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| ID | Type | Description | Link |
|---|---|---|---|
| 2020-003272-41 | EudraCT Number | ||
| 2023-508842-17-00 | EU Trial (CTIS) Number |
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This study is researching the experimental drugs called pozelimab and cemdisiran, and cemdisiran monotherapy. The study is focused on patients with generalized Myasthenia Gravis (gMG). Myasthenia gravis is a disease that causes weakness and fatigue in muscles in the body because the nerves and muscles are not communicating properly.
The aim of the study is to see how effective pozelimab and cemdisiran are when used in combination and when pozelimab and cemdisiran are used alone for patients with gMG.
The study is looking at several other research questions, including:
DBTP- Double Blind Treatment Period (24 weeks) ETP - Extension Treatment Period (28 weeks) OLTP- Open Label Treatment Period (68 weeks) FUP-Off treatment Follow Up Period (52 weeks)
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Group 1 | Experimental | Placebo in DBTP; Re-randomized to Combination or Cemdisiran in ETP and OLTP |
|
| Group 2 | Experimental | Combination regimen throughout the study. For participants who have not reached week 108 will receive cemdisiran monotherapy in the OLTP. |
|
| Group 3 | Experimental | Cemdisiran throughout the study |
|
| Group 4 | Experimental | Pozelimab monotherapy in DBTP followed by combination in ETP and OLTP. For participants who have not reached week 108 will receive cemdisiran monotherapy in the OLTP. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Pozelimab + Cemdisiran | Drug | Subcutaneous administration as described in the protocol |
|
| Measure | Description | Time Frame |
|---|---|---|
| Change in Myasthenia Gravis-Activities of Daily Living (MG-ADL) total score | The total MG-ADL score ranges from 0 to 24 points, with higher scores indicating greater functional impairment and disability | From baseline to week 24 |
| Measure | Description | Time Frame |
|---|---|---|
| Change from baseline in Quantitative Myasthenia Gravis (QMG) score | QMG total scores range from 0 to 39, with higher scores representing greater impairment | Week 24 |
| Achievement of a ≥3-point reduction (improvement) in MG-ADL total score |
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Key Inclusion Criteria:
Key Exclusion Criteria:
NOTE: Other protocol-defined Inclusion/ Exclusion Criteria apply
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| Name | Affiliation | Role |
|---|---|---|
| Clinical Trial Management | Regeneron Pharmaceuticals | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| HonorHealth Neurology 2018 | Scottsdale | Arizona | 85251 | United States | ||
| University of California Irvine |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 42030965 | Derived | Vu T, Habib AA, Jacob S, Mantegazza R, Murai H, Vissing J, Shaibani A, Levine T, Hussain Y, Meisel A, Adamczak-Ratajczak A, Ilkowski J, Sgobbi P, Guerreiro A, Luo S, Pavani R, Chaudhari U, Jalali N, DeVeaux M, Moore W, Meagher KA, Burczynski ME, Kokate S, Sherman S, Pawaskar D, Singh N, Souttou A, Sirulnik A, Perlee L, Yancopoulos GD, Howard JF Jr; NIMBLE Trial Investigators. Efficacy and safety of cemdisiran siRNA in myasthenia gravis (NIMBLE): a double-blind, randomised, placebo-controlled, phase 3 trial. Lancet. 2026 May 2;407(10540):1712-1725. doi: 10.1016/S0140-6736(26)00690-2. Epub 2026 Apr 21. |
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All Individual Patient Data (IPD) that underlie publicly available results will be considered for sharing
When Regeneron has:
Qualified researchers can submit a proposal for access to individual patient or aggregate level data from a Regeneron-sponsored clinical trial through Vivli. Regeneron's Independent Research Request Evaluation Criteria can be found at: https://www.regeneron.com/sites/default/files/Regeneron-External-Data-Sharing-Policy-and-Independent-Research-Request-Evaluation-Criteria.pdf
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| Cemdisiran | Drug | SC administration as described in the protocol |
|
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| Placebo | Other | SC administration as described in the protocol |
|
| Pozelimab | Drug | SC administration as described in the protocol |
|
|
| From baseline to week 24 |
| Achievement of a ≥5-point reduction (improvement) in QMG total score | From baseline to week 24 |
| Achievement of a consistent response on the MG-ADL | A participant with a ≥2-point reduction (improvement) in MG-ADL total score on 2 or more consecutive assessments spanning 4 or more weeks during the DBTP | From baseline to week 24 |
| Achievement of Minimal Symptom Expression (MSE) | Score of 0 to 1 on the MG-ADL | Week 24 |
| Change from baseline in the Myasthenia Gravis Composite (MGC) total score | MGC score ranges from 0 to 50, with higher score indicating higher impairment | Week 24 |
| Change from baseline in Myasthenia Gravis Quality of Life (MG-QOL15r) total score | Total score ranges from 0 to 30 points; a higher score represents greater impairment | Week 24 |
| Achievement of a ≥2-, 4-, 5-, 6-, 7-, 8-, 9-, or 10-point reduction on MG-ADL total score | From baseline to week 24 |
| Achievement of a ≥3-, 4-, 6-, 7-, 8-, 9-, or 10-point reduction on QMG | From baseline to week 24 |
| Incidence and severity of Treatment-Emergent Adverse Events (TEAEs) in participants treated with pozelimab + cemdisiran, cemdisiran monotherapy or placebo | Through week 24 |
| Incidence and severity of Serious Adverse Events (SAEs) in participants treated with pozelimab + cemdisiran, cemdisiran monotherapy or placebo | Through week 24 |
| Incidence and severity of Adverse Events of Special Interest (AESIs) in participants treated with pozelimab + cemdisiran, cemdisiran monotherapy or placebo | Through week 24 |
| Concentrations of total pozelimab in serum | Through study duration, approximate 172 weeks |
| Concentrations of total Complement component 5 (C5) in plasma | Through study duration, approximate 172 weeks |
| Concentrations of cemdisiran and its metabolites in plasma | Through study duration, approximate 172 weeks |
| Incidence of treatment-emergent Anti-Drug Antibodies (ADAs) to pozelimab over time | Through study duration, approximately 172 weeks |
| Incidence of treatment-emergent ADAs to cemdisiran over time | Through study duration, approximate 172 weeks |
| Change in total complement hemolysis activity assay (CH50) over time | Through study duration, approximately 172 weeks |
| Percent change in CH50 over time | Through study duration, approximately 172 weeks |
| Irvine |
| California |
| 92697 |
| United States |
| University of Southern California | Los Angeles | California | 90033 | United States |
| Colorado Springs Neurological Associates | Colorado Springs | Colorado | 80907-5307 | United States |
| SFM Clinical Research, LLC | Boca Raton | Florida | 33487 | United States |
| Diverse Clinical Research | Miami | Florida | 33175 | United States |
| Aqualane Clinical Research | Naples | Florida | 34105 | United States |
| Neurological Services of Orlando | Orlando | Florida | 32806 | United States |
| Medsol Clinical Research Center Inc | Port Charlotte | Florida | 33952 | United States |
| University of South Florida Morsani Center for Advanced Healthcare | Tampa | Florida | 33612 | United States |
| NorthShore University Health System | Glenview | Illinois | 60026-1301 | United States |
| St. Elizabeth's Hospital | O'Fallon | Illinois | 62269 | United States |
| Northwest Neurology Ltd. - Clinedge - PPDS | Rolling Meadows | Illinois | 60080 | United States |
| Wayne State University School of Medicine | Detroit | Michigan | 48201 | United States |
| Dayton Center for Neurological Disorders | Centerville | Ohio | 45459 | United States |
| University of Cincinnati Gardner Neuroscience Institute | Cincinnati | Ohio | 45219 | United States |
| Penn Medicine University City | Philadelphia | Pennsylvania | 19104 | United States |
| National Neuromuscular Research Institute | Austin | Texas | 78759 | United States |
| Nerve and Muscle Center of Texas | Houston | Texas | 77030 | United States |
| Universitair Ziekenhuis Antwerpen | Edegem | Antwerp | 2650 | Belgium |
| AZ Sint-Lucas | Ghent | Oost-Vlaanderen | 9000 | Belgium |
| IMV Pesquisa Neurologica | Porto Alegre | Rio Grande do Sul | 90110-000 | Brazil |
| Jordy Sinapse Medicina LTDA | Itapevi | São Paulo | 06655-250 | Brazil |
| Faculdade de Medicina Do ABC | Santo André | São Paulo | 09060-870 | Brazil |
| Pseg Centro de Pesquisa Clinica S.A | São Paulo | 04038-002 | Brazil |
| Hospital das Clinicas da Faculdade de Medicina da Universidade de Sao Paulo | São Paulo | 05403-010 | Brazil |
| University of Alberta | Edmonton | Alberta | T6G 2X8 | Canada |
| London Health Sciences Centre | London | Ontario | N6A 5A5 | Canada |
| Toronto General Hospital | Toronto | Ontario | M5G2C4 | Canada |
| Peking Union Medical College Hospital | Beijing | Beijing Municipality | 100032 | China |
| Fujian Medical University Union Hospital | Fuzhou | Fujian | 350001 | China |
| Guangdong Hospital of Traditional Chinese Medicine | Guangdong | Guangdong | 510120 | China |
| Nanfang Hospital Southern Medical University | Guangzhou | Guangdong | 510120 | China |
| The University of Hong Kong-Shenzhen Hospital | Shenzhen | Guangdong | 518053 | China |
| The First Affiliated Hospital of Guangzhou University of Chinese Medicine | Guangzhou | Guangzhou | 510405 | China |
| Hainan General Hospital | Haikou | Hainan | 570311 | China |
| Renmin Hospital of Wuhan University - Hubei Provincial People's Hospital | Wuhan | Hubei | 430060 | China |
| Xiangya Hospital Central South University | Changsha | Hunan | 410008 | China |
| The First Hospital of Jilin University | Changchun | Jilin | 130000 | China |
| Qilu Hospital of Shandong University (Qingdan) | Qingdao | Shandong | 266035 | China |
| Huashan Hospital Fudan University | Shanghai | Shanghai Municipality | 201107 | China |
| The Second Affiliated Hospital of the Chinese Peoples' Liberation Army Air Force Medical University | Xi’an | Shanxi | 710032 | China |
| Sichuan Provincial People's Hospital | Chengdu | Sichuan | 610072 | China |
| Tianjin Medical University General Hospital | Tianjin | Tianjin Municipality | 300050 | China |
| Aalborg Universitetshospital | Aalborg | North Denmark | 09000 | Denmark |
| Aarhus University Hospital | Aarhus N | 8200 | Denmark |
| Rigshospitalet, Copenhagen | Copenhagen | 2100 | Denmark |
| Odense Universitetshospital | Odense | 05000 | Denmark |
| Centre Hospitalier Universitaire de Nice | Nice | Alpes Maritimes | 6002 | France |
| CHU Bicetre | Le Kremlin-Bicêtre | 94275 | France |
| Centre Hospitalier Regional Universitaire (CHRU) de Nancy | Nancy | 54035 | France |
| Hopital de la Pitie Salpetriere | Paris | 75013 | France |
| Israeli-Georgian Medical Research Clinic Healthycore, LTD | Tbilisi | 0112 | Georgia |
| LTD New Hospitals | Tbilisi | 0114 | Georgia |
| Pineo Medical Ecosystem | Tbilisi | 0114 | Georgia |
| LTD National Center of Urology Named after L. Managadze | Tbilisi | 0144 | Georgia |
| Multiprofile Clinic Consilium Medulla | Tbilisi | 0186 | Georgia |
| Friedrich Baur Institute | München | Bavaria | 80336 | Germany |
| University Hospital Essen | Essen | North Rhine-Westphalia | 45127 | Germany |
| Universitatsklinikum Munster | Münster | North Rhine-Westphalia | 48149 | Germany |
| Universitatsklinikum Leipzig | Leipzig | Saxony | 04103 | Germany |
| Charite - Universitatsmedizin Berlin | Berlin | 10117 | Germany |
| Universitatsklinikum Jena | Jena | 07747 | Germany |
| Government General Hospital, Guntur | Guntur | Andhra Pradesh | 522001 | India |
| Institute of Neurosciences | Surat | Gujarat | 395001 | India |
| Nizam's Institute of Medical Sciences (NIMS) | Panjagutta | Hyderabad | 500082 | India |
| Kasturba Medical College (KMC) | Udupi | Karnataka | 576104 | India |
| Amrita Institute of Medical Sciences and Research Centre (AIMS) | Kochi | Kerala | 682041 | India |
| Deenanath Mangeshkar Hospital and Research Centre | Pune | Maharashtra | 411004 | India |
| Seth G.S. Medical College & K.E.M. Hospital, Mumbai | Pārel | Mumbai | 400012 | India |
| All India Institute of Medical Sciences New Delhi | New Delhi | National Capital Territory of Delhi | 110029 | India |
| Polakulath Narayanan Renai Medicity Multi Specialty Hospital, Kochi | Kochi | Punjab | 141008 | India |
| Christian Medical College & Hospital | Ludhiana | Punjab | 141008 | India |
| Apex Hospital | Jaipur | Rajasthan | 302017 | India |
| City Neuro Centre, Hyderabad | Hyderabad | Telangana | 500034 | India |
| Bangalore Medical College and Research Institute (BMCRI) - Victoria Hospital | Bangalore | 560002 | India |
| Postgraduate Institute of Medical Education & Research | Chandigarh | 160012 | India |
| Azienda Ospedaliera Sant'andrea | Rome | Lazio | 00189 | Italy |
| Fondazione IRCCS Di Rilievo Nazionale Istituto Nazionale Neurologico Carlo Besta | Milan | Lombardy | 20133 | Italy |
| Azienda Ospedaliero Universitaria Pisana | Pisa | Tuscany | 56126 | Italy |
| Azienda Ospedaliera Papa Giovanni XXIII | Bergamo | 24127 | Italy |
| Universita' Degli Studi di Roma La Sapienza | Roma | 00161 | Italy |
| Kobe City Medical Center General Hospital | Kobe | Hyōgo | 650-0047 | Japan |
| Kochi Medical School Hospital | Nankoku | Kochi | 783-8505 | Japan |
| Okinawa National Hospital | Ginowan-Shi | Okinawa | 901-2214 | Japan |
| Okinawa Prefectural Nanbu Medical Center and Children's Medical Center | Shimajiri | Okinawa | 901-1193 | Japan |
| Saitama Medical University, Saitama Medical Center | Kawagoe | Saitama | 350-8550 | Japan |
| Medical Hospital of Tokyo Medical and Dental University | Bunkyo | Tokyo | 113-8519 | Japan |
| Tokyo Medical University Hospital | Shinjuku | Tokyo | 160-0023 | Japan |
| Nihon University Itabashi Hospital | tabashi City | Tokyo | 173-8610 | Japan |
| Yamaguchi University Hospital | Ube | Yamaguchi | 755-8505 | Japan |
| Chiba University Hospital | Chiba | 260-8677 | Japan |
| Hiroshima City Hiroshima Citiz | Hiroshima | 730-8518 | Japan |
| Japanese Red Cross Osaka Hospital | Osaka | 543-8555 | Japan |
| Clinical Research Center Spolka z Ograniczona Odpowiedzialnoscia Medic-R Sp.k | Poznan | Greater Poland Voivodeship | 61-731 | Poland |
| NZOZ Neuro-kard | Poznan | Greater Poland Voivodeship | 61-853 | Poland |
| Centrum Neurologii Klinicznej, Krakowska Akademia Neurologii | Krakow | Lesser Poland Voivodeship | 31-505 | Poland |
| NeuroProtect | Warsaw | Mazovian | 01-684 | Poland |
| Uniwersyteckie Centrum Kliniczne WUM, Centralny Szpital Kliniczny | Warsaw | Mosavian | 02-097 | Poland |
| Gdanski Uniwersytet Medyczny | Gdansk | Pomeranian Voivodeship | 80-952 | Poland |
| University Clinical Centre Nis | Niš | 700461 | Serbia |
| Kyungpook National University Chilgok Hospital | Daegu | 41404 | South Korea |
| Samsung Medical Center | Seoul | 06351 | South Korea |
| Hospital de la Santa Creu i Sant Pau | Barcelona | 08041 | Spain |
| Hospital Universitari i Politecnic La Fe | Valencia | 46026 | Spain |
| Chang Gung Memorial Foundation Kaohsiung Branch | Kaohsiung City | 833 | Taiwan |
| China Medical University | Taichung | 40447 | Taiwan |
| National Cheng Kung University Hospital | Tainan | 701 | Taiwan |
| Shin Kong Wu Ho Su Memorial Hospital | Taipei | 111 | Taiwan |
| Chang Gung Memorial Hospital at Linkou | Taoyuan | 333 | Taiwan |
| Kocaeli University Hospital | Izmit | Van | 41380 | Turkey (Türkiye) |
| Dokuz Eylul University Medical Faculty | Izmir | 35340 | Turkey (Türkiye) |
| Ondokuz Mayis Universitesi Tip Fakultesi Hastanesi | Samsun | 55239 | Turkey (Türkiye) |
| Karadeniz Technical University Farabi Hospital | Trabzon | 61080 | Turkey (Türkiye) |
| Sheffield Teaching Hospital NHS Foundation Trust | Sheffield | South Yorkshire | 511 9NE | United Kingdom |
| University Hospital Birmingham | Birmingham | B15 2TH | 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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