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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.
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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| tazbentetol | Experimental | synthetic small molecule |
|
| Placebo | Placebo Comparator | placebo |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| 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. |
| Measure | Description | Time Frame |
|---|---|---|
| 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 |
| Measure | Description | Time Frame |
|---|---|---|
| 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. | 36 weeks |
| Phase 2: Change from baseline in Clinician Global Impression-Inhibitory (CGI-I) |
| Measure | Description | Time Frame |
|---|---|---|
| Phase 2: ALSFRS-R total score rate of decline | To further assess the effect of tazbentetol on the progression of ALS | 36 weeks |
| Phase 2: Proportion of patients alive and survival time | To further assess the effect of tazbentetol on the progression of ALS |
Inclusion Criteria:
Exclusion Criteria:
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| Name | Role | Phone | Extension | |
|---|---|---|---|---|
| Study Contact | Contact | 503 915 1400 | contact@spinogenix.com |
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| ID | Term |
|---|---|
| D000690 | Amyotrophic Lateral Sclerosis |
| ID | Term |
|---|---|
| D013118 | Spinal Cord Diseases |
| D002493 | Central Nervous System Diseases |
| D009422 | Nervous System Diseases |
| D016472 | Motor Neuron Disease |
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|
| placebo | Drug | participants in double blind placebo controlled phase will be randomized to received placebo tablets |
|
Change in clinician rated measures on scale of 1 to 7, higher value indicates more severe symptom presentation |
| 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. | 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. | 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. | 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. | 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 | 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. | 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 | 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. | 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. | 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. | 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. | 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 | 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. | 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 | 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. | 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. | 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. | 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. | 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 | 36 weeks |
| 36 weeks |
| Phase 2 substudy: To compare electroencephalogram (EEG) outcomes of two dose levels of tazbentetol to placebo in participants with ALS | To measure the change from baseline in EEG delta and power bands at resting state at Weeks 8, 24, and 36 | 36 weeks |
| Phase 3: ALSFRS-R total score rate of decline | To further assess the effect of tazbentetol on the progression of ALS | 36 weeks |
| Phase 3: Proportion of patients alive and survival time | To further assess the effect of tazbentetol on the progression of ALS | 36 weeks |
| OLE: ALSFRS-R total score rate of decline | To further assess the effect of tazbentetol on the progression of ALS | 36 weeks |
| OLE: Proportion of patients alive and survival time | To further assess the effect of tazbentetol on the progression of ALS | 36 weeks |
| D019636 | Neurodegenerative Diseases |
| D057177 | TDP-43 Proteinopathies |
| D009468 | Neuromuscular Diseases |
| D057165 | Proteostasis Deficiencies |
| D008659 | Metabolic Diseases |
| D009750 | Nutritional and Metabolic Diseases |