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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. mai 2026 oppdatert 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

Studieoversikt

Status

Rekruttering

Forhold

Detaljert beskrivelse

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.

Studietype

Observasjonsmessig

Registrering (Antatt)

100

Kontakter og plasseringer

Denne delen inneholder kontaktinformasjon for de som utfører studien, og informasjon om hvor denne studien blir utført.

Studiekontakt

Studiesteder

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

Deltakelseskriterier

Forskere ser etter personer som passer til en bestemt beskrivelse, kalt kvalifikasjonskriterier. Noen eksempler på disse kriteriene er en persons generelle helsetilstand eller tidligere behandlinger.

Kvalifikasjonskriterier

Alder som er kvalifisert for studier

  • Voksen
  • Eldre voksen

Tar imot friske frivillige

Nei

Prøvetakingsmetode

Ikke-sannsynlighetsprøve

Studiepopulasjon

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.

Beskrivelse

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

Denne delen gir detaljer om studieplanen, inkludert hvordan studien er utformet og hva studien måler.

Hvordan er studiet utformet?

Designdetaljer

Hva måler studien?

Primære resultatmål

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

Sekundære resultatmål

Resultatmål
Tiltaksbeskrivelse
Tidsramme
Changes in QMG scores over time
Tidsramme: 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
Tidsramme: Up to 12 months
Up to 12 months
Changes in PROMIS Fatigue SF 4a scores over time
Tidsramme: 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
Tidsramme: 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
Tidsramme: 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
Tidsramme: 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
Tidsramme: 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
Tidsramme: 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
Tidsramme: 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
Tidsramme: 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

Samarbeidspartnere og etterforskere

Det er her du vil finne personer og organisasjoner som er involvert i denne studien.

Sponsor

argenx

Studierekorddatoer

Disse datoene sporer fremdriften for innsending av studieposter og sammendragsresultater til ClinicalTrials.gov. Studieposter og rapporterte resultater gjennomgås av National Library of Medicine (NLM) for å sikre at de oppfyller spesifikke kvalitetskontrollstandarder før de legges ut på det offentlige nettstedet.

Studer hoveddatoer

Studiestart (Faktiske)

25. februar 2026

Primær fullføring (Antatt)

1. november 2027

Studiet fullført (Antatt)

1. juli 2028

Datoer for studieregistrering

Først innsendt

9. mars 2026

Først innsendt som oppfylte QC-kriteriene

12. mai 2026

Først lagt ut (Faktiske)

19. mai 2026

Oppdateringer av studieposter

Sist oppdatering lagt ut (Faktiske)

19. mai 2026

Siste oppdatering sendt inn som oppfylte QC-kriteriene

12. mai 2026

Sist bekreftet

1. mai 2026

Mer informasjon

Begreper knyttet til denne studien

Ytterligere relevante MeSH-vilkår

Andre studie-ID-numre

  • ARGX-113-NIS-2411-ITA

Plan for individuelle deltakerdata (IPD)

Planlegger du å dele individuelle deltakerdata (IPD)?

NEI

Legemiddel- og utstyrsinformasjon, studiedokumenter

Studerer et amerikansk FDA-regulert medikamentprodukt

Nei

Studerer et amerikansk FDA-regulert enhetsprodukt

Nei

produkt produsert i og eksportert fra USA

Nei

Denne informasjonen ble hentet direkte fra nettstedet clinicaltrials.gov uten noen endringer. Hvis du har noen forespørsler om å endre, fjerne eller oppdatere studiedetaljene dine, vennligst kontakt register@clinicaltrials.gov. Så snart en endring er implementert på clinicaltrials.gov, vil denne også bli oppdatert automatisk på nettstedet vårt. .

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