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| ID | Type | Description | Link |
|---|---|---|---|
| 2024-511796-15-00 | Other Identifier | CTIS number |
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The primary purpose of this study is to measure the efficacy and safety of efgartigimod IV compared to placebo in participants with Acetylcholine Receptor Binding Antibody (AChR-Ab) seronegative Generalized Myasthenia Gravis (gMG). The study consists of a Part A where participants will be randomized to receive either efgartigimod IV or placebo IV and a Part B where participants completing part A will receive open-label efgartigimod IV. Participants will be in the study for up to (approximately) 2.5 years.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Efgartigimod IV | Experimental | Patients receiving efgartigimod IV in both part A and part B |
|
| Placebo | Placebo Comparator | Patients receiving placebo during part A and receiving efgartigimod IV during part B |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Efgartigimod IV | Biological | Intravenous infusion of efgartigimod |
|
| Measure | Description | Time Frame |
|---|---|---|
| MG-ADL total score change from baseline | The Myasthenia Gravis Activities of Daily Living (MG- ADL) scale is an 8-item instrument used to assess MG symptoms and their effects on daily activities. The total score ranges from 0 (normal symptoms) to 24 (most severe symptoms) | Up to 29 days (part A) |
| Measure | Description | Time Frame |
|---|---|---|
| QMG total score change from baseline | The Quantitative myasthenia gravis (QMG) includes 13 items that measure endurance or fatigability and accounts for fluctuations in disease state. The total score ranges from 0 (no disease severity) to 39 (highest disease severity) | Up to 8 weeks (part A) + 2 years (part B) |
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Inclusion Criteria:
The participant is at least 18 years of age and the local legal age of consent for clinical studies when signing the ICF.
The participant is capable of providing signed informed consent and following with protocol requirements.
The participant agrees to use contraceptive measures consistent with local regulations and the women of child-bearing potential (WOCBP) must have a negative serum pregnancy test result at screening and a negative urine pregnancy test result at baseline before receiving the study drug.
The participant has no known weakness in infancy and later develop fatigable weakness after aged 16 years and diagnosed with acquired gMG of both of the following:
The participant is receiving a stable dose of MG therapy before screening that includes acetylcholinesterase (AChE) inhibitors, steroids, or nonsteroidal immunosuppressive therapies (NSISTs) in combination or alone.
Exclusion Criteria:
The complete list of exclusion criteria can be found in the protocol.
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| HonorHealth Neurology - Bob Bove Neuroscience Institute | Scottsdale | Arizona | 85251 | United States | ||
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| Label | URL |
|---|---|
| US patient website | View source |
| US HCP website | View source |
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| Placebo IV | Other | Intravenous infusion of placebo |
|
| Proportion of participants who are both MG-ADL and QMG responders in part A |
| Up to 8 weeks (part A) |
| Proportion of participants with MSE | Minimal symptom expression (MSE) is defined as an MG-ADL total score of 0 or 1 | Up to 8 weeks (part A) + 2 years (part B) |
| Proportion of participants who are MG-ADL responders in part A | The Myasthenia Gravis Activities of Daily Living (MG- ADL) scale is an 8-item instrument used to assess MG symptoms and their effects on daily activities. The total score ranges from 0 (normal symptoms) to 24 (most severe symptoms). | Up to 8 weeks (part A) |
| Proportion of participants who are QMG responders in part A | The Quantitative myasthenia gravis (QMG) includes 13 items that measure endurance or fatigability and accounts for fluctuations in disease state. The total score ranges from 0 (no disease severity) to 39 (highest disease severity) | Up to 8 weeks (part A) |
| Proportion of participants who are early MG-ADL responders in part A | The Myasthenia Gravis Activities of Daily Living (MG- ADL) scale is an 8-item instrument used to assess MG symptoms and their effects on daily activities. The total score ranges from 0 (normal symptoms) to 24 (most severe symptoms). | Up to 8 weeks (part A) |
| MG-ADL total score change from baseline over time in part A and part A+B | The Myasthenia Gravis Activities of Daily Living (MG- ADL) scale is an 8-item instrument used to assess MG symptoms and their effects on daily activities. The total score ranges from 0 (normal symptoms) to 24 (most severe symptoms). | Up to 8 weeks (part A) + 2 years (part B) |
| MG-QoL15r change from baseline over time in part A and part A+B | The Myasthenia Gravis Quality of Life-15 revised (MG-QoL15r) is a patient-reported instrument that measures the impact of MG symptoms on quality of life. The total scores range from 0 (best outcome) to 30 (worst outcome) | Up to 8 weeks (part A) + 2 years (part B) |
| EQ-5D-5L VAS change from baseline over time in part A and part A+B | The EQ-5D-5L is a participant-reported measure of health status developed by the EuroQol Group. The questionnaire comprises 5 dimensions: mobility, self-care, usual activities, pain/discomfort, and anxiety/depression. Current health status is rated on vertical visual analog score (VAS) from 0 to 100, with a score of 0 corresponding to "the worst health you can imagine" and 100 corresponding to "the best health you can imagine." | Up to 8 weeks (part A) + 2 years (part B) |
| Incidence of AEs and SAEs in part A and part A+B | AE: adverse event; SAE: serious adverse event | Up to 8 weeks (part A) + 2 years (part B) |
| Total IgG concentrations percent changes from baseline over time in part A and part A+B | Up to 8 weeks (part A) + 2 years (part B) |
| Loma Linda University Health |
| Fresno |
| California |
| 93701-2234 |
| United States |
| First Choice Neurology Boca Raton | Boca Raton | Florida | 33428 | United States |
| SFM Clinical Research LLC | Boca Raton | Florida | 33487 | United States |
| The Neurology Institute / Healthcare Innovations Institute - Coral Springs | Coral Springs | Florida | 33067 | United States |
| Neurology Associates PA | Maitland | Florida | 32751 | United States |
| Desai Sethi Medical Center | Miami | Florida | 33136 | United States |
| Medsol Clinical Research Center Inc | Port Charlotte | Florida | 33952 | United States |
| BayCare - St. Anthony's Hospital | St. Petersburg | Florida | 33705-1410 | United States |
| University of South Florida (USF) Health - Morsani Center for Advanced Healthcare | Tampa | Florida | 33612 | United States |
| Wellstar - Augusta University Medical Center | Augusta | Georgia | 30912 | United States |
| Kansas University Medical Center - Kansas City | Fairway | Kansas | 66205 | United States |
| Rutgers-Robert Wood Johnson Medical School | New Brunswick | New Jersey | 08901-1962 | United States |
| University of North Carolina - Chapel Hill | Chapel Hill | North Carolina | 27599-7025 | United States |
| Duke University School of Medicine - Duke Early Phase Clinical Research Unit | Durham | North Carolina | 27710 | United States |
| Cleveland Clinic | Cleveland | Ohio | 44195 | United States |
| Erlanger Neuroscience Institute | Chattanooga | Tennessee | 37403-2173 | United States |
| National Neuromuscular Research Institute | Austin | Texas | 78756 | United States |
| University of Washington Medical Center - Montlake | Seattle | Washington | 98195 | United States |
| Hôpital Erasme | Anderlecht | 1070 | Belgium |
| Cliniques Universitaires Saint-Luc | Brussels | 1200 | Belgium |
| AZ Sint-Lucas Gent | Ghent | 9000 | Belgium |
| UZ Leuven | Leuven | 3000 | Belgium |
| Heritage Medical Research Clinic | Calgary | T2N 4Z6 | Canada |
| Genge Partners Inc. | Montreal | H4A 3T2 | Canada |
| Ottawa Hospital - Civic Campus | Ottawa | K1H 8L6 | Canada |
| Xuanwu Hospital Capital Medical University | Beijing | 100053 | China |
| The First Hospital of Jilin University | Changchun | 130021 | China |
| Sichuan Academy of Medical Sciences & Sichuan Provincial People's Hospital | Chongqing | 400016 | China |
| The First Affiliated Hospital of Chongqing Medical University | Chongqing | 400016 | China |
| Fujian Medical University Union Hospital | Fuzhou | 350001 | China |
| The First Affiliated Hospital of Guangxi Medical University | Guangxi | 530021 | China |
| The First Affiliated Hospital of Guangzhou University Chinese Medicine | Guangzhou | 510405 | China |
| Guangdong Province Traditional Chinese Medical Hospital | Guangzhou | 518053 | China |
| The Affiliated Hospital of Guizhou Medical University | Guiyang | 550004 | China |
| Ningbo Medical Center Lihuili Hospital | Ningbo | 315041 | China |
| Qilu Hospital of Shandong University | Qingdao | 266035 | China |
| Huashan Hospital Fudan University | Shanghai | 200040 | China |
| First Hospital of Shanxi Medical University | Taiyuan | 030001 | China |
| Tongji Hospital Tongji Medical College Huazhong University of Science and Technology | Wuhan | 430030 | China |
| Cyprus Institute of Neurology and Genetics | Égkomi | 2371 | Cyprus |
| Fakultni nemocnice Brno | Brno | 625 00 | Czechia |
| Nemocnice Pardubickeho kraje, a.s., Pardubicka nemocnice | Pardubice | 530 03 | Czechia |
| Aarhus Universitetshospital | Aarhus | 8200 | Denmark |
| Rigshospitalet | Copenhagen | 2100 | Denmark |
| Odense Universitetshospital | Odense C | 5000 | Denmark |
| Turun Yliopistollinen Keskussairaala | Turku | 20540 | Finland |
| AP-HM- Hôpital de La Timone | Marseille | 13386 | France |
| CHU de Nice-Hôpital Pasteur | Nice | 06000 | France |
| AP-HP - Hôpital de la Pitié Salpétrière | Paris | 75013 | France |
| West Georgia Medical Center | Kutaisi | 4600 | Georgia |
| Petre Sarajishvili Institute of Neurology | Tbilisi | 0112 | Georgia |
| Pineo Medical Ecosystem | Tbilisi | 0112 | Georgia |
| New Hospitals | Tbilisi | 0114 | Georgia |
| LLC Caucasus Medical Centre | Tbilisi | 0186 | Georgia |
| Charité - Universitätsmedizin Berlin | Berlin | 13353 | Germany |
| Katholisches Klinikum Bochum - St. Josef Hospital | Bochum | 44791 | Germany |
| Universitätsklinikum Würzburg | Würzburg | 97080 | Germany |
| Eginitio Hospital | Athens | 115 28 | Greece |
| University General Hospital ''ATTIKON'' | Chaïdári | 124 62 | Greece |
| University General Hospital of Patras | Pátrai | 265 04 | Greece |
| AHEPA University General Hospital of Thessaloniki | Thessaloniki | 546 36 | Greece |
| Semmelweis Egyetem Genomikai Medicina és Ritka Betegségek Intézete | Budapest | 1082 | Hungary |
| Leiden University Medical Center | Leiden | 2333 ZA | Netherlands |
| Universitair Medisch Centrum Utrecht | Utrecht | 3584 CX | Netherlands |
| Haukeland Universitetssykehus | Bergen | 5021 | Norway |
| Oslo Universitetssykehus HF, Ullevål | Oslo | 424 | Norway |
| MICS Centrum Medyczne Bydgoszcz | Bydgoszcz | 85-065 | Poland |
| Centrum Medyczne Neurologia Slaska | Katowice | 40-689 | Poland |
| Szpital Uniwersytecki w Krakowie | Krakow | 31-503 | Poland |
| Krakowska Akademia Neurologii Sp. z o.o. | Krakow | 31-505 | Poland |
| CLINIREM Sp z o.o. | Lublin | 20-883 | Poland |
| Uniwersytecki Szpital Kliniczny im. Jana Mikulicza Radeckiego we Wroclawiu | Wroclaw | 50-556 | Poland |
| ULS de Santa Maria, EPE - Hospital de Santa Maria | Lisbon | 1150-199 | Portugal |
| ULS de Lisboa Ocidental, EPE - Hospital Egas Moniz | Lisbon | 1349-019 | Portugal |
| Hospital de Santo António - Unidade Local de Saúde de Santo António | Porto | 4050-011 | Portugal |
| Cluj County Emergency Clinical Hospital | Cluj-Napoca | 400012 | Romania |
| Sibiu Emergency County Clinical Hospital | Sibiu | 550166 | Romania |
| King Faisal Specialist Hospital & Research Center | Riyadh | 11211 | Saudi Arabia |
| National Guard Riyadh | Riyadh | 14611 | Saudi Arabia |
| Opsta bolnica MSB - Medicinski Sistemi Beograd | Belgrade | 11000 | Serbia |
| University Clinical Center Kragujevac | Kragujevac | 34000 | Serbia |
| Hospital General Universitario Dr. Balmis | Alicante | 03010 | Spain |
| Hospital Universitario Vall d'Hebron - PPDS | Barcelona | 8035 | Spain |
| Hospital de La Santa Creu i Sant Pau | Barcelona | 8041 | Spain |
| Hospital Universitari Arnau de Vilanova | Lleida | 25198 | Spain |
| Hospital Universitario Ramon y Cajal | Madrid | 28304 | Spain |
| Hospital Clinico Universitario de Santiago | Santiago de Compostela | 15706 | Spain |
| Hospital Universitari i Politecnic La Fe de Valencia | Valencia | 46026 | Spain |
| Panthera Biopartners | Glasgow | G51 4TY | United Kingdom |
| Leeds General Infirmary | Leeds | LS1 3EX | United Kingdom |
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| Release Date | Unrelease Date | Unrelease Date Unknown | Reset Date | MCP Release Number |
|---|---|---|---|---|
| Jun 30, 2026 |
| 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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