anti-AQP4 Ab+ Neuromyelitis Optica Spectrum Disorder(NMOSD)
Conditions
Interventions
Experimental Group:Ravulizumab injection will be administered at a weight-based dose, with an induction dose given on Day 1, followed by a maintenance dose on Day 15, and every 8 weeks (q8w) thereafte
Sponsors
Huashan Hospital, Fudan University
Eligibility
Sex/Gender
All
Age
18 Years to No maximum
Inclusion criteria
Inclusion criteria: 1.Male or female participants = 18 years old at the time of signing the informed consent. 2.Participants with diagnosis of NMOSD as defined by the 2015 international consensus diagnostic criteria (Wingerchuk, 2015). 3.Anti-AQP4 Ab+ at Screening. 4.At least 1 attack or relapse in the last 12 months prior to Screening. Note: Participants with a single lifetime attack will be considered to satisfy Inclusion Criterion 4 if the attack occurred in the last 12 months. 5.EDSS score = 6 months and have been on a stable dose for >= 2 months prior to Screening. (2) If participants who enter the study are receiving other ISTs (eg, MMF, methotrexate, or tacrolimus), they must have been on the IST for >= 3 months and have been on a stable dose for >= 4 weeks prior to Screening. (3) If participants who enter the study are receiving oral corticosteroids, they must have been on a stable dose for = 4 weeks prior to Screening. (4) If a participant enters the study 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. 7.Body weight >= 40 kg. 8.Female participants of childbearing potential must have a negative pregnancy test (serum human chorionic gonadotropin at Screening). Female participants of childbearing potential must practice an effective, reliable, and medically approved contraceptive regimen as outlined in Section 10.5 during the study and for at least 8 months following discontinuation of the study intervention. 9.Male participants are eligible to participate if they agree to the following during the study intervention Treatment Period and for at least 8 months after the last dose of study intervention: Refrain from donating fresh unwashed semen. PLUS, either, be abstinent from heterosexual intercourse as their preferred and usual lifestyle (abstinent on a long-term and persistent basis) and agree to remain abstinent. OR Must agree to use barrier as detailed below: Agree to use a male condom when having sexual intercourse with a woman of childbearing potential who is not currently pregnant. 10.Capable of giving signed informed consent which includes compliance with the requirements and restrictions listed in the ICF and in this protocol. 11.Participants must be willing and able to comply with the protocol requirements for the duration of the study. 12.To reduce the risk of meningococcal infection (N meningitidis), all participants must be vaccinated against meningococcal infections from serogroups A, C, W, Y (and B where available) within the 3 years prior to study intervention administration on Day 1. If vaccination occurs < 2 weeks from the first dose of study intervention, participants must receive appropriate prophylactic antibiotics for at least 2 weeks after the vaccination.
Exclusion criteria
Exclusion criteria: 1.Pregnant, breastfeeding, or intending to conceive during the study. 2.Prior history of N meningitidis infection or unresolved meningococcal disease. 3.Active systemic bacterial, viral, or fungal infection within 14 days prior to study intervention administration on Day 1. 4.Presence of fever >= 38°C within 7 days prior to study intervention administration on Day 1. 5.Hypersensitivity to murine proteins or to one of the excipients of ravulizumab. 6.Any medical condition that, in the opinion of the Investigator, might interfere with the participant’s participation in the study, poses any added risk for the participant, or confounds the assessment of the participants. 7.Known history of HIV, active hepatitis B infection, or active hepatitis C infection. 8.Use of rituximab, inebilizumab, or other B cell-depleting therapy within 3 months prior to Screening. 9.Use of mitoxantrone or satralizumab within 3 months prior to Screening. 10.Use of IVIg within 3 weeks prior to Screening. 11.Previously or currently treated with a complement inhibitor. 12.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 investigational drug, whichever is greater.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Adjudicated On-Trial ARR; | — |
Secondary
| Measure | Time frame |
|---|---|
| Changes in the incidence rates of treatment?emergent adverse events (TEAEs), treatment?emergent serious adverse events (TESAEs), and adverse events of special interest (AESIs) over time.;Clinically significant deterioration in EDSS from baseline;Clinically significant change in Hauser Ambulation Index(HAI) from baseline;Change from baseline to end in EQ-5D (index and VAS);Change from baseline in vital signs and laboratory assessments;To characterize the PD of ravulizumab in adult participants with NMOSD;To characterize the immunogenicity of romolizumab in adult participants with NMOSD;To characterize the PK of ravulizumab in adult participants with NMOSD; | — |
Countries
China
Contacts
Public ContactChao Quan
Huashan Hospital, Fudan University
Outcome results
None listed