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

Stopped early · Phase 2/Phase 3

Conditions studied: Neuromyelitis Optica, Neuromyelitis Optica Spectrum Disorder

In brief

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).

Key facts

Study ID

NCT04155424

Run by

Alexion Pharmaceuticals, Inc.

People needed

5

Starts

2020-01-14

Expected to finish

2023-07-31

Last updated by the study team

2024-09-25

Who can join

Age: any. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• 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.
• 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.

You may not qualify if…

• Parent or legal guardian is an Alexion employee.
• 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.
• Use of immunomodulatory therapies for multiple sclerosis within 3 months prior to Screening.
• Has previously received treatment with eculizumab or other complement inhibitors.

Where it is running

• 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
• Research Site — New Brunswick, New Jersey, United States
• Research Site — New York, New York, United States
• Clinical Trial Site — Chapel Hill, North Carolina, United States
• Clinical Trial Site — Philadelphia, Pennsylvania, United States
• Research Site — Dallas, Texas, United States
• Research Site — Calgary, Alberta, Canada
• Clinical Trial Site — Edmonton, Alberta, Canada
• Research Site — Toronto, Ontario, Canada
• Clinical Trial Site — Toronto, Ontario, Canada
• Research Site — Montreal, Quebec, Canada
• Clinical Trial Site — Regensburger Straße, Goettingen, Germany
• Research Site — Datteln, Germany
• Research Site — Münster, Germany
• Research Site — Catania, Italy
• Research Site — Gallarate, Italy

Full record on ClinicalTrials.gov

Trial information comes from ClinicalTrials.gov and is refreshed daily. TrialsForMe does not provide medical care and does not run the studies it lists.