Phase 3 Study to Assess the Efficacy and Safety of Batoclimab as Induction and Maintenance Therapy in Adult Participants With Generalized Myasthenia Gravis
- Conditions
- Generalized Myasthenia Gravis
- Interventions
- Registration Number
- NCT05403541
- Lead Sponsor
- Immunovant Sciences GmbH
- 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.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ACTIVE_NOT_RECRUITING
- Sex
- All
- Target Recruitment
- 240
- Are ≥ 18 years of age at the Screening Visit.
- Have mild to severe gMG by Myasthenia Gravis Foundation of America (MGFA) classification Class II, III, or IVa at the Screening Visit.
- Have a QMG score ≥ 11 at the Screening and Baseline Visits.
- Have a MG-ADL score of ≥ 5 at the Screening and Baseline Visits.
- Additional inclusion criteria are defined in the protocol.
- Have experienced myasthenic crisis within 3 months of the Screening Visit.
- Have had a thymectomy performed < 6 months prior to the Screening Visit or have a planned thymectomy during the study period.
- Have any active or untreated malignant thymoma.
- 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.
- 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.
- Additional exclusion criteria are defined in the protocol.
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- PARALLEL
- Arm && Interventions
Group Intervention Description Batoclimab Maintenance Dose 2 (Period 2) Batoclimab 340 mg SC bi-weekly - Batoclimab Maintenance Dose 1 (Period 2) Batoclimab 340 mg SC weekly - Batoclimab Induction Dose 1 (Period 1) Batoclimab 680 mg SC weekly - Placebo Induction Dose (Period 1) Matching Placebo SC - Batoclimab Induction Dose 2 (Period 1) Batoclimab 340 mg SC weekly - Placebo Maintenance Dose (Period 2) Matching Placebo SC -
- Primary Outcome Measures
Name Time Method Change from Baseline in Myasthenia Gravis Activities of Daily Living (MG-ADL) score in acetylcholine receptor (AChR) Ab seropositive (AChRAb+) participants Baseline (Day 1) to Week 12 MG-ADL is an 8-item, participant-reported questionnaire that assesses gMG symptoms and their effects on activities of daily living. Each item is assessed on a 4-point scale where a score of 0 represents normal function and a score of 3 represents loss of ability to perform that function. Total score ranges from 0 to 24, with higher scores indicating greater functional impairment and disability.
- Secondary Outcome Measures
Name Time Method Percentage of AChRAb+ participants achieving MG-ADL score of 0 or 1 by Week 12 Up to Week 12 Percentage of participants with clinical laboratory-related TEAEs or treatment emergent laboratory abnormalities. Up to 76 Weeks Change from Baseline in Quantitative Myasthenia Gravis (QMG) score in AChRAb+ participants Baseline (Day 1) to Week 12 QMG is clinician-reported assessment to evaluate muscle weakness in participants with MG. The QMG consists of 13 items ranging from 0 to 3 with 3 being the most severe. Total score ranges from 0 to 39, with higher scores representing greater impairment.
Change from Baseline in MG-ADL score for AChRAb+ randomized withdrawal participants Baseline (Week 12) to Week 24 Percentage of AChRAb+ participants with greater than equal to (>=) 3-point improvement in QMG score Up to Week 12 Change from Baseline in MG-ADL score in AChRAB- (AChRAB negative) participants Baseline (Day 1) to Week 12 Percentage of Participants With Clinically Significant Changes in Vital Sign Measurements Up to 76 Weeks Vital signs, including systolic and diastolic blood pressures, pulse rate, respiratory rate, and temperature will be obtained and recorded at specified timepoints. All vital sign measures will be obtained with the participant in the supine position and having rested for at least 5 minutes.
Number of Participants with Clinically Significant Changes in Laboratory Results Up to 76 Weeks Blood samples will be collected at specified timepoints for the analysis of laboratory parameters including clinical chemistry, hematology and urinalysis.
Percentage of Participants with Treatment Emergent Adverse Events (TEAEs) Up to 76 Weeks An adverse event (AE) is defined as any untoward medical occurrence in a participant who has either been administered a study drug or has undergone study procedures.
Trial Locations
- Locations (105)
Site Number - 1028
🇺🇸Port Charlotte, Florida, United States
Site Number -1016
🇺🇸Dallas, Texas, United States
Site Number - 2002
🇨🇦Toronto, Ontario, Canada
Site Number - 4020
🇯🇵Asahikawa-shi, Japan
Site Number -4501
🇰🇷Seoul, Korea, Republic of
Site Number -7501
🇷🇴Targu Mures, Romania
Site Number - 4015
🇯🇵Yokohama, Japan
Site Number -1002
🇺🇸Carlsbad, California, United States
Site Number -1009
🇺🇸Irvine, California, United States
Site Number -1017
🇺🇸Boca Raton, Florida, United States
Site Number -1007
🇺🇸Clearwater, Florida, United States
Site Number - 1021
🇺🇸Buffalo, New York, United States
Site Number -1010
🇺🇸Maitland, Florida, United States
Site Number - 1012
🇺🇸Olive View, California, United States
Site Number - 1023
🇺🇸Philadelphia, Pennsylvania, United States
Site Number - 6506
🇩🇪Bochum, Germany
Site Number -8002
🇬🇪Tbilisi, Georgia
Site Number - 5501
🇧🇷Ribeirão Preto, Brazil
Site Number - 4014
🇯🇵Fuchu-shi, Japan
Site Number - 4009
🇯🇵Kawasaki-shi, Japan
Site Number - 3008
🇵🇱Lublin, Poland
Site Number - 3005
🇵🇱Warszawa, Poland
Site Number - 7001
🇬🇧Liverpool, United Kingdom
Site Number -3501
🇪🇸Madrid, Spain
Site Number -1011
🇺🇸Fairway, Kansas, United States
Site Number - 9002
🇷🇸Niš, Serbia
Site Number - 4001
🇯🇵Osaka, Japan
Site Number - 4005
🇯🇵Tokyo, Japan
Site Number -8004
🇬🇪Tbilisi, Georgia
Site Number -8003
🇬🇪Tbilisi, Georgia
Site Number -3002
🇵🇱Kraków, Poland
Site Number -7502
🇷🇴Constanta, Romania
Site Number -1029
🇺🇸Scottsdale, Arizona, United States
Site Number - 5002
🇦🇷Buenos Aires, Argentina
Site Number - 5003
🇦🇷Rosario, Argentina
Site Number - 2004
🇨🇦Montreal, Quebec, Canada
Site Number - 8005
🇬🇪Tbilisi, Georgia
Site Number - 4003
🇯🇵Narita-shi, Japan
Site Number - 2601
🇲🇽Mexico City, Mexico
Site Number -3004
🇵🇱Krakow, Poland
Site Number - 7552
🇭🇺Kistarcsa, Hungary
Site Number - 4012
🇯🇵Koriyama-shi, Japan
Site Number - 4011
🇯🇵Matsuyama-shi, Japan
Site Number - 4010
🇯🇵Osaka, Japan
Site Number - 4004
🇯🇵Osaka, Japan
Site Number - 4018
🇯🇵Toyama-shi, Japan
Site Number - 4019
🇯🇵Yokohama-shi, Japan
Site Number - 3003
🇵🇱Krakow, Poland
Site Number -3006
🇵🇱Poznań, Poland
Site Number -7553
🇭🇺Budapest, Hungary
Site Number - 5001
🇦🇷Buenos Aires, Argentina
Site Number - 4013
🇯🇵Higashimatsushima, Japan
Site Number - 4016
🇯🇵Yokohama-shi, Japan
Site Number - 3007
🇵🇱Gdańsk, Poland
Site Number - 9001
🇷🇸Belgrade, Serbia
Site Number -8001
🇬🇪Tbilisi, Georgia
Site Number - 4002
🇯🇵Hanamaki-shi, Japan
Site Number - 4006
🇯🇵Kagawa, Japan
Site Number - 4017
🇯🇵Yonago-shi, Japan
Site Number -7503
🇷🇴Timişoara, Romania
Site Number -2001
🇨🇦Edmonton, Alberta, Canada
Site Number - 2003
🇨🇦Vancouver, British Columbia, Canada
Site Number - 1026
🇺🇸Memphis, Tennessee, United States
Site Number -6006
🇮🇹Brescia, Italy
Site Number - 6003
🇮🇹Milano, Italy
Site Number -6005
🇮🇹Roma, Italy
Site Number -1022
🇺🇸Phoenix, Arizona, United States
Site Number -1032
🇺🇸San Francisco, California, United States
Site Number -1006
🇺🇸Portland, Oregon, United States
Site Number - 1020
🇺🇸Miami, Florida, United States
Site Number - 1024
🇺🇸Minneapolis, Minnesota, United States
Site Number - 1008
🇺🇸Durham, North Carolina, United States
Site Number - 1034
🇺🇸Houston, Texas, United States
Site Number - 1015
🇺🇸Tampa, Florida, United States
Site Number - 1014
🇺🇸Round Rock, Texas, United States
Site Number -1004
🇺🇸Cleveland, Ohio, United States
Site Number -1003
🇺🇸Lexington, Kentucky, United States
Site Number -1013
🇺🇸East Lansing, Michigan, United States
Site Number -1031
🇺🇸Murray, Utah, United States
Site Number -1005
🇺🇸Charlottesville, Virginia, United States
Site Number- 5503
🇧🇷Rio De Janeiro, Brazil
Site Number - 6507
🇩🇪Berlin, Germany
Site Number - 6501
🇩🇪Hamburg, Germany
Site Number -6504
🇩🇪Leipzig, Germany
Site Number - 6503
🇩🇪Jena, Germany
Site Number -6502
🇩🇪Würzburg, Germany
Site Number - 6004
🇮🇹Bergamo, Italy
Site Number - 6002
🇮🇹Genova, Italy
Site Number -6001
🇮🇹Napoli, Italy
Site Number - 6007
🇮🇹Napoli, Italy
Site Number - 4007
🇯🇵Koshigaya-shi, Japan
Site Number - 4008
🇯🇵Miyagi, Japan
Site Number -4505
🇰🇷Daegu, Korea, Republic of
Site Number -3001
🇵🇱Katowice, Poland
Site Number -3504
🇪🇸Cordoba, Spain
Site Number -3502
🇪🇸Barcelona, Spain
Site Number -3505
🇪🇸Barcelona, Spain
Site Number - 7002
🇬🇧Sheffield, United Kingdom
Site Number - 1027
🇺🇸Aurora, Colorado, United States
Site Number -1019
🇺🇸Orlando, Florida, United States
Site Number -3503
🇪🇸Madrid, Spain
Site Number - 1025
🇺🇸New Haven, Connecticut, United States
Site Number - 1018
🇺🇸Chapel Hill, North Carolina, United States
Site Number - 1030
🇺🇸Charleston, South Carolina, United States
Site Number -1001
🇺🇸Austin, Texas, United States