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).



Eligible Ages
Between 2 Years and 17 Years
Eligible Genders
Accepts Healthy Volunteers

Inclusion Criteria

  1. Male or female participants aged 2 years to < 18 years with body weight ≥ 10 kilograms (kg). 2. 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. 3. 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. 4. Anti-aquaporin-4 antibody-positive and diagnosis of NMOSD as defined by the 2015 International Panel for Neuromyelitis Optica Diagnosis criteria. 5. 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. 6. EDSS score ≤ 7. 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. 8. 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. 9. Male participants with a female spouse/partner of childbearing potential or a pregnant or breastfeeding spouse or partner must agree to use double barrier contraception (male condom plus appropriate barrier method for the female partner) while on treatment and for at least 5 months after the last dose of eculizumab.

Exclusion Criteria

  1. Parent or legal guardian is an Alexion employee. 2. Pregnant, breastfeeding, or intending to conceive during the course of the study. 3. Participants known to be human immunodeficiency virus positive or with congenital immunodeficiency. 4. Unresolved meningococcal or other serious infection. 5. 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. 6. Use of rituximab or other biologicals such as tocilizumab within 6 months prior to Screening. 7. Use of mitoxantrone within 3 months prior to Screening. 8. Use of intravenous immunoglobulin or plasma exchange within 3 weeks prior to Screening. 9. Use of immunomodulatory therapies for multiple sclerosis within 3 months prior to Screening. 10. Has previously received treatment with eculizumab or other complement inhibitors.

Study Design

Phase 2/Phase 3
Study Type
Intervention Model
Single Group Assignment
Primary Purpose
None (Open Label)

Arm Groups

ArmDescriptionAssigned Intervention
All participants will receive open-label eculizumab by intravenous infusion during the Primary Treatment Period, starting on Day 1 and for a total of 52/53 weeks. The dosing regimen will be based on the participant's body weight. As body weight changes during the study, the participant's weight cohort and dose may change accordingly. After completing the 52/53-week Primary Treatment Period, participants may continue receiving eculizumab in the Extension Treatment Period for 104 weeks.
  • Drug: Eculizumab
    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.
    Other names:
    • Soliris

Recruiting Locations

Massachusetts General Hospital
Boston, Massachusetts 02114

More Details

Alexion Pharmaceuticals

Study Contact

Alexion Pharmaceuticals, Inc.


Study information shown on this site is derived from ClinicalTrials.gov (a public registry operated by the National Institutes of Health). The listing of studies provided is not certain to be all studies for which you might be eligible. Furthermore, study eligibility requirements can be difficult to understand and may change over time, so it is wise to speak with your medical care provider and individual research study teams when making decisions related to participation.