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

June 17, 2026 updated by: Alexion Pharmaceuticals, Inc.

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)

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.

Study Overview

Status

Recruiting

Conditions

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.

Study Type

Observational

Enrollment (Estimated)

122

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Name: Alexion Pharmaceuticals, Inc. (Sponsor)
Phone Number: 1-855-752-2356
Email: clinicaltrials@alexion.com

Study Locations

Argentina
- - Buenos Aires, Argentina
    - Recruiting
    - Clinical Trial Site
    - Contact:
      
      Veronica Tkachuk
  - Buenos Aires, Argentina
    - Not yet recruiting
    - Clinical Trial Site
    - Contact:
      
      Daniel Munoz, MD
Canada
- British Columbia
  - Burnaby, British Columbia, Canada
    - Recruiting
    - Clinical Trial Site
    - Contact:
      
      Galina Vorobeychik, MD
China
- - Beijing, China
    - Not yet recruiting
    - Clinical Trial Site
    - Contact:
      
      Fudong Shi
  - Guangzhou, China
    - Not yet recruiting
    - Clinical Trial Site
    - Contact:
      
      Wei Qiu
  - Henan, China
    - Not yet recruiting
    - Clinical Trial Site
    - Contact:
      
      Yue Huang
  - Shanghai, China
    - Not yet recruiting
    - Clinical Trial Site
    - Contact:
      
      Qian Cao
  - Sichuan, China
    - Not yet recruiting
    - Clinical Trial Site
    - Contact:
      
      Hong Tan
  - Wuhan, China
    - Not yet recruiting
    - Clinical Trial Site
    - Contact:
      
      Daishi Tian, MD
Germany
- - Dresden, Germany
    - Recruiting
    - Clinical Trial Site
    - Contact:
      
      Tjalf Ziemssen
  - Essen, Germany
    - Recruiting
    - Clinical Trial Site
    - Contact:
      
      Refik Pul
  - München, Germany
    - Not yet recruiting
    - Clinical Trial Site
    - Contact:
      
      Achim Berthele
Italy
- - Naples, Italy
    - Recruiting
    - Clinical Trial Site
    - Contact:
      
      Roberta Lanzillo
Japan
- - Fukuoka, Japan
    - Recruiting
    - Clinical Trial Site
    - Contact:
      
      Katsuhisa Masaki
  - Kawasaki, Japan
    - Recruiting
    - Clinical Trial Site
    - Contact:
      
      Kenzo Sakurai
  - Miyagi, Japan
    - Recruiting
    - Clinical Trial Site
    - Contact:
      
      Ichiro Nakashima
  - Tokyo, Japan
    - Not yet recruiting
    - Clinical Trial Site
    - Contact:
      
      Jin Nakahara
South Korea
- - Daejeon, South Korea
    - Recruiting
    - Clinical Trial Site
    - Contact:
      
      Eun Hee Sohn
  - Gyeonggi-do, South Korea
    - Recruiting
    - Clinical Trial Site
    - Contact:
      
      Ho Jin Kim
  - Seoul, South Korea
    - Recruiting
    - Clinical Trial Site
    - Contact:
      
      Jun-Soon Kim
United States
- District of Columbia
  - Washington D.C., District of Columbia, United States, 20010
    - Recruiting
    - Clinical Trial Site
    - Contact:
      
      Benjamin Osborne
- Massachusetts
  - Boston, Massachusetts, United States, 02114
    - Recruiting
    - Research Site
- North Carolina
  - Chapel Hill, North Carolina, United States, 27599
    - Not yet recruiting
    - Clinical Trial Site
    - Contact:
      
      Irena Dujmovic-Basuroski
- Ohio
  - Columbus, Ohio, United States, 43215
    - Recruiting
    - Clinical Trial Site
    - Contact:
      
      Jacqueline Nicholas
- Texas
  - Plano, Texas, United States, 75024
    - Recruiting
    - Clinical Trial Site
    - Contact:
      
      Annette Okai
- Wisconsin
  - Milwaukee, Wisconsin, United States, 53215
    - Recruiting
    - Clinical Trial Site
    - Contact:
      
      Bhupendra Khatri

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

Adult
Older Adult

Accepts Healthy Volunteers

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.

Description

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.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Annualized Relapse Rate (ARR) Time Frame: Up to approximately 5 years	The ARR is defined as the total number of relapses divided by the total number of participant-years.	Up to approximately 5 years

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Alexion Pharmaceuticals, Inc.

Investigators

Principal Investigator: Pablo Lopez, Hospital Aleman

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

February 1, 2024

Primary Completion (Estimated)

September 27, 2029

Study Completion (Estimated)

September 27, 2029

Study Registration Dates

First Submitted

July 7, 2023

First Submitted That Met QC Criteria

July 21, 2023

First Posted (Actual)

July 28, 2023

Study Record Updates

Last Update Posted (Actual)

June 18, 2026

Last Update Submitted That Met QC Criteria

June 17, 2026

Last Verified

June 1, 2026

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

D9282C00003

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

YES

IPD Sharing Supporting Information Type

STUDY_PROTOCOL
SAP
CSR

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.