Epidemiological Study of Treatment Approaches in AChR-Antibody Positive Generalized Myasthenia Gravis in Russia

A Multicenter Non Interventional Single Arm Retrospective-prospective Observational Study in Therapeutic Approaches in AChR-Antibody Positive Generalized Myasthenia Gravis (gMG) in Real Clinical Practice in Russia

This is a multicenter, non-interventional, retrospective-prospective, single-arm observational study designed to describe real-world treatment approaches and clinical outcomes among adults with acetylcholine receptor (AChR) antibody-positive generalized myasthenia gravis (gMG) in routine clinical practice in Russia.

Study Overview

• Status: Recruiting
• Conditions: Rare Diseases; Generalized Myasthenia Gravis (gMG)

Detailed Description

This is a multicenter, non-interventional, retrospective-prospective, single-arm observational study designed to describe real-world treatment approaches and clinical outcomes among adults with acetylcholine receptor (AChR) antibody-positive generalized myasthenia gravis (gMG) in routine clinical practice in Russia.

The primary objective is to characterize the demographic and clinical profile of adult AChR-Ab-positive gMG patients. Key secondary objectives include assessing disease severity over time (MG-ADL, QMG, MGFA class), evaluating rates, duration, and reasons for all-cause and gMG-related hospitalizations, describing diagnostic pathways and treatment strategies (including first-line regimens and use of therapies such as anticholinesterase agents, corticosteroids, immunosuppressants, IVIG, plasmapheresis/plasma filtration, thymectomy, rituximab, and complement C5 inhibitors), exploring patterns and outcomes of myasthenic crises, and documenting meningococcal vaccination and any prophylactic antibiotic use prior to initiation of C5 inhibitor therapy.

Approximately 100 adults will be enrolled consecutively across about 10 specialized sites. The study will sequentially include only those patients who have signed the informed consent form (ICF). Eligible patients will be enrolled consecutively at each site to minimize selection bias. Data are collected from existing paper/electronic medical records (secondary data collection) and recorded into an eCRF at seven timepoints: baseline (with retrospective abstraction, including medical history from diagnosis) and prospective follow-up every 6 months up to Month 36 (±1 month windows). No study-mandated interventions, tests, or visit schedules are imposed; all care follows routine practice.

• Study Type: Observational
• Enrollment (Estimated): 450

Contacts and Locations

• Study Contact: Name: AstraZeneca Clinical Study Information Center; Phone Number: 1-877-240-9479; Email: information.center@astrazeneca.com

Study Locations

• Russia
  • Kazan', Russia
    • Recruiting
    • Research Site
  • Moscow, Russia
    • Recruiting
    • Research Site
  • Novosibirsk, Russia
    • Recruiting
    • Research Site
  • Rostov-on-Don, Russia
    • Recruiting
    • Research Site
  • Saint Petersburg, Russia
    • Not yet recruiting
    • Research Site
  • Samara, Russia
    • Recruiting
    • Research Site

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: No

• Sampling Method: Non-Probability Sample

Study Population

Participants of both sexes aged 18 years and older with diagnosis of AChR-antibody positive generalized MG will be enrolled in various clinical institutions in Russia that provide treatment for gMG patients.

Description

Inclusion Criteria:

1. Adults (≥18 years) diagnosed with generalized MG positive for acetylcholine receptor (AChR) antibodies.
2. Provision of signed and dated written informed consent.

Exclusion Criteria:

1. Participants currently enrolled in clinical studies for treatment of gMG.
2. Ocular MG only.

Study Plan

How is the study designed?

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Age at baseline (years)	Patient age in full years at study enrollment (ICF signing). Summarized as a quantitative variable and as categorical distribution by ≤50 years vs >50 years.	Baseline (Month 0; at informed consent and enrollment)
Age at gMG diagnosis (years)	Patient age in full years at the date of generalized myasthenia gravis (gMG) diagnosis. Summarized as a quantitative variable and as categorical distribution by ≤50 years vs >50 years.	Baseline (retrospective abstraction at Month 0)
Sex and ethnicity distribution	Proportion of patients by sex (male, female) and ethnicity (White, Asian, Black, other) recorded at enrollment.	Baseline (Month 0)
Body Mass Index (BMI)	BMI in kg/m^2 calculated as weight (kg)/height (m)^2 using the most recent values closest to enrollment, summarized as mean, SD, median, IQR, minimum, maximum. Height and weight are abstracted from medical records.	Baseline (Month 0; last available measurements closest to ICF signing)
Disease duration (days)	Time between the date of documented gMG diagnosis and the date of informed consent/enrollment, summarized as median and IQR (and mean, SD, min, max). Calculated in months/years based on dates abstracted from medical records to the eCRF.	Baseline (Month 0)
Clinical symptom profile of gMG	Proportion of patients presenting each predefined gMG symptom category at enrollment (ocular, bulbar, respiratory, chewing/facial, limb/trunk/neck weakness, autonomic dysfunction subdomains); summarized as counts and percentages.	Baseline (Month 0)
Comorbidity profile	Proportion of patients with specified comorbidities in history or present at enrollment: autoimmune thyroiditis/hyperthyroidism, systemic lupus erythematosus, rheumatoid arthritis, autoimmune encephalitis, arterial hypertension, hyperlipidemia.	Baseline (Month 0)
Number and proportion of patients with gMG-related hospitalizations	Patients with ≥1 hospitalization attributed to gMG (investigator judgment) from date of gMG diagnosis to ICF signing.	Retrospective period from gMG diagnosis to Baseline (Month 0)
Duration of gMG-related hospitalizations (days)	Sum of days of gMG-related hospitalizations per patient from gMG diagnosis to ICF signing.	Retrospective period from gMG diagnosis to Baseline (Month 0)

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
MG-ADL total score and change from baseline	Patient-reported MG-ADL total score (range 0-24; higher=worse). Report descriptive statistics at each time point and change from Baseline (visit value minus Baseline). Scores are abstracted from routine care records; only total scores documented in source are collected.	Baseline (Month 0) and Months 6, 12, 18, 24, 30, 36 (±1 month)
QMG total score and change from baseline	Physician-reported QMG total score (0-39; higher=worse). Metrics: absolute score and change from baseline. Summarized descriptively. Collected only if present in routine practice records.	Baseline (Month 0) and Months 6, 12, 18, 24, 30, 36 (±1 month)
Distribution of MGFA clinical classification over time	Proportion of participants in each MGFA class (I, IIa/IIb, IIIa/IIIb, IVa/IVb, V) at each time point. Also report proportion improving (lower class) or worsening (higher class) versus Baseline. MGFA class is abstracted only if recorded in routine practice.	Baseline (Month 0) and Months 6, 12, 18, 24, 30, 36 (±1 month)
Number and proportion of patients with all-cause hospitalization	Patients experiencing ≥1 hospitalization for any cause during prospective follow-up. Metric: count and proportion (percentage) of patients with ≥1 event. Each participant's status (yes/no) assessed across the follow-up period.	From Baseline through Months 6, 12, 18, 24, 30, 36 (±1 month)
Duration of all-cause hospitalizations (days per participant)	Total days hospitalized for any cause per participant during prospective follow-up. Metric: sum of hospital-day durations per participant; summarized descriptively.	From Baseline through Months 6, 12, 18, 24, 30, 36 (±1 month)
Number and proportion of patients with gMG-related hospitalization	Patients with ≥1 hospitalization attributed to gMG during follow-up. Categorize reasons: related to MG exacerbation (myasthenic crisis) vs not related (thymectomy, investigation, social reason, other). Record ICU admission for gMG-related hospitalizations (yes/no). Report count and proportion (percentage) of affected patients overall and by categories.	From Baseline through Months 6, 12, 18, 24, 30, 36 (±1 month)
Duration of gMG-related hospitalizations (days per participant)	Total days hospitalized for gMG-related reasons per participant during prospective follow-up. Metric: sum of hospital-day durations for gMG-related stays; summarized descriptively.	From Baseline through Months 6, 12, 18, 24, 30, 36 (±1 month)
Time from MG symptom onset to gMG diagnosis (months)	Interval between the date of first MG symptoms/suspicion (if known) and the date of generalized MG (gMG) diagnosis. Metric: duration in months (or days if available); summarized descriptively. Evaluated only for patients with a documented onset date.	Retrospective period prior to Baseline
Prior misdiagnoses before gMG diagnosis (number and proportion by category)	Proportion of participants with ≥1 prior misdiagnosis and distribution by category (myopathies, polyneuropathies, Lambert-Eaton, mitochondrial encephalomyopathies, congenital myasthenic syndromes, cerebrovascular accidents, brainstem encephalitis, brain tumors, ALS).	Retrospective period prior to Baseline
Diagnostic examinations for gMG confirmation and available results	Proportion with each examination and latest available result: AChR-Ab (required positive), CT/MRI chest/mediastinum, brain/spinal MRI, ENMG, single-fiber EMG, RNS. Report counts, percentages (95% CI), and result categories if available.	Retrospective period prior to Baseline
Diagnostic test usage and results	Proportion with each test performed and latest result (positive/negative/unknown) as documented in routine care; report counts and percentages with 95% CI.	Retrospective period prior to Baseline
Drug therapies received (proportion by class)	Proportion of patients receiving each therapy class: anticholinesterase (neostigmine), IVIG, corticosteroids (prednisolone/methylprednisolone), complement inhibitors (ravulizumab/eculizumab), diuretics, potassium, immunosuppressants (e.g., azathioprine, mycophenolate, cyclosporine), rituximab. Summarized as counts/percentages by class.	12 months pre-Baseline and Baseline to Month 36 (±1 month)
Duration of drug therapies (days per participant)	For each therapy class received, total days on therapy per participant during the assessment window; summarized descriptively.	12 months pre-Baseline and Baseline to Month 36 (±1 month)
Mean daily corticosteroid dose (prednisolone equivalent)	Among CS users, average daily dose recorded in mg; convert methylprednisolone to prednisolone equivalent for analysis. Summarized with descriptive statistics.	12 months pre-Baseline and Baseline to Month 36 (±1 month)
Plasmapheresis/plasma exchange/plasmafiltration use (proportion)	Proportion of patients receiving plasmapheresis, plasma exchange, or plasmafiltration for gMG; summarized as counts and percentages.	12 months pre-Baseline and Baseline to Month 36. (±1 month)
Duration of plasmapheresis/plasma exchange/plasmafiltration (days)	Total treatment days per participant for plasmapheresis, plasma exchange, or plasmafiltration within the assessment window; summarized descriptively.	12 months pre-Baseline and Baseline to Month 36 (±1 month)
Thymectomy performed	Proportion of patients who underwent thymectomy for gMG; summarized as counts and percentages.	12 months pre-Baseline and Baseline to Month 36 (±1 month)
Invasive lung ventilation required	Proportion of patients requiring invasive mechanical ventilation for gMG; summarized as counts and percentages.	12 months pre-Baseline and Baseline to Month 36 (±1 month)
First-line treatment regimen at gMG diagnosis	Distribution of initial treatment components at diagnosis (same therapy categories as above); summarized as counts and percentages by regimen.	At gMG diagnosis (retrospective abstraction at Baseline)
Myasthenic crisis in prior 12 months	Proportion with ≥1 crisis and distribution by number of episodes (1, 2, ≥3) during the 12 months prior to enrollment; counts and percentages from medical records.	12 months prior to Baseline (Month 0)
Myasthenic crisis during follow-up	Proportion with ≥1 crisis and distribution by number of episodes (1, 2, ≥3) during prospective follow-up; counts and percentages from medical records.	From Baseline to Month 36 (±1 month)
Crisis precipitants-physical stressors	Proportion of crisis episodes associated with predefined physical precipitants: aspiration pneumonitis, infection, perimenstrual state, pregnancy, sleep deprivation, surgery, environmental stressors, emotional stress, pain, temperature extremes, tapering of immune-modulating medications.	12 months pre-Baseline and Baseline to Month 36 (±1 month)
Crisis precipitants-medications	Proportion of crisis episodes associated with predefined medications: α-interferon; antibiotics (e.g., aminoglycosides, macrolides, quinolones, polymyxin); antiepileptics (e.g., gabapentin, phenytoin, carbamazepine, high-dose benzodiazepines); β-blockers; calcium channel antagonists; contrast media; magnesium; hormonal drugs (e.g., corticosteroids, ACTH, oral contraceptives, oxytocin, thyroid hormones); antithyroid agents; antiarrhythmics (e.g., procainamide, quinidine).	12 months pre-Baseline and Baseline to Month 36 (±1 month)
Crisis outcomes by MGFA class change	Change in MGFA class from the most recent pre-crisis assessment to the nearest post-crisis scheduled assessment; proportion improved, unchanged, or worsened.	Months 6, 12, 18, 24, 30, 36 (±1 month)
Initiation of complement C5 inhibitor therapy (C5IT)	Proportion of participants who initiated C5IT during observation; counts and percentages; with start/stop dates abstracted from records.	From gMG diagnosis to Month 36 (±1 month)
Meningococcal vaccination prior to C5IT initiation	Among those who initiated C5IT, proportion with documented meningococcal vaccination before first dose; time from gMG diagnosis to first vaccination; record revaccinations. Report counts, percentages, and time-to-vaccination summary.	From gMG diagnosis to C5IT start (vaccination events abstracted during follow-up)
Prophylactic antibiotic use prior to C5IT initiation	Among those who initiated C5IT, proportion with documented prophylactic antibiotic use prior to first dose; time from gMG diagnosis to first prophylactic use; record repeated prophylaxis. Report counts, percentages, and time-to-prophylaxis summary.	From gMG diagnosis to C5IT start (antibiotic events abstracted during follow-up)

Collaborators and Investigators

• Sponsor: AstraZeneca

Study record dates

Study Major Dates

• Study Start (Actual): December 23, 2025
• Primary Completion (Estimated): December 31, 2029
• Study Completion (Estimated): December 31, 2029

Study Registration Dates

• First Submitted: November 18, 2025
• First Submitted That Met QC Criteria: November 18, 2025
• First Posted (Actual): November 25, 2025

Study Record Updates

• Last Update Posted (Actual): May 13, 2026
• Last Update Submitted That Met QC Criteria: May 12, 2026
• Last Verified: May 1, 2026

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Nervous System Diseases; Pathologic Processes; Neoplasms by Site; Neoplasms; Neuromuscular Diseases; Disease Attributes; Autoimmune Diseases; Immune System Diseases; Autoimmune Diseases of the Nervous System; Neurodegenerative Diseases; Paraneoplastic Syndromes, Nervous System; Nervous System Neoplasms; Paraneoplastic Syndromes; Neuromuscular Junction Diseases; Pathological Conditions, Signs and Symptoms; Rare Diseases; Myasthenia Gravis
• Other Study ID Numbers: D9281R00003

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: YES
• IPD Plan Description: Qualified researchers can request access to anonymized individual patient-level data from AstraZeneca group of companies sponsored clinical trials via the request portal Vivli.org. All requests will be evaluated as per the AZ disclosure commitment: https://astrazenecagrouptrials.pharmacm.com/ST/Submission/Disclosure. Yes, indicates that AZ are accepting requests for IPD, but this does not mean all requests will be shared
• IPD Sharing Time Frame: AstraZeneca will meet or exceed data availability as per the commitments made to the EFPIA PhRMA Data Sharing Principles. For details of our timelines, please rerefer to our disclosure commitment at https://astrazenecagrouptrials.pharmacm.com/ST/Submission/Disclosure.
• IPD Sharing Access Criteria: When a request has been approved AstraZeneca will provide access to the anonymized individual patient-level data via secure research environment Vivli.org. Signed Data Usage Agreement (non-negotiable contract for data accessors) must be in place before accessing requested information.