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
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)
Change in Myasthenia Gravis-Activities of Daily Living (MG-ADL) total score — From baseline to week 24 The total MG-ADL score ranges from 0 to 24 points, with higher scores indicating greater functional impairment and disability
Trial sites (116)
Facility
City
Region
Status
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.
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.
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