Myasthenia Gravis, Generalized
Conditions
Keywords
Ravulizumab, Generalized Myasthenia Gravis, gMG, observational
Brief summary
The Polish multicentre observational (non-interventional) study aiming to collect data on the management and clinical outcomes of patients with gMG that received ravulizumab.
Detailed description
The Polish, prospective, multicenter, observational, cohort study designed to collect longitudinal data on the management and clinical outcomes of patients with gMG naive to complement inhibitors who received ravulizumab in the scope of routine clinical practice within the frames of National Drug Program (NDP) for up to 42 months (\ 6 months baseline and \ 36 months follow-up).
Interventions
DRUGRavulizumab
Ravulizumab, concentrate for solution for infusion
Sponsors
AstraZeneca
Study design
Observational model
COHORT
Time perspective
PROSPECTIVE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* Adult (aged ≥18 years) patients with gMG, naive to complement inhibitors, receiving ravulizumab treatment in the frames of NDP in Poland. * Patients willing to participate in the study and signed Informed Consent Form (ICF). * Vaccination against N. meningitidis
Exclusion criteria
* Those who plan to participate in gMG clinical trial on/after the date of first ravulizumab infusion through NDP. * Cognitive incapacity, unwillingness or language barriers precluding adequate understanding or cooperation.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Change from baseline in Myasthenia Gravis-Activities of Daily Living (MG-ADL) total score | at 6 month |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Change from baseline in Myasthenia Gravis-Activities of Daily Living (MG-ADL) total score | at month 12, 24, 36 | — |
| Change from baseline in MG-ADL subcomponent scores | at month 6, 12, 24, 36 | Subcomponent scores - bulbar, limbs, respiratory, and ocular |
| Change from baseline in Myasthenia Gravis (MG) composite scale | at month 6, 12, 24, 36 | — |
| Proportion of patients who achieved response | at month 6, 12, 24, 36 | Response - an improvement of ≥ 2 points in MG-ADL total score (proportion of patients with ≥ 3 improvement will be also presented) |
| Change in the Revised 15-Component Myasthenia Gravis Quality of Life (MG-QOL15r) score | at month 6, 12, 24, 36 | — |
| Change from baseline in Work Productivity and Activity Impairment: Specific Health Problem (WPAI:SHP) | at month 6, 12, 24, 36 | — |
| Change from baseline in Patient Global Impression of Change (PGI-C) | at month 6, 12, 24, 36 | — |
| Change from baseline in Neuro-Qol Short Form v1.0 - Fatigue | at month 6, 12, 24, 36 | — |
| Change from baseline in Patient Health Questionnaire - 9 (PHQ-9) | at month 6, 12, 24, 36 | — |
Countries
Poland
Contacts
CONTACTAstraZeneca Clinical Study Information Center
Outcome results
None listed