Pediatric NMOSD Observational Study

Study Overview
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Brief Summary
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This study is being done to develop a database of pediatric patients in order to study the cause, early detection and best treatment for neuromyelitis optica spectrum disorder (NMOSD) in pediatric patients.

Detailed Description
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This study is being done to collect information on the natural history of NMOSD in pediatric AQP4-IgG seropositive patients. A major restriction in performing drug studies in pediatric patients with NMOSD is limited information on the course of the disease in these patients. Collecting clinical information over the course of a 1 year observational study would inform on the natural history of the disease in these patients. A repository of pediatric patients with rare diseases can increase knowledge on the natural history of the specific disease, assist in identifying appropriate patients fulfilling specified criteria for drug studies and potentially serve as a control group. Timepoints: Baseline, 3mo, 6mo, 9mo, 12mo (+/- 1 mo for each time point). Baseline data: Demographics [age/sex/ethnicity], Clinical presentation information including date of initial diagnosis, Clinical phenotype Immunotherapy used current and past, Family history of autoimmune diseases, Serological data results Radiologic data as available Self-Report Assessments will be: Current impairment, as measured by the expanded disability status scale (EDSS) score self-reported using Ratzker (1997) EDSS Self Report form, Quality of life as measured by the EQ-5D and Varni's (1998) PedsQL over the phone/mail/email. At follow-up visits 3mo, 6mo, 9mo, 12mo (+/- 1 mo for each time point): Attacks/relapses Any hospitalizations Confirm medications and update records if changes Self-Report Assessments at follow-up will be: Current impairment, as measured by the EDSS score self-reported using Ratzker (1997) EDSS Self Report form, Quality of life as measured by the EQ-5D and PedsQL over the phone/mail/email.

Official Title
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Pediatric Neuromyelitis Optica Spectrum Disorder (NMOSD) 1 Year Observational Study

Conditions
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Neuromyelitis Optica, NMO Spectrum Disorder

Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: AQP4 positive Ability to give informed consent by patient or caregiver Exclusion Criteria: - Inability to complete required forms via phone, mail, or email.

Ages Eligible for Study
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Minimum Age: 2 Years
Maximum Age: 17 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Pediatric Neuromyelitis Optica<br>Participants under the age of 18 that are positive for Aquaporin-4 antibody. | |

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Time to relapse | Relapse events of NMOSD defined by clinical worsening of neurological symptoms and treatment. | One year. | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Pediatric, Aquaporin-4, AQP4, NMOSD, NMO, Neuromyelitis Optica