Registry for AQP4+ NMOSD Patients on Alexion C5 Inhibitors

This is an observational study, called a registry, for people with AQP4+ NMOSD (Neuromyelitis Optica Spectrum Disorder, a rare autoimmune disease affecting the brain and spinal cord). It's collecting information on how Alexion C5 inhibitor therapies (C5IT) affect patients in the real world. Researchers will look at your medical history and how often you experience relapses (Annualized Relapse Rate or ARR) over approximately five years. They will also gather information on your quality of life. To join, you must be at least 18 years old, have a confirmed diagnosis of AQP4+ NMOSD, and already be receiving an Alexion C5 inhibitor therapy for relapse prevention. The current status of this study is unclear.

Study design
This is an observational registry study with a planned enrollment of 122 participants. It is designed to collect real-world data on patients already receiving treatment.
What's involved
At enrollment, your past medical records and AQP4+ NMOSD treatment history from one year before starting ALXN-C5IT will be reviewed. Data collection will continue for approximately five years from the last participant's enrollment.
Compensation
Not stated in the trial record.
Follow-up
Participants' data will be collected for approximately five years from the day the last participant is enrolled.

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NCT05966467

Registry of Patients With AQP4+ NMOSD Treated With Alexion C5 Inhibitor Therapies

Recruiting
Not specifiedAges 18+Observational
Alexion Pharmaceuticals, Inc.
~122 participants
Updated 2026-06-18 on ClinicalTrials.gov

At a glance

Recruiting sites
23 of 33 listed sites are recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
Annualized Relapse Rate (ARR)
Measured over Up to approximately 5 years
Neuromyelitis Optica Spectrum Disorder
NMOSD
AQP4+ NMOSD
33 sites across 13 states
South Korea8
China6
Argentina4
Japan4
Germany3
District of Columbia1
Massachusetts1
North Carolina1
  • Pablo Lopez · PRINCIPAL_INVESTIGATOR · Hospital Aleman
Alexion Pharmaceuticals, Inc. (Sponsor)
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Eligibility criteria

Inclusion

Participant is ≥ 18 years of age at the time of enrollment in the Registry.
Participant must have a confirmed diagnosis of AQP4+ NMOSD.
At the time of enrollment in the Registry, participants must be receiving treatment with ALXN-C5IT for the purpose of chronic relapse prevention in a manner consistent with the local label. Specifically, they should have received at least 1 dose of eculizumab within 4 weeks prior to enrollment or at least 1 dose of ravulizumab within 12 weeks prior to enrollment.
Participants must have both the following historical data available to be enrolled in the Registry: ALXN-C5IT dosing information since initiation and number and types of relapses from 1 year prior to ALXN-C5IT initiation through Registry enrollment.

Exclusion

Participants currently enrolled in an interventional clinical study for the treatment of AQP4+ NMOSD in which the intervention is a drug.
  • Annualized Relapse Rate (ARR)Up to approximately 5 years

    The ARR is defined as the total number of relapses divided by the total number of participant-years.