AQP4+ NMOSD, Neuromyelitis Optica Spectrum Disorder, NMOSD
Conditions
Keywords
Neuromyelitis Optica Spectrum Disorder, NMOSD, C5 Inhibitor Therapies, AQP4+ NMOSD, Patient Registry, Alexion C5IT, Soliris
Brief summary
Long-term, multicenter, multinational, observational, registry of patients with AQP4+ NMOSD that is designed to collect data on clinical outcomes and safety in patients prescribed Alexion C5 inhibitor therapies (C5IT). The registry will also collect data on patient reported outcomes (PROs), quality of life (QoL), and targeted AQP4+ NMOSD therapies used to provide evidence on the real-world impact of ALXN-C5IT on patients with AQP4+ NMOSD.
Detailed description
At the time of enrollment in the Registry, participant records will be queried for retrospective information about the participant's medical history and AQP4+ NMOSD treatment history for the time period beginning 1 year prior to ALXN-C5IT initiation through Registry enrollment. The duration of data collection for the Registry will be approximately 5 years from the day the last participant is enrolled.
Interventions
None listed
Sponsors
Alexion Pharmaceuticals, Inc.
Study design
Observational model
COHORT
Time perspective
PROSPECTIVE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* 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 criteria
* Participants currently enrolled in an interventional clinical study for the treatment of AQP4+ NMOSD in which the intervention is a drug.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| 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. |
Countries
Argentina, Canada, China, Germany, Italy, Japan, South Korea, United States
Contacts
CONTACTAlexion Pharmaceuticals, Inc. (Sponsor)
PRINCIPAL_INVESTIGATORPablo Lopez
Hospital Aleman
Outcome results
None listed