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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 opdateret af: 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

Studieoversigt

Status

Rekruttering

Betingelser

Detaljeret 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.

Undersøgelsestype

Observationel

Tilmelding (Anslået)

100

Kontakter og lokationer

Dette afsnit indeholder kontaktoplysninger for dem, der udfører undersøgelsen, og oplysninger om, hvor denne undersøgelse udføres.

Studiekontakt

Studiesteder

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

Deltagelseskriterier

Forskere leder efter personer, der passer til en bestemt beskrivelse, kaldet berettigelseskriterier. Nogle eksempler på disse kriterier er en persons generelle helbredstilstand eller tidligere behandlinger.

Berettigelseskriterier

Aldre berettiget til at studere

  • Voksen
  • Ældre voksen

Tager imod sunde frivillige

Ingen

Prøveudtagningsmetode

Ikke-sandsynlighedsprøve

Studiebefolkning

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

Dette afsnit indeholder detaljer om studieplanen, herunder hvordan undersøgelsen er designet, og hvad undersøgelsen måler.

Hvordan er undersøgelsen tilrettelagt?

Design detaljer

Hvad måler undersøgelsen?

Primære resultatmål

Resultatmål
Foranstaltningsbeskrivelse
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
Foranstaltningsbeskrivelse
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

Samarbejdspartnere og efterforskere

Det er her, du vil finde personer og organisationer, der er involveret i denne undersøgelse.

Sponsor

argenx

Datoer for undersøgelser

Disse datoer sporer fremskridtene for indsendelser af undersøgelsesrekord og resumeresultater til ClinicalTrials.gov. Studieregistreringer og rapporterede resultater gennemgås af National Library of Medicine (NLM) for at sikre, at de opfylder specifikke kvalitetskontrolstandarder, før de offentliggøres på den offentlige hjemmeside.

Studer store datoer

Studiestart (Faktiske)

25. februar 2026

Primær færdiggørelse (Anslået)

1. november 2027

Studieafslutning (Anslået)

1. juli 2028

Datoer for studieregistrering

Først indsendt

9. marts 2026

Først indsendt, der opfyldte QC-kriterier

12. maj 2026

Først opslået (Faktiske)

19. maj 2026

Opdateringer af undersøgelsesjournaler

Sidste opdatering sendt (Faktiske)

19. maj 2026

Sidste opdatering indsendt, der opfyldte kvalitetskontrolkriterier

12. maj 2026

Sidst verificeret

1. maj 2026

Mere information

Begreber relateret til denne undersøgelse

Yderligere relevante MeSH-vilkår

Andre undersøgelses-id-numre

  • ARGX-113-NIS-2411-ITA

Plan for individuelle deltagerdata (IPD)

Planlægger du at dele individuelle deltagerdata (IPD)?

INGEN

Lægemiddel- og udstyrsoplysninger, undersøgelsesdokumenter

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Ingen

Studerer et amerikansk FDA-reguleret enhedsprodukt

Ingen

produkt fremstillet i og eksporteret fra U.S.A.

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Disse oplysninger blev hentet direkte fra webstedet clinicaltrials.gov uden ændringer. Hvis du har nogen anmodninger om at ændre, fjerne eller opdatere dine undersøgelsesoplysninger, bedes du kontakte register@clinicaltrials.gov. Så snart en ændring er implementeret på clinicaltrials.gov, vil denne også blive opdateret automatisk på vores hjemmeside .

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