Идёт набор NCT07247279

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

— Наблюдательное Rare Diseases Generalized Myasthenia Gravis (gMG)

Ориентир для пациента и семьи

Простыми словами

Автоматическая сводка по структурированным данным реестра. Она помогает сориентироваться, но не заменяет официальный протокол или оценку врача.

Что изучают: Это наблюдательное исследование: исследуемое лечение участникам по протоколу не назначают.
Кому может быть актуально: Состояния в реестре: Rare Diseases, Generalized Myasthenia Gravis (gMG). Базовые параметры: от 18 лет · Все.
Что важно проверить: Возраст, диагноз и пол — только базовые ориентиры. Предыдущее лечение, анализы и другие обязательные условия указаны ниже в критериях участия.
Где проводится: Россия
Следующий шаг: Сохраните исследование, покажите его лечащему врачу и уточните актуальный статус у исследовательского центра. Расходы, документы и поездка →

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Официальное название

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

Обзор

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.

Подробное описание

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.

Первичные конечные точки

Age at baseline (years) [Срок оценки: Baseline (Month 0; at informed consent and enrollment)]
Age at gMG diagnosis (years) [Срок оценки: Baseline (retrospective abstraction at Month 0)]
Sex and ethnicity distribution [Срок оценки: Baseline (Month 0)]
Body Mass Index (BMI) [Срок оценки: Baseline (Month 0; last available measurements closest to ICF signing)]
Disease duration (days) [Срок оценки: Baseline (Month 0)]
Clinical symptom profile of gMG [Срок оценки: Baseline (Month 0)]
Comorbidity profile [Срок оценки: Baseline (Month 0)]
Number and proportion of patients with gMG-related hospitalizations [Срок оценки: Retrospective period from gMG diagnosis to Baseline (Month 0)]
Duration of gMG-related hospitalizations (days) [Срок оценки: Retrospective period from gMG diagnosis to Baseline (Month 0)]

Вторичные конечные точки (12)

MG-ADL total score and change from baseline [Срок оценки: Baseline (Month 0) and Months 6, 12, 18, 24, 30, 36 (±1 month)]
QMG total score and change from baseline [Срок оценки: Baseline (Month 0) and Months 6, 12, 18, 24, 30, 36 (±1 month)]
Distribution of MGFA clinical classification over time [Срок оценки: Baseline (Month 0) and Months 6, 12, 18, 24, 30, 36 (±1 month)]
Number and proportion of patients with all-cause hospitalization [Срок оценки: From Baseline through Months 6, 12, 18, 24, 30, 36 (±1 month)]
Duration of all-cause hospitalizations (days per participant) [Срок оценки: From Baseline through Months 6, 12, 18, 24, 30, 36 (±1 month)]
Number and proportion of patients with gMG-related hospitalization [Срок оценки: From Baseline through Months 6, 12, 18, 24, 30, 36 (±1 month)]
Duration of gMG-related hospitalizations (days per participant) [Срок оценки: From Baseline through Months 6, 12, 18, 24, 30, 36 (±1 month)]
Time from MG symptom onset to gMG diagnosis (months) [Срок оценки: Retrospective period prior to Baseline]
Prior misdiagnoses before gMG diagnosis (number and proportion by category) [Срок оценки: Retrospective period prior to Baseline]
Diagnostic examinations for gMG confirmation and available results [Срок оценки: Retrospective period prior to Baseline]
Diagnostic test usage and results [Срок оценки: Retrospective period prior to Baseline]
Drug therapies received (proportion by class) [Срок оценки: 12 months pre-Baseline and Baseline to Month 36 (±1 month)]

Критерии участия

Критерии включения

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