Efficacy and Safety Study of Ravulizumab IV in Pediatric Participants With NMOSD

Launched by ALEXION PHARMACEUTICALS, INC. · Apr 20, 2022

Keywords

ClinConnect Summary

This clinical trial is investigating a medication called ravulizumab to see how safe and effective it is for children and teenagers with Neuromyelitis Optica Spectrum Disorder (NMOSD), a rare condition that affects the nervous system. The trial is currently looking for participants who are between specific ages and have tested positive for a particular antibody related to NMOSD. To join, participants must have experienced at least one attack or relapse in the past year and have a certain level of disability. It’s important that they have not received certain treatments recently and are up to date on vaccinations to prevent infections.

If your child is eligible and decides to participate, they will receive ravulizumab and be closely monitored by the study team for any side effects or changes in their condition. This study will help researchers understand how well ravulizumab works in children with NMOSD and contribute to better treatment options in the future. If you have any questions or want to know more about the trial, it’s a good idea to talk to your doctor.

Gender

ALL

Eligibility criteria

• Inclusion Criteria:
• • Participants must be anti-AQP4 Ab-positive and have a diagnosis of NMOSD as defined by the 2015 international consensus diagnostic criteria.
• • Complement inhibitor treatment-naïve participants must have had at least 1 attack or relapse in the last 12 months prior to the Screening Period.
• • Expanded Disability Status Scale (EDSS) score ≤ 7.
• • Eculizumab-experienced participants must be clinically stable per Investigator for 30 days and have been treated with eculizumab in Study ECU-NMO-303 for at least 90 days prior to screening with no missed doses within 2 months prior to Day 1.
• • Participants who enter the study receiving supportive IST(s) (eg, corticosteroid, azathioprine \[AZA\], mycophenolate mofetil \[MMF\], methotrexate \[MTX\], tacrolimus \[TAC\], cyclosporin \[CsA\], or cyclophosphamide \[CYC\]) for the prevention of relapse, either in combination or monotherapy, must be on a stable dosing regimen of adequate duration prior to Screening and remain on a stable dosing regimen during the Screening Period.
• • To reduce the risk of meningococcal infection (Neisseria meningitidis), all participants must be vaccinated against meningococcal infection.
• • Documented vaccination for Hib and S pneumoniae at least 14 days prior to Day 1 according to national/local guidelines for the applicable age group.
• Exclusion Criteria:
• • Use of rituximab within 3 months prior to screening.
• • Currently treated with a biologic medications (other than eculizumab) that may affect immune system functioning, or has stopped treatment with a biologic medication that may affect immune system functioning, and 5 half lives of the medication have not elapsed by the time of the Screening Visit.
• • Use of intravenous immunoglobulin (IVIg) or plasma exchange (PE) within 3 weeks prior to Screening.
• • Participation in another investigational drug or investigational device study (other than Study ECU-NMO-303) within 5 half lives of that investigational product (if known) or 30 days before initiation of the first dose of study drug, whichever is longer.
• • Use of immunomodulatory therapies for multiple sclerosis within 3 months prior to Screening.