Efficacy and Safety of Tazbentetol in ALS Participants

Phase 3Not Yet RecruitingNCT07325591

Spinogenix430 enrolled

Overview

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.

Study Type

INTERVENTIONAL

Allocation

RANDOMIZED

Purpose

TREATMENT

Masking

QUADRUPLE

Enrollment

430

Conditions

Amyotrophic Lateral Sclerosis

Interventions

TazbentetolDRUG

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

Eligibility

Sex: ALLMin age: 18 YearsMax age: 80 Years

Medical Language ↔ Plain English

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

Outcomes

Primary Outcomes

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.

Time frame: 36 weeks

Phase 3: Absolute change from baseline in Amyotrophic Lateral Sclerosis Functional Rating Scale-revised (ALSFRS-R) total score

Time frame: 36 weeks

OLE: Absolute change from baseline in Amyotrophic Lateral Sclerosis Functional Rating Scale-revised (ALSFRS-R) total score

Time frame: 36 weeks

Secondary Outcomes

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.

Time frame: 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

Central Contacts

Study Contact

CONTACT

503 915 1400 contact@spinogenix.com

Data from ClinicalTrials.gov

This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.

Time frame: 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.

Time frame: 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.

Time frame: 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.

Time frame: 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.

Time frame: 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

Time frame: 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.

Time frame: 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

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

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

Time frame: 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.

Time frame: 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

Time frame: 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.

Time frame: 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

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

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

Time frame: 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.

Time frame: 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

Time frame: 36 weeks