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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)

2026年5月12日 更新者：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

研究概览

地位

招聘中

条件

详细说明

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.

研究类型

观察性的

注册 (估计的)

100

联系人和位置

本节提供了进行研究的人员的详细联系信息，以及有关进行该研究的地点的信息。

学习联系方式

学习地点

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

参与标准

研究人员寻找符合特定描述的人，称为资格标准。这些标准的一些例子是一个人的一般健康状况或先前的治疗。

资格标准

适合学习的年龄

  • 成人
  • 年长者

接受健康志愿者

不

取样方法

非概率样本

研究人群

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.

描述

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.

学习计划

本节提供研究计划的详细信息，包括研究的设计方式和研究的衡量标准。

研究是如何设计的？

设计细节

研究衡量的是什么？

主要结果指标

结果测量
措施说明
大体时间
Changes in MG-ADL scores from baseline
大体时间：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
大体时间：Up to 12 months
Minimal Symptom Expression (MSE) is defined as an MG-ADL score ≤ 1
Up to 12 months

次要结果测量

结果测量
措施说明
大体时间
Changes in QMG scores over time
大体时间：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
大体时间：Up to 12 months
Up to 12 months
Changes in PROMIS Fatigue SF 4a scores over time
大体时间：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
大体时间：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
大体时间：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
大体时间：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
大体时间：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
大体时间：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
大体时间：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
大体时间：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

合作者和调查者

在这里您可以找到参与这项研究的人员和组织。

赞助

argenx

研究记录日期

这些日期跟踪向 ClinicalTrials.gov 提交研究记录和摘要结果的进度。研究记录和报告的结果由国家医学图书馆 (NLM) 审查，以确保它们在发布到公共网站之前符合特定的质量控制标准。

研究主要日期

学习开始 (实际的)

2026年2月25日

初级完成 (估计的)

2027年11月1日

研究完成 (估计的)

2028年7月1日

研究注册日期

首次提交

2026年3月9日

首先提交符合 QC 标准的

2026年5月12日

首次发布 (实际的)

2026年5月19日

研究记录更新

最后更新发布 (实际的)

2026年5月19日

上次提交的符合 QC 标准的更新

2026年5月12日

最后验证

2026年5月1日

更多信息

与本研究相关的术语

其他相关的 MeSH 术语

其他研究编号

  • ARGX-113-NIS-2411-ITA

计划个人参与者数据 (IPD)

计划共享个人参与者数据 (IPD)？

不

药物和器械信息、研究文件

研究美国 FDA 监管的药品

不

研究美国 FDA 监管的设备产品

不

在美国制造并从美国出口的产品

不

此信息直接从 clinicaltrials.gov 网站检索，没有任何更改。如果您有任何更改、删除或更新研究详细信息的请求，请联系 register@clinicaltrials.gov. clinicaltrials.gov 上实施更改，我们的网站上也会自动更新.

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