Neuromyelitis Optica Spectrum Disorder
Conditions
Interventions
Eligible patients will enroll into the study to receive intravenous infusions
of ravulizumab.
The ravulizumab dose for each patient will be based on body weight.
The dosing regimen consists of a lo
Sponsors
Alexion Pharmaceuticals
Eligibility
Age
18 Years to 99 Years
Inclusion criteria
Inclusion criteria: Participants are eligible to be included in the study only if all of the following criteria apply: 1. Patient must be 18 years of age or older, at the time of signing the informed consent. 2. At least 1 attack or relapse in the last 12 months prior to the Screening Period NOTE: Patients with a single life-time attack will be considered to satisfy inclusion criterion #3 if the attack occurred in the last 12 months. 3. Expanded Disability Status Scale (EDSS) score = 6 months and have been on a stable dose for >= 2 months prior to Screening. b. If patients who enter the study are receiving other ISTs (eg, MMF, MTX, or TAC), they must have been on the IST for >= 3 months and have been on a stable dose for >= 4 weeks prior to Screening. c. If patients who enter the study are receiving oral corticosteroids, they must have been on a stable dose for >= 4 weeks prior to Screening. d. If a patient enters the trial receiving oral corticosteroid(s) with or without other IST(s), the daily corticosteroid dose must be no more than prednisone 20 mg/day (or equivalent) prior to Screening. 5. Vaccinated against N. meningitidis within 3 years prior to, or at the time of, initiating ravulizumab. Patients 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. Please see detailed list of inclusion criteria in the protocol
Exclusion criteria
Exclusion criteria: Patients are excluded from the study if any of the following criteria apply: 1. History of N. meningitidis infection. 2. Human immunodeficiency virus (HIV) infection (evidenced by HIV-1 or HIV-2 antibody titer) 3. History of unexplained infections 4. Active systemic bacterial, viral, or fungal infection within 14 days prior to study drug administration on Day 1 5. Previously or currently treated with a complement inhibitor. 6. Use of rituximab within 3 months prior to Screening 7. Use of mitoxantrone within 3 months prior to Screening 8. Use of Intravenous Immunoglobulin (IVIg) within 3 weeks prior to Screening Prior/Concurrent Clinical Study Experience 9. Participation in any other investigational drug study or exposure to an investigational drug or device within 30 days of Screening or 5 half-lives of the study drug, whichever is greater Other Exclusions 10. Pregnant, breastfeeding, or intending to conceive during the course of the study Please see detailed list of exclusion criteria in the protocol.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| • Time to first adjudicated On-Trial Relapse and relapse risk reduction | — |
Secondary
| Measure | Time frame |
|---|---|
| • Incidence of treatment-emergent adverse events (TEAEs), Treatment-emergent serious adverse events (TESAEs), and TEAEs leading to study drug discontinuation • Adjudicated On-Trial ARR (analyzed relapse rate) • Clinically important worsening in expanded disability status scale (EDSS) • Change from baseline in EuroQoL-5D (EQ-5D) • Clinically important change in Hauser ambulation index (HAI) • Change in serum ravulizumab concentration over the study duration • Change in serum free C5 concentration over the study duration • Presence and titer of anti-drug antibodies (ADAs) over the study duration | — |
Countries
Netherlands
Outcome results
None listed