Skip to main content
OpenTrials
Not Yet Recruiting

NCT Number: NCT07325591

Efficacy and Safety of Tazbentetol in ALS Participants

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.

Not Yet Recruiting

Trial opening soon.

Get Notified

Key information

Conditions

Amyotrophic Lateral Sclerosis Central Nervous System Diseases Metabolic Diseases Motor Neuron Disease Nervous System Diseases Neurodegenerative Diseases Neuromuscular Diseases Nutritional and Metabolic Diseases Proteostasis Deficiencies Spinal Cord Diseases TDP-43 Proteinopathies

Age range

18 year–80 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2 / Phase 3

About this study

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.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • 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

Exclusion criteria

  • 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

Treatment and study plan

Tazbentetol

Drug

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.

Other names: SPG302

Placebo

Drug

participants in double blind placebo controlled phase will be randomized to received placebo tablets

Primary outcomes

  1. 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.

  2. 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.

  3. 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.

Secondary outcomes

  1. Phase 2: Combined Assessment of Function and Survival (CAFS) scores

    Time frame: 36 weeks

    This assessment ranks clinical outcome based on changes to ALSFRS-R score. A higher score indicates a better clinical outcome.

  2. Phase 2: Change from baseline in Clinician Global Impression-Inhibitory (CGI-I)

    Time frame: 36 weeks

    Change in clinician rated measures on scale of 1 to 7, higher value indicates more severe symptom presentation

  3. Phase 2: Change from baseline in Edinburgh Cognitive and Behavioural ALS Screen (ECAS)

    Time frame: 36 weeks

    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.

  4. Phase 2: Change from baseline in ALS Assessment Questionnaire 40 items (ALSAQ-40)

    Time frame: 36 weeks

    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.

  5. Phase 2: Change from baseline in Patient Global Impression (PGI) of Improvement

    Time frame: 36 weeks

    This will measure all aspects of patient health and improvement or decline. A higher scale indicates greater disability.

  6. Phase 2: Change from baseline in Rasch Overall ALS Disability (ROADS).

    Time frame: 36 weeks

    This will measure patient reported outcomes of overall disability. A lower score indicates greater disability.

  7. Phase 2: Incidence, nature, and severity of treatment emergent adverse events (TEAEs) and serious adverse events (SAEs)

    Time frame: 36 weeks

    This will assess the safety and tolerability of tazbentetol in of participants with ALS

  8. Phase 3: Combined Assessment of Function and Survival (CAFS) scores

    Time frame: 36 weeks

    This assessment ranks clinical outcome based on changes to ALSFRS-R score. A higher score indicates a better clinical outcome.

  9. Phase 3: Change from baseline in Clinician Global Impression-Inhibitory (CGI-I)

    Time frame: 36 weeks

    Change in clinician rated measures on scale of 1 to 7, higher value indicates more severe symptom presentation

  10. Phase 3: Change from baseline in Edinburgh Cognitive and Behavioural ALS Screen (ECAS)

    Time frame: 36 weeks

    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.

  11. Phase 3: Change from baseline in ALS Assessment Questionnaire 40 items (ALSAQ-40)

    Time frame: 36 weeks

    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.

  12. Phase 3: Change from baseline in Patient Global Impression (PGI) of Improvement

    Time frame: 36 weeks

    This will measure all aspects of patient health and improvement or decline. A higher scale indicates greater disability.

  13. Phase 3: Change from baseline in Rasch Overall ALS Disability (ROADS).

    Time frame: 36 weeks

    This will measure patient reported outcomes of overall disability. A lower score indicates greater disability.

  14. Phase 3: Incidence, nature, and severity of treatment emergent adverse events (TEAEs) and serious adverse events (SAEs)

    Time frame: 36 weeks

    This will assess the safety and tolerability of tazbentetol in of participants with ALS

  15. OLE: Combined Assessment of Function and Survival (CAFS) scores

    Time frame: 36 weeks

    This assessment ranks clinical outcome based on changes to ALSFRS-R score. A higher score indicates a better clinical outcome.

  16. OLE: Change from baseline in Clinician Global Impression-Inhibitory (CGI-I)

    Time frame: 36 weeks

    Change in clinician rated measures on scale of 1 to 7, higher value indicates more severe symptom presentation

  17. OLE: Change from baseline in ALS Assessment Questionnaire 40 items (ALSAQ-40)

    Time frame: 36 weeks

    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.

  18. OLE: Change from baseline in Edinburgh Cognitive and Behavioural ALS Screen (ECAS)

    Time frame: 36 weeks

    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.

  19. OLE: Change from baseline in Patient Global Impression (PGI) of Improvement

    Time frame: 36 weeks

    This will measure all aspects of patient health and improvement or decline. A higher scale indicates greater disability.

  20. OLE: Change from baseline in Rasch Overall ALS Disability (ROADS).

    Time frame: 36 weeks

    This will measure patient reported outcomes of overall disability. A lower score indicates greater disability.

  21. OLE: Incidence, nature, and severity of treatment emergent adverse events (TEAEs) and serious adverse events (SAEs)

    Time frame: 36 weeks

    This will assess the safety and tolerability of tazbentetol in of participants with ALS

Other outcomes

  1. Phase 2: ALSFRS-R total score rate of decline

    Time frame: 36 weeks

    To further assess the effect of tazbentetol on the progression of ALS

  2. Phase 2: Proportion of patients alive and survival time

    Time frame: 36 weeks

    To further assess the effect of tazbentetol on the progression of ALS

  3. Phase 2 substudy: To compare electroencephalogram (EEG) outcomes of two dose levels of tazbentetol to placebo in participants with ALS

    Time frame: 36 weeks

    To measure the change from baseline in EEG delta and power bands at resting state at Weeks 8, 24, and 36

  4. Phase 3: ALSFRS-R total score rate of decline

    Time frame: 36 weeks

    To further assess the effect of tazbentetol on the progression of ALS

  5. Phase 3: Proportion of patients alive and survival time

    Time frame: 36 weeks

    To further assess the effect of tazbentetol on the progression of ALS

  6. OLE: ALSFRS-R total score rate of decline

    Time frame: 36 weeks

    To further assess the effect of tazbentetol on the progression of ALS

  7. OLE: Proportion of patients alive and survival time

    Time frame: 36 weeks

    To further assess the effect of tazbentetol on the progression of ALS

Study contacts

Contact information is provided by the study sponsor or research team.

Study Contact

CONTACT

[email protected]

503 915 1400

Sponsors and collaborators

Lead sponsor

Spinogenix

Industry

Registry information

Official study 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)

Important dates

Study start
2026
Primary completion
2027
Study completion
2028
First posted
Jan 8, 2026
Registry last updated
Mar 2, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

Related clinical trials

Published trials that share one or more normalized conditions with this study.