A Trial to Evaluate the Safety and Activity of Eculizumab in PediatricPatients with Relapsing Neuromyelitis Optica Spectrum Disorder (NMOSD)

Recruitment & Eligibility

Status: ot Recruiting

Sex: All

Target Recruitment: 12

Inclusion Criteria

• Male or female patients aged 2 years to < 18 years at time of assent/consent.
• Anti-AQP4 Ab-positive and diagnosis of NMOSD as defined by the 2015 IPND 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.
• Expanded Disability Status Scale score = 7
• Patients who enter the study receiving supportive immunosuppressive therapies for the prevention of relapse, either in combination or monotherapy, must be on a stable dosing regimen of adequate duration prior to Screening and remain on a stable dosing regimen during the
Screening Period.
• Vaccinated against N meningitidis within 3 years prior to, or at the time of initiating eculizumab. Patients 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 Hib and S pneumoniae infections 2 weeks prior to dosing as per local and country-specific immunization guidelines for the appropriate age group.
• Willing and capable to give informed assent (if applicable, as determined by the central Institutional Review Boards (IRBs)/Independent Ethics Committees (IECs) and in accordance with local requirements) and whose parent/legal guardian are willing and able to give written informed consent, which includes compliance with the requirements and restrictions listed in the informed consent form (ICF) and in the protocol.
Are the trial subjects under 18? yes
Number of subjects for this age range: 12
F.1.2 Adults (18-64 years) no
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

• Patients known to be human immunodeficiency virus (HIV) positive or with congenital immunodeficiency.
• Unresolved meningococcal or other serious infection.
•Active systemic bacterial, viral, or fungal infection within 14 days prior to study drug administration on Day 1.
• Hypersensitivity to murine proteins or to one of the excipients of eculizumab.
• 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 (IVIg) or plasma exchange (PE) within 3 weeks prior to Screening.
• Use of immunomodulatory therapies for multiple sclerosis within 3 months prior to Screening.
• Patient is currently treated with a biologic medication that may affect immune system functioning, or has stopped treatment with a biologic medication that may affect immune system functioning, and 5 half-lives of the medication have not elapsed by the time of the Screening Visit, unless otherwise specified in the protocol.

Study & Design

Study Type: Interventional clinical trial of medicinal product

Study Design: Not specified

Primary Outcome Measures

Name	Time	Method

Secondary Outcome Measures

Name	Time	Method

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