A Study of the Safety and Activity of Eculizumab in Pediatric Participants With Relapsing Neuromy… (NCT04155424) | Clinical Trial Compass
TerminatedPhase 2/3
A Study of the Safety and Activity of Eculizumab in Pediatric Participants With Relapsing Neuromyelitis Optica Spectrum Disorder
Stopped: Pediatric clinical study is not feasible.
United States5 participantsStarted 2020-01-14
Plain-language summary
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).
Who can participate
SexALL
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Inclusion criteria
✓. Male or female participants aged 2 years to \< 18 years 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.
✓. EDSS score ≤ 7.
✓. Participants who enter the study receiving supportive immunosuppressive therapies (ISTs) for the prevention of relapse, either in combination or monotherapy, must be on a stable dosing regimen of adequate duration.
✓. Female participants of childbearing potential must have a negative pregnancy test (serum human chorionic gonadotropin) at Screening and follow protocol-specified contraception guidance for avoiding pregnancy while on treatment and for 5 months after the last dose of eculizumab.
Exclusion criteria
✕. Parent or legal guardian is an Alexion employee.
What they're measuring
1
Change Between the Baseline Annualized Relapse Rate (ARR) and the On-Trial ARR at Week 52/53
✕. Pregnant, breastfeeding, or intending to conceive during the course of the study.
✕. Participants known to be human immunodeficiency virus positive or with congenital immunodeficiency.
✕. Unresolved meningococcal or other serious infection.
✕. Any unresolved acute or chronic systemic bacterial or other infection that is clinically significant in the opinion of the Investigator and has not been treated with appropriate antibiotics.
✕. Use of rituximab or other biologicals such as tocilizumab within 6 months prior to Screening.
✕. Use of mitoxantrone within 3 months prior to Screening.
✕. Use of intravenous immunoglobulin or plasma exchange within 3 weeks prior to Screening.