Efficacy and Safety of a New Formulation of Oral Cladribine Compared With Placebo in Participants With Generalized Myasthenia Gravis (MyClad)
NCT Number: NCT06463587
Status: RECRUITING
Study Type: INTERVENTIONAL
Phases: PHASE3
Conditions: Generalized Myasthenia Gravis
Interventions: OTHER: Placebo|DRUG: Cladribine Low Dose|DRUG: Cladribine High Dose
Sponsor: Merck Healthcare KGaA, Darmstadt, Germany, an affiliate of Merck KGaA, Darmstadt, Germany
Collaborators: EMD Serono Research & Development Institute, Inc.
Sex: ALL
Age: ADULT, OLDER_ADULT
Enrollment: 240
Study Design: Allocation: RANDOMIZED|Intervention Model: SEQUENTIAL|Masking: QUADRUPLE (PARTICIPANT, CARE_PROVIDER, INVESTIGATOR, OUTCOMES_ASSESSOR)|Primary Purpose: TREATMENT
Start Date: 2024-06-25
Completion Date: 2030-07-23
Locations: SFM Clinical Research, LLC, Boca Raton, Florida, 33487, United States|Hakodate Municipal Hospital - Dept of Neurology, Hakodate-shi, Hokkaido, 392006, Japan|General Hanamaki Hospital - Dept of Neurology, Hanamaki-shi, Iwate-Ken, 025-0082, Japan|Keimyung University Dongsan Hospital, Seoul, 02841, Korea, Republic of|Samsung Medical Center, Seoul, 06351, Korea, Republic of

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.

Primary Outcome Measures: 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, Baseline, Week 24
Secondary Outcome Measures: Change from Baseline in Quantitative Myasthenia Gravis (QMG) Scale Score at Week 24 During the Double-Blind Placebo Controlled (DBPC) Period, Baseline, Week 24|Percentage of MG-ADL Responders at Week 24 During the Double-Blind Placebo Controlled (DBPC) Period, At Week 24|Change from Baseline in Myasthenia Gravis Composite (MGC) Scale Score at Week 24 During the Double-Blind Placebo Controlled (DBPC) Period, Baseline, Week 24|Percentage of Quantitative Myasthenia Gravis (QMG) Scale Responders at Week 24 During the Double-Blind Placebo Controlled (DBPC) Period, At Week 24|Time From Initial Cladribine Full Dose Treatment to First Retreatment of Rescue Treatment up to end of Study, Up to End of Study (Week 144)|Number of Participants With Adverse Events (AEs) and Adverse Events of Special Interest (AESIs), 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, Up to End of Study (Week 144)|Number of Participants with Abnormal Laboratory Variables including Absolute Lymphocyte Count and Vital Signs, Up to End of Study (Week 144)|Pharmacokinetic (PK) Plasma Concentration of Cladribine, 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, Baseline, Week 24