Amyotrophic Lateral SclerosisPhase 1/2NCT04326283
Trial of Safety, Tolerability and Efficacy of Trametinib (SNR1611) in Patients With Amyotrophic Lateral Sclerosis (ALS)
A Phase 1/2 study testing Trametinib (0.5 mg), Trametinib (1 mg) and Riluzole (100 mg) in people with Amyotrophic Lateral Sclerosis, run by Genuv Inc. at 5 sites in South Korea.
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. Read more about this study
- Who is it for?
- Ages 19 to 75 years
- Conditions the study is for
- Amyotrophic Lateral Sclerosis
- What is being tested?
- Trametinib (0.5 mg), Trametinib (1 mg) and Riluzole (100 mg)
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 23 participants
- Where
- 5 sitesSouth Korea
- Study dates
- 2 Apr 2020to 28 Apr 2023
- Phase
- Phase 1/2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 19 to 75 years
- All sexes
- No healthy volunteers
- Adult, Older adult
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 locations and contacts
5 sites in 1 country.
5 sites in 1 country
South Korea5 sites
Inje University Busan Paik Hospital
Busan
Asan Medical Center
Seoul
Korea University Anam Hospital
Seoul
Samsung Medical Center
Seoul
Severance Hospital, Yonsei University Health System
Seoul
About this study
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
The purpose of this study is to evaluate the safety, tolerability and efficacy of trametinib (SNR1611) in the treatment of amyotrophic lateral sclerosis.
Read the 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.
- Conditions
- Amyotrophic Lateral Sclerosis
- Being tested
- Trametinib (0.5 mg), Trametinib (1 mg), Riluzole (100 mg)
- Keywords
- ALS, Lou Gehrig's disease
- Medical subject headings
- Amyotrophic Lateral Sclerosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Trametinib (0.5 mg)Drug
Also called Meqsel, SNR1611
0.5 mg/day
Trametinib (1 mg)Drug
Also called Meqsel, SNR1611
1 mg/day
Riluzole (100 mg)Drug
Also called Yooritek
100 mg/day (50 mg twice)
| Study group | Role | What they receive |
|---|---|---|
| Trametinib (0.5 mg) | Experimental | One tablet of trametinib 0.5 mg per day Receives: Drug: Trametinib (0.5 mg) |
| Trametinib (1 mg) | Experimental | Two tablets of trametinib 0.5 mg per day Receives: Drug: Trametinib (1 mg) |
| Riluzole (100 mg) | Active comparator | One tablet of riluzole 50 mg taken twice per day Receives: Drug: Riluzole (100 mg) |
What is being measured
The primary outcome is the main question the study is designed to answer.
Safety and tolerability of SNR1611: adverse events
Observation of adverse events
Measured: 24-week (24-week extension and additional 48-week are optional)
Secondary outcomes (5)
K-ALSFRS-R score
Change in Korean Amyotrophic Lateral Sclerosis Functional Rating Scale Revised (K-ALSFRS-R) score from baseline
Measured: 24-week (24-week extension and additional 48-week are optional)
FVC
Change in Forced Vital Capacity (FVC) from baseline
Measured: 24-week (24-week extension and additional 48-week are optional)
CSF trough concentrations of SNR1611
Trough concentrations of SNR1611 in cerebrospinal fluid (CSF)
Measured: 24-week (24-week extension and additional 48-week are optional)
Plasma trough concentrations of SNR1611
Trough concentrations of SNR1611 in plasma
Measured: 24-week (24-week extension and additional 48-week are optional)
Milestone
Time to event milestones
Measured: Additional 48-week (optional)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted30 Mar 2020
- Study start2 Apr 2020
- Primary completion28 Apr 2023
- Study completion28 Apr 2023
Why it stopped: Terminated as it is considered that necessary data have been collected
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Amyotrophic Lateral SclerosisStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- 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
- Single blind
- Who is masked
- Outcomes assessor
- Enrolment
- 23 participants (actual)
Sponsor and investigators
- Lead sponsor
- Genuv Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Byoung Joon Kim, Principal investigator, Samsung Medical Center, Seoul, Republic of Korea
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04326283
- Sponsor's protocol ID
- CT1SNR1611ALS1
- Status last verified
- May 2023
- First submitted
- 24 Mar 2020
- Last updated
- 9 May 2023
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 9 May 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT04326283
- Acronym / sponsor ID
- CT1SNR1611ALS1
- Conditions
- Amyotrophic Lateral Sclerosis
- Keywords
- ALS, Lou Gehrig's disease
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Terminated as it is considered that necessary data have been collected
- Enrolment
- 23 participants (actual)
- Lead sponsor
- Genuv Inc.Industry (pharma, biotech, devices)
- Study start
- 2 Apr 2020 (actual)
- Primary completion
- 28 Apr 2023 (actual)
- Study completion
- 28 Apr 2023 (actual)
- First posted
- 30 Mar 2020 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Single blind (outcomes assessor)
- Number of arms
- 3
- 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.
| Arm | Type | Receives |
|---|---|---|
| Trametinib (0.5 mg) | Experimental | Drug: Trametinib (0.5 mg)One tablet of trametinib 0.5 mg per day |
| Trametinib (1 mg) | Experimental | Drug: Trametinib (1 mg)Two tablets of trametinib 0.5 mg per day |
| Riluzole (100 mg) | Active comparator | Drug: Riluzole (100 mg)One tablet of riluzole 50 mg taken twice per day |
Who may qualify
- Ages
- 19 to 75 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Trametinib (0.5 mg)Also: Meqsel, SNR16110.5 mg/day | Drug | Trametinib (0.5 mg) |
| Trametinib (1 mg)Also: Meqsel, SNR16111 mg/day | Drug | Trametinib (1 mg) |
| Riluzole (100 mg)Also: Yooritek100 mg/day (50 mg twice) | Drug | Riluzole (100 mg) |
Primary outcome
Safety and tolerability of SNR1611: adverse events
Time frame: 24-week (24-week extension and additional 48-week are optional)
Observation of adverse events
5 secondary outcomes
K-ALSFRS-R score
Time frame: 24-week (24-week extension and additional 48-week are optional)
FVC
Time frame: 24-week (24-week extension and additional 48-week are optional)
CSF trough concentrations of SNR1611
Time frame: 24-week (24-week extension and additional 48-week are optional)
Plasma trough concentrations of SNR1611
Time frame: 24-week (24-week extension and additional 48-week are optional)
Milestone
Time frame: Additional 48-week (optional)
Results and safety
The study was stopped early (reason given: Terminated as it is considered that necessary data have been collected). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 9 May 2023.
- Individual participant dataSharing planned: No
Related trials for Amyotrophic Lateral Sclerosis
RecruitingPhase 1/2
Study is to Assess the Safety and Tolerability of VTx-002 in Participants With ALS
Amyotrophic Lateral Sclerosis
Not yet recruiting
A Study on the Addition of Alsum30 for the Treatment of Amyotrophic Lateral Sclerosis
Amyotrophic Lateral Sclerosis (ALS), Bacillus Coagulans
Active, not recruiting
Functional Outcomes and Control Using Synchron BCI - Australia
Neurologic Disorder, Neurologic Diseases
Not yet recruitingPhase 1
Safety, Tolerability, and Pharmacodynamic Exploration of Orelabrutinib in Patients With Amyotrophic Lateral Sclerosis (ALS)
Amyotrophic Lateral Sclerosis (ALS)
Recruiting
Clinical Outcome Assessment for AT & BCI
Spinal Cord Injury, ALS (Amyotrophic Lateral Sclerosis)
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Byoung Joon Kim principal investigator
Samsung Medical Center, Seoul, Republic of Korea
5 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing Trametinib (0.5 mg), Trametinib (1 mg) and Riluzole (100 mg) in people with Amyotrophic Lateral Sclerosis, run by Genuv Inc. at 5 sites in South Korea.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: stopped early, as of the sponsor's last update on 9 May 2023. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 5 sites in South Korea. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study was stopped early (reason given: Terminated as it is considered that necessary data have been collected). Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Safety and tolerability of SNR1611: adverse events (time frame: 24-week (24-week extension and additional 48-week are optional)). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 9 May 2023.