Monitor the Evolution of Myasthenia Gravis Symptoms in Real-life in Patients With Anti-AChR and Anti-MUSK Generalised Myasthenia Gravis in Therapy With RYSTIGGO® (Rozanolixizumab) (REAL MG PRO)
An Observational, Prospective, Multicenter, Open-label Study to Monitor the Evolution of Myasthenia Gravis Symptoms in Real-life in Patients With Anti-AChR and Anti-MUSK Generalised Myasthenia Gravis in Therapy With RYSTIGGO® (Rozanolixizumab)
Generalized Myasthenia Gravis (gMG) is a rare autoimmune disease (a disease in which the body attacks its own tissues) that causes muscle weakness and significant fatigue.
Current treatments (corticosteroids, plasma exchange, intravenous immunoglobulin infusions) improve symptoms in many patients. However, many continue to suffer from fatigue and fatigability that are not well measured by standard tools. Moreover, these treatments can cause significant long-term side effects, reducing quality of life.
New treatments such as Rozanolixizumab (ROZ) are now available. They act rapidly and are well tolerated, allowing better symptom control while reducing the risks associated with conventional treatments.
To properly evaluate these new treatments, it is essential to understand patients' perspectives on their effectiveness. The scales used by physicians do not always capture all the symptoms experienced by patients, particularly fatigability. This is why a new tool has been developed: the MG symptoms PRO. This questionnaire allows patients to assess their own symptoms (fatigue, weakness of the eyes, mouth, breathing, muscle fatigability) in detail.
This research aims to better understand the effectiveness of treatments from the patients' perspective in order to improve their care.
The goal of the study is to evaluate the impact of Rozanolixizumab administration in real-world practice through the MG symptoms PRO questionnaire. This is an observational study, meaning that the medication is prescribed by the physician according to current regulations, and the study simply collects routine medical data during your follow-up, over a period of approximately 9 months.
研究概览
地位
条件
研究类型
注册 (估计的)
联系人和位置
学习地点
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Angers、法国、49933
- 尚未招聘
- CHU d'Angers
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接触：
- Marco Spinazzi, PH
- 电话号码：+33241357933
- 邮箱：marco.spinazzi@chu-angers.fr
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Boulogne-sur-Mer、法国、62231
- 尚未招聘
- Ch de Boulogne sur Mer
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接触：
- Mathieu BATAILLE, PH
- 电话号码：+321993056
- 邮箱：m.bataille@ch-boulogne.fr
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Créteil、法国、94000
- 尚未招聘
- Hopital Henri Mondor
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接触：
- Edoardo MALFATTI, PU-PH
- 电话号码：+33149812111
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Garches、法国、92380
- 尚未招聘
- Hôpital Raymond Pointcarré
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接触：
- Diane FRIEDMAN, PH
- 电话号码：+33147107785
- 邮箱：friedman.diane@aphp.fr
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Grenoble、法国、38700
- 尚未招聘
- CHU de GRENOBLE
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接触：
- Emmeline LAGRANGE, PH
- 邮箱：elagrange@chu-grenoble.fr
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Lille、法国、59037
- 尚未招聘
- CHU de Lille
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接触：
- Céline TARD, PH
- 电话号码：+330320444145
- 邮箱：celine.tard@chu-lille.fr
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Lille、法国、59000
- 尚未招聘
- GHICL Saint Vincent de Paul
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接触：
- Julien Poupart, PH
- 电话号码：+33320874901
- 邮箱：poupart.julien@ghicl.net
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Limoges、法国、87042
- 尚未招聘
- Chu Dupuytren 1
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接触：
- Laurent MAGY, PU-PH
- 电话号码：+33555056561
- 邮箱：laurent.magy@unlim.fr
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Rouen、法国、76031
- 尚未招聘
- CHU Charles Nicolle
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接触：
- Anne-Laure BEDAT-MILLET, PH
- 电话号码：+33232885721
- 邮箱：anne-laure.millet@chu-rouen.fr
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Toulouse、法国、31059
- 尚未招聘
- CHU de Toulouse
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接触：
- Blandine ACKET, PH
- 电话号码：+33561779989
- 邮箱：acket.b@chu-toulouse.fr
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Alpes-maritimes
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Nice、Alpes-maritimes、法国、06000
- 招聘中
- Chu De Nice
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接触：
- Sabrina SACCONI
- 电话号码：0492035757
- 邮箱：sacconi.s@chu-nice.fr
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接触：
- Sabrina SACCONI, pu-ph
- 电话号码：+33 4 92 03 57 57
- 邮箱：sacconi.s@chu-nice.fr
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参与标准
资格标准
适合学习的年龄
- 成人
- 年长者
接受健康志愿者
取样方法
研究人群
描述
Inclusion Criteria:
- Male or female subjects aged ≥18 years;
- Diagnosed with MG with confirmed documentation and supported by a physical exam and confirmed seropositivity for anti AChR or anti MUSK antibodies;
- Meets the clinical criteria as defined by the Myasthenia Gravis Foundation of America (MFGA) for generalized MG class IIa, IIb, IIIa, and IIIb;
- Patient suitable for ROZ treatment (IgG ≥ 5,5 g/l);
- Patient naïve for ROZ treatment, or receiving the last dose more than 3 months prior inclusion;
- MG-ADL score ≥ 3 (with ≥ 3 points of non-ocular symptoms);
- Patient must have received all mandatory vaccinations according to local regulations prior to study enrollment;
- Capable of understanding the written informed consent, and providing signed, dated, and witnessed written informed consent;
- Willing and able to comply with scheduled visits, treatment plan, study restrictions, laboratory tests, contraceptive guidelines, and other study procedures;
- Patient affiliated to a European social security system.
Exclusion Criteria:
- Any current mental condition (psychiatric disorder, senility, or dementia) that, in the opinion of the investigator, may affect study compliance or prevent understanding of the aims, investigational procedures, or possible consequences of the study; and/or patient with a Mini-mental State evaluation (MMSE) of <25;
- History of any illness or any clinical condition that, in the opinion of the investigator, might confound the results of the study or pose an additional risk in administering study drug to the subject;
- History of ongoing malignancy (except basal cell and squamous cell carcinomas of the skin, or in situ carcinoma of the cervix uteri that have been completely excised and cured);
- Clinically significant uncontrolled active or chronic bacterial, viral, or fungal infection at inclusion;
- Patient receiving concomitant treatment with rituximab, anti-FcRn, complement inhibitors and/or PLEX;
- Patients discontinued from rituximab without respecting the administration rules of ROZ;
- Patient with end-stage diseases or with a disease that will enable him to be evaluated and/or treated;
- Patients with non-treated active infections;
- Patients with hypersensitivity to the active substance(s) or to any of the excipients, specifically: histidine, histidine hydrochloride monohydrate, proline, polysorbate 80, or water for injections.
- Subject, or close relative of the subject, is the investigator or a sub-investigator, research assistant, pharmacist, study coordinator, or other staff directly involved with the conduct of the study at that site;
- Patient protected by law, under guardianship or curator ship, or not able to participate in a clinical study according to the article L.1121-16 of the French Public Health Code
学习计划
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 |
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To assess in real-life the impact of the ROZ on MG symptoms PRO score in generalized MG patients with anti-AChR or anti-MUSK antibodies
大体时间：7 days after the end of every cycle of treatment for 9 months
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To monitor 7 days after the end of every cycle of treatment for 9 months the change from baseline of MG symptoms PRO score (frome non to severe)
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7 days after the end of every cycle of treatment for 9 months
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次要结果测量
结果测量 |
措施说明 |
大体时间 |
|---|---|---|
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Evaluate the impact of real life on MG symptoms PRO global sub-scores physical fatigue
大体时间：7 days after the end of every cycle of treatment for 9 months
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To monitor 7 days after the end of every cycle of treatment for 9 months the change from baseline of MG symptoms PRO sub-score physical fatigue (from none of the time to all the time)
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7 days after the end of every cycle of treatment for 9 months
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Evaluate the impact of real life on quality of life measured by MG-QOL15r
大体时间：7 days after the end of every cycle of treatment for 9 months
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To monitor 7 days after the end of every cycle of treatment for 9 months the change from baseline of MG-QOL15r (MYASTHENIA GRAVIS QUALITY OF LIFE 15 REVISED) (from not at all to very much)
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7 days after the end of every cycle of treatment for 9 months
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Evaluate the impact of real life on patient general state, measured by the Patient-Acceptable Symptom State (PASS)
大体时间：7 days after the end of every cycle of treatment for 9 months
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To monitor 7 days after the end of every cycle of treatment for 9 months the change from baseline of PASS (PATIENT-ACCEPTABLE SYMPTOM STATE)
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7 days after the end of every cycle of treatment for 9 months
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Evaluate the impact of real life on the evolution of gMG after each cycle measured by MG-ADL score
大体时间：7 days after the end of every cycle of treatment for 9 months
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To monitor 7 days after the end of every cycle of treatment for 9 months the change from baseline of MG-ADL score (MYASTHENIA GRAVIS ACTIVITIES OF DAILY LIVING) (from normal to most severe)
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7 days after the end of every cycle of treatment for 9 months
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合作者和调查者
研究记录日期
研究主要日期
学习开始 (实际的)
初级完成 (估计的)
研究完成 (估计的)
研究注册日期
首次提交
首先提交符合 QC 标准的
首次发布 (实际的)
研究记录更新
最后更新发布 (实际的)
上次提交的符合 QC 标准的更新
最后验证
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
- 25-PP-23
计划个人参与者数据 (IPD)
计划共享个人参与者数据 (IPD)？
药物和器械信息、研究文件
研究美国 FDA 监管的药品
研究美国 FDA 监管的设备产品
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