Neuromyelitis Optica Clinical Trial
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
Verified date | May 2024 |
Source | Alexion Pharmaceuticals, Inc. |
Contact | n/a |
Is FDA regulated | No |
Health authority | |
Study type | Interventional |
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).
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. |
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 |
Lead Sponsor | Collaborator |
---|---|
Alexion Pharmaceuticals, Inc. |
United States, Canada, Germany, Italy, Japan, Korea, Republic of, Spain,
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 |
Status | Clinical Trial | Phase | |
---|---|---|---|
Completed |
NCT02283671 -
Treatment of Multiple Sclerosis and Neuromyelitis Optica With Regulatory Dendritic Cell: Clinical Trial Phase 1 B
|
Phase 1 | |
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 |