An observational study in Rare Diseases and Generalized Myasthenia Gravis (gMG), sponsored by AstraZeneca. Recruiting at 7 sites in Russia. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2026-10-14.
Sponsored by AstraZeneca · Observational
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.
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.
592 studies on the registry are indexed under Rare Diseases; 125 are open to participants now.
This study's planned enrollment of 450 is above the median of 133 across 208 observational studies indexed under Rare Diseases.
Browse Rare Diseases studies →AstraZeneca is the lead sponsor of 3,499 studies on the registry; 270 are open to participants now.
Of its 368 completed or terminated interventional studies of FDA-regulated products, 178 (48%) have results posted.
Counted across the registry records on this site, refreshed daily.
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.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Exclusion Criteria:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
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.
Time frame: 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.
Time frame: 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.
Time frame: 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.
Time frame: 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.
Time frame: 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.
Time frame: 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.
Time frame: 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.
Time frame: 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.
Time frame: Retrospective period from gMG diagnosis to Baseline (Month 0)
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.
Time frame: 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.
Time frame: 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.
Time frame: 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.
Time frame: 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.
Time frame: 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.
Time frame: 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.
Time frame: 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.
Time frame: 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).
Time frame: 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.
Time frame: 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.
Time frame: 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.
Time frame: 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.
Time frame: 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.
Time frame: 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.
Time frame: 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.
Time frame: 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.
Time frame: 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.
Time frame: 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.
Time frame: 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.
Time frame: 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.
Time frame: 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.
Time frame: 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).
Time frame: 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.
Time frame: 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.
Time frame: 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.
Time frame: 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.
Time frame: From gMG diagnosis to C5IT start (antibiotic events abstracted during follow-up)
Plan to share: Yes — 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
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.
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.
Data sharing details ↗ · vivli.org
No publications or documents are linked to this record.
Started Dec 2025 (actual). Primary completion is estimated for Dec 2029, 48 months after the start.
Of 88 observational studies under Rare Diseases, half reached primary completion within 118 months of starting (the median), counting those still running; a quarter within 39 and three quarters within 183. This study's planned 48 months is below that.
Rare Diseases covers many different conditions, not one, so the studies compared here vary widely. Read these figures as a rough guide.
How these figures are worked out, and which studies are left out of them.
From the registry record's own update history. This site started tracking changes on Sep 25, 2026; for anything earlier, see the record history on ClinicalTrials.gov ↗
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