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

Genuv

Status and phase: Terminated; Phase 2; Phase 1

Conditions: Amyotrophic Lateral Sclerosis

Treatments: Drug: Riluzole (100 mg); Drug: Trametinib (1 mg); Drug: Trametinib (0.5 mg)

Study type: Interventional

Funder types: Industry

Identifiers

NCT04326283

CT1SNR1611ALS1

Details and patient eligibility

About

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

Full 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.

Enrollment: 23 patients

Sex: All

Ages: 19 to 75 years old

Volunteers: No Healthy Volunteers

Inclusion and exclusion criteria

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.

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Single Blind

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23 participants in 3 patient groups

Trametinib (0.5 mg)

Experimental group

Description:

One tablet of trametinib 0.5 mg per day

Treatment:

Drug: Trametinib (0.5 mg)

Trametinib (1 mg)

Experimental group

Description:

Two tablets of trametinib 0.5 mg per day

Treatment:

Drug: Trametinib (1 mg)

Riluzole (100 mg)

Active Comparator group

Description:

One tablet of riluzole 50 mg taken twice per day

Treatment:

Drug: Riluzole (100 mg)