Registry of Participants With Generalized Myasthenia Gravis Treated With Alexion C5 Inhibition Therapies (C5ITs)

Recruiting

• Conditions: Generalized Myasthenia Gravis

• Registration Number: NCT04202341
• Lead Sponsor: Alexion Pharmaceuticals, Inc.

Brief Summary

Long-term, multicenter, multinational, observational, registry of patients with gMG that is designed to collect data on clinical outcomes and safety in patients prescribed Alexion C5 inhibitor therapies (C5IT) such as eculizumab (Soliris®) and ravulizumab (Ultomiris®).

Detailed Description

At the time of enrollment in the Registry, participant records will be queried for retrospective information about the participants' medical history and gMG disease treatment history. Following enrollment, prospective data collection will be performed using data obtained as part of the routine clinical care and through patient-reported outcome methods in use. Data will be collected using an electronic data capture system. The duration of data collection for the Registry will be up to 5 years from the day of enrollment.

Study Timeline

• Study Start: 2019-12-02
• Study First Submitted: 2019-12-16
• Study First Submitted QC: 2019-12-16
• Study First Posted: 2019-12-17
• Status Verified: 2025-04
• Last Update Submitted: 2025-04-28
• Last Update Posted: 2025-04-29
• Primary Completion: 2029-12-31
• Study Completion: 2029-12-31

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 500

Inclusion Criteria

1. Participants with a diagnosis of gMG who are treated with Alexion C5IT at the time of enrollment, including patients previously treated with Soliris or Ultomiris and withdrawn from treatment.
2. Capable of giving signed informed consent, which includes compliance with the protocol requirements and restrictions.
3. Participants must have myasthenia gravis historical data, such as MG-ADL and MGFA class, available to be enrolled in the Registry.

Exclusion Criteria

1. Participants currently enrolled in an Alexion-sponsored interventional clinical study for treatment of gMG cannot be enrolled in the Alexion gMG Registry while enrolled/participating in the clinical study for gMG therapy.

Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Change in Myasthenia Gravis Activities of Daily Living (MG-ADL) Score	Approximately 5 years from enrollment	The MG-ADL is an eight-item patient-reported outcome measure assessing MG symptoms and functional activities related to activities of daily living.48 Each of the items is scored from 0 (normal) to 3 (most severe), providing a total MG-ADL score ranging from 0 to 24, where higher scores indicate greater severity of symptoms.

Trial Locations

Locations (2)

Clinical Trial Site; 🇮🇹 Naples, Italy

Clincal Trial Site; 🇺🇸 Gainesville, Florida, United States