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

May 12, 2026 updated by: argenx

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

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

Study Overview

Status

Recruiting

Conditions

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.

Study Type

Observational

Enrollment (Estimated)

100

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Locations

  • Italy
      • Acquaviva delle Fonti, Italy, 70021
        • Recruiting
        • Ospedale Generale Regionale F. Miulli
        • Contact:
      • Ancona, Italy, 60126
        • Recruiting
        • Azienda Ospedaliero Universitaria delle Marche
        • Contact:
      • Bari, Italy, 70124
        • Recruiting
        • A.O.U. Consorziale Policlinico
        • Contact:
      • Bergamo, Italy, 24127
      • Bologna, Italy, 40139
        • Recruiting
        • Ospedale Bellaria
        • Contact:
      • Catanzaro, Italy, 88100
        • Recruiting
        • A.O.U. Renato Dulbecco
        • Contact:
      • Florence, Italy, 50134
      • Genova, Italy, 16132
        • Recruiting
        • IRCCS Ospedale Policlinico San Martino
        • Contact:
      • Messina, Italy, 98124
        • Recruiting
        • A.O.U. Policlinico G. Martino
        • Contact:
      • Milan, Italy, 20132
      • Milan, Italy, 20133
        • Recruiting
        • IRCCS Istituto Neurologico Carlo Besta
        • Contact:
      • Milan, Italy, 20162
      • Milan, Italy, 20122
        • Recruiting
        • IRCCS Cà Granda Ospedale Maggiore Policlinico
        • Contact:
      • Monserrato, Italy, 09042
        • Recruiting
        • A.O.U. Policlinico Universitario
        • Contact:
      • Naples, Italy, 80131
        • Recruiting
        • AORN A. Cardarelli
        • Contact:
      • Naples, Italy, 80138
        • Recruiting
        • A.O.U. Università della Campania L. Vanvitelli
        • Contact:
      • Orbassano, Italy, 10043
        • Recruiting
        • A.O.U. San Luigi Gonzaga
        • Contact:
      • Padova, Italy, 35128
        • Recruiting
        • Azienda Ospedale Universita Padova
        • Contact:
      • Pavia, Italy, 27100
        • Recruiting
        • IRCCS Fondazione Mondino
        • Contact:
      • Perugia, Italy, 06132
        • Recruiting
        • Azienda Ospedaliera Perugia
        • Contact:
      • Pisa, Italy, 56124
        • Recruiting
        • A.O.U. Pisana
        • Contact:
      • Rimini, Italy, 47923
        • Recruiting
        • Ospedale degli Infermi
        • Contact:
      • Roma, Italy, 00168
        • Recruiting
        • Fondazione Policlinico Universitario Agostino Gemelli IRCCS
        • Contact:
      • Roma, Italy, 00189
      • Roma, Italy, 00133
        • Recruiting
        • Fondazione PTV Policlinico Tor Vergata
        • Contact:
      • Salerno, Italy, 84131
        • Recruiting
        • A.O.U. San Giovanni di Dio e Ruggi D'Aragona
        • Contact:
      • San Giovanni Rotondo, Italy, 71013
        • Recruiting
        • IRCCS Fondazione Casa Sollievo della Sofferenza
        • Contact:
      • Sassari, Italy, 07100
      • Treviso, Italy, 31100
        • Recruiting
        • Ospedale Ca' Foncello
        • Contact:
      • Verona, Italy, 37134
        • Recruiting
        • AOUI Verona - Policlinico G.B. Rossi
        • Contact:

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Sampling Method

Non-Probability Sample

Study Population

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.

Description

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.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Changes in MG-ADL scores from baseline
Time Frame: Up to 12 months
The Myasthenia Gravis Activities of Living (MG-ADL) scale is an 8-item patient-reported scale that measures MG symptoms and functional status across 4 domains. The total score ranges from 0 to 24, with higher scores indicating greater disability.
Up to 12 months
Percentage of patients achieving MSE
Time Frame: Up to 12 months
Minimal Symptom Expression (MSE) is defined as an MG-ADL score ≤ 1
Up to 12 months

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Changes in QMG scores over time
Time Frame: Up to 12 months
The quantitative myasthenia gravis (QMG) score is a commonly used scale for evaluating muscle weakness associated with myasthenia gravis. It consists in a 13-item direct physician assessment scoring system that quantifies disease severity, based on impairments of body functions and structures. The total QMG score ranges from 0 to 39, where higher scores indicated greater disease severity.
Up to 12 months
Changes in MG-QoL-15r scores over time
Time Frame: Up to 12 months
Up to 12 months
Changes in PROMIS Fatigue SF 4a scores over time
Time Frame: Up to 12 months
The PROMIS Fatigue Short Form 4a is a four-item questionnaire used to assess the experience of fatigue over the past seven days. The items are designed to capture different aspects of fatigue, including the impact of fatigue on physical and mental activities. The raw score can range from 4 to 20, with higher scores indicating more severe fatigue.
Up to 12 months
Changes in FSS over time
Time Frame: Up to 12 months
The Fatigue Severity Scale (FSS) is a self-administered questionnaire with 9 items (questions) investigating the severity of fatigue in different situations during the past week. The final score ranges from 1 to 7.
Up to 12 months
Changes in HADS-Anxiety scores over time
Time Frame: Up to 12 months
The Hospital Anxiety and Depression Scale (HADS) is a widely used screening tool that helps to identify possible or probable instances of anxiety and depression among patients in non-psychiatric hospital settings. It consists of 14 items, with 7 items related to anxiety (HADS-A) and 7 related to depression (HADS-D), collecting how the patient was feeling in the previous week. The score for each subscale ranges from 0 to 21.
Up to 12 months
Changes in HADS-Depression scores over time
Time Frame: Up to 12 months
The Hospital Anxiety and Depression Scale (HADS) is a widely used screening tool that helps to identify possible or probable instances of anxiety and depression among patients in non-psychiatric hospital settings. It consists of 14 items, with 7 items related to anxiety (HADS-A) and 7 related to depression (HADS-D), collecting how the patient was feeling in the previous week. The score for each subscale ranges from 0 to 21.
Up to 12 months
Changes in PSQI scores over time
Time Frame: Up to 12 months
The Pittsburgh Sleep Quality Index (PSQI) is a self-rated questionnaire which assesses sleep quality and disturbances over a 1-month time interval. Nineteen individual items generate seven "component" scores: subjective sleep quality, sleep latency, sleep duration, habitual sleep efficiency, sleep disturbances, use of sleeping medication, and daytime dysfunction. The sum of scores for these seven components yields one global score, ranging from 0 to 21, with higher scores indicating worse sleep quality.
Up to 12 months
TANDEM - patient version scores over time
Time Frame: Up to 12 months
The Treatment Assessment in Dual Evaluation Model (TANDEM) - patient version is a non-validated, exploratory PRO designed to assess patient satisfaction with efgartigimod alfa treatment and symptom control in MG. It includes 8 items on therapeutic alignment, treatment experience, and symptom management.
Up to 12 months
TANDEM - clinician version scores over time
Time Frame: Up to 12 months
The Treatment Assessment in Dual Evaluation Model (TANDEM) - clinician version is a non-validated, exploratory PRO designed to assess clinician satisfaction with the management of efgartigimod alfa treatment and symptom control in patients with MG. It includes 8 items on therapeutic personalization, treatment effectiveness, and symptom stability.
Up to 12 months
CBI scores over time
Time Frame: Up to 12 months
The Caregiver Burden Inventory (CBI) is a 24-item questionnaire designed to assess the following different aspects of caregiver burden. Scores range from 0 to 96, with higher scores indicating greater feelings of burden.
Up to 12 months

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

argenx

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

February 25, 2026

Primary Completion (Estimated)

November 1, 2027

Study Completion (Estimated)

July 1, 2028

Study Registration Dates

First Submitted

March 9, 2026

First Submitted That Met QC Criteria

May 12, 2026

First Posted (Actual)

May 19, 2026

Study Record Updates

Last Update Posted (Actual)

May 19, 2026

Last Update Submitted That Met QC Criteria

May 12, 2026

Last Verified

May 1, 2026

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • ARGX-113-NIS-2411-ITA

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

product manufactured in and exported from the U.S.

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

Clinical Trials on gMG

Search Similar Trials

Sponsors and Collaborators

Medical Conditions

Drug Interventions

CROs by country

CROs in Colombia

Conditions

Rare Diseases

Drug Interventions

Dietary Supplements

Sponsor/Collaborators

Locations

Subscribe