Anticipating Irreversible Disability in Neuromyelitis Optica Spectrum Disorder: a Study to Assess Disease Activity in Apparently Stable Patients

Официальное название

Anticipating Irreversible Disability in Neuromyelitis Optica Spectrum Disorder: a Multicenter Prospective Observational Study to Assess Disease Activity in Apparently Stable Patients

Обзор

The present research is an observational clinical study. The project aims to investigate astrocytic damage, assessed through biological findings such as an increase in GFAP level and/or MRI water index, in patients with NMOSD and its potential role in predicting demyelinating relapses or affecting disability outcomes. It will also explore predictors of astrocytic relapses, focusing on demographic, clinical, and immunological biomarkers like T cell responses, B cell repopulation, and cytokine levels. The goal is to identify unrecognized disease activity, providing insights for future research and clinical trials. The study will involve 8 sites in Italy: 6 NMOSD clinical centers for patient enrolment and 2 centers for bioengineering and biological analysis. Centralized analysis of the MRI images of all patients enrolled in the clinical centers will be performed by the Neuroimaging Research Unit Fase 1 of San Raffaele Hospital. A total of 50 patients will be included and they will be followed for 12 months. Comprehensive evaluation of patients, including clinical assessment, bioengineering evaluation, MRI, and blood samples, will be conducted at baseline, month 6, and month 12. To assess silent astrocytic relapses, a specialized evaluation will take place at months 3 and 9, including clinical analysis, blood samples to assess biomarkers like GFAP, a reduced MRI protocol to assess MRI water index, and bioengineering evaluation. In the case of a classical relapse, a dedicated visit will occur within 5 days of symptom onset, using the same evaluation protocol as at months 3 and 9 (MRI and biomarkers will be evaluated if not done in the month before).

Вмешательства

• Диагностический тест MRI
  MRI will be performed every 3 months from baseline to 1 year.
• Диагностический тест Immunological Factors
  Immunological factors will be performed every 3 months from baseline to 1 year.

Первичные конечные точки

• Identifying predictors of clinical relapse [Срок оценки: From enrollment to 12 months]

Критерии участия

Критерии включения

• Adult patients (age ≥18 years);
• NMOSD diagnosis with AQP4 IgG positive status assessed with a cell-based assay;
• Under rituximab treatment (standard of care) from at least 1 year and for not more than 5 years;
• Ability to provide written informed consent.

Критерии исключения

• History of a clinical relapse or new/enlarging T2 lesion during the 6 months preceding the enrollment;
• Steroids treatment during 30 days before the enrollment;
• Any contraindication to MRI;
• Patients currently participating in a different clinical trial;
• Patients currently pregnant.

Критерии приведены из реестра в оригинале (на английском). Окончательную оценку соответствия проводит исследовательский центр.

Здоровые добровольцы: Нет

Дизайн исследования

Модель наблюдения

Когортное

Центры проведения

Италия · 1 центр

• San Raffaele Hospital — Milan

Публикации

• Masuda H, Mori M, Hirano S, Uzawa A, Uchida T, Muto M, Ohtani R, Aoki R, Kuwabara S. Silent progression of brain atrophy in aquaporin-4 antibody-positive neuromyelitis optica spectrum disorder. J Neurol Neurosurg Psychiatry. 2022 Jan;93(1):32-40. doi: 10.1136/jnnp-2021-326386. Epub 2021 Aug 6. PMID 34362853
• Margoni M, Gueye M, Meani A, Pagani E, Valsasina P, Storelli L, Preziosa P, Moiola L, Rocca MA, Filippi M. Substrates of 8.5-year clinical outcomes in aquaporin-4 IgG-positive neuromyelitis optica spectrum disorders. J Neurol. 2026 Feb 2;273(2):112. doi: 10.1007/s00415-026-13647-x. PMID 41627519
• Lorefice L, Carotenuto A, Fenu G. Silent burden: recognising and managing invisible symptoms in neuromyelitis optica. J Neurol Neurosurg Psychiatry. 2025 Jul 16;96(8):744-752. doi: 10.1136/jnnp-2025-336041. PMID 40506118
• Cacciaguerra L, Pagani E, Radaelli M, Mesaros S, Martinelli V, Ivanovic J, Drulovic J, Filippi M, Rocca MA. MR T2-relaxation time as an indirect measure of brain water content and disease activity in NMOSD. J Neurol Neurosurg Psychiatry. 2022 Apr 28:jnnp-2022-328956. doi: 10.1136/jnnp-2022-328956. Online ahead of print. PMID 35483915

Идентификаторы

NCT: NCT07526298 · ISS 20247046 Uplitza

Первоисточники (государственные реестры)

• ClinicalTrials.gov (NIH, США)

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