Current Pediatric Research Studies

/in /by



SRNA helps with research to find new ways to treat ADEM, AFM, MOGAD, NMOSD, ON, and TM. Research is important to advance our understanding of rare neuroimmune disorders and develop better treatments. Participating in research is a way to contribute to these efforts and make a difference for the whole community of people diagnosed with rare neuroimmune disorders. If you want to help by participating in these studies, you can contact the study coordinator listed for more information.

 Study of Inebilizumab in Pediatric Subjects With Neuromyelitis Optica Spectrum Disorder

Horizon Therapeutics has a new study available for pediatric patients diagnosed with NMOSD. This is a Phase 2, open-label, multicenter study to evaluate the pharmacokinetics (PK), pharmacodynamics (PD), and safety of inebilizumab. 

Participant criteria includes:

  • Must be between 2-18 years of age
  • Recently active recently active neuromyelitis optica spectrum disorder (NMOSD)
  • Seropositive for autoantibodies against aquaporin-4 (AQP4-immunoglobulin [Ig]G)

Find more details and contact information here. 

Pediatric NMOSD 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.

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. Any pediatric patients that have tested positive for AQP4 antibody regardless of geographic location can participate in this study.

Find more details and contact information here.

PEDIATRIC SONICS: Pediatric Study of Neuropsychology and Imaging in CNS Demyelinating Syndromes (SONICS)

Researchers are asking teens with ADEM, NMOSD, MOGAD, and TM and their siblings without a CNS demyelinating disorder between the ages of 12-18 to participate in an observational study.  You don’t have to have a sibling to participate. With an MRI using a stronger magnet, a specialized picture of the brain, researchers at UTSW might predict if an individual may have difficulty with learning.

Participant criteria includes:

  • Diagnosis of Multiple Sclerosis , ADEM, NMOSD, TM, or anti-MOG antibody associated CNS demyelination
  • Age 12 to 18 inclusive at time of enrollment

Exclusion Criteria: 

  • Known history of traumatic brain injury that required medical care
  • Non-English speaking (based on standardized neuropsychological testing and questionnaires)
  • Claustrophobic, pregnant, the presence of metallic braces, implants or medical devices that are unsafe at 3T or 7T and/or interfere with the MRI/MRS signals 

Find more details and contact information here.

Acute Flaccid Myelitis and Host Genetics

If interested in participating in the Johns Hopkins Acute Flaccid Myelitis study please contact Dr. Duggal directly at [email protected]. This study enrolls individuals with AFM and their families in a genetic study where their DNA is compared to other cases of AFM, and to their family members. Enrollment in the study is done via phone and mail, no travel is required. DNA is extracted from saliva, using saliva collection tubes.

For more information on the study, listen to our Ask the Expert podcast overview of the study. 

A Study of the Safety and Activity of Eculizumab in Pediatric Participants With Relapsing Neuromyelitis Optica Spectrum Disorder

Alexion Pharmaceuticals is conducting this clinical trial. The objective of this study is to evaluate the safety and efficacy of eculizumab in pediatric participants (aged 2 to < 18 years) with relapsing neuromyelitis optica spectrum disorder (NMOSD).

Participant criteria includes:

  • Male or female participants between the ages of 2 and 17 with body weight ≥ 10 kilograms (kg).
  • Vaccinated against Neisseria meningitidis within 3 years prior to, or at the time of initiating eculizumab. Participants who initiate study drug treatment less than 2 weeks after receiving a meningococcal vaccine must receive appropriate prophylactic antibiotics until 2 weeks after the vaccination.
  • Documented vaccination against haemophilus influenzae type b and streptococcus pneumoniae infections at least 2 weeks prior to dosing as per local and country-specific immunization guidelines for the appropriate age group.
  • Anti-aquaporin-4 antibody-positive and diagnosis of NMOSD as defined by the 2015 International Panel for Neuromyelitis Optica Diagnosis criteria.
  • Historical Relapse Rate of at least 2 relapses in the last 2 years, and with at least 1 relapse in the year prior to Screening.

Find more details and contact information here.

Neuroimaging and Neurobehavioral Outcomes of Pediatric Neuromyelitis Optica Spectrum Disorder: A Pilot Study

The primary objective of this study is to determine how well specific neuroimaging modalities detect the different aspects of anomalous white matter development associated with pediatric NMOSD. Our purpose is to acquire data using neuroimaging obtained at 3.0 Tesla as well as neurobehavioral data to better characterize neuroimaging features and function in this rare population.

This study takes part in two phases. For the first part, participants will undergo neuropsychological (cognitive) testing and have a “mock” or practice magnetic resonance imaging (MRI) scan”. The purpose of this “mock” scan is to improve comfort, decrease potential anxiety, and to train you to lie still while in the scanner. After the “mock”/practice scan, participants will have the MRI exam. The MRI scan could take up to 1 hour. Participants are in this study for one day for approximately 3-4 hours.

Participant criteria includes:

  • Individuals aged 13-18 years with NMOSD may join. We will also be enrolling typically developing individuals of the same ages without NMOSD, to serve as a control group.

Find more details and contact information here.