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Recruiting NCT07595653

A Study to Evaluate the Impact of Efgartigimod on Overall Disease Experience of People Suffering From Generalized Myasthenia Gravis (gMG) in Italy

— Observational Generalized Myasthenia Gravis (gMG) gMG

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
This is an observational study: the protocol does not assign a study treatment.
Who it may be relevant to
Registry conditions: Generalized Myasthenia Gravis (gMG), gMG. 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
Italy
Next step
Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →

Unsure about the terms? Read our patient guide →

Official title

Non-interventional, Longitudinal Hybrid Data Sources Study to Evaluate the Impact of Efgartigimod on Overall Disease Experience of People Suffering From Generalized Myasthenia Gravis (gMG) in Italy

Overview

This study aims to generate real world evidence (RWE) from Italian clinical practice on the impact of efgartigimod alfa in gMG patients encompassing clinical outcomes and patient reported experiences. The study population will consist in adult patients with a documented diagnosis of gMG who are AChR-antibody positive and for whom the decision of treatment with efgartigimod alfa for gMG has been made independently of study participation as part of routine clinical care. The total study duration will be up to 23 months

Detailed description

Primary data collection will be performed during the baseline visit and follow-up will be at month 6 and 12 after baseline.

Secondary data collection from medical charts, information on myasthenia gravis (MG) related therapies (including rescue therapies), MG exacerbations, and MG-related hospitalizations will be collected for the 12 months preceding baseline. Additionally, the patient's MG history and overall medical history will be documented.

Primary outcome measures

  • Changes in MG-ADL scores from baseline [Time frame: Up to 12 months]
  • Percentage of patients achieving MSE [Time frame: Up to 12 months]
Secondary outcome measures (10)
  • Changes in QMG scores over time [Time frame: Up to 12 months]
  • Changes in MG-QoL-15r scores over time [Time frame: Up to 12 months]
  • Changes in PROMIS Fatigue SF 4a scores over time [Time frame: Up to 12 months]
  • Changes in FSS over time [Time frame: Up to 12 months]
  • Changes in HADS-Anxiety scores over time [Time frame: Up to 12 months]
  • Changes in HADS-Depression scores over time [Time frame: Up to 12 months]
  • Changes in PSQI scores over time [Time frame: Up to 12 months]
  • TANDEM - patient version scores over time [Time frame: Up to 12 months]
  • TANDEM - clinician version scores over time [Time frame: Up to 12 months]
  • CBI scores over time [Time frame: Up to 12 months]

Eligibility criteria

Inclusion criteria

  • At least 18 years old at signing of informed Consent Form (ICF) and privacy form (PF)
  • Documented diagnosis of gMG
  • AChR-antibody positive
  • The treating physician has decided to initiate efgartigimod alfa as part of routine clinical care and in accordance with product labelling, independently from the study

Exclusion criteria

  • Hypersensitivity to the active substance or to any of the excipients listed: sodium dihydrogen phosphate, monohydrate; disodium hydrogen phosphate, anhydrous; arginine hydrochloride; polysorbate 80, hyaluronidase, histidine, histidine hydrochloride monohydrate, methionine, polysorbate 20, sucrose.
  • Current or planned participation in an interventional clinical trial.

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

Italy · 30 centers
  • Ospedale Generale Regionale F. Miulli — Acquaviva delle Fonti
  • Azienda Ospedaliero Universitaria delle Marche — Ancona
  • A.O.U. Consorziale Policlinico — Bari
  • ASST Papa Giovanni XXIII — Bergamo
  • Ospedale Bellaria — Bologna
  • A.O.U. Renato Dulbecco — Catanzaro
  • A.O.U. Careggi — Florence
  • IRCCS Ospedale Policlinico San Martino — Genova
  • … and 22 more centers

Identifiers

NCT: NCT07595653 · ARGX-113-NIS-2411-ITA

Primary sources (government registries)

View this study on ClinicalTrials.gov ↗