Pediatric NMOSD Observational Study

April 8, 2024 updated by: Sean Pittock, Mayo Clinic

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

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.

Study Overview

Status

Completed

Conditions

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.

Study Type

Observational

Enrollment (Actual)

100

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 Locations

  • United States
    • Minnesota
      • Rochester, Minnesota, United States, 55905
        • Mayo Clinic

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

2 years to 17 years (Child)

Accepts Healthy Volunteers

No

Sampling Method

Non-Probability Sample

Study Population

Any pediatric patients that have tested positive for AQP4 antibody regardless of geographic location.

Description

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.

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

  • Observational Models: Cohort
  • Time Perspectives: Prospective

Cohorts and Interventions

Group / Cohort
Pediatric Neuromyelitis Optica
Participants under the age of 18 that are positive for Aquaporin-4 antibody.

What is the study measuring?

Primary Outcome Measures

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

Collaborators and Investigators

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

Sponsor

Mayo Clinic

Collaborators

Alexion Pharmaceuticals, Inc.

Investigators

  • Principal Investigator: Sean J Pittock, M.D., Mayo Clinic

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.

Helpful Links

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 (Actual)

February 1, 2018

Primary Completion (Actual)

April 3, 2024

Study Completion (Actual)

April 3, 2024

Study Registration Dates

First Submitted

December 4, 2018

First Submitted That Met QC Criteria

December 4, 2018

First Posted (Actual)

December 6, 2018

Study Record Updates

Last Update Posted (Actual)

April 10, 2024

Last Update Submitted That Met QC Criteria

April 8, 2024

Last Verified

April 1, 2024

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • 17-005618

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

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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