Esta página se tradujo automáticamente y no se garantiza la precisión de la traducción. por favor refiérase a versión inglesa para un texto fuente.

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)

31 de agosto de 2026 actualizado por: Centre Hospitalier Universitaire de Nice

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.

Descripción general del estudio

Estado

Reclutamiento

Condiciones

Intervención / Tratamiento

Tipo de estudio

De observación

Inscripción (Estimado)

50

Contactos y Ubicaciones

Esta sección proporciona los datos de contacto de quienes realizan el estudio e información sobre dónde se lleva a cabo este estudio.

Ubicaciones de estudio

  • Francia
      • Angers, Francia, 49933
        • Aún no reclutando
        • CHU d'Angers
        • Contacto:
      • Boulogne-sur-Mer, Francia, 62231
        • Aún no reclutando
        • Ch de Boulogne sur Mer
        • Contacto:
      • Créteil, Francia, 94000
        • Aún no reclutando
        • Hopital Henri Mondor
        • Contacto:
          • Edoardo MALFATTI, PU-PH
          • Número de teléfono: +33149812111
      • Garches, Francia, 92380
        • Aún no reclutando
        • Hôpital Raymond Pointcarré
        • Contacto:
      • Grenoble, Francia, 38700
      • Lille, Francia, 59037
        • Aún no reclutando
        • CHU de Lille
        • Contacto:
      • Lille, Francia, 59000
        • Aún no reclutando
        • GHICL Saint Vincent de Paul
        • Contacto:
      • Limoges, Francia, 87042
        • Aún no reclutando
        • Chu Dupuytren 1
        • Contacto:
      • Rouen, Francia, 76031
        • Aún no reclutando
        • CHU Charles Nicolle
        • Contacto:
      • Toulouse, Francia, 31059
        • Aún no reclutando
        • CHU de Toulouse
        • Contacto:
    • Alpes-maritimes
      • Nice, Alpes-maritimes, Francia, 06000
        • Reclutamiento
        • Chu De Nice
        • Contacto:
        • Contacto:
          • Sabrina SACCONI, pu-ph
          • Número de teléfono: +33 4 92 03 57 57
          • Correo electrónico: sacconi.s@chu-nice.fr

Criterios de participación

Los investigadores buscan personas que se ajusten a una determinada descripción, denominada criterio de elegibilidad. Algunos ejemplos de estos criterios son el estado de salud general de una persona o tratamientos previos.

Criterio de elegibilidad

Edades elegibles para estudiar

  • Adulto
  • Adulto Mayor

Acepta Voluntarios Saludables

No

Método de muestreo

Muestra no probabilística

Población de estudio

Patients affected by generalized Myasthenia Gravis with anti-AChR and anti-MUSK and treated with ROZ

Descripción

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

Plan de estudios

Esta sección proporciona detalles del plan de estudio, incluido cómo está diseñado el estudio y qué mide el estudio.

¿Cómo está diseñado el estudio?

Detalles de diseño

¿Qué mide el estudio?

Medidas de resultado primarias

Medida de resultado
Medida Descripción
Periodo de tiempo
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
Periodo de tiempo: 7 days after the end of every cycle of treatment for 9 months
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)
7 days after the end of every cycle of treatment for 9 months

Medidas de resultado secundarias

Medida de resultado
Medida Descripción
Periodo de tiempo
Evaluate the impact of real life on MG symptoms PRO global sub-scores physical fatigue
Periodo de tiempo: 7 days after the end of every cycle of treatment for 9 months
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)
7 days after the end of every cycle of treatment for 9 months
Evaluate the impact of real life on quality of life measured by MG-QOL15r
Periodo de tiempo: 7 days after the end of every cycle of treatment for 9 months
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)
7 days after the end of every cycle of treatment for 9 months
Evaluate the impact of real life on patient general state, measured by the Patient-Acceptable Symptom State (PASS)
Periodo de tiempo: 7 days after the end of every cycle of treatment for 9 months
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)
7 days after the end of every cycle of treatment for 9 months
Evaluate the impact of real life on the evolution of gMG after each cycle measured by MG-ADL score
Periodo de tiempo: 7 days after the end of every cycle of treatment for 9 months
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)
7 days after the end of every cycle of treatment for 9 months

Colaboradores e Investigadores

Aquí es donde encontrará personas y organizaciones involucradas en este estudio.

Patrocinador

Centre Hospitalier Universitaire de Nice

Fechas de registro del estudio

Estas fechas rastrean el progreso del registro del estudio y los envíos de resultados resumidos a ClinicalTrials.gov. Los registros del estudio y los resultados informados son revisados ​​por la Biblioteca Nacional de Medicina (NLM) para asegurarse de que cumplan con los estándares de control de calidad específicos antes de publicarlos en el sitio web público.

Fechas importantes del estudio

Inicio del estudio (Actual)

15 de junio de 2026

Finalización primaria (Estimado)

1 de marzo de 2028

Finalización del estudio (Estimado)

1 de marzo de 2028

Fechas de registro del estudio

Enviado por primera vez

30 de abril de 2026

Primero enviado que cumplió con los criterios de control de calidad

30 de abril de 2026

Publicado por primera vez (Actual)

6 de mayo de 2026

Actualizaciones de registros de estudio

Última actualización publicada (Actual)

1 de septiembre de 2026

Última actualización enviada que cumplió con los criterios de control de calidad

31 de agosto de 2026

Última verificación

1 de agosto de 2026

Más información

Términos relacionados con este estudio

Términos MeSH relevantes adicionales

Otros números de identificación del estudio

  • 25-PP-23

Plan de datos de participantes individuales (IPD)

¿Planea compartir datos de participantes individuales (IPD)?

NO

Información sobre medicamentos y dispositivos, documentos del estudio

Estudia un producto farmacéutico regulado por la FDA de EE. UU.

No

Estudia un producto de dispositivo regulado por la FDA de EE. UU.

No

Esta información se obtuvo directamente del sitio web clinicaltrials.gov sin cambios. Si tiene alguna solicitud para cambiar, eliminar o actualizar los detalles de su estudio, comuníquese con register@clinicaltrials.gov. Tan pronto como se implemente un cambio en clinicaltrials.gov, también se actualizará automáticamente en nuestro sitio web. .

Suscribir