Clinical trial NCT03766347: Pediatric NMOSD Observational Study
Official title: Pediatric Neuromyelitis Optica Spectrum Disorder (NMOSD) 1 Year Observational Study
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Neuromyelitis Optica
NMO Spectrum Disorder
Keywords: Pediatric
Aquaporin-4
AQP4
NMOSD
NMO
Neuromyelitis Optica
Lead sponsor: Mayo Clinic
Sponsor class: OTHER
Collaborators: name: Alexion Pharmaceuticals, Inc.
First posted: 2018-12-06
Enrollment: 100
Sex: ALL
Minimum age (years): 2
Maximum age (years): 17
Standard ages: CHILD
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Any pediatric patients that have tested positive for AQP4 antibody regardless of geographic location.

Brief summary: 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: 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.

Eligibility criteria: 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.

Primary outcomes:
measure: Time to relapse
timeFrame: One year.
Secondary outcomes:
Other outcomes:
Locations:
city: Rochester
country: United States
facility: Mayo Clinic
state: Minnesota
Study officials:
affiliation: Mayo Clinic
name: Sean J Pittock, M.D.
role: PRINCIPAL_INVESTIGATOR
References: