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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT04326283
Recruitment Status : Recruiting
First Posted : March 30, 2020
Last Update Posted : May 8, 2020
Sponsor:
Information provided by (Responsible Party):
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.

Condition or disease Intervention/treatment Phase
Amyotrophic Lateral Sclerosis Drug: Trametinib (0.5 mg) Drug: Trametinib (1 mg) Drug: Trametinib (2 mg) Drug: Riluzole (100 mg) Phase 1 Phase 2

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.

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 30 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Intervention Model Description: Patients will be randomly assigned 4:1 to receive SNR1611 (0.5, 1 or 2 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: Single (Outcomes Assessor)
Masking Description: K-ALSFRS-R score will be measured by independent outcome assessors.
Primary Purpose: Treatment
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
Actual Study Start Date : April 2, 2020
Estimated Primary Completion Date : March 2022
Estimated Study Completion Date : March 2022


Arm Intervention/treatment
Experimental: Trametinib (0.5 mg)
One tablet of trametinib 0.5 mg per day
Drug: Trametinib (0.5 mg)
0.5 mg/day
Other Names:
  • Meqsel
  • SNR1611

Experimental: Trametinib (1 mg)
Two tablets of trametinib 0.5 mg per day
Drug: Trametinib (1 mg)
1 mg/day
Other Names:
  • Meqsel
  • SNR1611

Experimental: Trametinib (2 mg)
One tablet of trametinib 2 mg per day
Drug: Trametinib (2 mg)
2 mg/day
Other Names:
  • Meqsel
  • SNR1611

Active Comparator: Riluzole (100 mg)
One tablet of riluzole 50 mg taken twice per day
Drug: Riluzole (100 mg)
100 mg/day (50 mg twice)
Other Name: Yooritek




Primary Outcome Measures :
  1. Safety and tolerability of SNR1611: adverse events [ Time Frame: 24-week (additional 24-week is optional) ]
    Observation of adverse events


Secondary Outcome Measures :
  1. K-ALSFRS-R score [ Time Frame: 24-week (additional 24-week is optional) ]
    Change in Korean Amyotrophic Lateral Sclerosis Functional Rating Scale Revised (K-ALSFRS-R) score from baseline

  2. FVC [ Time Frame: 24-week (additional 24-week is optional) ]
    Change in Forced Vital Capacity (FVC) from baseline

  3. CSF trough concentrations of SNR1611 [ Time Frame: 24-week (additional 24-week is optional) ]
    Trough concentrations of SNR1611 in cerebrospinal fluid (CSF)

  4. Plasma trough concentrations of SNR1611 [ Time Frame: 24-week (additional 24-week is optional) ]
    Trough concentrations of SNR1611 in plasma



Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


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Ages Eligible for Study:   19 Years to 75 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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.

Exclusion Criteria:

  • Patients with primary lateral sclerosis, progressive muscular atrophy or lower motor neuron disease.
  • Patients who have history of ALS treatment of riluzole, 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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04326283


Contacts
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Contact: Inho Seo +82-2-542-0318 info@genuv.com

Locations
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Korea, Republic of
Asan Medical Center Recruiting
Seoul, Korea, Republic of
Contact: Kwang-Kuk Kim         
Korea University Anam Hospital Recruiting
Seoul, Korea, Republic of
Contact: Byung-Jo Kim         
Samsung Medical Center Recruiting
Seoul, Korea, Republic of
Contact: Byoung Joon Kim         
Severance Hospital, Yonsei University Health System Recruiting
Seoul, Korea, Republic of
Contact: Seung Woo Kim         
Sponsors and Collaborators
Genuv Inc.
Investigators
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Principal Investigator: Byoung Joon Kim Samsung Medical Center, Seoul, Republic of Korea
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Responsible Party: Genuv Inc.
ClinicalTrials.gov Identifier: NCT04326283    
Other Study ID Numbers: CT1SNR1611ALS1
First Posted: March 30, 2020    Key Record Dates
Last Update Posted: May 8, 2020
Last Verified: May 2020
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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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
Keywords provided by Genuv Inc.:
ALS
Lou Gehrig's disease
Additional relevant MeSH terms:
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Motor Neuron Disease
Amyotrophic Lateral Sclerosis
Sclerosis
Pathologic Processes
Neurodegenerative Diseases
Nervous System Diseases
Neuromuscular Diseases
Spinal Cord Diseases
Central Nervous System Diseases
TDP-43 Proteinopathies
Proteostasis Deficiencies
Metabolic Diseases
Trametinib
Riluzole
Antineoplastic Agents
Protein Kinase Inhibitors
Enzyme Inhibitors
Molecular Mechanisms of Pharmacological Action
Anticonvulsants
Excitatory Amino Acid Antagonists
Excitatory Amino Acid Agents
Neurotransmitter Agents
Physiological Effects of Drugs
Neuroprotective Agents
Protective Agents