A Phase 3, Randomized, Double-Blind, Placebo-Controlled, 3-Arm, 3-Period Study to Assess the Efficacy and Safety of a New Formulation of Oral Cladribine Compared With Placebo in Participants With Generalized Myasthenia Gravis (MyClad)
Summary
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. Furthermore, in trial interviews will be conducted as a sub-study to MyClad with a sub-set of participants to gain an in depth understanding of the participant cladribine treatment and study experience.
Arms & interventions
- OtherPlacebo
Participants will receive placebo matched to cladribine in two courses separated by 4 weeks.
- DrugCladribine Low Dose
Participants will receive cladribine low dose in two courses separated by 4 weeks.
- DrugCladribine High Dose
Participants will receive cladribine high dose in two courses separated by 4 weeks.
Outcome measures
Primary
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
Secondary
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
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
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
Number of Participants with Abnormal ECG parameters
Time frame: Up to End of Study (Week 144)
Eligibility criteria
Study locations (21)
Arizona Neuroscience Research, LLC
Phoenix, Arizona, 85032
Advanced Neurosciences Research LLC
Longmont, Colorado, 80501
University of Connecticut Health Center - Department of Medicine
Farmington, Connecticut, 06030-5550
The George Washington University Medical Faculty Associates Foggy Bottom South Pavilion
Washington D.C., District of Columbia, 20037
Neurology of Central Florida Research Center, LLC
Altamonte Springs, Florida, 32714
SFM Clinical Research, LLC
Boca Raton, Florida, 33487
University of Florida Health Science Center - 300120311
Jacksonville, Florida, 32209
Neurology Associates, P. A.
Maitland, Florida, 32751
University of South Florida - PARENT - PARENT
Tampa, Florida, 33612
University of Kansas Medical Center Research Institute, Inc. - 3901 Rainbow (MAIN)
Kansas City, Kansas, 66160
UNC Hospitals - PARENT
Chapel Hill, North Carolina, 27599
Atrium Health Wake Forest Baptist
Winston-Salem, North Carolina, 27157-1078
Miami Valley Hospital South
Centerville, Ohio, 45459
University of Cincinnati
Cincinnati, Ohio, 45219
Clinical Trials of South Carolina - Charleston
Charleston, South Carolina, 29406
Erlanger Health, Inc. - 1123255
Chattanooga, Tennessee, 37403
Baylor Scott & White Research Institute - Baylor Scott & White Research Institute
Dallas, Texas, 75246
ANESC Research
El Paso, Texas, 79912
Baylor Scott & White Neurology - McKinney
McKinney, Texas, 75071
The University of Vermont Medical Center
Burlington, Vermont, 05401
Integrated Neurology Services - Dr. Simon Fishman's Office
Falls Church, Virginia, 22043