Last updated 9 days ago
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
235 patients around the world
Available in Spain, Brazil, United States
DBTP- Double blind treatment period (24 weeks) ETP - Extension treatment period (28
weeks) OLTP- Open label treatment period (68 weeks) Off-treatment follow up period (52
weeks)
Regeneron Pharmaceuticals
2Research sites
235Patients around the world
This study is for people with
Myasthenia gravis
Myasthenia gravis in adults
Requirements for the patient
From 18 Years
All Gender
Medical requirements
- Male or female patients ≥18 years of age at screening (or ≥ legal age of adulthood based on local regulations, whichever is older).Patient 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-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.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 patient 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).
- 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.History of thymectomy within 12 months prior to screening or planned during the study.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.Myasthenic crisis or Myasthenia Gravis Foundation of America (MGFA) Class V within 1 month of screening.No documented meningococcal vaccination within 5 years prior to screening visit unless vaccination will be administered during the screening period and prior to initiation of study treatment.Known contraindication to meningococcal vaccines (group ACWY conjugate and group B vaccines) as described in the protocol.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.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 HIV infection or a positive test at screening per local requirements.
Sites
Jordy Sinapse Medicina LTDA ME
Recruiting
Hospital das Clínicas da Faculdade de Medicina da São Paulo - USP
Recruiting
StudyNIMBLE
SponsorRegeneron Pharmaceuticals
Conditions
Myasthenia gravis in adults
Requirements
From 18 YearsAll Gender
Unique study IDclinicaltrials.gov
NCT05070858
