treatment dose
timeFrame: Every 6 months up to 3 years
measure: Treatment frequency
timeFrame: Every 6 months up to 3 years
Secondary outcomes:
measure: Demographic factors
timeFrame: Baseline
measure: Healthcare resource utilization
timeFrame: Every 6 months up to 3 years
measure: Cormobidity
timeFrame: Every 6 months up to 3 years
measure: Healthcare resource utilization
timeFrame: Every 6 months up to 3 years
measure: MG diagnosis history
timeFrame: Every 6 months up to 3 years
measure: Vital sign - blood pressure
timeFrame: Every 6 months up to 3 years
measure: Laboratory test - blood cell test
timeFrame: Every 6 months up to 3 years
measure: Laboratory tests etc.) - MG-related antibodies
timeFrame: Every 6 months up to 3 years
measure: Vital sign - pulse
timeFrame: Every 6 months up to 3 years
Other outcomes:
Locations:
city: Beijing
country: China
facility: Research Site
city: Chengdu
country: China
facility: Research Site
city: Fuzhou
country: China
facility: Research Site
city: Guangzhou
country: China
facility: Research Site
city: Guiyang
country: China
facility: Research Site
city: Harbin
country: China
facility: Research Site
city: Hebei
country: China
facility: Research Site
city: Henan
country: China
facility: Research Site
city: Inner Mongolia
country: China
facility: Research Site
city: Jiangsu
country: China
facility: Research Site
city: Lanzhou
country: China
facility: Research Site
city: Nanning
country: China
facility: Research Site
city: Qingdao
country: China
facility: Research Site
city: Taiyuan
country: China
facility: Research Site
city: Tianjin
country: China
facility: Research Site
city: Urumqi
country: China
facility: Research Site
city: Wenzhou
country: China
facility: Research Site
city: Wuhan
country: China
facility: Research Site
city: XI An
country: China
facility: Research Site
city: Xi'an
country: China
facility: Research Site
city: Yinchuan
country: China
facility: Research Site
Study officials:
affiliation: Beijing Tiantan Hospital
name: Fudong Shi, MD, PhD
role: PRINCIPAL_INVESTIGATOR
References: