A Study of the Safety and Activity of Eculizumab in Pediatric Participants With Relapsing Neuromyelitis Optica Spectrum Disorder
NCT ID: NCT04155424
Last Updated: 2024-09-25
Study Results
Outcome measurements, participant flow, baseline characteristics, and adverse events have been published for this study.
View full resultsBasic Information
Get a concise snapshot of the trial, including recruitment status, study phase, enrollment targets, and key timeline milestones.
TERMINATED
PHASE2/PHASE3
5 participants
INTERVENTIONAL
2020-01-14
2023-07-31
Brief Summary
Review the sponsor-provided synopsis that highlights what the study is about and why it is being conducted.
Related Clinical Trials
Explore similar clinical trials based on study characteristics and research focus.
Efficacy and Safety Study of Ravulizumab IV in Pediatric Participants With NMOSD
NCT05346354
An Open Label Extension Trial of Eculizumab in Relapsing NMO Patients
NCT02003144
A Randomized Controlled Trial of Eculizumab in AQP4 Antibody-positive Participants With NMO (PREVENT Study)
NCT01892345
Eculizumab For Acute Attack of Neuromyelitis Optica Spectrum Disorder
NCT06673394
Efficacy, Safety, PK, PD, and ADA of Eculizumab in Chinese Adults With NMOSD
NCT06724809
Detailed Description
Dive into the extended narrative that explains the scientific background, objectives, and procedures in greater depth.
Conditions
See the medical conditions and disease areas that this research is targeting or investigating.
Study Design
Understand how the trial is structured, including allocation methods, masking strategies, primary purpose, and other design elements.
NON_RANDOMIZED
SINGLE_GROUP
TREATMENT
NONE
Study Groups
Review each arm or cohort in the study, along with the interventions and objectives associated with them.
Eculizumab
All participants will receive open-label eculizumab by intravenous infusion during the Primary Treatment Period, starting on Day 1 and for a total of 52/53 weeks. The dosing regimen will be based on the participant's body weight. As body weight changes during the study, the participant's weight cohort and dose may change accordingly.
After completing the 52/53-week Primary Treatment Period, participants may continue receiving eculizumab in the Extension Treatment Period for 104 weeks.
Eculizumab
Following a weight-based weekly dose of eculizumab during an induction phase, participants will receive weight-based doses of eculizumab every 2 weeks during the Primary Treatment Period and Extension Treatment Period.
Interventions
Learn about the drugs, procedures, or behavioral strategies being tested and how they are applied within this trial.
Eculizumab
Following a weight-based weekly dose of eculizumab during an induction phase, participants will receive weight-based doses of eculizumab every 2 weeks during the Primary Treatment Period and Extension Treatment Period.
Other Intervention Names
Discover alternative or legacy names that may be used to describe the listed interventions across different sources.
Eligibility Criteria
Check the participation requirements, including inclusion and exclusion rules, age limits, and whether healthy volunteers are accepted.
Inclusion Criteria
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
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.
ALL
No
Sponsors
Meet the organizations funding or collaborating on the study and learn about their roles.
Alexion Pharmaceuticals, Inc.
INDUSTRY
Responsible Party
Identify the individual or organization who holds primary responsibility for the study information submitted to regulators.
Locations
Explore where the study is taking place and check the recruitment status at each participating site.
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
Clinical Trial Site
Genoa, , Italy
Research Site
Genova, , Italy
Research Site
Rome, , Italy
Clinical Trial Site
Rome, , Italy
Research Site
Yokohama, , Japan
Clinical Trial Site
Yokohama, , Japan
Clinical Trial Site
Seoul, , South Korea
Clinical Trial Site
Barcelona, , Spain
Research Site
Esplugues de Llobregat, , Spain
Clinical Trial Site
Seville, , Spain
Countries
Review the countries where the study has at least one active or historical site.
Provided Documents
Download supplemental materials such as informed consent forms, study protocols, or participant manuals.
Document Type: Study Protocol
Document Type: Statistical Analysis Plan
Related Links
Access external resources that provide additional context or updates about the study.
Other Identifiers
Review additional registry numbers or institutional identifiers associated with this trial.
2019-001829-26
Identifier Type: EUDRACT_NUMBER
Identifier Source: secondary_id
ECU-NMO-303
Identifier Type: -
Identifier Source: org_study_id
More Related Trials
Additional clinical trials that may be relevant based on similarity analysis.