Fact CheckerSign in

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
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

Full criteria on ClinicalTrials.gov

Sites (116)

Sites in registry order. Pick your city on the disease page to sort them by distance.

SiteCityCountrySite status
Universitair Ziekenhuis Antwerpen
Coordinator of this site? Confirm its status
EdegemBelgiumUnknown status
AZ Sint-Lucas
Coordinator of this site? Confirm its status
GhentBelgiumUnknown status
Jordy Sinapse Medicina LTDA
Coordinator of this site? Confirm its status
ItapeviBrazilUnknown status
IMV Pesquisa Neurológica
Coordinator of this site? Confirm its status
Porto AlegreBrazilUnknown status
Faculdade de Medicina do ABC
Coordinator of this site? Confirm its status
Santo AndréBrazilUnknown status
Hospital das Clinicas da Faculdade de Medicina da Universidade de São Paulo
Coordinator of this site? Confirm its status
São PauloBrazilUnknown status
PSEG Centro de Pesquisa Clinica S.A.
Coordinator of this site? Confirm its status
São PauloBrazilUnknown status
University of Alberta
Coordinator of this site? Confirm its status
EdmontonCanadaUnknown status
London Health Sciences Centre
Coordinator of this site? Confirm its status
LondonCanadaUnknown status
Toronto General Hospital
Coordinator of this site? Confirm its status
TorontoCanadaUnknown status
Peking Union Medical College Hospital
Coordinator of this site? Confirm its status
BeijingChinaUnknown status
The First Hospital of Jilin University
Coordinator of this site? Confirm its status
ChangchunChinaUnknown status
Xiangya Hospital Central South University
Coordinator of this site? Confirm its status
ChangshaChinaUnknown status
Sichuan Provincial People's Hospital
Coordinator of this site? Confirm its status
ChengduChinaUnknown status
Fujian Medical University Union Hospital
Coordinator of this site? Confirm its status
FuzhouChinaUnknown status
Guangdong Hospital of Traditional Chinese Medicine
Coordinator of this site? Confirm its status
GuangdongChinaUnknown status
The First Affiliated Hospital of Guangzhou University of Chinese Medicine
Coordinator of this site? Confirm its status
GuangzhouChinaUnknown status
Nanfang Hospital, Southern Medical University
Coordinator of this site? Confirm its status
GuangzhouChinaUnknown status
Hainan General Hospital
Coordinator of this site? Confirm its status
HaikouChinaUnknown status
Qilu Hospital of Shandong University (Qingdan)
Coordinator of this site? Confirm its status
QingdaoChinaUnknown status
Huashan Hospital, Fudan University
Coordinator of this site? Confirm its status
ShanghaiChinaUnknown status
The University of Hong Kong-Shenzhen Hospital
Coordinator of this site? Confirm its status
ShenzhenChinaUnknown status
Tianjin Medical University General Hospital
Coordinator of this site? Confirm its status
TianjinChinaUnknown status
Renmin Hospital of Wuhan University - Hubei Provincial People's Hospital
Coordinator of this site? Confirm its status
WuhanChinaUnknown status
The Second Affiliated Hospital of the Chinese Peoples' Liberation Army Air Force Medical University
Coordinator of this site? Confirm its status
Xi’anChinaUnknown status
Charité Universitätsmedizin Berlin
Coordinator of this site? Confirm its status
BerlinGermanyUnknown status
University Hospital Essen
Coordinator of this site? Confirm its status
EssenGermanyUnknown status
Universitätsklinikum Jena
Coordinator of this site? Confirm its status
JenaGermanyUnknown status
Universitatsklinikum Leipzig
Coordinator of this site? Confirm its status
LeipzigGermanyUnknown status
Friedrich Baur Institute
Coordinator of this site? Confirm its status
MünchenGermanyUnknown status
Universitätsklinikum Münster
Coordinator of this site? Confirm its status
MünsterGermanyUnknown status
Aalborg Universitetshospital
Coordinator of this site? Confirm its status
AalborgDenmarkUnknown status
Aarhus University Hospital
Coordinator of this site? Confirm its status
Aarhus NDenmarkUnknown status
Rigshospitalet, Copenhagen
Coordinator of this site? Confirm its status
CopenhagenDenmarkUnknown status
Odense Universitetshospital
Coordinator of this site? Confirm its status
OdenseDenmarkUnknown status
Hospital de la Santa Creu i Sant Pau
Coordinator of this site? Confirm its status
BarcelonaSpainUnknown status
Hospital Universitari i Politécnic La Fe
Coordinator of this site? Confirm its status
ValenciaSpainUnknown status
CHU Bicêtre
Coordinator of this site? Confirm its status
Le Kremlin-BicêtreFranceUnknown status
Centre Hospitalier Regional Universitaire (CHRU) de Nancy
Coordinator of this site? Confirm its status
NancyFranceUnknown status
Centre Hospitalier Universitaire de Nice
Coordinator of this site? Confirm its status
NiceFranceUnknown status
Hôpital de la Pitié-Salpêtrière
Coordinator of this site? Confirm its status
ParisFranceUnknown status
University Hospital Birmingham
Coordinator of this site? Confirm its status
BirminghamUnited KingdomUnknown status
Sheffield Teaching Hospital NHS Foundation Trust
Coordinator of this site? Confirm its status
SheffieldUnited KingdomUnknown status
Israeli-Georgian Medical Research Clinic Healthycore, LTD
Coordinator of this site? Confirm its status
TbilisiGeorgiaUnknown status
Pineo Medical Ecosystem
Coordinator of this site? Confirm its status
TbilisiGeorgiaUnknown status
Multiprofile Clinic Consilium Medulla
Coordinator of this site? Confirm its status
TbilisiGeorgiaUnknown status
LTD "New Hospitals"
Coordinator of this site? Confirm its status
TbilisiGeorgiaUnknown status
LTD National Center of Urology Named after L. Managadze
Coordinator of this site? Confirm its status
TbilisiGeorgiaUnknown status
Bangalore Medical College and Research Institute (BMCRI) - Victoria Hospital
Coordinator of this site? Confirm its status
BangaloreIndiaUnknown status
Postgraduate Institute of Medical Education & Research
Coordinator of this site? Confirm its status
ChandigarhIndiaUnknown status
Government General Hospital Guntur
Coordinator of this site? Confirm its status
GunturIndiaUnknown status
City Neuro Centre, Hyderabad
Coordinator of this site? Confirm its status
HyderabadIndiaUnknown status
Apex Hospital
Coordinator of this site? Confirm its status
JaipurIndiaUnknown status
Polakulath Narayanan Renai Medicity Multi Specialty Hospital, Kochi
Coordinator of this site? Confirm its status
KochiIndiaUnknown status
Amrita Institute of Medical Sciences and Research Centre (AIMS)
Coordinator of this site? Confirm its status
KochiIndiaUnknown status
Christian Medical College & Hospital
Coordinator of this site? Confirm its status
LudhianaIndiaUnknown status
All India Institute of Medical Sciences, New Delhi
Coordinator of this site? Confirm its status
New DelhiIndiaUnknown status
Nizam's Institute of Medical Sciences (NIMS)
Coordinator of this site? Confirm its status
PanjaguttaIndiaUnknown status
Seth G.S. Medical College & K.E.M. Hospital, Mumbai
Coordinator of this site? Confirm its status
ParelIndiaUnknown status
Deenanath Mangeshkar Hospital and Research Centre
Coordinator of this site? Confirm its status
PuneIndiaUnknown status
Institute of Neurosciences
Coordinator of this site? Confirm its status
SuratIndiaUnknown status
Kasturba Medical College (KMC)
Coordinator of this site? Confirm its status
UdupiIndiaUnknown status
Azienda Ospedaliera Papa Giovanni XXIII
Coordinator of this site? Confirm its status
BergamoItalyUnknown status
Fondazione IRCCS Di Rilievo Nazionale Istituto Nazionale Neurologico Carlo Besta
Coordinator of this site? Confirm its status
MilanItalyUnknown status
Azienda Ospedaliero Universitaria Pisana
Coordinator of this site? Confirm its status
PisaItalyUnknown status
Universita' Degli Studi di Roma La Sapienza
Coordinator of this site? Confirm its status
RomaItalyUnknown status
Azienda Ospedaliera Sant'Andrea
Coordinator of this site? Confirm its status
RomeItalyUnknown status
Medical Hospital of Tokyo Medical and Dental University
Coordinator of this site? Confirm its status
BunkyoJapanUnknown status
Chiba University Hospital
Coordinator of this site? Confirm its status
ChibaJapanUnknown status
Okinawa National Hospital
Coordinator of this site? Confirm its status
Ginowan-ShiJapanUnknown status
Hiroshima City Hiroshima Citiz
Coordinator of this site? Confirm its status
HiroshimaJapanUnknown status
Saitama Medical University, Saitama Medical Center
Coordinator of this site? Confirm its status
KawagoeJapanUnknown status
Kobe City Medical Center General Hospital
Coordinator of this site? Confirm its status
KobeJapanUnknown status
Kochi Medical School Hospital
Coordinator of this site? Confirm its status
NankokuJapanUnknown status
Japanese Red Cross Osaka Hospital
Coordinator of this site? Confirm its status
OsakaJapanUnknown status
Okinawa Prefectural Nanbu Medical Center and Children's Medical Center
Coordinator of this site? Confirm its status
ShimajiriJapanUnknown status
Tokyo Medical University Hospital
Coordinator of this site? Confirm its status
ShinjukuJapanUnknown status
Nihon University Itabashi Hospital
Coordinator of this site? Confirm its status
tabashi CityJapanUnknown status
Yamaguchi University Hospital
Coordinator of this site? Confirm its status
UbeJapanUnknown status
Kyungpook National University Chilgok Hospital
Coordinator of this site? Confirm its status
DaeguSouth KoreaUnknown status
Samsung Medical Center
Coordinator of this site? Confirm its status
SeoulSouth KoreaUnknown status
Gdański Uniwersytet Medyczny
Coordinator of this site? Confirm its status
GdanskPolandUnknown status
Centrum Neurologii Klinicznej Krakowska Akademia Neurologii
Coordinator of this site? Confirm its status
KrakowPolandUnknown status
Clinical Research Center Spólka z Ograniczona Odpowiedzialnoscia Medic-R sp. k.
Coordinator of this site? Confirm its status
PoznanPolandUnknown status
NZOZ Neuro-Kard
Coordinator of this site? Confirm its status
PoznanPolandUnknown status
NeuroProtect
Coordinator of this site? Confirm its status
WarsawPolandUnknown status
Uniwersyteckie Centrum Kliniczne WUM Centralny Szpital Kliniczny
Coordinator of this site? Confirm its status
WarsawPolandUnknown status
University Clinical Centre Nis
Coordinator of this site? Confirm its status
NišSerbiaUnknown status
Chang Gung Memorial Foundation Kaohsiung Branch
Coordinator of this site? Confirm its status
Kaohsiung CityTaiwanUnknown status
China Medical University
Coordinator of this site? Confirm its status
TaichungTaiwanUnknown status
National Cheng Kung University Hospital
Coordinator of this site? Confirm its status
TainanTaiwanUnknown status
Shin Kong Wu Ho Su Memorial Hospital
Coordinator of this site? Confirm its status
TaipeiTaiwanUnknown status
Chang Gung Memorial Hospital at Linkou
Coordinator of this site? Confirm its status
TaoyuanTaiwanUnknown status
National Neuromuscular Research Institute
Coordinator of this site? Confirm its status
AustinUnited StatesUnknown status
SFM Clinical Research LLC
Coordinator of this site? Confirm its status
Boca RatonUnited StatesUnknown status
Dayton Center for Neurological Disorders
Coordinator of this site? Confirm its status
CentervilleUnited StatesUnknown status
University of Cincinnati Gardner Neuroscience Institute
Coordinator of this site? Confirm its status
CincinnatiUnited StatesUnknown status
Colorado Springs Neurological Associates
Coordinator of this site? Confirm its status
Colorado SpringsUnited StatesUnknown status
Wayne State University School of Medicine
Coordinator of this site? Confirm its status
DetroitUnited StatesUnknown status
Northshore University Health System
Coordinator of this site? Confirm its status
GlenviewUnited StatesUnknown status
Nerve and Muscle Center of Texas
Coordinator of this site? Confirm its status
HoustonUnited StatesUnknown status
University of California, Irvine
Coordinator of this site? Confirm its status
IrvineUnited StatesUnknown status
University of Southern California
Coordinator of this site? Confirm its status
Los AngelesUnited StatesUnknown status
Diverse Clinical Research
Coordinator of this site? Confirm its status
MiamiUnited StatesUnknown status
Aqualane Clinical Research
Coordinator of this site? Confirm its status
NaplesUnited StatesUnknown status
St. Elizabeth's Hospital
Coordinator of this site? Confirm its status
O'FallonUnited StatesUnknown status
Neurological Services of Orlando
Coordinator of this site? Confirm its status
OrlandoUnited StatesUnknown status
Penn Medicine University City
Coordinator of this site? Confirm its status
PhiladelphiaUnited StatesUnknown status
Medsol Clinical Research Center Inc
Coordinator of this site? Confirm its status
Port CharlotteUnited StatesUnknown status
Northwest Neurology Ltd. - Clinedge - PPDS
Coordinator of this site? Confirm its status
Rolling MeadowsUnited StatesUnknown status
HonorHealth Neurology 2018
Coordinator of this site? Confirm its status
ScottsdaleUnited StatesUnknown status
University of South Florida Morsani Center for Advanced Healthcare
Coordinator of this site? Confirm its status
TampaUnited StatesUnknown status
Dokuz Eylul University Medical Faculty
Coordinator of this site? Confirm its status
IzmirTurkey (Türkiye)Unknown status
Kocaeli University Hospital
Coordinator of this site? Confirm its status
İzmitTurkey (Türkiye)Unknown status
Ondokuz Mayis Universitesi Tip Fakultesi Hastanesi
Coordinator of this site? Confirm its status
SamsunTurkey (Türkiye)Unknown status
Karadeniz Technical University Farabi Hospital
Coordinator of this site? Confirm its status
TrabzonTurkey (Türkiye)Unknown status

This page is information, not medical advice. Discuss any trial with your doctor.