Neuromyelitis Optica Spectrum Disorder Clinical Trial
Official title:
A Phase 2/3, Open-label, Historical-controlled, Single-arm, Multicenter Study to Evaluate the Efficacy, Pharmacokinetics, Pharmacodynamics, and Safety of Ravulizumab in Children and Adolescents With Aquaporin-4 Antibody Positive (AQP4-Ab [+]) Neuromyelitis Optica Spectrum Disorder (NMOSD)
The primary purpose of this study is to evaluate the safety and efficacy of ravulizumab in pediatric participants with Neuromyelitis Optica Spectrum Disorder (NMOSD).
Status | Recruiting |
Enrollment | 12 |
Est. completion date | March 31, 2028 |
Est. primary completion date | March 31, 2026 |
Accepts healthy volunteers | No |
Gender | All |
Age group | N/A and older |
Eligibility | Inclusion Criteria: - Participants must be anti-AQP4 Ab-positive and have a diagnosis of NMOSD as defined by the 2015 international consensus diagnostic criteria. - Complement inhibitor treatment-naïve participants must have had at least 1 attack or relapse in the last 12 months prior to the Screening Period. - Expanded Disability Status Scale (EDSS) score = 7. - Eculizumab-experienced participants must be clinically stable per Investigator for 30 days and have been treated with eculizumab in Study ECU-NMO-303 for at least 90 days prior to screening with no missed doses within 2 months prior to Day 1. - Participants who enter the study receiving supportive IST(s) (eg, corticosteroid, azathioprine [AZA], mycophenolate mofetil [MMF], methotrexate [MTX], tacrolimus [TAC], cyclosporin [CsA], or cyclophosphamide [CYC]) for the prevention of relapse, either in combination or monotherapy, must be on a stable dosing regimen of adequate duration prior to Screening and remain on a stable dosing regimen during the Screening Period. - To reduce the risk of meningococcal infection (Neisseria meningitidis), all participants must be vaccinated against meningococcal infection. - Documented vaccination for Hib and S pneumoniae at least 14 days prior to Day 1 according to national/local guidelines for the applicable age group. Exclusion Criteria: - Use of rituximab within 6 months prior to Day 1. - Currently treated with a biologic medications (other than eculizumab) that may affect immune system functioning, or has stopped treatment with a biologic medication that may affect immune system functioning, and 5 half lives of the medication have not elapsed by the time of the Screening Visit. - Use of intravenous immunoglobulin (IVIg) or plasma exchange (PE) within 3 weeks prior to Screening. - Participation in another investigational drug or investigational device study (other than Study ECU-NMO-303) within 5 half lives of that investigational product (if known) or 30 days before initiation of the first dose of study drug, whichever is longer. - Use of immunomodulatory therapies for multiple sclerosis within 3 months prior to Screening. |
Country | Name | City | State |
---|---|---|---|
Canada | Research Site | Edmonton | Alberta |
Canada | Research Site | Montreal | Quebec |
Canada | Research Site | Toronto | Ontario |
France | Research Site | Le Kremlin-Bicêtre | |
France | Research Site | Marseille | |
France | Research Site | Montpellier Cedex 5 | |
Germany | Research Site | Bochum | |
Germany | Research Site | Giessen | |
Italy | Research Site | Catania | |
Italy | Research Site | Chieti | |
Italy | Research Site | Gallarate | |
Italy | Research Site | Roma | |
Japan | Research Site | Yokohama-shi | |
Korea, Republic of | Research Site | Goyang-si | |
Spain | Research Site | Esplugues de Llobregat | |
United States | Research Site | Boston | Massachusetts |
United States | Research Site | Durham | North Carolina |
United States | Research Site | Miami | Florida |
United States | Research Site | Philadelphia | Pennsylvania |
United States | Research Site | Saint Louis | Missouri |
United States | Research Site | Washington | District of Columbia |
Lead Sponsor | Collaborator |
---|---|
Alexion Pharmaceuticals, Inc. |
United States, Canada, France, Germany, Italy, Japan, Korea, Republic of, Spain,
Type | Measure | Description | Time frame | Safety issue |
---|---|---|---|---|
Primary | Change From Baseline in the Annualized Relapse Rate at Week 50 | Baseline, Week 50 | ||
Primary | Time to First Adjudicated On-trial Relapse through Week 50 | Baseline through Week 50 | ||
Secondary | Change From Baseline in Expanded Disability Status Scale Score At Week 50 | The score ranges are 0 to 10, higher score indicates worse outcome. | Baseline, Week 50 | |
Secondary | Change From Baseline in Hauser Ambulation Index at Week 50 | The score ranges are 0 to 9, higher score indicates worse outcome. | Baseline, Week 50 | |
Secondary | Change From Baseline in Visual Acuity at Week 50 | Baseline, Week 50 | ||
Secondary | Change From Baseline in Confrontational Visual Fields at Week 50 | Baseline, Week 50 | ||
Secondary | Change From Baseline in Color Vision at Week 50 | Baseline, Week 50 | ||
Secondary | Serum Ravulizumab Concentration | Predose and postdose (at end of infusion) on Day 1, Weeks 2, 10, 18, 26, and 42, and predose on Week 50 | ||
Secondary | Change from Baseline in Free Serum Complement Component 5 (C5) Concentration Over Time Through Week 50 | Baseline through Week 50 |
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