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Clinical Trials in the USA / NCT05070858
Active, not recruiting Phase 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

NCT05070858 · tracked via the Priya Life Science USA tracker
Sponsor
Regeneron Pharmaceuticals
Phase
Phase 3
Started
2021-12-14
Last updated
2026-07-08

Condition(s) studied

Generalized Myasthenia Gravis

Investigational drug(s) / intervention(s)

Pozelimab + CemdisiranCemdisiranPlaceboPozelimab

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

Study summary

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)

Eligibility

Sex
ALL
Min age
18 Years
Max age
Healthy volunteers
No
Key Inclusion Criteria: 1. Male or female participants ≥18 years of age at screening (or ≥ legal age of adulthood based on local regulations, whichever is older) 2. Participant 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-Lipoprotein Receptor-related Protein 4 (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 participant 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 Key Exclusion Criteria: 1. Patients 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 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 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. 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 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 Human Immunodeficiency Virus (HIV) infection or a positive test at screening per local requirements NOTE: Other protocol-defined Inclusion/ Exclusion Criteria apply

Primary outcome measure(s)

Trial sites (116)

FacilityCityRegionStatus
HonorHealth Neurology 2018 Scottsdale Arizona
University of California Irvine Irvine California
University of Southern California Los Angeles California
Colorado Springs Neurological Associates Colorado Springs Colorado
SFM Clinical Research, LLC Boca Raton Florida
Diverse Clinical Research Miami Florida
Aqualane Clinical Research Naples Florida
Neurological Services of Orlando Orlando Florida
Medsol Clinical Research Center Inc Port Charlotte Florida
University of South Florida Morsani Center for Advanced Healthcare Tampa Florida
NorthShore University Health System Glenview Illinois
St. Elizabeth's Hospital O'Fallon Illinois
Northwest Neurology Ltd. - Clinedge - PPDS Rolling Meadows Illinois
Wayne State University School of Medicine Detroit Michigan
Dayton Center for Neurological Disorders Centerville Ohio
University of Cincinnati Gardner Neuroscience Institute Cincinnati Ohio
Penn Medicine University City Philadelphia Pennsylvania
National Neuromuscular Research Institute Austin Texas
Nerve and Muscle Center of Texas Houston Texas
Universitair Ziekenhuis Antwerpen Edegem Antwerp
AZ Sint-Lucas Ghent Oost-Vlaanderen
IMV Pesquisa Neurologica Porto Alegre Rio Grande do Sul
Jordy Sinapse Medicina LTDA Itapevi São Paulo
Faculdade de Medicina Do ABC Santo André São Paulo
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
London Health Sciences Centre London Ontario
Toronto General Hospital Toronto Ontario
Peking Union Medical College Hospital Beijing Beijing Municipality
Fujian Medical University Union Hospital Fuzhou Fujian
Guangdong Hospital of Traditional Chinese Medicine Guangdong Guangdong
Nanfang Hospital Southern Medical University Guangzhou Guangdong
The University of Hong Kong-Shenzhen Hospital Shenzhen Guangdong
The First Affiliated Hospital of Guangzhou University of Chinese Medicine Guangzhou Guangzhou
Hainan General Hospital Haikou Hainan
Renmin Hospital of Wuhan University - Hubei Provincial People's Hospital Wuhan Hubei
Xiangya Hospital Central South University Changsha Hunan
The First Hospital of Jilin University Changchun Jilin
Qilu Hospital of Shandong University (Qingdan) Qingdao Shandong

+ 76 more sites — see the full list on the official registry below.

Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT05070858 on ClinicalTrials.gov ↗ ← All trials in the USA