NCT05070858Active, not recruitingPhase 3
A Study to Test How Safe Pozelimab and Cemdisiran Combination Therapy and Cemdisiran Alone Are and How Well They Work in Adult Patients With Generalized Myasthenia Gravis
Efficacy and Safety of Pozelimab and Cemdisiran Combination Therapy and Cemdisiran Monotherapy in Patients With Symptomatic Generalized Myasthenia Gravis
Source: ClinicalTrials.gov · Last updated 28 Oct 2026
Brief summary (registry, in English)
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: * What side effects may happen from taking the study drugs * How the study drugs work inside the body * How much of the study drugs are in the blood at different times * Whether the body makes antibodies against pozelimab and cemdisiran (which could make the drugs less effective or could lead to side effects)
- Status (registry)
- Active, not recruiting
- Phase (registry)
- Phase 3
- Study type
- Interventional
- Enrollment
- 288 (actual)
- First submitted
- 27 Sept 2021
- Start date
- 14 Dec 2021
- Primary completion date
- 8 Jul 2025
- Lead sponsor
- Regeneron Pharmaceuticals
- Diseases
- Myasthenia gravis
- NCT number
- NCT05070858
- EU CT number
- 2023-508842-17-00
- EudraCT number
- 2020-003272-41
What you commit to
- Study type: Interventional · Phase 3
- Participants enrolled: 288
- From 14 Dec 2021 to the primary completion date, 8 Jul 2025
- 116 sites in 19 countries
- Visits and tests: ask the trial team, the registry does not list them
Built from the registry fields of ClinicalTrials.gov. It describes the study, not your situation.
Who cannot take part
Copied verbatim from ClinicalTrials.gov and grouped by topic. Only the trial team decides who can take part.
Prior treatment
- Participants with antibody profile that is only positive for Muscle-Specific tyrosine Kinase (MuSK) (MuSK positivity is based on a documented prior history of positive serologic test for antibodies to MuSK or a positive result during screening
- History of thymectomy within 12 months prior to screening or planned during the study
- Not meeting meningococcal vaccination requirements and, at a minimum, documentation of quadrivalent meningococcal vaccination within 5 years prior to randomization and serotype B vaccine (when available) within 3 years prior to randomization as described in the protocol
- Known contraindication to meningococcal vaccines (group ACWY conjugate and group B vaccines) as described in the protocol
Other illness
- History of malignant thymoma (patients with stage 1 may be enrolled), or history of cancer within the past 5 years, except for adequately treated basal cell skin cancer, squamous cell skin cancer, or in situ cervical cancer
- Participants who require antibiotics for meningococcal prophylaxis and have a contraindication, warning, or precaution precluding the use of penicillin class and penicillin-alternative antibiotics planned to be used for prophylaxis, or a history of intolerance leading to the discontinuation of these antibiotics
- Positive hepatitis B surface antigen or hepatitis C virus Ribonucleic Acid (RNA) during screening. NOTE: Cases with unclear interpretation should be discussed with the medical monitor
- History of Human Immunodeficiency Virus (HIV) infection or a positive test at screening per local requirements
- NOTE: Other protocol-defined Inclusion/ Exclusion Criteria apply
Other
- Myasthenic crisis or MGFA Class V within 1 month of screening
Who the study is looking for
- Male or female participants ≥18 years of age at screening (or ≥ legal age of adulthood based on local regulations, whichever is older)
- Participant with documented diagnosis of Myasthenia Gravis (MG) based on medical history and supported by previous evaluations as described in the protocol
- Documented prior history of positive serologic test or a positive result during screening of Anti-acetylcholine Receptor (AChR) antibodies or anti-Lipoprotein Receptor-related Protein 4 (LRP4) antibodies
- Myasthenia Gravis Foundation of America (MGFA) Clinical Classification Class II to IVa at screening
- Myasthenia Gravis-Activities of Daily Living (MG-ADL) score ≥6 at screening. Ocular items should not contribute more than 50% of MG-ADL total score as described in the protocol
- Currently receiving an acetylcholinesterase inhibitor or documented reason for not using acetylcholinesterase inhibitor therapy per investigator
- Currently receiving an Immunosuppressive Therapy (IST) for MG, or documented reason why the participant is not taking an IST per investigator
- If currently receiving an IST, not anticipated to have IST dosage changed before randomization or during Double-Blind Treatment Period (DBTP)
- Willing and able to comply with clinic visits and study-related procedures, including completion of the primary series of the meningococcal vaccinations required per protocol
- For the optional PK sub-study: Must have a Body Mass Index (BMI) ≥ 18.5 kg/m\^2 and ≤ 30 kg/m\^2 at screening
Age in the registry: 18 years to - · Sex: All · Does not accept healthy volunteers
Sites (116)
Sites in registry order. Pick your city on the disease page to sort them by distance.
| Site | City | Country | Site status |
|---|---|---|---|
| Universitair Ziekenhuis Antwerpen Coordinator of this site? Confirm its status | Edegem | Belgium | Unknown status |
| AZ Sint-Lucas Coordinator of this site? Confirm its status | Ghent | Belgium | Unknown status |
| Jordy Sinapse Medicina LTDA Coordinator of this site? Confirm its status | Itapevi | Brazil | Unknown status |
| IMV Pesquisa Neurológica Coordinator of this site? Confirm its status | Porto Alegre | Brazil | Unknown status |
| Faculdade de Medicina do ABC Coordinator of this site? Confirm its status | Santo André | Brazil | Unknown status |
| Hospital das Clinicas da Faculdade de Medicina da Universidade de São Paulo Coordinator of this site? Confirm its status | São Paulo | Brazil | Unknown status |
| PSEG Centro de Pesquisa Clinica S.A. Coordinator of this site? Confirm its status | São Paulo | Brazil | Unknown status |
| University of Alberta Coordinator of this site? Confirm its status | Edmonton | Canada | Unknown status |
| London Health Sciences Centre Coordinator of this site? Confirm its status | London | Canada | Unknown status |
| Toronto General Hospital Coordinator of this site? Confirm its status | Toronto | Canada | Unknown status |
| Peking Union Medical College Hospital Coordinator of this site? Confirm its status | Beijing | China | Unknown status |
| The First Hospital of Jilin University Coordinator of this site? Confirm its status | Changchun | China | Unknown status |
| Xiangya Hospital Central South University Coordinator of this site? Confirm its status | Changsha | China | Unknown status |
| Sichuan Provincial People's Hospital Coordinator of this site? Confirm its status | Chengdu | China | Unknown status |
| Fujian Medical University Union Hospital Coordinator of this site? Confirm its status | Fuzhou | China | Unknown status |
| Guangdong Hospital of Traditional Chinese Medicine Coordinator of this site? Confirm its status | Guangdong | China | Unknown status |
| The First Affiliated Hospital of Guangzhou University of Chinese Medicine Coordinator of this site? Confirm its status | Guangzhou | China | Unknown status |
| Nanfang Hospital, Southern Medical University Coordinator of this site? Confirm its status | Guangzhou | China | Unknown status |
| Hainan General Hospital Coordinator of this site? Confirm its status | Haikou | China | Unknown status |
| Qilu Hospital of Shandong University (Qingdan) Coordinator of this site? Confirm its status | Qingdao | China | Unknown status |
| Huashan Hospital, Fudan University Coordinator of this site? Confirm its status | Shanghai | China | Unknown status |
| The University of Hong Kong-Shenzhen Hospital Coordinator of this site? Confirm its status | Shenzhen | China | Unknown status |
| Tianjin Medical University General Hospital Coordinator of this site? Confirm its status | Tianjin | China | Unknown status |
| Renmin Hospital of Wuhan University - Hubei Provincial People's Hospital Coordinator of this site? Confirm its status | Wuhan | China | Unknown status |
| The Second Affiliated Hospital of the Chinese Peoples' Liberation Army Air Force Medical University Coordinator of this site? Confirm its status | Xi’an | China | Unknown status |
| Charité Universitätsmedizin Berlin Coordinator of this site? Confirm its status | Berlin | Germany | Unknown status |
| University Hospital Essen Coordinator of this site? Confirm its status | Essen | Germany | Unknown status |
| Universitätsklinikum Jena Coordinator of this site? Confirm its status | Jena | Germany | Unknown status |
| Universitatsklinikum Leipzig Coordinator of this site? Confirm its status | Leipzig | Germany | Unknown status |
| Friedrich Baur Institute Coordinator of this site? Confirm its status | München | Germany | Unknown status |
| Universitätsklinikum Münster Coordinator of this site? Confirm its status | Münster | Germany | Unknown status |
| Aalborg Universitetshospital Coordinator of this site? Confirm its status | Aalborg | Denmark | Unknown status |
| Aarhus University Hospital Coordinator of this site? Confirm its status | Aarhus N | Denmark | Unknown status |
| Rigshospitalet, Copenhagen Coordinator of this site? Confirm its status | Copenhagen | Denmark | Unknown status |
| Odense Universitetshospital Coordinator of this site? Confirm its status | Odense | Denmark | Unknown status |
| Hospital de la Santa Creu i Sant Pau Coordinator of this site? Confirm its status | Barcelona | Spain | Unknown status |
| Hospital Universitari i Politécnic La Fe Coordinator of this site? Confirm its status | Valencia | Spain | Unknown status |
| CHU Bicêtre Coordinator of this site? Confirm its status | Le Kremlin-Bicêtre | France | Unknown status |
| Centre Hospitalier Regional Universitaire (CHRU) de Nancy Coordinator of this site? Confirm its status | Nancy | France | Unknown status |
| Centre Hospitalier Universitaire de Nice Coordinator of this site? Confirm its status | Nice | France | Unknown status |
| Hôpital de la Pitié-Salpêtrière Coordinator of this site? Confirm its status | Paris | France | Unknown status |
| University Hospital Birmingham Coordinator of this site? Confirm its status | Birmingham | United Kingdom | Unknown status |
| Sheffield Teaching Hospital NHS Foundation Trust Coordinator of this site? Confirm its status | Sheffield | United Kingdom | Unknown status |
| Israeli-Georgian Medical Research Clinic Healthycore, LTD Coordinator of this site? Confirm its status | Tbilisi | Georgia | Unknown status |
| Pineo Medical Ecosystem Coordinator of this site? Confirm its status | Tbilisi | Georgia | Unknown status |
| Multiprofile Clinic Consilium Medulla Coordinator of this site? Confirm its status | Tbilisi | Georgia | Unknown status |
| LTD "New Hospitals" Coordinator of this site? Confirm its status | Tbilisi | Georgia | Unknown status |
| LTD National Center of Urology Named after L. Managadze Coordinator of this site? Confirm its status | Tbilisi | Georgia | Unknown status |
| Bangalore Medical College and Research Institute (BMCRI) - Victoria Hospital Coordinator of this site? Confirm its status | Bangalore | India | Unknown status |
| Postgraduate Institute of Medical Education & Research Coordinator of this site? Confirm its status | Chandigarh | India | Unknown status |
| Government General Hospital Guntur Coordinator of this site? Confirm its status | Guntur | India | Unknown status |
| City Neuro Centre, Hyderabad Coordinator of this site? Confirm its status | Hyderabad | India | Unknown status |
| Apex Hospital Coordinator of this site? Confirm its status | Jaipur | India | Unknown status |
| Polakulath Narayanan Renai Medicity Multi Specialty Hospital, Kochi Coordinator of this site? Confirm its status | Kochi | India | Unknown status |
| Amrita Institute of Medical Sciences and Research Centre (AIMS) Coordinator of this site? Confirm its status | Kochi | India | Unknown status |
| Christian Medical College & Hospital Coordinator of this site? Confirm its status | Ludhiana | India | Unknown status |
| All India Institute of Medical Sciences, New Delhi Coordinator of this site? Confirm its status | New Delhi | India | Unknown status |
| Nizam's Institute of Medical Sciences (NIMS) Coordinator of this site? Confirm its status | Panjagutta | India | Unknown status |
| Seth G.S. Medical College & K.E.M. Hospital, Mumbai Coordinator of this site? Confirm its status | Parel | India | Unknown status |
| Deenanath Mangeshkar Hospital and Research Centre Coordinator of this site? Confirm its status | Pune | India | Unknown status |
| Institute of Neurosciences Coordinator of this site? Confirm its status | Surat | India | Unknown status |
| Kasturba Medical College (KMC) Coordinator of this site? Confirm its status | Udupi | India | Unknown status |
| Azienda Ospedaliera Papa Giovanni XXIII Coordinator of this site? Confirm its status | Bergamo | Italy | Unknown status |
| Fondazione IRCCS Di Rilievo Nazionale Istituto Nazionale Neurologico Carlo Besta Coordinator of this site? Confirm its status | Milan | Italy | Unknown status |
| Azienda Ospedaliero Universitaria Pisana Coordinator of this site? Confirm its status | Pisa | Italy | Unknown status |
| Universita' Degli Studi di Roma La Sapienza Coordinator of this site? Confirm its status | Roma | Italy | Unknown status |
| Azienda Ospedaliera Sant'Andrea Coordinator of this site? Confirm its status | Rome | Italy | Unknown status |
| Medical Hospital of Tokyo Medical and Dental University Coordinator of this site? Confirm its status | Bunkyo | Japan | Unknown status |
| Chiba University Hospital Coordinator of this site? Confirm its status | Chiba | Japan | Unknown status |
| Okinawa National Hospital Coordinator of this site? Confirm its status | Ginowan-Shi | Japan | Unknown status |
| Hiroshima City Hiroshima Citiz Coordinator of this site? Confirm its status | Hiroshima | Japan | Unknown status |
| Saitama Medical University, Saitama Medical Center Coordinator of this site? Confirm its status | Kawagoe | Japan | Unknown status |
| Kobe City Medical Center General Hospital Coordinator of this site? Confirm its status | Kobe | Japan | Unknown status |
| Kochi Medical School Hospital Coordinator of this site? Confirm its status | Nankoku | Japan | Unknown status |
| Japanese Red Cross Osaka Hospital Coordinator of this site? Confirm its status | Osaka | Japan | Unknown status |
| Okinawa Prefectural Nanbu Medical Center and Children's Medical Center Coordinator of this site? Confirm its status | Shimajiri | Japan | Unknown status |
| Tokyo Medical University Hospital Coordinator of this site? Confirm its status | Shinjuku | Japan | Unknown status |
| Nihon University Itabashi Hospital Coordinator of this site? Confirm its status | tabashi City | Japan | Unknown status |
| Yamaguchi University Hospital Coordinator of this site? Confirm its status | Ube | Japan | Unknown status |
| Kyungpook National University Chilgok Hospital Coordinator of this site? Confirm its status | Daegu | South Korea | Unknown status |
| Samsung Medical Center Coordinator of this site? Confirm its status | Seoul | South Korea | Unknown status |
| Gdański Uniwersytet Medyczny Coordinator of this site? Confirm its status | Gdansk | Poland | Unknown status |
| Centrum Neurologii Klinicznej Krakowska Akademia Neurologii Coordinator of this site? Confirm its status | Krakow | Poland | Unknown status |
| Clinical Research Center Spólka z Ograniczona Odpowiedzialnoscia Medic-R sp. k. Coordinator of this site? Confirm its status | Poznan | Poland | Unknown status |
| NZOZ Neuro-Kard Coordinator of this site? Confirm its status | Poznan | Poland | Unknown status |
| NeuroProtect Coordinator of this site? Confirm its status | Warsaw | Poland | Unknown status |
| Uniwersyteckie Centrum Kliniczne WUM Centralny Szpital Kliniczny Coordinator of this site? Confirm its status | Warsaw | Poland | Unknown status |
| University Clinical Centre Nis Coordinator of this site? Confirm its status | Niš | Serbia | Unknown status |
| Chang Gung Memorial Foundation Kaohsiung Branch Coordinator of this site? Confirm its status | Kaohsiung City | Taiwan | Unknown status |
| China Medical University Coordinator of this site? Confirm its status | Taichung | Taiwan | Unknown status |
| National Cheng Kung University Hospital Coordinator of this site? Confirm its status | Tainan | Taiwan | Unknown status |
| Shin Kong Wu Ho Su Memorial Hospital Coordinator of this site? Confirm its status | Taipei | Taiwan | Unknown status |
| Chang Gung Memorial Hospital at Linkou Coordinator of this site? Confirm its status | Taoyuan | Taiwan | Unknown status |
| National Neuromuscular Research Institute Coordinator of this site? Confirm its status | Austin | United States | Unknown status |
| SFM Clinical Research LLC Coordinator of this site? Confirm its status | Boca Raton | United States | Unknown status |
| Dayton Center for Neurological Disorders Coordinator of this site? Confirm its status | Centerville | United States | Unknown status |
| University of Cincinnati Gardner Neuroscience Institute Coordinator of this site? Confirm its status | Cincinnati | United States | Unknown status |
| Colorado Springs Neurological Associates Coordinator of this site? Confirm its status | Colorado Springs | United States | Unknown status |
| Wayne State University School of Medicine Coordinator of this site? Confirm its status | Detroit | United States | Unknown status |
| Northshore University Health System Coordinator of this site? Confirm its status | Glenview | United States | Unknown status |
| Nerve and Muscle Center of Texas Coordinator of this site? Confirm its status | Houston | United States | Unknown status |
| University of California, Irvine Coordinator of this site? Confirm its status | Irvine | United States | Unknown status |
| University of Southern California Coordinator of this site? Confirm its status | Los Angeles | United States | Unknown status |
| Diverse Clinical Research Coordinator of this site? Confirm its status | Miami | United States | Unknown status |
| Aqualane Clinical Research Coordinator of this site? Confirm its status | Naples | United States | Unknown status |
| St. Elizabeth's Hospital Coordinator of this site? Confirm its status | O'Fallon | United States | Unknown status |
| Neurological Services of Orlando Coordinator of this site? Confirm its status | Orlando | United States | Unknown status |
| Penn Medicine University City Coordinator of this site? Confirm its status | Philadelphia | United States | Unknown status |
| Medsol Clinical Research Center Inc Coordinator of this site? Confirm its status | Port Charlotte | United States | Unknown status |
| Northwest Neurology Ltd. - Clinedge - PPDS Coordinator of this site? Confirm its status | Rolling Meadows | United States | Unknown status |
| HonorHealth Neurology 2018 Coordinator of this site? Confirm its status | Scottsdale | United States | Unknown status |
| University of South Florida Morsani Center for Advanced Healthcare Coordinator of this site? Confirm its status | Tampa | United States | Unknown status |
| Dokuz Eylul University Medical Faculty Coordinator of this site? Confirm its status | Izmir | Turkey (Türkiye) | Unknown status |
| Kocaeli University Hospital Coordinator of this site? Confirm its status | İzmit | Turkey (Türkiye) | Unknown status |
| Ondokuz Mayis Universitesi Tip Fakultesi Hastanesi Coordinator of this site? Confirm its status | Samsun | Turkey (Türkiye) | Unknown status |
| Karadeniz Technical University Farabi Hospital Coordinator of this site? Confirm its status | Trabzon | Turkey (Türkiye) | Unknown status |
This page is information, not medical advice. Discuss any trial with your doctor.