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

April 10, 2026 updated by: Prof. Massimo Filippi, IRCCS San Raffaele

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

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).

Study Overview

Status

Not yet recruiting

Conditions

Intervention / Treatment

Study Type

Observational

Enrollment (Estimated)

50

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Contact Backup

Study Locations

  • Italy
    • Milan
      • Milan, Milan, Italy, 20132
        • San Raffaele Hospital
        • Contact:

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Sampling Method

Non-Probability Sample

Study Population

The study will include adult patients with NMOSD.

Description

Inclusion Criteria:

  • 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.

Exclusion Criteria:

  • 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.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

Cohorts and Interventions

Group / Cohort
Intervention / Treatment
Adult patients with NMOSD
Diagnostic test: MRI
MRI will be performed every 3 months from baseline to 1 year.
Diagnostic test: Immunological Factors
Immunological factors will be performed every 3 months from baseline to 1 year.

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Identifying predictors of clinical relapse
Time Frame: From enrollment to 12 months
The primary outcomes measures are: percentage of patients with relapse, defined by the occurrence of a novel/worsening NMOSD symptom (detected by patient reported outcomes or clinical judgment or bioengineering indexes) PLUS biological findings of astrocyte damage (increase of GFAP level and/or MRI water index).
From enrollment to 12 months

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

IRCCS San Raffaele

Collaborators

Amgen

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Estimated)

April 1, 2026

Primary Completion (Estimated)

April 1, 2027

Study Completion (Estimated)

April 1, 2027

Study Registration Dates

First Submitted

March 6, 2026

First Submitted That Met QC Criteria

April 10, 2026

First Posted (Actual)

April 13, 2026

Study Record Updates

Last Update Posted (Actual)

April 13, 2026

Last Update Submitted That Met QC Criteria

April 10, 2026

Last Verified

February 1, 2026

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • ISS 20247046 Uplitza

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

product manufactured in and exported from the U.S.

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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