A Pediatric NMOSD Observational Study opportunity is open for enrollment to any pediatric patient, age 2 to 17 years, that has tested positive for the AQP4 antibody, regardless of their location. The study, led by Dr. Sean Pittock of Mayo Clinic along with Alexion Pharmaceuticals, is being done to collect information to develop a database on the natural history of NMOSD in pediatric patients who test positive for AQP4-IgG.
The lack of information on the course and natural history of NMOSD in children is a limiting factor in performing drug studies for children with this disorder. Participation in the study will allow for the collection of clinical information over a one-year period to help inform on the natural history of NMOSD and to build a repository of pediatric patients. A repository or database of information will allow the rare disease community, and medical and scientific researchers, to increase knowledge of NMOSD. It will also identify appropriate individuals for specific drug studies and may also help serve as a control group for future research.
This is an observational, prospective study, and information (e.g., baseline data, self-report assessments, etc.) is collected over the course of one year: at enrollment, 3 months, 6 months, 9 months, and 12 months (+/- 1 month for each time point). Travel to Mayo Clinic is not necessary for participation!
A full description of data collected, self-reporting assessments used, and timepoints for data collection can be found here.
Inclusion Criteria include the following:
- AQP4 positive.
- Ability to give informed consent by patient or caregiver.
Inability to complete required forms via phone, mail, or email.
If you or your child have questions or are interested in participating, please contact Cara L Thomas, AA, at 507-722-5563, or via email [email protected], or Katie Dunlay, BA, at 507-538-5418, or via email at [email protected].
It is through your active consideration and participation in research that assists our medical and scientific community with identifying and carrying out research priorities for our children diagnosed with NMOSD. Thank you for your thoughtful consideration!