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

Study Identifier:
ECU-NMO-303
ClinicalTrials.gov Identifier:
NCT04155424
EudraCT Identifier:
2019-001829-26
EU CT ID:
N/A
Sponsor:
Alexion Pharmaceuticals, Inc.
Study Contact Information:
N/A
Other

Available Documents

ProtocolStatistical Analysis Plan

Study Details

Medical Condition
  • Neuromyelitis Optica
  • Neuromyelitis Optica Spectrum Disorder
Study Drug
  • Drug: Eculizumab
Date
Jan 2020 - Jul 2023
Phase 1
Phase 2
Phase 3
Phase 4
N/A
Patient Requirements
Sex: Female & Male
Age: N/A
Requirements Information
Inclusion and Exclusion Criteria
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.
  • 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
  • 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.
Sex
Female & Male
Age
N/A

Study Details

Medical Condition
  • Neuromyelitis Optica
  • Neuromyelitis Optica Spectrum Disorder
Study Drug
  • Drug: Eculizumab
Date
Jan 2020 - Jul 2023
Phase 1
Phase 2
Phase 3
Phase 4
N/A
Patient Requirements
Sex: Female & Male
Age: N/A years
Requirements Information
Inclusion and Exclusion Criteria
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.
  • 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
  • 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.

Protocol 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).

Trial Locations

Location
Status
Location
Clinical Trial Site
San Francisco, California, United States, 94016
Status
N/A
Location
Clinical Trial Site
Washington D.C., District of Columbia, United States, 20001
Status
N/A
Location
Clinical Trial Site
Miami, Florida, United States, 33101
Status
N/A
Location
Clinical Trial Site
Atlanta, Georgia, United States, 30301
Status
N/A
Location
Research Site
Baltimore, Maryland, United States, 21287
Status
N/A
Location
Clinical Trial Site
Rockville, Maryland, United States, 20847
Status
N/A
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