Trial of Safety, Tolerability and Efficacy of Trametinib (SNR1611) in Patients With Amyotrophic Lateral Sclerosis (ALS)

Phase 1

Terminated

• Conditions: Amyotrophic Lateral Sclerosis
• Interventions: Drug: Trametinib (0.5 mg); Drug: Trametinib (1 mg); Drug: Riluzole (100 mg)

• Registration Number: NCT04326283
• Lead Sponsor: Genuv Inc.

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 Timeline

• Study First Submitted: 2020-03-24
• Study First Submitted QC: 2020-03-27
• Study First Posted: 2020-03-30
• Study Start: 2020-04-02
• Primary Completion: 2023-04-28
• Study Completion: 2023-04-28
• Status Verified: 2023-05
• Last Update Submitted: 2023-05-07
• Last Update Posted: 2023-05-09

Recruitment & Eligibility

• Status: TERMINATED
• Sex: All
• Target Recruitment: 23

Inclusion Criteria

Not provided

Exclusion Criteria

Not provided

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
Trametinib (0.5 mg)	Trametinib (0.5 mg)	One tablet of trametinib 0.5 mg per day
Trametinib (1 mg)	Trametinib (1 mg)	Two tablets of trametinib 0.5 mg per day
Riluzole (100 mg)	Riluzole (100 mg)	One tablet of riluzole 50 mg taken twice per day

Primary Outcome Measures

Name	Time	Method
Safety and tolerability of SNR1611: adverse events	24-week (24-week extension and additional 48-week are optional)	Observation of adverse events

Secondary Outcome Measures

Name	Time	Method
K-ALSFRS-R score	24-week (24-week extension and additional 48-week are optional)	Change in Korean Amyotrophic Lateral Sclerosis Functional Rating Scale Revised (K-ALSFRS-R) score from baseline
FVC	24-week (24-week extension and additional 48-week are optional)	Change in Forced Vital Capacity (FVC) from baseline
Plasma trough concentrations of SNR1611	24-week (24-week extension and additional 48-week are optional)	Trough concentrations of SNR1611 in plasma
Milestone	Additional 48-week (optional)	Time to event milestones
CSF trough concentrations of SNR1611	24-week (24-week extension and additional 48-week are optional)	Trough concentrations of SNR1611 in cerebrospinal fluid (CSF)

Trial Locations

Locations (5)

Inje University Busan Paik Hospital; 🇰🇷 Busan, Korea, Republic of

Samsung Medical Center; 🇰🇷 Seoul, Korea, Republic of

Asan Medical Center; 🇰🇷 Seoul, Korea, Republic of

Korea University Anam Hospital; 🇰🇷 Seoul, Korea, Republic of

Severance Hospital, Yonsei University Health System; 🇰🇷 Seoul, Korea, Republic of