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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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Batoclimab Induction Dose 1 (Period 1) | Experimental |
| |
| Batoclimab Induction Dose 2 (Period 1) | Experimental |
| |
| Placebo Induction Dose (Period 1) | Placebo Comparator |
| |
| Batoclimab Maintenance Dose 1 (Period 2) | Experimental |
| |
| Batoclimab Maintenance Dose 2 (Period 2) | Experimental |
| |
| Placebo Maintenance Dose (Period 2) | Placebo Comparator |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Batoclimab 680 mg SC weekly | Drug | Batoclimab is a fully human anti-neonatal fragment crystallizable receptor (FcRn) monoclonal antibody |
|
| Measure | Description | Time Frame |
|---|---|---|
| Change from Baseline in Myasthenia Gravis Activities of Daily Living (MG-ADL) score in acetylcholine receptor (AChR) Ab seropositive (AChRAb+) participants | 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. | Baseline (Day 1) to Week 12 |
| Measure | Description | Time Frame |
|---|---|---|
| Change from Baseline in Quantitative Myasthenia Gravis (QMG) score in AChRAb+ participants | 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. | Baseline (Day 1) to Week 12 |
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Inclusion Criteria:
Exclusion Criteria:
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Site Number -1022 | Phoenix | Arizona | 85028 | United States | ||
| Site Number -1029 |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 38268752 | Derived | Benatar M, Wiendl H, Nowak R, Zheng Y, Macias W. Batoclimab as induction and maintenance therapy in patients with myasthenia gravis: rationale and study design of a phase 3 clinical trial. BMJ Neurol Open. 2024 Jan 10;6(1):e000536. doi: 10.1136/bmjno-2023-000536. eCollection 2024. |
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Sponsor Staff is also masked, so the study is Quadruple-blind.
|
| Batoclimab 340 mg SC weekly | Drug | Batoclimab is a fully human anti-neonatal fragment crystallizable receptor (FcRn) monoclonal antibody |
|
|
| Matching Placebo SC | Drug | Placebo |
|
| Batoclimab 340 mg SC bi-weekly | Drug | Batoclimab is a fully human anti-neonatal fragment crystallizable receptor (FcRn) monoclonal antibody |
|
|
| 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 |
| Percentage of AChRAb+ participants achieving MG-ADL score of 0 or 1 by Week 12 | Up to Week 12 |
| Change from Baseline in MG-ADL score in AChRAb- (AChRAb negative) participants | Baseline (Day 1) to Week 12 |
| Scottsdale |
| Arizona |
| 85251 |
| United States |
| Site Number -1002 | Carlsbad | California | 92011 | United States |
| Site Number -1009 | Irvine | California | 92697 | United States |
| Site Number - 1012 | Olive View | California | 91342 | United States |
| Site Number -1032 | San Francisco | California | 94143 | United States |
| Site Number - 1027 | Aurora | Colorado | 80045 | United States |
| Site Number - 1025 | New Haven | Connecticut | 06519 | United States |
| Site Number -1007 | Clearwater | Florida | 33761 | United States |
| Site Number -1010 | Maitland | Florida | 32751 | United States |
| Site Number - 1020 | Miami | Florida | 33136 | United States |
| Site Number - 1019 | Orlando | Florida | 32806 | United States |
| Site Number - 1028 | Port Charlotte | Florida | 33952 | United States |
| Site Number - 1015 | Tampa | Florida | 33612 | United States |
| Site Number -1011 | Fairway | Kansas | 66205 | United States |
| Site Number -1003 | Lexington | Kentucky | 40536 | United States |
| Site Number -1013 | East Lansing | Michigan | 48824 | United States |
| Site Number - 1024 | Minneapolis | Minnesota | 55414 | United States |
| Site Number - 1021 | Buffalo | New York | 14203 | United States |
| Site Number - 1018 | Chapel Hill | North Carolina | 27599 | United States |
| Site Number - 1008 | Durham | North Carolina | 27710 | United States |
| Site Number -1004 | Cleveland | Ohio | 44195 | United States |
| Site Number -1006 | Portland | Oregon | 97239 | United States |
| Site Number - 1023 | Philadelphia | Pennsylvania | 19104 | United States |
| Site Number - 1026 | Memphis | Tennessee | 38120 | United States |
| Site Number -1001 | Austin | Texas | 78759 | United States |
| Site Number -1016 | Dallas | Texas | 75206 | United States |
| Site Number - 1034 | Houston | Texas | 77030 | United States |
| Site Number - 1014 | Round Rock | Texas | 78681 | United States |
| Site Number -1005 | Charlottesville | Virginia | 22908 | United States |
| Site Number - 5002 | Buenos Aires | 4190 | Argentina |
| Site Number - 5001 | Buenos Aires | C1428AQK | Argentina |
| Site Number - 5003 | Rosario | S2000 | Argentina |
| Site Number - 5501 | Ribeirão Preto | 14051-140 | Brazil |
| Site Number- 5503 | Rio de Janeiro | 22640-100 | Brazil |
| Site Number - 2003 | Vancouver | British Columbia | V5Z 1M9 | Canada |
| Site Number - 2002 | Toronto | Ontario | M5G 2C4 | Canada |
| Site Number - 2004 | Montreal | Quebec | H3A 2B4 | Canada |
| Site Number -8002 | Tbilisi | 00114 | Georgia |
| Site Number -8004 | Tbilisi | 00114 | Georgia |
| Site Number -8001 | Tbilisi | 00160 | Georgia |
| Site Number -8003 | Tbilisi | 00186 | Georgia |
| Site Number - 8005 | Tbilisi | 0179 | Georgia |
| Site Number - 6507 | Berlin | 10117 | Germany |
| Site Number - 6505 | Gummersbach | 51643 | Germany |
| Site Number - 6503 | Jena | 7747 | Germany |
| Site Number -6504 | Leipzig | 04103 | Germany |
| Site Number -6502 | Würzburg | 97074 | Germany |
| Site Number -7553 | Budapest | 1082 | Hungary |
| Site Number - 7552 | Kistarcsa | 2143 | Hungary |
| Site Number - 6004 | Bergamo | 24127 | Italy |
| Site Number -6006 | Brescia | 25123 | Italy |
| Site Number - 6002 | Genova | 16132 | Italy |
| Site Number - 6003 | Milan | 20133 | Italy |
| Site Number - 6007 | Naples | 80131 | Italy |
| Site Number -6001 | Naples | 80131 | Italy |
| Site Number - 4020 | Asahikawa-shi | 078-8510 | Japan |
| Site Number - 4014 | Fuchu-shi | 183-0042 | Japan |
| Site Number - 4002 | Hanamaki-shi | 025-0082 | Japan |
| Site Number - 4013 | Higashimatsushima | 355-0005 | Japan |
| Site Number - 4009 | Kawasaki-shi | 216-8511 | Japan |
| Site Number - 4012 | Koriyama-shi | 963-8563 | Japan |
| Site Number - 4007 | Koshigaya-shi | 343-8555 | Japan |
| Site Number - 4011 | Matsuyama | 791-8026 | Japan |
| Site Number - 4008 | Miyagi | 983-8520 | Japan |
| Site Number - 4003 | Narita-shi | 286-8520 | Japan |
| Site Number - 4010 | Osaka | 534-0021 | Japan |
| Site Number - 4001 | Osaka | 589-8511 | Japan |
| Site Number - 4005 | Tokyo | 160-0023 | Japan |
| Site Number - 4016 | Yokohama | 222-0036 | Japan |
| Site Number - 4019 | Yokohama | 230-8765 | Japan |
| Site Number - 4015 | Yokohama | 236-0004 | Japan |
| Site Number - 2601 | Mexico City | 06700 | Mexico |
| Site Number - 3007 | Gdansk | 80-214 | Poland |
| Site Number -3001 | Katowice | 40-123 | Poland |
| Site Number -3004 | Krakow | 31-324 | Poland |
| Site Number - 3003 | Krakow | 31-503 | Poland |
| Site Number -3002 | Krakow | 31-505 | Poland |
| Site Number - 3008 | Lublin | 20-701 | Poland |
| Site Number -3006 | Poznan | 61-731 | Poland |
| Site Number - 3005 | Warsaw | 01-684 | Poland |
| Site Number -7502 | Constanța | 900628 | Romania |
| Site Number -7501 | Târgu Mureş | 540136 | Romania |
| Site Number -7503 | Timișoara | 300723 | Romania |
| Site Number - 9001 | Belgrade | 11000 | Serbia |
| Site Number - 9002 | Niš | 1800 | Serbia |
| Site Number - 4505 | Daegu | 41944 | South Korea |
| Site Number - 4501 | Seoul | 06351 | South Korea |
| Site Number -3502 | Barcelona | 08195 | Spain |
| Site Number -3505 | Barcelona | 8041 | Spain |
| Site Number -3501 | Madrid | 028034 | Spain |
| Site Number - 3503 | Madrid | 28046 | Spain |
| Site Number - 7002 | Sheffield | S102JF | United Kingdom |
| ID | Term |
|---|---|
| D009157 | Myasthenia Gravis |
| ID | Term |
|---|---|
| D020361 | Paraneoplastic Syndromes, Nervous System |
| D009423 | Nervous System Neoplasms |
| D009371 | Neoplasms by Site |
| D009369 | Neoplasms |
| D010257 | Paraneoplastic Syndromes |
| D020274 | Autoimmune Diseases of the Nervous System |
| D009422 | Nervous System Diseases |
| D019636 | Neurodegenerative Diseases |
| D020511 | Neuromuscular Junction Diseases |
| D009468 | Neuromuscular Diseases |
| D001327 | Autoimmune Diseases |
| D007154 | Immune System Diseases |
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