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

Phase 1

• Conditions: euromyelitis Optica Spectrum Disorder (NMOSD); MedDRA version: 21.1 Level: LLT Classification code 10077879 Term: Neuromyelitis optica spectrum disorder relapse System Organ Class: 100000004852; Therapeutic area: Diseases [C] - Nervous System Diseases [C10]

• Registration Number: EUCTR2019-001829-26-ES
• Lead Sponsor: Alexion Pharmaceuticals Inc.

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2019-11-06
• Last Update Posted: 1 February 2020

Recruitment & Eligibility

• Status: Authorised-recruitment may be ongoing or finished
• Sex: Not specified
• Target Recruitment: 15

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.
• 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: 15
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.
• Any unresolved acute or chronic systemic bacterial or other infection which is clinically significant in the opinion of the Investigator and has not been treated with appropriate antibiotics.
• 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.

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified