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).
Study Type
INTERVENTIONAL
Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
5
Following a weight-based weekly dose of eculizumab during an induction phase, participants will receive weight-based doses of eculizumab every 2 weeks during the Primary Treatment Period and Extension Treatment Period.
Change Between the Baseline Annualized Relapse Rate (ARR) and the On-Trial ARR at Week 52/53
ARR was calculated as the number of relapses for each participant divided by the number of years of treatment for that participant. Baseline ARR was based on 24 months prior to screening.
Time frame: Baseline, Week 52/53
Time to First On-trial Relapse
Time to First Relapse was defined as beginning at the time the participant's first dose of eculizumab was administered until the participant's first on-trial relapse was reported by the Investigator. Participants who did not experience an on-trial relapse were censored at the end of the study period.
Time frame: Baseline up to Week 52/53
Change From Baseline in Expanded Disability Status Scale (EDSS) Score at Week 52/53
Disease-related disability was measured by the EDSS. The EDSS is an ordinal clinical rating scale that ranges from 0 (normal neurologic examination) to 10 (death) in half-point increments. A decrease in score indicates improvement.
Time frame: Baseline, Week 52/53
Change From Baseline in the Hauser Ambulation Index (HAI) Score at Week 52/53
The HAI evaluates gait and was used to assess the time and effort used by the participant to walk 25 feet (8 meters). The scale ranged from 0 to 9, with 0 being the best score (asymptomatic; fully ambulatory with no assistance) and 9 being the worst (restricted to wheel chair; unable to transfer self independently). A decrease in score indicates improvement.
Time frame: Baseline, Weeks 52/53
Change From Baseline in Pediatric Quality of Life Inventory (PedsQL) Score at Week 52/53
PedsQL included a child self-report for participants 5 to 18 years with a 23-item PedsQL Generic Core Scales report. The PedsQL Generic Core Scales report included 4 scales, physical functioning, emotional functioning, social functioning, and school functioning. Each item used a 5-point rating scale (from 0=never to 4=almost always). Items are reverse scored and linearly transformed to a 0 (almost always) -100 (never) scale. All summary/total scores were mean of specific items where higher score indicated better HRQoL.
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Clinical Trial Site
San Francisco, California, United States
Clinical Trial Site
Washington D.C., District of Columbia, United States
Clinical Trial Site
Miami, Florida, United States
Clinical Trial Site
Atlanta, Georgia, United States
Research Site
Baltimore, Maryland, United States
Clinical Trial Site
Rockville, Maryland, United States
Clinical Trial Site
Boston, Massachusetts, United States
Research Site
Boston, Massachusetts, United States
Research Site
St Louis, Missouri, United States
Research Site
Hackensack, New Jersey, United States
...and 25 more locations
Time frame: Baseline, Weeks 52/53
Number of Participants With Shift From Baseline in Visual Acuity (VA)
The Snellen chart was used to assess VA. The Snellen chart quantifies ability to read letters of varying sizes at a fixed distance in relation to the distance at which a participant with normal vision could read the same letters. The test was performed at a standard distance, typically 6 meters or 20 feet. The Snellen chart is typically recorded as acuity ratio distance (6 meters or 20 feet), so for normal VA it would be recorded as 20/20 or 6/6. Visual acuity was summarized according to the eye with greater worsening at the end of primary treatment period (Week 52/53). Data are presented for number of participants with a shift from baseline in VA presented per the different levels of acuity ratio distance. Baseline was defined as the last available assessment prior to the first IP study drug infusion for all participants regardless of treatment group. Visual Acuity data are only reported for the categories with available data at Baseline and Week 52/53.
Time frame: Baseline, Week 52/53
Number of Participants With Shift From Baseline in Confrontational Visual Fields (VF)
Confrontational visual fields were summarized according to the number of quadrants with deficits across both eyes. Baseline was defined as the last available assessment prior to the first IP study drug infusion for all participants regardless of treatment group.
Time frame: Baseline, Weeks 52/53
Number of Participants With Shift From Baseline in Color Vision
Color vision was evaluated as the shift from baseline and described for participants with normal color vision at baseline in at least one eye. Participants with 13 or less correctly identified Ishihara plates were considered as having abnormal color vision, participants with 14 or more correctly identified plates were considered as having normal color vision. Baseline was defined as the last available assessment prior to the first IP study drug infusion for all participants regardless of treatment group.
Time frame: Baseline, Weeks 52/53
Serum Eculizumab Concentration at Week 52
Time frame: Week 52
Change From Baseline in Serum Free Complement Protein 5 (C5) Concentrations at Week 52
Time frame: Baseline, Week 52