Clinical trial NCT05966467: Registry of Patients With AQP4+ NMOSD Treated With Alexion C5 Inhibitor Therapies
Official title: A Long-Term, Prospective, Observational, Registry of Patients With Anti-Aquaporin 4 Antibody-Positive (AQP4+) Neuromyelitis Optica Spectrum Disorder (NMOSD) Treated With Alexion Complement Component 5 (C5) Inhibitor Therapies (ALXN-C5IT)
Study type: OBSERVATIONAL
Overall status: RECRUITING
Conditions: Neuromyelitis Optica Spectrum Disorder
NMOSD
AQP4+ NMOSD
Keywords: Neuromyelitis Optica Spectrum Disorder
NMOSD
C5 Inhibitor Therapies
AQP4+ NMOSD
Patient Registry
Alexion C5IT
Soliris
Lead sponsor: Alexion Pharmaceuticals, Inc.
Sponsor class: INDUSTRY
First posted: 2023-07-28
Enrollment: 122
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Participants with a diagnosis of AQP4+ NMOSD who are being treated with ALXN-C5IT at the time of enrollment.

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.

Eligibility criteria: 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.

Primary outcomes:
measure: Annualized Relapse Rate (ARR)
timeFrame: Up to approximately 5 years
Secondary outcomes:
Other outcomes:
Locations:
city: Washington
country: United States
facility: Clinical Trial Site
state: District of Columbia
city: Boston
country: United States
facility: Research Site
state: Massachusetts
city: Chapel Hill
country: United States
facility: Clinical Trial Site
state: North Carolina
city: Columbus
country: United States
facility: Clinical Trial Site
state: Ohio
city: Plano
country: United States
facility: Clinical Trial Site
state: Texas
city: Milwaukee
country: United States
facility: Clinical Trial Site
state: Wisconsin
city: Buenos Aires
country: Argentina
facility: Clinical Trial Site
city: Buenos Aires
country: Argentina
facility: Clinical Trial Site
city: Buenos Aires
country: Argentina
facility: Clinical Trial Site
city: Buenos Aires
country: Argentina
facility: Clinical Trial Site
city: Burnaby
country: Canada
facility: Clinical Trial Site
state: British Columbia
city: Beijing
country: China
facility: Clinical Trial Site
city: Guangzhou
country: China
facility: Clinical Trial Site
city: Henan
country: China
facility: Clinical Trial Site
city: Shanghai
country: China
facility: Clinical Trial Site
city: Sichuan
country: China
facility: Clinical Trial Site
city: Wuhan
country: China
facility: Clinical Trial Site
city: Dresden
country: Germany
facility: Clinical Trial Site
city: Essen
country: Germany
facility: Clinical Trial Site
city: Munchen
country: Germany
facility: Clinical Trial Site
city: Naples
country: Italy
facility: Clinical Trial Site
city: Fukuoka
country: Japan
facility: Clinical Trial Site
city: Kawasaki
country: Japan
facility: Clinical Trial Site
city: Miyagi
country: Japan
facility: Clinical Trial Site
city: Tokyo
country: Japan
facility: Clinical Trial Site
city: Daejeon
country: Korea, Republic of
facility: Clinical Trial Site
city: Gyeonggi-do
country: Korea, Republic of
facility: Clinical Trial Site
city: Seoul
country: Korea, Republic of
facility: Clinical Trial Site
city: Seoul
country: Korea, Republic of
facility: Clinical Trial Site
city: Seoul
country: Korea, Republic of
facility: Clinical Trial Site
city: Seoul
country: Korea, Republic of
facility: Clinical Trial Site
city: Seoul
country: Korea, Republic of
facility: Clinical Trial Site
city: Seoul
country: Korea, Republic of
facility: Clinical Trial Site
Study officials:
affiliation: Hospital Aleman
name: Pablo Lopez
role: PRINCIPAL_INVESTIGATOR
References: