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

Neuromyelitis Optica Spectrum Disorder Clinical Trial

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)

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT05966467
• Other study ID #: D9282C00003
• Status: Recruiting
• Start date: August 30, 2023
• Est. completion date: September 30, 2026

Study information

• Verified date: January 2024
• Source: Alexion Pharmaceuticals, Inc.
• Contact: Alexion Pharmaceuticals, Inc. (Sponsor)
• Phone: 1-855-752-2356
• Email: clinicaltrials[@]alexion.com
• Is FDA regulated: No
• Study type: Observational [Patient Registry]

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 122
• Est. completion date: September 30, 2026
• Est. primary completion date: September 30, 2026
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

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.

Related Conditions & MeSH terms

• Neuromyelitis Optica
• Neuromyelitis Optica Spectrum Disorder

Locations

Country	Name	City	State
United States	Research Site	Boston	Massachusetts

Sponsors (1)

Lead Sponsor	Collaborator
Alexion Pharmaceuticals, Inc.

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Annualized Relapse Rate (ARR)	The ARR is defined as the total number of relapses divided by the total number of participant-years.	Up to approximately 5 years