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Recruiting NCT06909253

Ravulizumab Treatment Outcomes in Patients With Generalized Myasthenia Gravis

Observational Myasthenia Gravis, Generalized

For patients and families

In plain language

An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.

What is being studied
The protocol lists: Ravulizumab.
Who it may be relevant to
Registry conditions: Myasthenia Gravis, Generalized. Basic parameters: from 18 years · All.
What needs checking
Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
Where it takes place
Poland
Next step
Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
Official title

Ravulizumab Treatment Outcomes in Patients With Generalized Myasthenia Gravis (gMG) Naive to Complement Inhibitors. Prospective, Multicenter, Non-interventional Study (MG-ARCADIA).

Overview

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

  • Drug Ravulizumab
    Ravulizumab, concentrate for solution for infusion

Primary outcome measures

  • Change from baseline in Myasthenia Gravis-Activities of Daily Living (MG-ADL) total score [Time frame: at 6 month]
Secondary outcome measures (9)
  • Change from baseline in Myasthenia Gravis-Activities of Daily Living (MG-ADL) total score [Time frame: at month 12, 24, 36]
  • Change from baseline in MG-ADL subcomponent scores [Time frame: at month 6, 12, 24, 36]
  • Change from baseline in Myasthenia Gravis (MG) composite scale [Time frame: at month 6, 12, 24, 36]
  • Proportion of patients who achieved response [Time frame: at month 6, 12, 24, 36]
  • Change in the Revised 15-Component Myasthenia Gravis Quality of Life (MG-QOL15r) score [Time frame: at month 6, 12, 24, 36]
  • Change from baseline in Work Productivity and Activity Impairment: Specific Health Problem (WPAI:SHP) [Time frame: at month 6, 12, 24, 36]
  • Change from baseline in Patient Global Impression of Change (PGI-C) [Time frame: at month 6, 12, 24, 36]
  • Change from baseline in Neuro-Qol Short Form v1.0 - Fatigue [Time frame: at month 6, 12, 24, 36]
  • Change from baseline in Patient Health Questionnaire - 9 (PHQ-9) [Time frame: at month 6, 12, 24, 36]

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.

Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.

Healthy volunteers: No

Study design

Observational model
Cohort

Study locations

Poland · 12 centers
  • Research Site — Bydgoszcz
  • Research Site — Gdansk
  • Research Site — Katowice
  • Research Site — Krakow
  • Research Site — Lodz
  • Research Site — Lublin
  • Research Site — Poznan
  • Research Site — Poznan
  • … and 4 more centers

Identifiers

NCT: NCT06909253 · D9281R00004

Primary sources (government registries)

View this study on ClinicalTrials.gov ↗