Ravulizumab Treatment Outcomes in Patients With Generalized Myasthenia Gravis

Recruiting

• Conditions: Myasthenia Gravis, Generalized
• Interventions: Drug: Ravulizumab

• Registration Number: NCT06909253
• Lead Sponsor: AstraZeneca

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).

Study Timeline

• Study First Submitted: 2025-03-27
• Study First Submitted QC: 2025-03-27
• Study First Posted: 2025-04-03
• Status Verified: 2025-05
• Study Start: 2025-05-15
• Last Update Submitted: 2025-05-20
• Last Update Posted: 2025-05-21
• Primary Completion: 2029-12-31
• Study Completion: 2029-12-31

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 50

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.

Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Arm && Interventions

Group	Intervention	Description
Ravulizumab	Ravulizumab	Open-lable arm with gMG patients who received ravulizumab treatment in the frames of NDP in Poland.

Primary Outcome Measures

Name	Time	Method
Change from baseline in Myasthenia Gravis-Activities of Daily Living (MG-ADL) total score	at 6 month

Secondary Outcome Measures

Name	Time	Method
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

Trial Locations

Locations (1)

Research Site; 🇵🇱 Wroclaw, Poland