Neuromyelitis Optica Spectrum Disorder MedDRA version: 21.1 Level: PT Classification code 10077875 Term: Neuromyelitis optica spectrum disorder System Organ Class: 10029205 - Nervous system disorders
Conditions
Sponsors
Alexion Pharmaceuticals, Inc.
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1. Patient must be 18 years of age or older, at the time of signing the informed consent. Anti-AQP4 Ab-positive and a diagnosis of NMOSD as defined by the 2015 international consensus diagnostic criteria (Wingerchuk, 2015). 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 = 7 4. Patients who enter the trial receiving supportive IST (eg, corticosteroids, azathioprine [AZA], mycophenolate mofetil [MMF], methotrexate [MTX], and tacrolimus [TAC]) for the prevention of relapse, either in combination or monotherapy, must be on a stable dosing regimen of adequate duration prior to Screening with no plan to change the dose during the study period as follows: a. If patients who enter the study are receiving AZA, they must have been on AZA for = 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 the detailed list of inclusion criteria in the protocol. Are the trial subjects under 18? no Number of subjects for this age range: F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 50 F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range 5
Exclusion criteria
Exclusion criteria: 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 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 10. Pregnant, breastfeeding, or intending to conceive during the course of the study Please see the detailed list of exclusion criteria in the protocol.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To evaluate the effect of ravulizumab on adjudicated On-Trial Relapses in adult patients with NMOSD;Secondary Objective: To evaluate the safety of ravulizumab in adult patients with NMOSD To evaluate the effect of ravulizumab on adjudicated annualized response rate (ARR) in adult patients with NMOSD To characterize the PK of ravulizumab in adult patients with NMOSD To characterize the pharmacodynamics (PD) of ravulizumab in adult patients with NMOSD;Primary end point(s): Time to first adjudicated On-Trial Relapse and relapse risk reduction;Timepoint(s) of evaluation of this end point: On-Trial Relapses will be monitored throughout the study. The Investigator or a qualified designee will review the signs and symptoms of a potential relapse with the patient in detail at each visit. | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): 1. Adjudicated On Trial ARR 2. Clinically important worsening in expanded disability status scale (EDSS) 3. Change from baseline in EuroQoL-5D (EQ-5D) 4. Clinically important change in Hauser ambulation index (HAI) 5. Change in serum ravulizumab concentration over the study duration 6. Change in serum free C5 concentration over the study duration 7. Presence and titer of ADAs over the study duration;Timepoint(s) of evaluation of this end point: 1. Time of on-trial relapse 2. Throughout the study 3. Throughout the study 4. Throughout the study 5. Throughout the study 6. Throughout the study 7. Throughout the study | — |
Countries
Australia, Austria, Canada, Denmark, France, Germany, Italy, Japan, Korea, Republic of, Netherlands, Poland, Russian Federation, Spain, Turkey, United Kingdom, United States
Contacts
Public ContactRosa Enrique
Alexion Pharma Spain
Outcome results
None listed