The purpose of this study is to evaluate the safety, tolerability and efficacy of trametinib (SNR1611) in the treatment of amyotrophic lateral sclerosis.
Trametinib (SNR1611) is a MEK inhibitor that downregulates the MAPK/ERK pathway. In this study, the potential of MAPK/ERK pathway downregulation through trametinib (SNR1611) as a therapeutic treatment for amyotrophic lateral sclerosis (ALS) will be evaluated.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
SINGLE
Enrollment
23
0.5 mg/day
1 mg/day
100 mg/day (50 mg twice)
Inje University Busan Paik Hospital
Busan, South Korea
Asan Medical Center
Seoul, South Korea
Korea University Anam Hospital
Seoul, South Korea
Samsung Medical Center
Seoul, South Korea
Safety and tolerability of SNR1611: adverse events
Observation of adverse events
Time frame: 24-week (24-week extension and additional 48-week are optional)
K-ALSFRS-R score
Change in Korean Amyotrophic Lateral Sclerosis Functional Rating Scale Revised (K-ALSFRS-R) score from baseline
Time frame: 24-week (24-week extension and additional 48-week are optional)
FVC
Change in Forced Vital Capacity (FVC) from baseline
Time frame: 24-week (24-week extension and additional 48-week are optional)
CSF trough concentrations of SNR1611
Trough concentrations of SNR1611 in cerebrospinal fluid (CSF)
Time frame: 24-week (24-week extension and additional 48-week are optional)
Plasma trough concentrations of SNR1611
Trough concentrations of SNR1611 in plasma
Time frame: 24-week (24-week extension and additional 48-week are optional)
Milestone
Time to event milestones
Time frame: Additional 48-week (optional)
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Severance Hospital, Yonsei University Health System
Seoul, South Korea