Clinical trial NCT04202341: Registry of Participants With Generalized Myasthenia Gravis Treated With Alexion C5 Inhibition Therapies (C5ITs)
Official title: Long-Term, Observational, Global Registry of Patients With Generalized Myasthenia Gravis Who Have Received Treatment With Complement C5 Inhibition Therapies (C5ITs)
Study type: OBSERVATIONAL
Overall status: RECRUITING
Conditions: Generalized Myasthenia Gravis
Keywords: gMG
Soliris
Ultomiris
Alexion C5 Inhibition Therapy
Alexion C5IT
Patient Registry
Generalized Myasthenia Gravis
Alexion Registry
Lead sponsor: Alexion Pharmaceuticals, Inc.
Sponsor class: INDUSTRY
First posted: 2019-12-17
Enrollment: 500
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 gMG who are treated with Alexion C5IT at the time of enrollment, or have ever been treated with Alexion C5IT prior to enrollment, and meet the inclusion criteria and exclusion criteria are eligible for registry participation.

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.

Eligibility criteria: 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with Alexion C5IT at the time of enrollment, including patients previously treated with Soliris or Ultomiris and withdrawn from treatment.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.

Primary outcomes:
measure: Change in Myasthenia Gravis Activities of Daily Living (MG-ADL) Score
timeFrame: Approximately 5 years from enrollment
Secondary outcomes:
Other outcomes:
Locations:
city: Birmingham
country: United States
facility: Clinical Trial Site
state: Alabama
city: Scottsdale
country: United States
facility: Clinical Trial Site
state: Arizona
city: Fresno
country: United States
facility: Clinical Trial Site
state: California
city: Orange
country: United States
facility: Clinical Trial Site
state: California
city: Rancho Mirage
country: United States
facility: Clinical Trial Site
state: California
city: Sylmar
country: United States
facility: Clinical Trial Site
state: California
city: Colorado Springs
country: United States
facility: Clinical Trial Site
state: Colorado
city: Fort Collins
country: United States
facility: Clinical Trial Site
state: Colorado
city: New Haven
country: United States
facility: Clinical Trial Site
state: Connecticut
city: Washington
country: United States
facility: Clinical Trial Site
state: District of Columbia
city: Clearwater
country: United States
facility: Clinical Trial Site
state: Florida
city: Gainesville
country: United States
facility: Clincal Trial Site
state: Florida
city: Jacksonville
country: United States
facility: Clinical Trial Site
state: Florida
city: Tampa
country: United States
facility: Clinical Trial Site
state: Florida
city: Augusta
country: United States
facility: Clinical Trial Site
state: Georgia
city: Iowa City
country: United States
facility: Clinical Trial Site
state: Iowa
city: Lexington
country: United States
facility: Clinical Trial Site
state: Kentucky
city: Boston
country: United States
facility: Clinical Trial Site
state: Massachusetts
city: Lansing
country: United States
facility: Clinical Trial Site
state: Michigan
city: Columbia
country: United States
facility: Clinical Trial Site
state: Missouri
city: New Hyde Park
country: United States
facility: Clinical Trial Site
state: New York
city: Chapel Hill
country: United States
facility: Clinical Trial Site
state: North Carolina
city: Charlotte
country: United States
facility: Clinical Trial Site
state: North Carolina
city: Durham
country: United States
facility: Clinical Trial Site
state: North Carolina
city: Dayton
country: United States
facility: Clinical Trial Site
state: Ohio
city: Philadelphia
country: United States
facility: Clinical Trial Site
state: Pennsylvania
city: Chattanooga
country: United States
facility: Clinical Trial Site
state: Tennessee
city: Knoxville
country: United States
facility: Clinical Trial Site
state: Tennessee
city: Dallas
country: United States
facility: Clinical Trial Site
state: Texas
city: Houston
country: United States
facility: Clinical Trial Site
state: Texas
city: Burlington
country: United States
facility: Clinical Trial Site
state: Vermont
city: Milwaukee
country: United States
facility: Clinical Trial Site
state: Wisconsin
city: Vienna
country: Austria
facility: Clinical Trial Site
city: Brampton
country: Canada
facility: Clinical Trial Site
state: Ontario
city: London
country: Canada
facility: Clinical Trial Site
state: Ontario
city: Montreal
country: Canada
facility: Clinical Trial Site
state: Quebec
city: Chengdu
country: China
facility: Clinical Trial Site
state: Sichuan
city: Hanover
country: Germany
facility: Clinical Trial Site
state: Hannover
city: Milano
country: Italy
facility: Clinical Trial Site
city: Naples
country: Italy
facility: Clinical Trial Site
Study officials:
References: