Clinical trial NCT06909253: Ravulizumab Treatment Outcomes in Patients With Generalized Myasthenia Gravis
Official title: Ravulizumab Treatment Outcomes in Patients With Generalized Myasthenia Gravis (gMG) Naive to Complement Inhibitors. Prospective, Multicenter, Non-interventional Study (MG-ARCADIA).
Study type: OBSERVATIONAL
Overall status: RECRUITING
Conditions: Myasthenia Gravis, Generalized
Keywords: Ravulizumab
Generalized Myasthenia Gravis
gMG
observational
Lead sponsor: AstraZeneca
Sponsor class: INDUSTRY
First posted: 2025-04-03
Enrollment: 50
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Adult (aged ≥18 years) patients with gMG, naive to complement inhibitors, receiving ravulizumab treatment within the frames of NDP in Poland.

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).

Eligibility criteria: 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.

Primary outcomes:
measure: Change from baseline in Myasthenia Gravis-Activities of Daily Living (MG-ADL) total score
timeFrame: at 6 month
Secondary outcomes:
measure: Change from baseline in Myasthenia Gravis-Activities of Daily Living (MG-ADL) total score
timeFrame: at month 12, 24, 36
measure: Change from baseline in MG-ADL subcomponent scores
timeFrame: at month 6, 12, 24, 36
measure: Change from baseline in Myasthenia Gravis (MG) composite scale
timeFrame: at month 6, 12, 24, 36
measure: Proportion of patients who achieved response
timeFrame: at month 6, 12, 24, 36
measure: Change in the Revised 15-Component Myasthenia Gravis Quality of Life (MG-QOL15r) score
timeFrame: at month 6, 12, 24, 36
measure: Change from baseline in Work Productivity and Activity Impairment: Specific Health Problem (WPAI:SHP)
timeFrame: at month 6, 12, 24, 36
measure: Change from baseline in Patient Global Impression of Change (PGI-C)
timeFrame: at month 6, 12, 24, 36
measure: Change from baseline in Neuro-Qol Short Form v1.0 - Fatigue
timeFrame: at month 6, 12, 24, 36
measure: Change from baseline in Patient Health Questionnaire - 9 (PHQ-9)
timeFrame: at month 6, 12, 24, 36
Other outcomes:
Locations:
city: Bydgoszcz
country: Poland
facility: Research Site
city: Gdansk
country: Poland
facility: Research Site
city: Katowice
country: Poland
facility: Research Site
city: Krakow
country: Poland
facility: Research Site
city: Lublin
country: Poland
facility: Research Site
city: Poznan
country: Poland
facility: Research Site
city: Szczecin
country: Poland
facility: Research Site
city: Warsaw
country: Poland
facility: Research Site
city: Wroclaw
country: Poland
facility: Research Site
Study officials:
References: