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Clinical Trial Details — Status: Terminated

Administrative data

NCT number NCT04155424
Other study ID # ECU-NMO-303
Secondary ID
Status Terminated
Phase Phase 2/Phase 3
First received
Last updated
Start date January 14, 2020
Est. completion date July 31, 2023

Study information

Verified date May 2024
Source Alexion Pharmaceuticals, Inc.
Contact n/a
Is FDA regulated No
Health authority
Study type Interventional

Clinical Trial 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).


Recruitment information / eligibility

Status Terminated
Enrollment 5
Est. completion date July 31, 2023
Est. primary completion date July 31, 2023
Accepts healthy volunteers No
Gender All
Age group 2 Years to 17 Years
Eligibility 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.

Study Design


Related Conditions & MeSH terms


Intervention

Drug:
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.

Locations

Country Name City State
Canada Research Site Calgary Alberta
Canada Clinical Trial Site Edmonton Alberta
Canada Research Site Montreal Quebec
Canada Clinical Trial Site Toronto Ontario
Canada Research Site Toronto Ontario
Germany Research Site Datteln
Germany Research Site Munster
Germany Clinical Trial Site Regensburger Straße Goettingen
Italy Research Site Catania
Italy Research Site Gallarate
Italy Clinical Trial Site Genoa
Italy Research Site Genova
Italy Clinical Trial Site Rome
Italy Research Site Rome
Japan Clinical Trial Site Yokohama
Japan Research Site Yokohama-shi
Korea, Republic of Clinical Trial Site Seoul
Spain Clinical Trial Site Barcelona
Spain Research Site Esplugues de Llobregat
Spain Clinical Trial Site Sevilla
United States Clinical Trial Site Atlanta Georgia
United States Research Site Baltimore Maryland
United States Clinical Trial Site Boston Massachusetts
United States Research Site Boston Massachusetts
United States Clinical Trial Site Chapel Hill North Carolina
United States Research Site Dallas Texas
United States Research Site Hackensack New Jersey
United States Clinical Trial Site Miami Florida
United States Research Site New Brunswick New Jersey
United States Research Site New York New York
United States Clinical Trial Site Philadelphia Pennsylvania
United States Clinical Trial Site Rockville Maryland
United States Research Site Saint Louis Missouri
United States Clinical Trial Site San Francisco California
United States Clinical Trial Site Washington District of Columbia

Sponsors (1)

Lead Sponsor Collaborator
Alexion Pharmaceuticals, Inc.

Countries where clinical trial is conducted

United States,  Canada,  Germany,  Italy,  Japan,  Korea, Republic of,  Spain, 

Outcome

Type Measure Description Time frame Safety issue
Primary Change From Baseline In The Annualized Relapse Rate At 52/53 Weeks Baseline, Week 52/53
Primary Time To First Relapse Baseline up to Week 52/53
Secondary Change From Baseline In Expanded Disability Status Scale (EDSS) Score At 52/53 Weeks In Participants =5 Years Of Age Baseline, Week 52/53
Secondary Change From Baseline In The Hauser Ambulatory Index Score At 52/53 Weeks Baseline, Week 52/53
Secondary Change From Baseline In Pediatric Quality Of Life Inventory (PedsQL) At 52/53 Weeks In Participants =5 Years Of Age Baseline, Week 52/53
Secondary Change From Baseline In Pediatric Quality Of Life Inventory Parent Proxy (PedsQL Parent Proxy) At 52/53 Weeks In Participants < 5 Years Of Age Baseline, Week 52/53
Secondary Change From Baseline In Visual Acuity As Measured By The Snellen Or LEA Symbols Eye Chart Examination At 52/53 Weeks Baseline, Week 52/53
Secondary Change From Baseline In Confrontational Visual Fields As Measured During Ophthalmologic Examination At 52/53 Weeks Baseline, Week 52/53
Secondary Change From Baseline In Color Vision As Measured During Ophthalmologic Examination At 52/53 Weeks Baseline, Week 52/53
Secondary Serum Eculizumab Concentration Over Time Baseline through Week 52/53
Secondary Serum Free Complement Protein 5 Concentrations Over Time Baseline through Week 52/53
Secondary In Vitro Hemolytic Activity Over Time Baseline through Week 52/53
See also
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Recruiting NCT01024985 - Evaluation of Neural Loss in Multiple Sclerosis and Neuromyelitis Optica Using High Resolution Oct N/A
Recruiting NCT04601142 - Association Between the Effect of Glucocorticoid Pulse Therapy on Neuromyelitis Optica (NMO) and Gene Polymorphism
Completed NCT03942952 - PEDIATRIC SONICS: Pediatric Study of Neuropsychology and Imaging in CNS Demyelinating Syndromes.
Recruiting NCT05573711 - Restless Legs Syndrome in People With Neuromyelitis Optica Spectrum Disorder
Not yet recruiting NCT06118398 - Efgartigimod in Acute Neuromyelitis Optica Spectrum Disorders
Recruiting NCT05566769 - Performance and Safety of a Digital Tool for Unsupervised Self-assessment of NMOSD N/A
Withdrawn NCT03829566 - Autologous Transplant To End NMO Spectrum Disorder Phase 2/Phase 3
Recruiting NCT05403138 - Safety and Efficacy of Daratumumab in Patients With Anti-Aquaporin 4 Antibody Positive Neuromyelitis Optica Spectrum Disorders Phase 2/Phase 3
Completed NCT03350633 - Tocilizumab vs Azathioprine in Neuromyelitis Optica Spectrum Disorders Phase 2/Phase 3
Recruiting NCT05356858 - An Open Label Study of the Effects and Safety of Zanubrutinib in NMOSDs Adult Patients Phase 2
Recruiting NCT05982925 - Longitudinal Cortical Demyelination in Multiple Sclerosis and Related Disorders
Active, not recruiting NCT00445367 - Biobank For MS And Other Demyelinating Diseases
Terminated NCT02398994 - A Multicentre randomiSed Controlled TRial of IntraVEnous Immunoglobulin Versus Standard Therapy for Transverse Myelitis Phase 3
Withdrawn NCT02087813 - Pilot Study of alpha1-antitrypsin to Treat Neuromyelitis Optica Relapses Phase 1
Terminated NCT01339455 - Autologous Hematopoietic Stem Cell Transplant in Neuromyelitis Optica Phase 1/Phase 2
Recruiting NCT02021825 - Efficacy and Safety of Mitoxantrone in Patients With Refractory Neuromyelitis Optica and Spectrum Disorders Phase 4
Active, not recruiting NCT04614454 - High Frequency Impulse Therapy for Neuropathic Pain in NMOSD Phase 2
Recruiting NCT03370965 - Optic Neuritis Differential Diagnosis Study N/A
Completed NCT01777412 - Efficacy of Bevacizumab (Avastin) in Treatment of Acute NMO Exacerbations Phase 1