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
Purpose
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 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)
Condition
- Generalized Myasthenia Gravis
Eligibility
- Eligible Ages
- Over 18 Years
- Eligible Genders
- All
- Accepts Healthy Volunteers
- No
Inclusion Criteria
- Male or female patients ≥18 years of age at screening (or ≥ legal age of adulthood based on local regulations, whichever is older) 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
- 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 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 NOTE: Other protocol-defined Inclusion/ Exclusion Criteria apply
Study Design
- Phase
- Phase 3
- Study Type
- Interventional
- Allocation
- Randomized
- Intervention Model
- Parallel Assignment
- Primary Purpose
- Treatment
- Masking
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Arm Groups
| Arm | Description | Assigned Intervention |
|---|---|---|
|
Experimental Group 1 |
Placebo in DBTP; Re-randomized to Combination or Cemdisiran in ETP and OLTP |
|
|
Experimental Group 2 |
Combination regimen throughout the study |
|
|
Experimental Group 3 |
Cemdisiran throughout the study |
|
|
Experimental Group 4 |
Pozelimab monotherapy in DBTP followed by combination in ETP and OLTP |
|
Recruiting Locations
HonorHealth Neurology 2018
Scottsdale, Arizona 85251
Scottsdale, Arizona 85251
University of California Irvine
Irvine, California 92697
Irvine, California 92697
University of Southern California
Los Angeles, California 90033
Los Angeles, California 90033
Colorado Springs Neurological Associates
Colorado Springs, Colorado 80907-5307
Colorado Springs, Colorado 80907-5307
SFM Clinical Research, LLC
Boca Raton, Florida 33487
Boca Raton, Florida 33487
Diverse Clinical Research
Miami, Florida 33175
Miami, Florida 33175
Aqualane Clinical Research
Naples, Florida 34105
Naples, Florida 34105
Neurological Services of Orlando
Orlando, Florida 32806
Orlando, Florida 32806
Medsol Clinical Research Center Inc
Port Charlotte, Florida 33952
Port Charlotte, Florida 33952
University of South Florida Morsani Center for Advanced Healthcare
Tampa, Florida 33612
Tampa, Florida 33612
NorthShore University Health System
Glenview, Illinois 60026-1301
Glenview, Illinois 60026-1301
St. Elizabeth's Hospital
O'Fallon, Illinois 62269
O'Fallon, Illinois 62269
Wayne State University School of Medicine
Detroit, Michigan 48201
Detroit, Michigan 48201
Weill Cornell Medicine - Peripheral Neuropathy Center
New York, New York 10021
New York, New York 10021
Dayton Center for Neurological Disorders
Centerville, Ohio 45459
Centerville, Ohio 45459
University of Cincinnati Gardner Neuroscience Institute
Cincinnati, Ohio 45219
Cincinnati, Ohio 45219
Penn Medicine University City
Philadelphia, Pennsylvania 19104
Philadelphia, Pennsylvania 19104
National Neuromuscular Research Institute
Austin, Texas 78759
Austin, Texas 78759
Nerve and Muscle Center of Texas
Houston, Texas 77030
Houston, Texas 77030
More Details
- NCT ID
- NCT05070858
- Status
- Recruiting
- Sponsor
- Regeneron Pharmaceuticals
Detailed Description
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)