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)
DBTP- Double Blind Treatment Period (24 weeks) ETP - Extension Treatment Period (28 weeks) OLTP- Open Label Treatment Period (68 weeks) FUP-Off treatment Follow Up Period (52 weeks)
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Enrollment
288
Subcutaneous administration as described in the protocol
SC administration as described in the protocol
SC administration as described in the protocol
SC administration as described in the protocol
HonorHealth Neurology 2018
Scottsdale, Arizona, United States
University of California Irvine
Irvine, California, United States
University of Southern California
Los Angeles, California, United States
Colorado Springs Neurological Associates
Colorado Springs, Colorado, United States
SFM Clinical Research, LLC
Boca Raton, Florida, United States
Change in Myasthenia Gravis-Activities of Daily Living (MG-ADL) total score
The total MG-ADL score ranges from 0 to 24 points, with higher scores indicating greater functional impairment and disability
Time frame: From baseline to week 24
Change from baseline in Quantitative Myasthenia Gravis (QMG) score
QMG total scores range from 0 to 39, with higher scores representing greater impairment
Time frame: Week 24
Achievement of a ≥3-point reduction (improvement) in MG-ADL total score
Time frame: From baseline to week 24
Achievement of a ≥5-point reduction (improvement) in QMG total score
Time frame: From baseline to week 24
Achievement of a consistent response on the MG-ADL
A participant with a ≥2-point reduction (improvement) in MG-ADL total score on 2 or more consecutive assessments spanning 4 or more weeks during the DBTP
Time frame: From baseline to week 24
Achievement of Minimal Symptom Expression (MSE)
Score of 0 to 1 on the MG-ADL
Time frame: Week 24
Change from baseline in the Myasthenia Gravis Composite (MGC) total score
MGC score ranges from 0 to 50, with higher score indicating higher impairment
Time frame: Week 24
Change from baseline in Myasthenia Gravis Quality of Life (MG-QOL15r) total score
Total score ranges from 0 to 30 points; a higher score represents greater impairment
Time frame: Week 24
Achievement of a ≥2-, 4-, 5-, 6-, 7-, 8-, 9-, or 10-point reduction on MG-ADL total score
Time frame: From baseline to week 24
Achievement of a ≥3-, 4-, 6-, 7-, 8-, 9-, or 10-point reduction on QMG
Time frame: From baseline to week 24
Incidence and severity of Treatment-Emergent Adverse Events (TEAEs) in participants treated with pozelimab + cemdisiran, cemdisiran monotherapy or placebo
Time frame: Through week 24
Incidence and severity of Serious Adverse Events (SAEs) in participants treated with pozelimab + cemdisiran, cemdisiran monotherapy or placebo
Time frame: Through week 24
Incidence and severity of Adverse Events of Special Interest (AESIs) in participants treated with pozelimab + cemdisiran, cemdisiran monotherapy or placebo
Time frame: Through week 24
Concentrations of total pozelimab in serum
Time frame: Through study duration, approximate 172 weeks
Concentrations of total Complement component 5 (C5) in plasma
Time frame: Through study duration, approximate 172 weeks
Concentrations of cemdisiran and its metabolites in plasma
Time frame: Through study duration, approximate 172 weeks
Incidence of treatment-emergent Anti-Drug Antibodies (ADAs) to pozelimab over time
Time frame: Through study duration, approximately 172 weeks
Incidence of treatment-emergent ADAs to cemdisiran over time
Time frame: Through study duration, approximate 172 weeks
Change in total complement hemolysis activity assay (CH50) over time
Time frame: Through study duration, approximately 172 weeks
Percent change in CH50 over time
Time frame: Through study duration, approximately 172 weeks
This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.
Diverse Clinical Research
Miami, Florida, United States
Aqualane Clinical Research
Naples, Florida, United States
Neurological Services of Orlando
Orlando, Florida, United States
Medsol Clinical Research Center Inc
Port Charlotte, Florida, United States
University of South Florida Morsani Center for Advanced Healthcare
Tampa, Florida, United States
...and 106 more locations