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

Amyotrophic Lateral Sclerosis Clinical Trial

Official title:

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

Clinical Trial Details — Status: Terminated

Administrative data

• NCT number: NCT04326283
• Other study ID #: CT1SNR1611ALS1
• Status: Terminated
• Phase: Phase 1/Phase 2
• Start date: April 2, 2020
• Est. completion date: April 28, 2023

Study information

• Verified date: May 2023
• Source: Genuv Inc.
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

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

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.

Recruitment information / eligibility

• Status: Terminated
• Enrollment: 23
• Est. completion date: April 28, 2023
• Est. primary completion date: April 28, 2023
• Accepts healthy volunteers: No
• Gender: All
• Age group: 19 Years to 75 Years

Eligibility

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.

Related Conditions & MeSH terms

• Amyotrophic Lateral Sclerosis
• Motor Neuron Disease
• Sclerosis

Intervention

Drug:
• Trametinib (0.5 mg)
0.5 mg/day
• Trametinib (1 mg)
1 mg/day
• Riluzole (100 mg)
100 mg/day (50 mg twice)

Locations

Country	Name	City	State
Korea, Republic of	Inje University Busan Paik Hospital	Busan
Korea, Republic of	Asan Medical Center	Seoul
Korea, Republic of	Korea University Anam Hospital	Seoul
Korea, Republic of	Samsung Medical Center	Seoul
Korea, Republic of	Severance Hospital, Yonsei University Health System	Seoul

Sponsors (1)

Lead Sponsor	Collaborator
Genuv Inc.

Country where clinical trial is conducted

Korea, Republic of, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Safety and tolerability of SNR1611: adverse events	Observation of adverse events	24-week (24-week extension and additional 48-week are optional)
Secondary	K-ALSFRS-R score	Change in Korean Amyotrophic Lateral Sclerosis Functional Rating Scale Revised (K-ALSFRS-R) score from baseline	24-week (24-week extension and additional 48-week are optional)
Secondary	FVC	Change in Forced Vital Capacity (FVC) from baseline	24-week (24-week extension and additional 48-week are optional)
Secondary	CSF trough concentrations of SNR1611	Trough concentrations of SNR1611 in cerebrospinal fluid (CSF)	24-week (24-week extension and additional 48-week are optional)
Secondary	Plasma trough concentrations of SNR1611	Trough concentrations of SNR1611 in plasma	24-week (24-week extension and additional 48-week are optional)
Secondary	Milestone	Time to event milestones	Additional 48-week (optional)