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

Primary Purpose

Amyotrophic Lateral Sclerosis

Status
Terminated
Phase
Phase 1
Locations
Korea, Republic of
Study Type
Interventional
Intervention
Trametinib (0.5 mg)
Trametinib (1 mg)
Riluzole (100 mg)
Sponsored by
Genuv Inc.
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Amyotrophic Lateral Sclerosis focused on measuring ALS, Lou Gehrig's disease

Eligibility Criteria

19 Years - 75 Years (Adult, Older Adult)All SexesDoes not accept healthy volunteers

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.

Sites / Locations

  • Inje University Busan Paik Hospital
  • Asan Medical Center
  • Korea University Anam Hospital
  • Samsung Medical Center
  • Severance Hospital, Yonsei University Health System

Arms of the Study

Arm 1

Arm 2

Arm 3

Arm Type

Experimental

Experimental

Active Comparator

Arm Label

Trametinib (0.5 mg)

Trametinib (1 mg)

Riluzole (100 mg)

Arm Description

One tablet of trametinib 0.5 mg per day

Two tablets of trametinib 0.5 mg per day

One tablet of riluzole 50 mg taken twice per day

Outcomes

Primary Outcome Measures

Safety and tolerability of SNR1611: adverse events
Observation of adverse events

Secondary Outcome Measures

K-ALSFRS-R score
Change in Korean Amyotrophic Lateral Sclerosis Functional Rating Scale Revised (K-ALSFRS-R) score from baseline
FVC
Change in Forced Vital Capacity (FVC) from baseline
CSF trough concentrations of SNR1611
Trough concentrations of SNR1611 in cerebrospinal fluid (CSF)
Plasma trough concentrations of SNR1611
Trough concentrations of SNR1611 in plasma
Milestone
Time to event milestones

Full Information

First Posted
March 24, 2020
Last Updated
May 7, 2023
Sponsor
Genuv Inc.
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1. Study Identification

Unique Protocol Identification Number
NCT04326283
Brief Title
Trial of Safety, Tolerability and Efficacy of Trametinib (SNR1611) in Patients With Amyotrophic Lateral Sclerosis (ALS)
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
Study Type
Interventional

2. Study Status

Record Verification Date
May 2023
Overall Recruitment Status
Terminated
Why Stopped
Terminated as it is considered that necessary data have been collected
Study Start Date
April 2, 2020 (Actual)
Primary Completion Date
April 28, 2023 (Actual)
Study Completion Date
April 28, 2023 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Genuv Inc.

4. Oversight

Studies a U.S. FDA-regulated Drug Product
No
Studies a U.S. FDA-regulated Device Product
No
Product Manufactured in and Exported from the U.S.
No
Data Monitoring Committee
Yes

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Amyotrophic Lateral Sclerosis
Keywords
ALS, Lou Gehrig's disease

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 1, Phase 2
Interventional Study Model
Parallel Assignment
Model Description
Patients will be randomly assigned 4:1 to receive SNR1611 (0.5, 1 mg) or riluzole as an active comparator. The study will initiate with the first group of patients who will receive SNR1611 (0.5 mg, 8 patients) or riluzole (100 mg, 2 patients), while the initiation of the next dose groups will be decided by the IDMC (Independent Data Monitoring Committee) through safety assessment of the first 4-week periods of prior dose groups.
Masking
Outcomes Assessor
Masking Description
K-ALSFRS-R score will be measured by independent outcome assessors.
Allocation
Randomized
Enrollment
23 (Actual)

8. Arms, Groups, and Interventions

Arm Title
Trametinib (0.5 mg)
Arm Type
Experimental
Arm Description
One tablet of trametinib 0.5 mg per day
Arm Title
Trametinib (1 mg)
Arm Type
Experimental
Arm Description
Two tablets of trametinib 0.5 mg per day
Arm Title
Riluzole (100 mg)
Arm Type
Active Comparator
Arm Description
One tablet of riluzole 50 mg taken twice per day
Intervention Type
Drug
Intervention Name(s)
Trametinib (0.5 mg)
Other Intervention Name(s)
Meqsel, SNR1611
Intervention Description
0.5 mg/day
Intervention Type
Drug
Intervention Name(s)
Trametinib (1 mg)
Other Intervention Name(s)
Meqsel, SNR1611
Intervention Description
1 mg/day
Intervention Type
Drug
Intervention Name(s)
Riluzole (100 mg)
Other Intervention Name(s)
Yooritek
Intervention Description
100 mg/day (50 mg twice)
Primary Outcome Measure Information:
Title
Safety and tolerability of SNR1611: adverse events
Description
Observation of adverse events
Time Frame
24-week (24-week extension and additional 48-week are optional)
Secondary Outcome Measure Information:
Title
K-ALSFRS-R score
Description
Change in Korean Amyotrophic Lateral Sclerosis Functional Rating Scale Revised (K-ALSFRS-R) score from baseline
Time Frame
24-week (24-week extension and additional 48-week are optional)
Title
FVC
Description
Change in Forced Vital Capacity (FVC) from baseline
Time Frame
24-week (24-week extension and additional 48-week are optional)
Title
CSF trough concentrations of SNR1611
Description
Trough concentrations of SNR1611 in cerebrospinal fluid (CSF)
Time Frame
24-week (24-week extension and additional 48-week are optional)
Title
Plasma trough concentrations of SNR1611
Description
Trough concentrations of SNR1611 in plasma
Time Frame
24-week (24-week extension and additional 48-week are optional)
Title
Milestone
Description
Time to event milestones
Time Frame
Additional 48-week (optional)

10. Eligibility

Sex
All
Minimum Age & Unit of Time
19 Years
Maximum Age & Unit of Time
75 Years
Accepts Healthy Volunteers
No
Eligibility 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.
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Byoung Joon Kim
Organizational Affiliation
Samsung Medical Center, Seoul, Republic of Korea
Official's Role
Principal Investigator
Facility Information:
Facility Name
Inje University Busan Paik Hospital
City
Busan
Country
Korea, Republic of
Facility Name
Asan Medical Center
City
Seoul
Country
Korea, Republic of
Facility Name
Korea University Anam Hospital
City
Seoul
Country
Korea, Republic of
Facility Name
Samsung Medical Center
City
Seoul
Country
Korea, Republic of
Facility Name
Severance Hospital, Yonsei University Health System
City
Seoul
Country
Korea, Republic of

12. IPD Sharing Statement

Plan to Share IPD
No

Learn more about this trial

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

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