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| ID | Type | Description | Link |
|---|---|---|---|
| 2024-514133-38-00 | EU Trial (CTIS) Number |
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The purpose of this study is to evaluate the efficacy and safety of efgartigimod PH20 SC given by a pre-filled syringe in adult patients with ocular myasthenia gravis. The study consists of a part A (approximately 7 weeks) and a part B (up to 2 years). In part A, half of the participants will receive efgartigimod PH20 SC and the other half will receive placebo. In part B, all participants will receive efgartigimod PH20 SC. The participants will be in the study for about up to 2 years and 12 weeks.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Efgartigimod PH20 SC in part A+B | Experimental | Participants receiving efgartigimod PH20 SC during part A and part B |
|
| Placebo PH20 SC in Part A + Efgartigimod PH20 SC in Part B | Experimental | Participants receiving placebo PH20 SC in Part A and Efgartigimod PH20 SC in Part B |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Efgartigimod PH20 SC | Combination Product | Subcutaneous efgartigimod PH20 SC given by prefilled syringe |
|
| Measure | Description | Time Frame |
|---|---|---|
| MGII (PRO) ocular score change from baseline to day 29 in part A | The Myasthenia Gravis Impairment Index (MGII) is a scoring tool measuring disease severity. It consists of 22 patient-reported outcomes (PRO) and 6 physical examinations (PE). The Ocular PRO score varies between 0 and 18. The higher the score, the more severe the disease. | Up to 29 days |
| Measure | Description | Time Frame |
|---|---|---|
| MGII (PRO+PE) ocular score change from baseline to day 29 in part A | The Myasthenia Gravis Impairment Index (MGII) is a scoring tool measuring disease severity. It consists of 22 patient-reported outcomes (PRO) and 6 physical examinations (PE). The Ocular PRO score varies between 0 and 18. The ocular PE score varies between 0 and 5. The higher the score, the more severe the disease. | Up to 29 days |
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Inclusion Criteria:
Exclusion Criteria:
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| HonorHealth Neurology - Bob Bové Neuroscience Institute | Scottsdale | Arizona | 85251 | United States | ||
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| Placebo PH20 SC | Other | Subcutaneous placebo PH20 SC given by prefilled syringe |
|
| MG-ADL ocular domain score change from baseline to day 29 in part A | The Myasthenia Gravis Activities of Daily Living (MG-ADL) scale assesses MG symptoms and their effects on daily activities. The ocular score varies between 0 and 16 (with 16 the most severe). | Up to 29 days |
| MGII total score change from baseline to day 29 in part A | The Myasthenia Gravis Impairment Index (MGII) is a scoring tool measuring disease severity. It consists of 22 patient-reported outcomes (PRO) and 6 physical examinations (PE). The score varies between 0 and 84. The higher the score, the more severe the disease. | Up to 29 days |
| MGII (PE) ocular score change from baseline to day 29 in part A | The Myasthenia Gravis Impairment Index (MGII) is a scoring tool measuring disease severity. It consists of 6 physical examinations (PE). The score varies between 0 and 5. The higher the score, the more severe the disease. | Up to 29 days |
| MG-ADL total score change from baseline to day 29 in part A | The Myasthenia Gravis Activities of Daily Living (MG-ADL) scale assesses MG symptoms and their effects on daily activities. The score varies between 0 and 24 (with 24 the most severe). | Up to 29 days |
| MGII ocular scores changes (PRO) from baseline in part A+B | The Myasthenia Gravis Impairment Index (MGII) is a scoring tool measuring disease severity. It consists of 22 patient-reported outcomes (PRO) and 6 physical examinations (PE). The Ocular PRO score varies between 0 and 18. The ocular PE score varies between 0 and 5. The higher the score, the more severe the disease. | Up to 111 weeks |
| Incidence of (serious) adverse events | Up to 111 weeks |
| MG-QoL15r total score changes from baseline in part A+B | The Myasthenia Gravis Quality of Life 15-item scale revised (MG-QoL15r) questionnaire is a patient-reported instrument that measures the impact of MG symptoms on Quality of Life. The score varies between 0 and 30 (with 30 the lowest quality of life). | Up to 111 weeks |
| NEI VFQ-25 score changes from baseline in part A+B | The 25-item National Eye Institute Visual Function Questionnaire (NEI VFQ-25) is a 25-item vision-targeted questionnaire. The score varies between 0 and 100 (with 0 being the worst score) | Up to 111 weeks |
| Percent change from baseline in total IgG levels over time in part A+B | Up to 111 weeks |
| Percent change from baseline in AChR-Ab levels in AChR-Ab seropositive participants over time in part A+B | Up to 111 weeks |
| Incidence of Anti-Drug Antibodies and Neutralizing Antibodies against efgartigimod in part A+B | Up to 111 weeks |
| Incidence of antibodies and Neutralizing Antibodies against rHuPH20 in part A+B | Up to 111 weeks |
| Loma Linda University Health |
| Loma Linda |
| California |
| 92354 |
| United States |
| USC Roski Eye Institute - Los Angeles | Los Angeles | California | 90033 | United States |
| University of California San Francisco | San Francisco | California | 94143 | United States |
| Neurology Offices of South Florida | Boca Raton | Florida | 33428-4221 | United States |
| SFM Clinical Research, LLC | Boca Raton | Florida | 33487-2768 | United States |
| Neurology Associates PA | Maitland | Florida | 32751 | United States |
| University of South Florida | Tampa | Florida | 33620 | United States |
| Northwestern Memorial Hospital | Chicago | Illinois | 60611 | United States |
| University of California Irvine | Columbia | Maryland | 21044 | United States |
| Massachusetts General Hospital | Boston | Massachusetts | 02114 | United States |
| Henry Ford Health System | Detroit | Michigan | 48202 | United States |
| Robert Wood Johnson Medical School | New Brunswick | New Jersey | 08901 | United States |
| University of North Carolina at Chapel Hill | Chapel Hill | North Carolina | 27514 | United States |
| Duke Neurological Disorders Clinic | Durham | North Carolina | 27710 | United States |
| Cleveland Clinic | Cleveland | Ohio | 44195 | United States |
| Martha Morehouse Medical plaza | Columbus | Ohio | 43221 | United States |
| Penn Medicine University City | Philadelphia | Pennsylvania | 19104 | United States |
| National Neuromuscular Research Institute | Austin | Texas | 78759-8402 | United States |
| Houston Methodist Neurological Institute | Houston | Texas | 77030 | United States |
| Box Hill Hospital | Box Hill | 3128 | Australia |
| The Alfred Hospital | Melbourne | 3004 | Australia |
| Gold Coast University Hospital | Southport | 4215 | Australia |
| Universitätsklinikum AKH Wien | Vienna | 1090 | Austria |
| UZ Leuven | Leuven | 3000 | Belgium |
| Hôpital de la Citadelle | Liège | 4000 | Belgium |
| Maritime Neurology | Halifax | B3R 1V9 | Canada |
| London Health Sciences Centre | London | N6A 5A5 | Canada |
| Centre Hospitalier de l'Universite de Montreal | Montreal | H2X 0A9 | Canada |
| Ottawa Hospital Research Institute | Ottawa | K1Y 4E9 | Canada |
| Hopital de L'enfant Jesus | Québec | G1J 1Z4 | Canada |
| Toronto General Hospital | Toronto | M5G 2C4 | Canada |
| Xiangya Hospital Central South University | Changsha | 410008 | China |
| Sichuan Academy of Medical Sciences & Sichuan Provincial People's Hospital | Chengdu | 610072 | China |
| The First Affiliated Hospital of Chongqing Medical University | Chongqing | 400016 | China |
| Fujian Medical University Union Hospital | Fuzhou | 350001 | China |
| Guangdong Province Traditional Chinese Medical Hospital | Guangzhou | 510120 | China |
| The First Affiliated Hospital of Guangzhou University Chinese Medicine | Guangzhou | 510405 | China |
| Qilu Hospital of Shandong University | Jinan | 250012 | China |
| The First Affiliated Hospital of Nanchang University - Xianghu Campus | Nanchang | 330000 | China |
| Huashan Hospital Fudan University | Shanghai | 200040 | China |
| Shijiazhuang People's Hospital - Fangbeilu Campus | Shijiazhuang | 050011 | China |
| Tongji Hospital Tongji Medical College Huazhong University of Science and Technology | Wuhan | 430030 | China |
| Renmin Hospital of Wuhan University - Main Campus | Wuhan | 430060 | 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 | 532 03 | Czechia |
| Aarhus Universitetshospital | Aarhus N | 8200 | Denmark |
| Rigshospitalet | Copenhagen | 2100 | Denmark |
| Turun Yliopistollinen Keskussairaala | Turku | 20521 | Finland |
| Hôpital Fondation Adolphe de Rothschild | Paris | 75019 | France |
| Petre Sarajishvili Institute of Neurology | Tbilisi | 112 | Georgia |
| New Hospitals | Tbilisi | 114 | Georgia |
| Pineo Medical Ecosystem | Tbilisi | 114 | Georgia |
| LLC Caucasus Medical Centre | Tbilisi | 186 | Georgia |
| Charité - Universitätsmedizin Berlin | Berlin | 13353 | Germany |
| Universität Georg August | Göttingen | 37075 | Germany |
| Eginitio Hospital | Athens | 115 28 | Greece |
| University General Hospital of Patras | Pátrai | 265 04 | Greece |
| ASST Papa Giovanni XXIII - Ospedale Papa Giovanni XXIII | Bergamo | 24127 | Italy |
| IRCCS Istituto dell Scienze Neurologiche di Bologna | Bologna | 40139 | Italy |
| Ospedale San Raffaele S.r.l. | Milan | 20132 | Italy |
| Fondazione IRCCS Istituto Neurologico Carlo Besta | Milan | 20133 | Italy |
| AORN Antonio Cardarelli | Naples | 80131 | Italy |
| Azienda Ospedaliero-Universitaria Sant'Andrea | Roma | 00189 | Italy |
| Chiba University Hospital | Chūōku | 260-8677 | Japan |
| Kyushu University Hospital | Fukuoka | 812-8582 | Japan |
| General Hanamaki Hospital | Hanamaki-shi | 025-0075 | Japan |
| Hiroshima University Hospital | Hiroshima | 734-8551 | Japan |
| Saitama Medical Center | Kawagoe | 350-0844 | Japan |
| National Hospital Organization Utano National Hospital | Kyoto | 616-8255 | Japan |
| IUHW Narita Hospital | Narita | 286-8520 | Japan |
| National Hospital Organization Hokkaido Medical Center | Sapporo | 063-0005 | Japan |
| Kindai University Hospital | Sayama | 589-8511 | Japan |
| Tokyo Medical University Hospital | Shinjuku-Ku | 160-0023 | Japan |
| Osaka University Hospital | Suita-Shi | 565-0871 | Japan |
| Leids Universitair Medisch Centrum | Leiden | 2333 ZA | Netherlands |
| MICS Centrum Medyczne Bydgoszcz | Bydgoszcz | 85-065 | Poland |
| Centrum Medyczne Neurologia Slaska | Katowice | 40-689 | Poland |
| SP ZOZ Szpital Uniwersytecki | Krakow | 31-503 | Poland |
| Krakowska Akademia Neurologii | Krakow | 31-505 | Poland |
| CLINIREM Sp z o.o. | Lublin | 20-064 | Poland |
| ULS de Santa Maria, EPE - Hospital de Santa Maria | Lisbon | 1649-035 | Portugal |
| ULS de Santo António, EPE - Hospital de Santo António | Porto | 4099-001 | Portugal |
| University Clinical Center of Serbia | Belgrade | 11000 | Serbia |
| University Clinical Center Nis | Niš | 18 000 | Serbia |
| Kyungpook National University Chilgok hospital | Daegu | 41404 | South Korea |
| Korea University Anam Hospital | Seoul | 2841 | South Korea |
| Severance Hospital Yonsei University Health System | Seoul | 3722 | South Korea |
| Hospital Universitario Vall d'Hebron | Barcelona | 08035 | Spain |
| Hospital del Mar | Barcelona | 8003 | Spain |
| Hospital Clinico San Carlos | Madrid | 28040 | Spain |
| Hospital Universitario Ramon y Cajal | Madrid | 28304 | Spain |
| Hospital Universitario de Donostia | San Sebastián | 20014 | Spain |
| Hospital Clinico Universitario de Valencia | Valencia | 46010 | Spain |
| Sahlgrenska Universitetssjukhuset | Gothenburg | 413 45 | Sweden |
| Mediclinic Parkview | Dubai | 51122 | United Arab Emirates |
| Royal Sussex County Hospital | Brighton | BN2 5BE | United Kingdom |
| Panthera Biopartners - Glasgow | Glasgow | G51 4TY | United Kingdom |
| Leeds General Infirmary | Leeds | LS1 3EX | United Kingdom |
| Dementech Neurosciences Clinical Academic Centre | London | W1G 9ST | United Kingdom |
| Southampton General Hospital | Southampton | SO16 6YD | 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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