Clinical trial NCT04155424: A Study of the Safety and Activity of Eculizumab in Pediatric Participants With Relapsing Neuromyelitis Optica Spectrum Disorder
Official title: A Phase 2/3 Open-Label, Single-Arm Trial to Evaluate the Safety and Activity of Eculizumab in Pediatric Patients With Relapsing Neuromyelitis Optica Spectrum Disorder
Study type: INTERVENTIONAL
Overall status: TERMINATED
Phase: PHASE2
PHASE3
Conditions: Neuromyelitis Optica
Neuromyelitis Optica Spectrum Disorder
Keywords: Neuromyelitis Optica
Neuromyelitis Optica Spectrum Disorder
Devic's Disease
Transverse Myelitis
Optic Neuritis
Relapse
Eculizumab
Soliris
CNS Autoimmune Disorders
Demyelinating Disorders
NMO
NMOSD
Lead sponsor: Alexion Pharmaceuticals, Inc.
Sponsor class: INDUSTRY
First posted: 2019-11-07
Enrollment: 5
Sex: ALL
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief 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).


Eligibility criteria: 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.

Primary outcomes:
measure: Change Between the Baseline Annualized Relapse Rate (ARR) and the On-Trial ARR at Week 52/53
timeFrame: Baseline, Week 52/53
measure: Time to First On-trial Relapse
timeFrame: Baseline up to Week 52/53
Secondary outcomes:
measure: Change From Baseline in Expanded Disability Status Scale (EDSS) Score at Week 52/53
timeFrame: Baseline, Week 52/53
measure: Change From Baseline in the Hauser Ambulation Index (HAI) Score at Week 52/53
timeFrame: Baseline, Weeks 52/53
measure: Change From Baseline in Pediatric Quality of Life Inventory (PedsQL) Score at Week 52/53
timeFrame: Baseline, Weeks 52/53
measure: Number of Participants With Shift From Baseline in Visual Acuity (VA)
timeFrame: Baseline, Week 52/53
measure: Number of Participants With Shift From Baseline in Confrontational Visual Fields (VF)
timeFrame: Baseline, Weeks 52/53
measure: Number of Participants With Shift From Baseline in Color Vision
timeFrame: Baseline, Weeks 52/53
measure: Serum Eculizumab Concentration at Week 52
timeFrame: Week 52
measure: Change From Baseline in Serum Free Complement Protein 5 (C5) Concentrations at Week 52
timeFrame: Baseline, Week 52
Other outcomes:
Locations:
city: San Francisco
country: United States
facility: Clinical Trial Site
state: California
city: Washington
country: United States
facility: Clinical Trial Site
state: District of Columbia
city: Miami
country: United States
facility: Clinical Trial Site
state: Florida
city: Atlanta
country: United States
facility: Clinical Trial Site
state: Georgia
city: Baltimore
country: United States
facility: Research Site
state: Maryland
city: Rockville
country: United States
facility: Clinical Trial Site
state: Maryland
city: Boston
country: United States
facility: Clinical Trial Site
state: Massachusetts
city: Boston
country: United States
facility: Research Site
state: Massachusetts
city: Saint Louis
country: United States
facility: Research Site
state: Missouri
city: Hackensack
country: United States
facility: Research Site
state: New Jersey
city: New Brunswick
country: United States
facility: Research Site
state: New Jersey
city: New York
country: United States
facility: Research Site
state: New York
city: Chapel Hill
country: United States
facility: Clinical Trial Site
state: North Carolina
city: Philadelphia
country: United States
facility: Clinical Trial Site
state: Pennsylvania
city: Dallas
country: United States
facility: Research Site
state: Texas
city: Calgary
country: Canada
facility: Research Site
state: Alberta
city: Edmonton
country: Canada
facility: Clinical Trial Site
state: Alberta
city: Toronto
country: Canada
facility: Research Site
state: Ontario
city: Toronto
country: Canada
facility: Clinical Trial Site
state: Ontario
city: Montreal
country: Canada
facility: Research Site
state: Quebec
city: Regensburger Straße
country: Germany
facility: Clinical Trial Site
state: Goettingen
city: Datteln
country: Germany
facility: Research Site
city: Munster
country: Germany
facility: Research Site
city: Catania
country: Italy
facility: Research Site
city: Gallarate
country: Italy
facility: Research Site
city: Genoa
country: Italy
facility: Clinical Trial Site
city: Genova
country: Italy
facility: Research Site
city: Rome
country: Italy
facility: Research Site
city: Rome
country: Italy
facility: Clinical Trial Site
city: Yokohama-shi
country: Japan
facility: Research Site
city: Yokohama
country: Japan
facility: Clinical Trial Site
city: Seoul
country: Korea, Republic of
facility: Clinical Trial Site
city: Barcelona
country: Spain
facility: Clinical Trial Site
city: Esplugues de Llobregat
country: Spain
facility: Research Site
city: Sevilla
country: Spain
facility: Clinical Trial Site
Study officials:
References: