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

12 maj 2026 uppdaterad av: 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

Studieöversikt

Status

Rekrytering

Betingelser

Detaljerad beskrivning

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.

Studietyp

Observationell

Inskrivning (Beräknad)

100

Kontakter och platser

Det här avsnittet innehåller kontaktuppgifter för dem som genomför studien och information om var denna studie genomförs.

Studiekontakt

Studieorter

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

Deltagandekriterier

Forskare letar efter personer som passar en viss beskrivning, så kallade behörighetskriterier. Några exempel på dessa kriterier är en persons allmänna hälsotillstånd eller tidigare behandlingar.

Urvalskriterier

Åldrar som är berättigade till studier

  • Vuxen
  • Äldre vuxen

Tar emot friska volontärer

Nej

Testmetod

Icke-sannolikhetsprov

Studera befolkning

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.

Beskrivning

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.

Studieplan

Det här avsnittet ger detaljer om studieplanen, inklusive hur studien är utformad och vad studien mäter.

Hur är studien utformad?

Designdetaljer

Vad mäter studien?

Primära resultatmått

Resultatmått
Åtgärdsbeskrivning
Tidsram
Changes in MG-ADL scores from baseline
Tidsram: 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
Tidsram: Up to 12 months
Minimal Symptom Expression (MSE) is defined as an MG-ADL score ≤ 1
Up to 12 months

Sekundära resultatmått

Resultatmått
Åtgärdsbeskrivning
Tidsram
Changes in QMG scores over time
Tidsram: 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
Tidsram: Up to 12 months
Up to 12 months
Changes in PROMIS Fatigue SF 4a scores over time
Tidsram: 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
Tidsram: 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
Tidsram: 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
Tidsram: 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
Tidsram: 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
Tidsram: 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
Tidsram: 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
Tidsram: 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

Samarbetspartners och utredare

Det är här du hittar personer och organisationer som är involverade i denna studie.

Sponsor

argenx

Studieavstämningsdatum

Dessa datum spårar framstegen för inlämningar av studieposter och sammanfattande resultat till ClinicalTrials.gov. Studieposter och rapporterade resultat granskas av National Library of Medicine (NLM) för att säkerställa att de uppfyller specifika kvalitetskontrollstandarder innan de publiceras på den offentliga webbplatsen.

Studera stora datum

Studiestart (Faktisk)

25 februari 2026

Primärt slutförande (Beräknad)

1 november 2027

Avslutad studie (Beräknad)

1 juli 2028

Studieregistreringsdatum

Först inskickad

9 mars 2026

Först inskickad som uppfyllde QC-kriterierna

12 maj 2026

Första postat (Faktisk)

19 maj 2026

Uppdateringar av studier

Senaste uppdatering publicerad (Faktisk)

19 maj 2026

Senaste inskickade uppdateringen som uppfyllde QC-kriterierna

12 maj 2026

Senast verifierad

1 maj 2026

Mer information

Termer relaterade till denna studie

Ytterligare relevanta MeSH-villkor

Andra studie-ID-nummer

  • ARGX-113-NIS-2411-ITA

Plan för individuella deltagardata (IPD)

Planerar du att dela individuella deltagardata (IPD)?

NEJ

Läkemedels- och apparatinformation, studiedokument

Studerar en amerikansk FDA-reglerad läkemedelsprodukt

Nej

Studerar en amerikansk FDA-reglerad produktprodukt

Nej

produkt tillverkad i och exporterad från U.S.A.

Nej

Denna information hämtades direkt från webbplatsen clinicaltrials.gov utan några ändringar. Om du har några önskemål om att ändra, ta bort eller uppdatera dina studieuppgifter, vänligen kontakta register@clinicaltrials.gov. Så snart en ändring har implementerats på clinicaltrials.gov, kommer denna att uppdateras automatiskt även på vår webbplats .

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