Clinical trial NCT05403541: Phase 3 Study to Assess the Efficacy and Safety of Batoclimab as Induction and Maintenance Therapy in Adult Participants With Generalized Myasthenia Gravis
Official title: A Phase 3, Multi-center, Randomized, Quadruple-blind, Placebo-controlled Study to Assess the Efficacy and Safety of Batoclimab as Induction and Maintenance Therapy in Adult Participants With Generalized Myasthenia Gravis (gMG)
Study type: INTERVENTIONAL
Overall status: ACTIVE_NOT_RECRUITING
Phase: PHASE3
Conditions: Generalized Myasthenia Gravis
Keywords: monoclonal antibody
batoclimab
IMVT-1401
Myasthenia Gravis
acetylcholine receptor
RVT-1401
Lead sponsor: Immunovant Sciences GmbH
Sponsor class: INDUSTRY
First posted: 2022-06-03
Enrollment: 240
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: The purpose of this 4-period study is to confirm the efficacy and safety of batoclimab in participants with gMG. In Period 1, participants will be randomized 1:1:1 to receive batoclimab 680 milligrams (mg) subcutaneously (SC) once a week (QW) or 340 mg SC QW or placebo. The primary efficacy endpoint will be assessed by change in the myasthenia gravis activities of daily living (MG- ADL) score in acetylcholine receptor antibody seropositive (AChRAb+) participants. In Period 2, participants previously treated with batoclimab will be re-randomized to stay on batoclimab (340 mg SC QW or 340 mg SC every two weeks) or receive placebo treatment. The secondary endpoint of maintenance of efficacy will be assessed by change in the MG- ADL score in AChRAb+ participants. Participants demonstrating a response to batoclimab during either Period 1 or 2 may enter the long-term extension (Period 3). Participants who complete Period 3 are eligible to participate in Period 4 (Optional Long-Term extension) according to their treatment assignment in Period 3.


Eligibility criteria: Inclusion Criteria:

1. Are ≥ 18 years of age at the Screening Visit.
2. Have mild to severe gMG by Myasthenia Gravis Foundation of America (MGFA) classification Class II, III, or IVa at the Screening Visit.
3. Have a QMG score ≥ 11 at the Screening and Baseline Visits.
4. Have a MG-ADL score of ≥ 5 at the Screening and Baseline Visits.
5. Additional inclusion criteria are defined in the protocol.

Exclusion Criteria:

1. Have experienced myasthenic crisis within 3 months of the Screening Visit.
2. Have had a thymectomy performed \< 6 months prior to the Screening Visit or have a planned thymectomy during the study period.
3. Have any active or untreated malignant thymoma.
4. Have received any agent or therapy (exclusive of those identified within inclusion criteria) with immunosuppressive properties (e.g., stem cell therapy, chemotherapies) within the past year.
5. Have used anti-FcRn treatment within 3 months prior to the Screening Visit or have a documented history of non-response to prior anti-FcRn treatment.
6. Additional exclusion criteria are defined in the protocol.

Primary outcomes:
measure: Change from Baseline in Myasthenia Gravis Activities of Daily Living (MG-ADL) score in acetylcholine receptor (AChR) Ab seropositive (AChRAb+) participants
timeFrame: Baseline (Day 1) to Week 12
Secondary outcomes:
measure: Change from Baseline in Quantitative Myasthenia Gravis (QMG) score in AChRAb+ participants
timeFrame: Baseline (Day 1) to Week 12
measure: Change from Baseline in MG-ADL score for AChRAb+ randomized withdrawal participants
timeFrame: Baseline (Week 12) to Week 24
measure: Percentage of AChRAb+ participants with greater than equal to (>=) 3-point improvement in QMG score
timeFrame: Up to Week 12
measure: Percentage of AChRAb+ participants achieving MG-ADL score of 0 or 1 by Week 12
timeFrame: Up to Week 12
measure: Change from Baseline in MG-ADL score in AChRAB- (AChRAB negative) participants
timeFrame: Baseline (Day 1) to Week 12
measure: Percentage of Participants with Treatment Emergent Adverse Events (TEAEs)
timeFrame: Up to 76 Weeks
measure: Percentage of Participants With Clinically Significant Changes in Vital Sign Measurements
timeFrame: Up to 76 Weeks
measure: Number of Participants with Clinically Significant Changes in Laboratory Results
timeFrame: Up to 76 Weeks
measure: Percentage of participants with clinical laboratory-related TEAEs or treatment emergent laboratory abnormalities.
timeFrame: Up to 76 Weeks
Other outcomes:
Locations:
city: Phoenix
country: United States
facility: Site Number -1022
state: Arizona
city: Scottsdale
country: United States
facility: Site Number -1029
state: Arizona
city: Carlsbad
country: United States
facility: Site Number -1002
state: California
city: Irvine
country: United States
facility: Site Number -1009
state: California
city: Olive View
country: United States
facility: Site Number - 1012
state: California
city: San Francisco
country: United States
facility: Site Number -1032
state: California
city: Aurora
country: United States
facility: Site Number - 1027
state: Colorado
city: New Haven
country: United States
facility: Site Number - 1025
state: Connecticut
city: Boca Raton
country: United States
facility: Site Number -1017
state: Florida
city: Clearwater
country: United States
facility: Site Number -1007
state: Florida
city: Maitland
country: United States
facility: Site Number -1010
state: Florida
city: Miami
country: United States
facility: Site Number - 1020
state: Florida
city: Orlando
country: United States
facility: Site Number -1019
state: Florida
city: Port Charlotte
country: United States
facility: Site Number - 1028
state: Florida
city: Tampa
country: United States
facility: Site Number - 1015
state: Florida
city: Fairway
country: United States
facility: Site Number -1011
state: Kansas
city: Lexington
country: United States
facility: Site Number -1003
state: Kentucky
city: East Lansing
country: United States
facility: Site Number -1013
state: Michigan
city: Minneapolis
country: United States
facility: Site Number - 1024
state: Minnesota
city: Buffalo
country: United States
facility: Site Number - 1021
state: New York
city: Chapel Hill
country: United States
facility: Site Number - 1018
state: North Carolina
city: Durham
country: United States
facility: Site Number - 1008
state: North Carolina
city: Cleveland
country: United States
facility: Site Number -1004
state: Ohio
city: Portland
country: United States
facility: Site Number -1006
state: Oregon
city: Philadelphia
country: United States
facility: Site Number - 1023
state: Pennsylvania
city: Charleston
country: United States
facility: Site Number - 1030
state: South Carolina
city: Memphis
country: United States
facility: Site Number - 1026
state: Tennessee
city: Austin
country: United States
facility: Site Number -1001
state: Texas
city: Dallas
country: United States
facility: Site Number -1016
state: Texas
city: Houston
country: United States
facility: Site Number - 1034
state: Texas
city: Round Rock
country: United States
facility: Site Number - 1014
state: Texas
city: Murray
country: United States
facility: Site Number -1031
state: Utah
city: Charlottesville
country: United States
facility: Site Number -1005
state: Virginia
city: Buenos Aires
country: Argentina
facility: Site Number - 5002
city: Buenos Aires
country: Argentina
facility: Site Number - 5001
city: Rosario
country: Argentina
facility: Site Number - 5003
city: Ribeirão Preto
country: Brazil
facility: Site Number - 5501
city: Rio De Janeiro
country: Brazil
facility: Site Number- 5503
city: Edmonton
country: Canada
facility: Site Number -2001
state: Alberta
city: Vancouver
country: Canada
facility: Site Number - 2003
state: British Columbia
city: Toronto
country: Canada
facility: Site Number - 2002
state: Ontario
city: Montreal
country: Canada
facility: Site Number - 2004
state: Quebec
city: Tbilisi
country: Georgia
facility: Site Number -8002
city: Tbilisi
country: Georgia
facility: Site Number -8004
city: Tbilisi
country: Georgia
facility: Site Number -8001
city: Tbilisi
country: Georgia
facility: Site Number -8003
city: Tbilisi
country: Georgia
facility: Site Number - 8005
city: Berlin
country: Germany
facility: Site Number - 6507
city: Bochum
country: Germany
facility: Site Number - 6506
city: Hamburg
country: Germany
facility: Site Number - 6501
city: Jena
country: Germany
facility: Site Number - 6503
city: Leipzig
country: Germany
facility: Site Number -6504
city: Würzburg
country: Germany
facility: Site Number -6502
city: Budapest
country: Hungary
facility: Site Number -7553
city: Kistarcsa
country: Hungary
facility: Site Number - 7552
city: Bergamo
country: Italy
facility: Site Number - 6004
city: Brescia
country: Italy
facility: Site Number -6006
city: Genova
country: Italy
facility: Site Number - 6002
city: Milano
country: Italy
facility: Site Number - 6003
city: Napoli
country: Italy
facility: Site Number - 6007
city: Napoli
country: Italy
facility: Site Number -6001
city: Roma
country: Italy
facility: Site Number -6005
city: Asahikawa-shi
country: Japan
facility: Site Number - 4020
city: Fuchu-shi
country: Japan
facility: Site Number - 4014
city: Hanamaki-shi
country: Japan
facility: Site Number - 4002
city: Higashimatsushima
country: Japan
facility: Site Number - 4013
city: Kagawa
country: Japan
facility: Site Number - 4006
city: Kawasaki-shi
country: Japan
facility: Site Number - 4009
city: Koriyama-shi
country: Japan
facility: Site Number - 4012
city: Koshigaya-shi
country: Japan
facility: Site Number - 4007
city: Matsuyama-shi
country: Japan
facility: Site Number - 4011
city: Miyagi
country: Japan
facility: Site Number - 4008
city: Narita-shi
country: Japan
facility: Site Number - 4003
city: Osaka
country: Japan
facility: Site Number - 4010
city: Osaka
country: Japan
facility: Site Number - 4004
city: Osaka
country: Japan
facility: Site Number - 4001
city: Tokyo
country: Japan
facility: Site Number - 4005
city: Toyama-shi
country: Japan
facility: Site Number - 4018
city: Yokohama-shi
country: Japan
facility: Site Number - 4016
city: Yokohama-shi
country: Japan
facility: Site Number - 4019
city: Yokohama
country: Japan
facility: Site Number - 4015
city: Yonago-shi
country: Japan
facility: Site Number - 4017
city: Daegu
country: Korea, Republic of
facility: Site Number -4505
city: Seoul
country: Korea, Republic of
facility: Site Number -4501
city: Mexico City
country: Mexico
facility: Site Number - 2601
city: Gdańsk
country: Poland
facility: Site Number - 3007
city: Katowice
country: Poland
facility: Site Number -3001
city: Krakow
country: Poland
facility: Site Number -3004
city: Krakow
country: Poland
facility: Site Number - 3003
city: Kraków
country: Poland
facility: Site Number -3002
city: Lublin
country: Poland
facility: Site Number - 3008
city: Poznań
country: Poland
facility: Site Number -3006
city: Warszawa
country: Poland
facility: Site Number - 3005
city: Constanta
country: Romania
facility: Site Number -7502
city: Targu Mures
country: Romania
facility: Site Number -7501
city: Timişoara
country: Romania
facility: Site Number -7503
city: Belgrade
country: Serbia
facility: Site Number - 9001
city: Niš
country: Serbia
facility: Site Number - 9002
city: Barcelona
country: Spain
facility: Site Number -3502
city: Barcelona
country: Spain
facility: Site Number -3505
city: Cordoba
country: Spain
facility: Site Number -3504
city: Madrid
country: Spain
facility: Site Number -3501
city: Madrid
country: Spain
facility: Site Number -3503
city: Liverpool
country: United Kingdom
facility: Site Number - 7001
city: Sheffield
country: United Kingdom
facility: Site Number - 7002
Study officials:
References: