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
NCT ID: NCT05070858
Last Updated: 2025-08-22
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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ACTIVE_NOT_RECRUITING
PHASE3
288 participants
INTERVENTIONAL
2021-12-14
2028-11-11
Brief Summary
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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 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)
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Detailed Description
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Conditions
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Study Design
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RANDOMIZED
PARALLEL
TREATMENT
QUADRUPLE
Study Groups
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Group 1
Placebo in DBTP; Re-randomized to Combination or Cemdisiran in ETP and OLTP
Pozelimab + Cemdisiran
Subcutaneous administration as described in the protocol
Cemdisiran
SC administration as described in the protocol
Placebo
SC administration as described in the protocol
Group 2
Combination regimen throughout the study
Pozelimab + Cemdisiran
Subcutaneous administration as described in the protocol
Group 3
Cemdisiran throughout the study
Cemdisiran
SC administration as described in the protocol
Group 4
Pozelimab monotherapy in DBTP followed by combination in ETP and OLTP
Pozelimab
SC administration as described in the protocol
Interventions
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Pozelimab + Cemdisiran
Subcutaneous administration as described in the protocol
Cemdisiran
SC administration as described in the protocol
Placebo
SC administration as described in the protocol
Pozelimab
SC administration as described in the protocol
Other Intervention Names
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Eligibility Criteria
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Inclusion Criteria
2. Patient with documented diagnosis of myasthenia gravis (MG) based on medical history and supported by previous evaluations as described in the protocol
3. Documented prior history of positive serologic test or a positive result during screening of anti-acetylcholine receptor (AChR) antibodies or anti-LRP4 antibodies.
4. Myasthenia Gravis Foundation of America (MGFA) Clinical Classification Class II to IVa at screening
5. 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
6. Currently receiving an acetylcholinesterase inhibitor or documented reason for not using acetylcholinesterase inhibitor therapy per investigator
7. Currently receiving an immunosuppressive therapy (IST) for MG, or documented reason why the patient is not taking an IST per investigator
8. If currently receiving an IST, not anticipated to have IST dosage changed before randomization or during double-blind treatment period (DBTP).
9. 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
Exclusion Criteria
2. History of thymectomy within 12 months prior to screening or planned during the study
3. 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
4. Myasthenic crisis or Myasthenia Gravis Foundation of America (MGFA) Class V within 1 month of screening
5. 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
6. Known contraindication to meningococcal vaccines (group ACWY conjugate and group B vaccines) as described in the protocol
7. Patients 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
8. 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
9. History of HIV infection or a positive test at screening per local requirements
18 Years
ALL
No
Sponsors
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Regeneron Pharmaceuticals
INDUSTRY
Responsible Party
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Principal Investigators
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Clinical Trial Management
Role: STUDY_DIRECTOR
Regeneron Pharmaceuticals
Locations
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HonorHealth Neurology 2018
Scottsdale, Arizona, United States
University of California Irvine
Irvine, California, United States
University of Southern California
Los Angeles, California, United States
Colorado Springs Neurological Associates
Colorado Springs, Colorado, United States
SFM Clinical Research, LLC
Boca Raton, Florida, United States
Diverse Clinical Research
Miami, Florida, United States
Aqualane Clinical Research
Naples, Florida, United States
Neurological Services of Orlando
Orlando, Florida, United States
Medsol Clinical Research Center Inc
Port Charlotte, Florida, United States
University of South Florida Morsani Center for Advanced Healthcare
Tampa, Florida, United States
NorthShore University Health System
Glenview, Illinois, United States
St. Elizabeth's Hospital
O'Fallon, Illinois, United States
Northwest Neurology Ltd. - Clinedge - PPDS
Rolling Meadows, Illinois, United States
Wayne State University School of Medicine
Detroit, Michigan, United States
Dayton Center for Neurological Disorders
Centerville, Ohio, United States
University of Cincinnati Gardner Neuroscience Institute
Cincinnati, Ohio, United States
Penn Medicine University City
Philadelphia, Pennsylvania, United States
National Neuromuscular Research Institute
Austin, Texas, United States
Nerve and Muscle Center of Texas
Houston, Texas, United States
Universitair Ziekenhuis Antwerpen
Edegem, Antwerp, Belgium
AZ Sint-Lucas
Ghent, Oost-Vlaanderen, Belgium
IMV Pesquisa Neurologica
Porto Alegre, Rio Grande do Sul, Brazil
Jordy Sinapse Medicina LTDA
Itapevi, São Paulo, Brazil
Faculdade de Medicina Do ABC
Santo André, São Paulo, Brazil
Pseg Centro de Pesquisa Clinica S.A
São Paulo, , Brazil
Hospital das Clinicas da Faculdade de Medicina da Universidade de Sao Paulo
São Paulo, , Brazil
University of Alberta
Edmonton, Alberta, Canada
London Health Sciences Centre
London, Ontario, Canada
Toronto General Hospital
Toronto, Ontario, Canada
Peking Union Medical College Hospital
Beijing, Beijing Municipality, China
Fujian Medical University Union Hospital
Fuzhou, Fujian, China
Guangdong Hospital of Traditional Chinese Medicine
Guangdong, Guangdong, China
Nanfang Hospital Southern Medical University
Guangzhou, Guangdong, China
The University of Hong Kong-Shenzhen Hospital
Shenzhen, Guangdong, China
The First Affiliated Hospital of Guangzhou University of Chinese Medicine
Guangzhou, Guangzhou, China
Hainan General Hospital
Haikou, Hainan, China
Renmin Hospital of Wuhan University - Hubei Provincial People's Hospital
Wuhan, Hubei, China
Xiangya Hospital Central South University
Changsha, Hunan, China
The First Hospital of Jilin University
Changchun, Jilin, China
Qilu Hospital of Shandong University (Qingdan)
Qingdao, Shandong, China
Huashan Hospital Fudan University
Shanghai, Shanghai Municipality, China
The Second Affiliated Hospital of the Chinese Peoples' Liberation Army Air Force Medical University
Xi’an, Shanxi, China
Sichuan Provincial People's Hospital
Chengdu, Sichuan, China
Tianjin Medical University General Hospital
Tianjin, Tianjin Municipality, China
Aalborg Universitetshospital
Aalborg, North Denmark, Denmark
Aarhus University Hospital
Aarhus N, , Denmark
Rigshospitalet, Copenhagen
Copenhagen, , Denmark
Odense Universitetshospital
Odense, , Denmark
Centre Hospitalier Universitaire de Nice
Nice, Alpes Maritimes, France
CHU Bicetre
Le Kremlin-Bicêtre, , France
Centre Hospitalier Regional Universitaire (CHRU) de Nancy
Nancy, , France
Hopital de la Pitie Salpetriere
Paris, , France
Israeli-Georgian Medical Research Clinic Healthycore, LTD
Tbilisi, , Georgia
LTD New Hospitals
Tbilisi, , Georgia
Pineo Medical Ecosystem
Tbilisi, , Georgia
LTD National Center of Urology Named after L. Managadze
Tbilisi, , Georgia
Multiprofile Clinic Consilium Medulla
Tbilisi, , Georgia
Friedrich Baur Institute
München, Bavaria, Germany
University Hospital Essen
Essen, North Rhine-Westphalia, Germany
Universitatsklinikum Munster
Münster, North Rhine-Westphalia, Germany
Universitatsklinikum Leipzig
Leipzig, Saxony, Germany
Charite - Universitatsmedizin Berlin
Berlin, , Germany
Universitatsklinikum Jena
Jena, , Germany
Government General Hospital, Guntur
Guntur, Andhra Pradesh, India
Institute of Neurosciences
Surat, Gujarat, India
Nizam's Institute of Medical Sciences (NIMS)
Panjagutta, Hyderabad, India
Kasturba Medical College (KMC)
Udupi, Karnataka, India
Amrita Institute of Medical Sciences and Research Centre (AIMS)
Kochi, Kerala, India
Deenanath Mangeshkar Hospital and Research Centre
Pune, Maharashtra, India
Seth G.S. Medical College & K.E.M. Hospital, Mumbai
Pārel, Mumbai, India
All India Institute of Medical Sciences New Delhi
New Delhi, National Capital Territory of Delhi, India
Polakulath Narayanan Renai Medicity Multi Specialty Hospital, Kochi
Kochi, Punjab, India
Christian Medical College & Hospital
Ludhiana, Punjab, India
Apex Hospital
Jaipur, Rajasthan, India
City Neuro Centre, Hyderabad
Hyderabad, Telangana, India
Bangalore Medical College and Research Institute (BMCRI) - Victoria Hospital
Bangalore, , India
Postgraduate Institute of Medical Education & Research
Chandigarh, , India
Azienda Ospedaliera Sant'andrea
Rome, Lazio, Italy
Fondazione IRCCS Di Rilievo Nazionale Istituto Nazionale Neurologico Carlo Besta
Milan, Lombardy, Italy
Azienda Ospedaliero Universitaria Pisana
Pisa, Tuscany, Italy
Azienda Ospedaliera Papa Giovanni XXIII
Bergamo, , Italy
Universita' Degli Studi di Roma La Sapienza
Roma, , Italy
Kobe City Medical Center General Hospital
Kobe, Hyōgo, Japan
Kochi Medical School Hospital
Nankoku, Kochi, Japan
Okinawa National Hospital
Ginowan-Shi, Okinawa, Japan
Okinawa Prefectural Nanbu Medical Center and Children's Medical Center
Shimajiri, Okinawa, Japan
Saitama Medical University, Saitama Medical Center
Kawagoe, Saitama, Japan
Medical Hospital of Tokyo Medical and Dental University
Bunkyo, Tokyo, Japan
Tokyo Medical University Hospital
Shinjuku, Tokyo, Japan
Nihon University Itabashi Hospital
tabashi City, Tokyo, Japan
Yamaguchi University Hospital
Ube, Yamaguchi, Japan
Chiba University Hospital
Chiba, , Japan
Hiroshima City Hiroshima Citiz
Hiroshima, , Japan
Japanese Red Cross Osaka Hospital
Osaka, , Japan
Clinical Research Center Spolka z Ograniczona Odpowiedzialnoscia Medic-R Sp.k
Poznan, Greater Poland Voivodeship, Poland
NZOZ Neuro-kard
Poznan, Greater Poland Voivodeship, Poland
Centrum Neurologii Klinicznej, Krakowska Akademia Neurologii
Krakow, Lesser Poland Voivodeship, Poland
NeuroProtect
Warsaw, Mazovian, Poland
Uniwersyteckie Centrum Kliniczne WUM, Centralny Szpital Kliniczny
Warsaw, Mosavian, Poland
Gdanski Uniwersytet Medyczny
Gdansk, Pomeranian Voivodeship, Poland
University Clinical Centre Nis
Niš, , Serbia
Kyungpook National University Chilgok Hospital
Daegu, , South Korea
Samsung Medical Center
Seoul, , South Korea
Hospital de la Santa Creu i Sant Pau
Barcelona, , Spain
Hospital Universitari i Politecnic La Fe
Valencia, , Spain
Chang Gung Memorial Foundation Kaohsiung Branch
Kaohsiung City, , Taiwan
China Medical University
Taichung, , Taiwan
National Cheng Kung University Hospital
Tainan City, , Taiwan
Shin Kong Wu Ho Su Memorial Hospital
Taipei, , Taiwan
Chang Gung Memorial Hospital at Linkou
Taoyuan District, , Taiwan
Kocaeli University Hospital
Izmit, Van, Turkey (Türkiye)
Dokuz Eylul University Medical Faculty
Izmir, , Turkey (Türkiye)
Ondokuz Mayis Universitesi Tip Fakultesi Hastanesi
Samsun, , Turkey (Türkiye)
Karadeniz Technical University Farabi Hospital
Trabzon, , Turkey (Türkiye)
Sheffield Teaching Hospital NHS Foundation Trust
Sheffield, South Yorkshire, United Kingdom
University Hospital Birmingham
Birmingham, , United Kingdom
Countries
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Other Identifiers
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2020-003272-41
Identifier Type: EUDRACT_NUMBER
Identifier Source: secondary_id
2023-508842-17-00
Identifier Type: CTIS
Identifier Source: secondary_id
R3918-MG-2018
Identifier Type: -
Identifier Source: org_study_id
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