The purpose of this clinical study is to determine the efficacy and safety of a new oral cladribine formulation in participants with Generalized Myasthenia Gravis (gMG) in comparison to placebo. It will also investigate the sustained efficacy, the need for retreatment, and the long-term safety of oral cladribine in gMG. An additional component is included to characterize the Pharmacokinetics (PK) of the new cladribine formulation in gMG participants. This study is divided into 3 periods: the double-blind placebo control (DBPC) pivotal period, and 2 extensions, the blinded extension (BE) and the retreatment (RT) period.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Enrollment
264
Participants will receive placebo matched to cladribine in two courses separated by 4 weeks.
Participants will receive cladribine low dose in two courses separated by 4 weeks.
Participants will receive cladribine high dose in two courses separated by 4 weeks.
Arizona Neuroscience Research, LLC
Phoenix, Arizona, United States
RECRUITINGAdvanced Neurosciences Research LLC
Longmont, Colorado, United States
RECRUITINGThe George Washington University Medical Faculty Associates Foggy Bottom South Pavilion
Washington D.C., District of Columbia, United States
RECRUITINGNeurology of Central Florida Research Center, LLC
Altamonte Springs, Florida, United States
Change from Baseline in Myasthenia Gravis - Activities of Daily Living (MG-ADL) Scale Score at Week 24 During the Double-Blind Placebo Controlled (DBPC) Period
Time frame: Baseline, Week 24
Change from Baseline in Quantitative Myasthenia Gravis (QMG) Scale Score at Week 24 During the Double-Blind Placebo Controlled (DBPC) Period
Time frame: Baseline, Week 24
Percentage of MG-ADL Responders at Week 24 During the Double-Blind Placebo Controlled (DBPC) Period
MG-ADL responder is defined by achieving \>= 3 points decrease in MG-ADL score.
Time frame: At Week 24
Change from Baseline in Myasthenia Gravis Composite (MGC) Scale Score at Week 24 During the Double-Blind Placebo Controlled (DBPC) Period
Time frame: Baseline, Week 24
Percentage of Quantitative Myasthenia Gravis (QMG) Scale Responders at Week 24 During the Double-Blind Placebo Controlled (DBPC) Period
QMG responder is defined by achieving \>= 4 points decrease in QMG score.
Time frame: At Week 24
Time From Initial Cladribine Full Dose Treatment to First Retreatment or Rescue Treatment up to end of Study
Time frame: Up to End of Study (Week 144)
Number of Participants With Adverse Events (AEs) and Adverse Events of Special Interest (AESIs)
Time frame: Up to End of Study (Week 144)
Number of participants with Adverse Events (AEs) by Severity as per National Cancer Institute Common Terminology Criteria for Adverse Events, version 5.0
Time frame: Up to End of Study (Week 144)
Number of Participants with Abnormal Laboratory Variables including Absolute Lymphocyte Count and Vital Signs
Time frame: Up to End of Study (Week 144)
Pharmacokinetic (PK) Plasma Concentration of Cladribine
Time frame: Pre-dose, 0.25, 1, 2, 3, 4, 6, 8 and 24 hours post-dose
Change from Baseline in the Revised Myasthenia Gravis Quality of Life - 15 Scale (MG-Qol15r) Score at Week 24 During the Double-Blind Placebo Controlled (DBPC) Period
Time frame: Baseline, Week 24
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SFM Clinical Research, LLC
Boca Raton, Florida, United States
RECRUITINGUniversity of Florida Health Science Center - 300120311
Jacksonville, Florida, United States
RECRUITINGNeurology Associates, P. A.
Maitland, Florida, United States
RECRUITINGUniversity of South Florida - PARENT - PARENT
Tampa, Florida, United States
RECRUITINGUniversity of Kansas Medical Center Research Institute, Inc. - 3901 Rainbow (MAIN)
Kansas City, Kansas, United States
RECRUITINGAtrium Health Wake Forest Baptist
Winston-Salem, North Carolina, United States
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