Efficacy and Safety of Tazbentetol in ALS Participants
A Phase 2B/3, Randomized, Double-blind, Placebo-controlled Study to Evaluate the Efficacy and Safety of Tazbentetol in Participants With Amyotrophic Lateral Sclerosis (ALS)
1 other identifier
interventional
430
0 countries
N/A
Brief Summary
The objectives of this study are to examine the effects of tazbentetol on clinical measures of ALS, patient reported outcomes (PROs), long-term safety and tolerability.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P75+ for phase_2
Started Mar 2026
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
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Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
January 6, 2026
CompletedFirst Posted
Study publicly available on registry
January 8, 2026
CompletedStudy Start
First participant enrolled
March 1, 2026
CompletedPrimary Completion
Last participant's last visit for primary outcome
December 31, 2027
ExpectedStudy Completion
Last participant's last visit for all outcomes
March 1, 2028
March 2, 2026
February 1, 2026
1.8 years
January 6, 2026
February 26, 2026
Conditions
Keywords
Outcome Measures
Primary Outcomes (3)
Phase 2: Absolute change from baseline in Amyotrophic Lateral Sclerosis Functional Rating Scale-revised (ALSFRS-R) total score
Questionnaire administered by a clinician that measures participants' ability to function in certain daily activities. Each type of function is scored from 4 (normal) to 0 (no ability), with a maximum total score of 48 and a minimum total score of 0. Patients with higher scores have more physical function.
36 weeks
Phase 3: Absolute change from baseline in Amyotrophic Lateral Sclerosis Functional Rating Scale-revised (ALSFRS-R) total score
Questionnaire administered by a clinician that measures participants' ability to function in certain daily activities. Each type of function is scored from 4 (normal) to 0 (no ability), with a maximum total score of 48 and a minimum total score of 0. Patients with higher scores have more physical function.
36 weeks
OLE: Absolute change from baseline in Amyotrophic Lateral Sclerosis Functional Rating Scale-revised (ALSFRS-R) total score
Questionnaire administered by a clinician that measures participants' ability to function in certain daily activities. Each type of function is scored from 4 (normal) to 0 (no ability), with a maximum total score of 48 and a minimum total score of 0. Patients with higher scores have more physical function.
36 weeks
Secondary Outcomes (21)
Phase 2: Combined Assessment of Function and Survival (CAFS) scores
36 weeks
Phase 2: Change from baseline in Clinician Global Impression-Inhibitory (CGI-I)
36 weeks
Phase 2: Change from baseline in Edinburgh Cognitive and Behavioural ALS Screen (ECAS)
36 weeks
Phase 2: Change from baseline in ALS Assessment Questionnaire 40 items (ALSAQ-40)
36 weeks
Phase 2: Change from baseline in Patient Global Impression (PGI) of Improvement
36 weeks
- +16 more secondary outcomes
Other Outcomes (7)
Phase 2: ALSFRS-R total score rate of decline
36 weeks
Phase 2: Proportion of patients alive and survival time
36 weeks
Phase 2 substudy: To compare electroencephalogram (EEG) outcomes of two dose levels of tazbentetol to placebo in participants with ALS
36 weeks
- +4 more other outcomes
Study Arms (2)
tazbentetol
EXPERIMENTALsynthetic small molecule
Placebo
PLACEBO COMPARATORplacebo
Interventions
Participants in both Phase 2B and Phase 3 will be randomized to received study drug tazbentetol or placebo tablets. Participants in the open-label extension phase will receive the dose determined from Phase 2.
participants in double blind placebo controlled phase will be randomized to received placebo tablets
Eligibility Criteria
You may qualify if:
- Age 18-80
- ALS TRICALS risk score
- Stable dose of standard of care treatment
- Contraception use by men or women consistent with local regulations
- Able and willing to provide written informed consent
You may not qualify if:
- Underlying physical or psychological condition prohibiting study completion
- Clinically significant cardiac disease
- Active or history of malignancy in the past 5 years
- Serious infection within 1 month of screening
- Acute illness within 30 days of Day 1
- History of suicidal behavior or suicidal ideation
- Active cigarette smokers and users of nicotine-containing products
- Neurodegenerative disease
- External respiratory support or supplemental oxygen requirement
- HIV, hepatitis B and hepatitis C positive
- Vaccines within 14 days
- Other investigational products within 30 days
- Blood donation within 30 days
- Plasma donation within 7 days
- Pregnant or breastfeeding
Contact the study team to confirm eligibility.
Sponsors & Collaborators
- Spinogenixlead
MeSH Terms
Conditions
Condition Hierarchy (Ancestors)
Central Study Contacts
Study Design
- Study Type
- interventional
- Phase
- phase 2
- Allocation
- RANDOMIZED
- Masking
- QUADRUPLE
- Who Masked
- PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
- Purpose
- TREATMENT
- Intervention Model
- PARALLEL
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
January 6, 2026
First Posted
January 8, 2026
Study Start
March 1, 2026
Primary Completion (Estimated)
December 31, 2027
Study Completion (Estimated)
March 1, 2028
Last Updated
March 2, 2026
Record last verified: 2026-02
Data Sharing
- IPD Sharing
- Will not share