Pediatric NMOSD Observational Study
|The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.|
|ClinicalTrials.gov Identifier: NCT03766347|
Recruitment Status : Recruiting
First Posted : December 6, 2018
Last Update Posted : June 11, 2020
|Condition or disease|
|Neuromyelitis Optica NMO Spectrum Disorder|
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).
- 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):
- 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 [Patient Registry]|
|Estimated Enrollment :||100 participants|
|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, 2021|
|Estimated Study Completion Date :||February 1, 2021|
Pediatric Neuromyelitis Optica
Participants under the age of 18 that are positive for Aquaporin-4 antibody.
- Time to relapse [ Time Frame: One year. ]Relapse events of NMOSD defined by clinical worsening of neurological symptoms and treatment.
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
|Contact: Cara L Thomas, AAemail@example.com|
|Contact: Katie Dunlay, BAfirstname.lastname@example.org|
|United States, Minnesota|
|Rochester, Minnesota, United States, 55905|
|Contact: Sean J. Pittock, M.D. 507-266-3196 email@example.com|
|Principal Investigator: Sean J. Pittock, M.D.|
|Principal Investigator:||Sean J Pittock, M.D.||Mayo Clinic|