Amyotrophic Lateral SclerosisPhase 2/3NCT07325591
Efficacy and Safety of Tazbentetol in ALS Participants
A Phase 2/3 study testing Tazbentetol in people with Amyotrophic Lateral Sclerosis, run by Spinogenix.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 to 80 years
- Conditions the study is for
- Amyotrophic Lateral Sclerosis
- What is being tested?
- Tazbentetol
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 430 participants
- Where
- No sites listed yet
- Study dates
- 1 Oct 2026to 1 Mar 2028
- Phase
- Phase 2/3
- Are there results?
- Not yet: the study is under way
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 18 to 80 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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
Who cannot join
- 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
Show all 15Show fewer
- 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
Trial locations and contacts
No sites are listed yet. Contact the study team or a site near you to ask about taking part.
About this study
Official title: 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)
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.
Read the detailed description
This is a Phase 2B/3 adaptive design randomized, double-blind, placebo-controlled (DBPC) study to evaluate the efficacy, safety and tolerability of tazbentetol administered orally in participants with ALS.
The study consists of 2 parts, as follows
Phase 2 double-blind, placebo controlled (DBPC): Randomized, double-blind, placebo-controlled study. Participants will be randomized to receive tazbentetol or placebo for 36 weeks.
Phase 3 double blind, placebo controlled (DBPC): Randomized, double-blind, placebo-controlled study. Participants will be randomized to receive the dose determined from Phase 2 or placebo for 36 weeks.
Open-label extension: Eligible participants who complete 36 weeks in the DBPC of either Phase 2 or Phase 3 will be offered to enroll into the OLE, starting at the corresponding DBPC Week 36 visit, and receive tazbentetol for 36 weeks. The dose for this extension will be based on data from DBPC phases.
- Conditions
- Amyotrophic Lateral Sclerosis
- Being tested
- Tazbentetol, placebo
- Keywords
- regenerative, synapse, Amyotrophic Lateral Sclerosis, placebo-controlled
- 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.
TazbentetolDrug
Also called SPG302
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.
placeboDrug
participants in double blind placebo controlled phase will be randomized to received placebo tablets
| Study group | Role | What they receive |
|---|---|---|
| tazbentetol | Experimental | synthetic small molecule Receives: Drug: Tazbentetol |
| Placebo | Placebo comparator | placebo Receives: Drug: placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 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.
Measured: 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.
Measured: 36 weeks
Secondary outcomes (21)
Phase 2: Combined Assessment of Function and Survival (CAFS) scores
This assessment ranks clinical outcome based on changes to ALSFRS-R score. A higher score indicates a better clinical outcome.
Measured: 36 weeks
Phase 2: Change from baseline in Clinician Global Impression-Inhibitory (CGI-I)
Change in clinician rated measures on scale of 1 to 7, higher value indicates more severe symptom presentation
Measured: 36 weeks
Phase 2: Change from baseline in Edinburgh Cognitive and Behavioural ALS Screen (ECAS)
Questionnaire to assesses cognitive and behavioral changes in people with (ALS) through a 136-point test covering language, verbal fluency, executive function, memory, and visuospatial cognitive domains. A lower score indicates worsening of symptoms.
Measured: 36 weeks
Phase 2: Change from baseline in ALS Assessment Questionnaire 40 items (ALSAQ-40)
The ALSAQ40 score is a measure of QoL for patients with ALS. The ALSAQ40 evaluates domains that include physical mobility, activities of daily living (ADL) and independence, eating and drinking, communication, and emotional reactions. A higher score indicates higher disability.
Measured: 36 weeks
Phase 2: Change from baseline in Patient Global Impression (PGI) of Improvement
This will measure all aspects of patient health and improvement or decline. A higher scale indicates greater disability.
Measured: 36 weeks
Phase 2: Change from baseline in Rasch Overall ALS Disability (ROADS).
This will measure patient reported outcomes of overall disability. A lower score indicates greater disability.
Measured: 36 weeks
Phase 2: Incidence, nature, and severity of treatment emergent adverse events (TEAEs) and serious adverse events (SAEs)
This will assess the safety and tolerability of tazbentetol in of participants with ALS
Measured: 36 weeks
Phase 3: Combined Assessment of Function and Survival (CAFS) scores
This assessment ranks clinical outcome based on changes to ALSFRS-R score. A higher score indicates a better clinical outcome.
Measured: 36 weeks
Phase 3: Change from baseline in Clinician Global Impression-Inhibitory (CGI-I)
Change in clinician rated measures on scale of 1 to 7, higher value indicates more severe symptom presentation
Measured: 36 weeks
Phase 3: Change from baseline in Edinburgh Cognitive and Behavioural ALS Screen (ECAS)
Questionnaire to assesses cognitive and behavioral changes in people with (ALS) through a 136-point test covering language, verbal fluency, executive function, memory, and visuospatial cognitive domains. A lower score indicates worsening of symptoms.
Measured: 36 weeks
Phase 3: Change from baseline in ALS Assessment Questionnaire 40 items (ALSAQ-40)
The ALSAQ40 score is a measure of QoL for patients with ALS. The ALSAQ40 evaluates domains that include physical mobility, activities of daily living (ADL) and independence, eating and drinking, communication, and emotional reactions. A higher score indicates higher disability.
Measured: 36 weeks
Phase 3: Change from baseline in Patient Global Impression (PGI) of Improvement
This will measure all aspects of patient health and improvement or decline. A higher scale indicates greater disability.
Measured: 36 weeks
Phase 3: Change from baseline in Rasch Overall ALS Disability (ROADS).
This will measure patient reported outcomes of overall disability. A lower score indicates greater disability.
Measured: 36 weeks
Phase 3: Incidence, nature, and severity of treatment emergent adverse events (TEAEs) and serious adverse events (SAEs)
This will assess the safety and tolerability of tazbentetol in of participants with ALS
Measured: 36 weeks
OLE: Combined Assessment of Function and Survival (CAFS) scores
This assessment ranks clinical outcome based on changes to ALSFRS-R score. A higher score indicates a better clinical outcome.
Measured: 36 weeks
OLE: Change from baseline in Clinician Global Impression-Inhibitory (CGI-I)
Change in clinician rated measures on scale of 1 to 7, higher value indicates more severe symptom presentation
Measured: 36 weeks
OLE: Change from baseline in ALS Assessment Questionnaire 40 items (ALSAQ-40)
The ALSAQ40 score is a measure of QoL for patients with ALS. The ALSAQ40 evaluates domains that include physical mobility, activities of daily living (ADL) and independence, eating and drinking, communication, and emotional reactions. A higher score indicates higher disability.
Measured: 36 weeks
OLE: Change from baseline in Edinburgh Cognitive and Behavioural ALS Screen (ECAS)
Questionnaire to assesses cognitive and behavioral changes in people with (ALS) through a 136-point test covering language, verbal fluency, executive function, memory, and visuospatial cognitive domains. A lower score indicates worsening of symptoms.
Measured: 36 weeks
OLE: Change from baseline in Patient Global Impression (PGI) of Improvement
This will measure all aspects of patient health and improvement or decline. A higher scale indicates greater disability.
Measured: 36 weeks
OLE: Change from baseline in Rasch Overall ALS Disability (ROADS).
This will measure patient reported outcomes of overall disability. A lower score indicates greater disability.
Measured: 36 weeks
OLE: Incidence, nature, and severity of treatment emergent adverse events (TEAEs) and serious adverse events (SAEs)
This will assess the safety and tolerability of tazbentetol in of participants with ALS
Measured: 36 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted8 Jan 2026
- Study start1 Oct 2026 (estimated)
- Primary completion31 Dec 2027 (estimated)
- Study completion1 Mar 2028 (estimated)
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 2/3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 430 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Spinogenix
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07325591
- Sponsor's protocol ID
- SPG302-ALS-003 (SPARK)
- Status last verified
- Sep 2026
- First submitted
- 6 Jan 2026
- Last updated
- 10 Sep 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 10 Sep 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- 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)
- ClinicalTrials.gov ID
- NCT07325591
- Acronym / sponsor ID
- SPG302-ALS-003 (SPARK)
- Conditions
- Amyotrophic Lateral Sclerosis
- Keywords
- regenerative, synapse, Amyotrophic Lateral Sclerosis, placebo-controlled
- Phase
- Phase 2/3
- Study type
- Interventional
- Recruitment status
- Not yet recruiting
- Enrolment
- 430 participants (estimated)
- Lead sponsor
- SpinogenixIndustry (pharma, biotech, devices)
- Study start
- 1 Oct 2026 (estimated)
- Primary completion
- 31 Dec 2027 (estimated)
- Study completion
- 1 Mar 2028 (estimated)
- First posted
- 8 Jan 2026 (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 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.
Detailed description
This is a Phase 2B/3 adaptive design randomized, double-blind, placebo-controlled (DBPC) study to evaluate the efficacy, safety and tolerability of tazbentetol administered orally in participants with ALS.
The study consists of 2 parts, as follows
Phase 2 double-blind, placebo controlled (DBPC): Randomized, double-blind, placebo-controlled study. Participants will be randomized to receive tazbentetol or placebo for 36 weeks.
Phase 3 double blind, placebo controlled (DBPC): Randomized, double-blind, placebo-controlled study. Participants will be randomized to receive the dose determined from Phase 2 or placebo for 36 weeks.
Open-label extension: Eligible participants who complete 36 weeks in the DBPC of either Phase 2 or Phase 3 will be offered to enroll into the OLE, starting at the corresponding DBPC Week 36 visit, and receive tazbentetol for 36 weeks. The dose for this extension will be based on data from DBPC phases.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| tazbentetol | Experimental | Drug: Tazbentetolsynthetic small molecule |
| Placebo | Placebo comparator | Drug: placeboplacebo |
Who may qualify
- Ages
- 18 to 80 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (5 listed)
- 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
Key exclusion criteria (15 listed)
- 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
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 |
|---|---|---|
| TazbentetolAlso: SPG302Participants 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. | Drug | tazbentetol |
| placeboparticipants in double blind placebo controlled phase will be randomized to received placebo tablets | Drug | Placebo |
Primary outcome
Phase 2: Absolute change from baseline in Amyotrophic Lateral Sclerosis Functional Rating Scale-revised (ALSFRS-R) total score
Time frame: 36 weeks
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.
Phase 3: Absolute change from baseline in Amyotrophic Lateral Sclerosis Functional Rating Scale-revised (ALSFRS-R) total score
Time frame: 36 weeks
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.
OLE: Absolute change from baseline in Amyotrophic Lateral Sclerosis Functional Rating Scale-revised (ALSFRS-R) total score
Time frame: 36 weeks
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.
21 secondary outcomes
Phase 2: Combined Assessment of Function and Survival (CAFS) scores
Time frame: 36 weeks
Phase 2: Change from baseline in Clinician Global Impression-Inhibitory (CGI-I)
Time frame: 36 weeks
Phase 2: Change from baseline in Edinburgh Cognitive and Behavioural ALS Screen (ECAS)
Time frame: 36 weeks
Phase 2: Change from baseline in ALS Assessment Questionnaire 40 items (ALSAQ-40)
Time frame: 36 weeks
Phase 2: Change from baseline in Patient Global Impression (PGI) of Improvement
Time frame: 36 weeks
Phase 2: Change from baseline in Rasch Overall ALS Disability (ROADS).
Time frame: 36 weeks
Phase 2: Incidence, nature, and severity of treatment emergent adverse events (TEAEs) and serious adverse events (SAEs)
Time frame: 36 weeks
Phase 3: Combined Assessment of Function and Survival (CAFS) scores
Time frame: 36 weeks
Phase 3: Change from baseline in Clinician Global Impression-Inhibitory (CGI-I)
Time frame: 36 weeks
Phase 3: Change from baseline in Edinburgh Cognitive and Behavioural ALS Screen (ECAS)
Time frame: 36 weeks
Phase 3: Change from baseline in ALS Assessment Questionnaire 40 items (ALSAQ-40)
Time frame: 36 weeks
Phase 3: Change from baseline in Patient Global Impression (PGI) of Improvement
Time frame: 36 weeks
Phase 3: Change from baseline in Rasch Overall ALS Disability (ROADS).
Time frame: 36 weeks
Phase 3: Incidence, nature, and severity of treatment emergent adverse events (TEAEs) and serious adverse events (SAEs)
Time frame: 36 weeks
OLE: Combined Assessment of Function and Survival (CAFS) scores
Time frame: 36 weeks
OLE: Change from baseline in Clinician Global Impression-Inhibitory (CGI-I)
Time frame: 36 weeks
OLE: Change from baseline in ALS Assessment Questionnaire 40 items (ALSAQ-40)
Time frame: 36 weeks
OLE: Change from baseline in Edinburgh Cognitive and Behavioural ALS Screen (ECAS)
Time frame: 36 weeks
OLE: Change from baseline in Patient Global Impression (PGI) of Improvement
Time frame: 36 weeks
OLE: Change from baseline in Rasch Overall ALS Disability (ROADS).
Time frame: 36 weeks
OLE: Incidence, nature, and severity of treatment emergent adverse events (TEAEs) and serious adverse events (SAEs)
Time frame: 36 weeks
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 10 Sep 2026.
- 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.
Questions about this trial
What is this clinical trial studying?
A Phase 2/3 study testing Tazbentetol in people with Amyotrophic Lateral Sclerosis, run by Spinogenix.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 15 exclusion criteria. 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: opening soon, as of the sponsor's last update on 10 Sep 2026. Status can change between updates, so confirm with the study contact before making plans.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. 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 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Study Contact (503 915 1400, contact@spinogenix.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Phase 2: Absolute change from baseline in Amyotrophic Lateral Sclerosis Functional Rating Scale-revised (ALSFRS-R) total score (time frame: 36 weeks). The study lists 3 primary outcomes in all. 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 10 Sep 2026.