Clinical trial NCT03920293: Safety and Efficacy Study of Ravulizumab in Adults With Generalized Myasthenia Gravis
Official title: A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Multicenter Study to Evaluate the Safety and Efficacy of Ravulizumab in Complement-Inhibitor-Naïve Adult Patients With Generalized Myasthenia Gravis
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE3
Conditions: Generalized Myasthenia Gravis
Lead sponsor: Alexion Pharmaceuticals, Inc.
Sponsor class: INDUSTRY
First posted: 2019-04-18
Enrollment: 175
Sex: ALL
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: The primary purpose of this study is to evaluate the safety and efficacy of ravulizumab for the treatment of participants with generalized myasthenia gravis (gMG).


Eligibility criteria: Inclusion Criteria:

1. Diagnosed with Myasthenia Gravis at least 6 months (180 days) prior to the date of the Screening Visit as confirmed by specific criteria.
2. Myasthenia Gravis Foundation of America Clinical Classification Class II to IV at screening.
3. MG-ADL profile must be ≥ 6 at screening and randomization (Day 1).
4. Vaccinated against meningococcal infections within 3 years prior to, or at the time of, initiating study drug to reduce the risk of meningococcal infection (N meningitidis).

Exclusion Criteria:

Medical Conditions

1. Any active or untreated thymoma. History of thymic carcinoma or thymic malignancy unless deemed cured by adequate treatment with no evidence of recurrence for ≥ 5 years before screening.
2. History of thymectomy within the 12 months prior to screening.
3. History of N meningitidis infection.
4. Use of the following within the time period specified below:

   * IV immunoglobulin within 4 weeks of randomization
   * Use of plasma exchange within 4 weeks of randomization
   * Use of rituximab within 6 months of screening
5. Participants who have received previous treatment with complement inhibitors (for example, eculizumab).

Primary outcomes:
measure: Change From Baseline In Myasthenia Gravis-Activities Of Daily Living (MG-ADL) Total Score At Week 26
timeFrame: Baseline, Week 26
Secondary outcomes:
measure: Change From Baseline In The Quantitative Myasthenia Gravis (QMG) Total Score At Week 26
timeFrame: Baseline, Week 26
measure: Percentage of Participants With a Quantitative Myasthenia Gravis (QMG) Total Score Reduction of at Least 5 Points At Week 26
timeFrame: Week 26
measure: Change From Baseline In the Revised 15 Component Myasthenia Gravis Quality of Life (MG-QOL15r) At Week 26
timeFrame: Baseline, Week 26
measure: Change From Baseline in Neurological Quality of Life (Neuro-QoL) Fatigue Score at Week 26
timeFrame: Baseline, Week 26
measure: Percentage of Participants With a Myasthenia Gravis Activities of Daily Living (MG-ADL) Total Score Reduction of at Least 3 Points At Week 26
timeFrame: Week 26
Other outcomes:
Locations:
city: Phoenix
country: United States
facility: Research Site
state: Arizona
city: Phoenix
country: United States
facility: Research Site
state: Arizona
city: Phoenix
country: United States
facility: Research Site
state: Arizona
city: Irvine
country: United States
facility: Research Site
state: California
city: Loma Linda
country: United States
facility: Research Site
state: California
city: Palo Alto
country: United States
facility: Research Site
state: California
city: San Francisco
country: United States
facility: Research Site
state: California
city: Sylmar
country: United States
facility: Research Site
state: California
city: Aurora
country: United States
facility: Research Site
state: Colorado
city: Colorado Springs
country: United States
facility: Research Site
state: Colorado
city: Fort Collins
country: United States
facility: Research Site
state: Colorado
city: Fairfield
country: United States
facility: Research Site
state: Connecticut
city: Washington
country: United States
facility: Research Site
state: District of Columbia
city: Jacksonville
country: United States
facility: Research Site
state: Florida
city: Tampa
country: United States
facility: Research Site
state: Florida
city: Chicago
country: United States
facility: Research Site
state: Illinois
city: Rolling Meadows
country: United States
facility: Research Site
state: Illinois
city: Indianapolis
country: United States
facility: Research Site
state: Indiana
city: Kansas City
country: United States
facility: Research Site
state: Kansas
city: Lexington
country: United States
facility: Research Site
state: Kentucky
city: Baltimore
country: United States
facility: Research Site
state: Maryland
city: Boston
country: United States
facility: Research Site
state: Massachusetts
city: Burlington
country: United States
facility: Research Site
state: Massachusetts
city: Ann Arbor
country: United States
facility: Research Site
state: Michigan
city: East Lansing
country: United States
facility: Research Site
state: Michigan
city: Grand Rapids
country: United States
facility: Research Site
state: Michigan
city: Columbia
country: United States
facility: Research Site
state: Missouri
city: Las Vegas
country: United States
facility: Research Site
state: Nevada
city: Las Vegas
country: United States
facility: Research Site
state: Nevada
city: Great Neck
country: United States
facility: Research Site
state: New York
city: New York
country: United States
facility: Research Site
state: New York
city: Chapel Hill
country: United States
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state: North Carolina
city: Charlotte
country: United States
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state: North Carolina
city: Durham
country: United States
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state: North Carolina
city: Cincinnati
country: United States
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state: Ohio
city: Cleveland
country: United States
facility: Research Site
state: Ohio
city: Columbus
country: United States
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state: Ohio
city: Hershey
country: United States
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state: Pennsylvania
city: Philadelphia
country: United States
facility: Research Site
state: Pennsylvania
city: Pittsburgh
country: United States
facility: Research Site
state: Pennsylvania
city: Charleston
country: United States
facility: Research Site
state: South Carolina
city: North Charleston
country: United States
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state: South Carolina
city: Austin
country: United States
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state: Texas
city: Dallas
country: United States
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state: Texas
city: Houston
country: United States
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state: Texas
city: Round Rock
country: United States
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state: Texas
city: Burlington
country: United States
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state: Vermont
city: Richmond
country: United States
facility: Research Site
state: Virginia
city: Morgantown
country: United States
facility: Research Site
state: West Virginia
city: Vienna
country: Austria
facility: Research Site
city: Edmonton
country: Canada
facility: Research Site
state: Alberta
city: London
country: Canada
facility: Research Site
state: Ontario
city: Toronto
country: Canada
facility: Research Site
state: Ontario
city: Montreal
country: Canada
facility: Research Site
state: Quebec
city: Brno
country: Czechia
facility: Research Site
city: Ostrava
country: Czechia
facility: Research Site
city: Praha
country: Czechia
facility: Research Site
city: Arhus C
country: Denmark
facility: Research Site
city: Copenhagen
country: Denmark
facility: Research Site
city: Ålborg
country: Denmark
facility: Research Site
city: Garches
country: France
facility: Research Site
city: Lille
country: France
facility: Research Site
city: Marseille
country: France
facility: Research Site
city: Nice
country: France
facility: Research Site
city: Berlin
country: Germany
facility: Research Site
city: Essen
country: Germany
facility: Research Site
city: Hamburg
country: Germany
facility: Research Site
city: Leipzig
country: Germany
facility: Research Site
city: Münster
country: Germany
facility: Research Site
city: Würzburg
country: Germany
facility: Research Site
city: Haifa
country: Israel
facility: Research Site
city: Tel Aviv
country: Israel
facility: Research Site
city: Bergamo
country: Italy
facility: Research Site
city: Bologna
country: Italy
facility: Research Site
city: Milano
country: Italy
facility: Research Site
city: Napoli
country: Italy
facility: Research Site
city: Roma
country: Italy
facility: Research Site
city: Rome
country: Italy
facility: Research Site
city: Bunkyo-ku
country: Japan
facility: Research Site
city: Chiba-shi
country: Japan
facility: Research Site
city: Fukuoka City
country: Japan
facility: Research Site
city: Hanamaki-shi
country: Japan
facility: Research Site
city: Kawagoe-shi
country: Japan
facility: Research Site
city: Moriguchi-shi
country: Japan
facility: Research Site
city: Nagoya-shi
country: Japan
facility: Research Site
city: Niigata-shi
country: Japan
facility: Research Site
city: Osaka
country: Japan
facility: Research Site
city: Sapporo-shi
country: Japan
facility: Research Site
city: Sapporo-shi
country: Japan
facility: Research Site
city: Sendai-shi
country: Japan
facility: Research Site
city: Shinjuku-ku
country: Japan
facility: Research Site
city: Ube-shi
country: Japan
facility: Research Site
city: Daegu
country: Korea, Republic of
facility: Research Site
city: Seoul
country: Korea, Republic of
facility: Research Site
city: Seoul
country: Korea, Republic of
facility: Research Site
city: Seoul
country: Korea, Republic of
facility: Research Site
city: Seoul
country: Korea, Republic of
facility: Research Site
city: Yangsan-si
country: Korea, Republic of
facility: Research Site
city: Amsterdam
country: Netherlands
facility: Research Site
city: Leiden
country: Netherlands
facility: Research Site
city: Porto
country: Portugal
facility: Research Site
city: Barcelona
country: Spain
facility: Research Site
city: L'Hospitalet de Llobregat
country: Spain
facility: Research Site
city: Madrid
country: Spain
facility: Research Site
city: Murcia
country: Spain
facility: Research Site
city: Zürich
country: Switzerland
facility: Research Site
Study officials:
References: