A Sequential Dose-Escalation, Randomized, Active-Controlled, Multi-Center, Phase 1/2a Clinical Trial to Evaluate the Safety, Tolerability and Efficacy of SNR1611 in Patients With Amyotrophic Lateral Sclerosis
Trial Snapshot
- Phase
- Phase 1
- Status
- Terminated
- Sponsor
- Genuv Inc.
- Enrollment
- 23
- Locations
- 5
- Primary Endpoint
- Safety and tolerability of SNR1611: adverse events
Study Overview
Brief Summary
The purpose of this study is to evaluate the safety, tolerability and efficacy of trametinib (SNR1611) in the treatment of amyotrophic lateral sclerosis.
Detailed Description
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 Design
- Study Type
- Interventional
- Allocation
- Randomized
- Intervention Model
- Parallel
- Primary Purpose
- Treatment
- Masking
- Single (Outcomes Assessor)
Masking Description
K-ALSFRS-R score will be measured by independent outcome assessors.
Eligibility Criteria
- Ages
- 19 Years to 75 Years (Adult, Older Adult)
- Sex
- All
- Accepts Healthy Volunteers
- No
Inclusion Criteria
- Not provided
Exclusion Criteria
- Not provided
Arms & Interventions
Trametinib (0.5 mg)
One tablet of trametinib 0.5 mg per day
Intervention: Trametinib (0.5 mg) (Drug)
Trametinib (1 mg)
Two tablets of trametinib 0.5 mg per day
Intervention: Trametinib (1 mg) (Drug)
Riluzole (100 mg)
One tablet of riluzole 50 mg taken twice per day
Intervention: Riluzole (100 mg) (Drug)
Outcomes
Primary Outcomes
Safety and tolerability of SNR1611: adverse events
Time Frame: 24-week (24-week extension and additional 48-week are optional)
Observation of adverse events
Secondary Outcomes
- K-ALSFRS-R score(24-week (24-week extension and additional 48-week are optional))
- FVC(24-week (24-week extension and additional 48-week are optional))
- Plasma trough concentrations of SNR1611(24-week (24-week extension and additional 48-week are optional))
- Milestone(Additional 48-week (optional))
- CSF trough concentrations of SNR1611(24-week (24-week extension and additional 48-week are optional))