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Pediatric NMOSD Observational Study

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ClinicalTrials.gov Identifier: NCT03766347
Recruitment Status : Recruiting
First Posted : December 6, 2018
Last Update Posted : December 6, 2018
Sponsor:
Collaborator:
Alexion Pharmaceuticals
Information provided by (Responsible Party):
Sean Pittock, Mayo Clinic

Study Description
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Top of Page Study Description Study Design Groups and Cohorts Outcome Measures Eligibility Criteria Contacts and Locations More Information
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.

Condition or disease
Neuromyelitis Optica NMO Spectrum Disorder

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 Design
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Study Type : Observational [Patient Registry]
Estimated Enrollment : 100 participants
Observational Model: Cohort
Time Perspective: Prospective
Target Follow-Up Duration: 1 Year
Official Title: Pediatric Neuromyelitis Optica Spectrum Disorder (NMOSD) 1 Year Observational Study
Actual Study Start Date : February 1, 2018
Estimated Primary Completion Date : February 1, 2020
Estimated Study Completion Date : February 1, 2021

Resource links provided by the National Library of Medicine


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


Outcome Measures
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Primary Outcome Measures :
  1. Time to relapse [ Time Frame: One year. ]
    Relapse events of NMOSD defined by clinical worsening of neurological symptoms and treatment.


Eligibility Criteria
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Ages Eligible for Study:   2 Years to 17 Years   (Child)
Sexes Eligible for Study:   All
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.
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.

Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03766347


Contacts
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Contact: Cara L Thomas, AA 507-422-5563 thomas.cara@mayo.edu
Contact: Katie Dunlay, BA 507-538-5418 dunlay.katie@mayo.edu

Locations
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United States, Minnesota
Mayo Clinic Recruiting
Rochester, Minnesota, United States, 55905
Contact: Sean J. Pittock, M.D.    507-266-3196    pittock.sean@mayo.edu   
Principal Investigator: Sean J. Pittock, M.D.         
Sponsors and Collaborators
Mayo Clinic
Alexion Pharmaceuticals
Investigators
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Principal Investigator: Sean J Pittock, M.D. Mayo Clinic
More Information
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Additional Information:

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Responsible Party: Sean Pittock, Prinicpal Investigator, Mayo Clinic
ClinicalTrials.gov Identifier: NCT03766347     History of Changes
Other Study ID Numbers: 17-005618
First Posted: December 6, 2018    Key Record Dates
Last Update Posted: December 6, 2018
Last Verified: December 2018
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Undecided

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No

Keywords provided by Sean Pittock, Mayo Clinic:
Pediatric
Aquaporin-4
AQP4
 NMOSD
NMO
Neuromyelitis Optica

Additional relevant MeSH terms:
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Neuromyelitis Optica
Myelitis, Transverse
Demyelinating Autoimmune Diseases, CNS
Autoimmune Diseases of the Nervous System
Nervous System Diseases
Optic Neuritis
 Optic Nerve Diseases
Cranial Nerve Diseases
Demyelinating Diseases
Eye Diseases
Autoimmune Diseases
Immune System Diseases