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

Study Purpose:

The purpose of this study is to evaluate the safety, tolerability and efficacy of trametinib (SNR1611) in the treatment of amyotrophic lateral sclerosis.

Study Status:



Amyotrophic Lateral Sclerosis

Study Type:


Type of Intervention:


Intervention Name:

Trametinib (0.5 mg), Trametinib (1 mg), Riluzole (100 mg)




Phase 1/Phase 2

Study Chair(s)/Principal Investigator(s):

Byoung Joon Kim, Samsung Medical Center, Seoul, Republic of Korea

Clinicaltrials.gov ID:


Neals Affiliated?


Coordinating Center Contact Information

Inho Seo / email hidden; JavaScript is required / +82-2-542-0318

Full Study Summary:

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 Sponsor:

Genuv Inc.

Estimated Enrollment:


Estimated Study Start Date:

04 / 02 / 2020

Estimated Study Completion Date:

12 / 01 / 2024

Posting Last Modified Date:

09 / 15 / 2022

Date Study Added to neals.org:

03 / 30 / 2020

Minimum Age:

19 Years

Maximum Age:

75 Years

Main inclusion criteria:

- Patients diagnosed as definite, probable or probable-laboratory-supported ALS according to El Escorial Criteria.

- Patients of less than 2 years after the onset of ALS.

- Patients who meet the criteria of K-ALSFRS-R score and forced vital capacity.

Main exclusion criteria:

- Patients with primary lateral sclerosis, progressive muscular atrophy or lower motor neuron disease.

- Patients who have history of ALS treatment of edaravone or stem cell therapy within 16 weeks before screening.

- Patients who have permanently ceased the administration of riluzole due to lack of tolerability and/or efficacy.

- Patients in Class II to IV according to the New York Heart Association functional classification. Patients with myocardial infarction, unstable arrhythmia, and/or significant cardiovascular disease such as unstable angina within 12 weeks before screening.

- Patients who do not meet the criteria of laboratory tests and medical/operation history.

Inje University Busan Paik Hospital | Recruiting

Seong Il Oh

Korea, Republic of

Asan Medical Center | Recruiting

Young-Min Lim

Korea, Republic of

Korea University Anam Hospital | Recruiting

Byung-Jo Kim

Korea, Republic of

Samsung Medical Center | Recruiting

Byoung Joon Kim

Korea, Republic of

Severance Hospital, Yonsei University Health System | Recruiting

Ha Young Shin

Korea, Republic of