Efficacy and Safety of Tazbentetol in ALS Participants

NCT ID: NCT07325591

Last Updated: 2026-01-08

Basic Information

• Recruitment Status: NOT_YET_RECRUITING
• Clinical Phase: PHASE2
• Total Enrollment: 336 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2026-03-01
• Study Completion Date: 2028-03-01

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.

Detailed Description

This is a Phase 2b 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

Double-blind, placebo controlled (DBPC): Randomized, double-blind, placebo-controlled with 3 parallel treatments arms. Participants will be randomized in a 1:1:1 ratio to receive either tazbentetol 300 mg once daily (QD), or 300 mg twice daily (BID), or placebo for 36 weeks.

Open-label extension: Eligible participants who complete 36 weeks in the DBPC 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.

Conditions

Amyotrophic Lateral Sclerosis

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: QUADRUPLE

Study Groups

tazbentetol 300 mg once daily

synthetic small molecule

Group Type: EXPERIMENTAL

Tazbentetol 300 mg once daily

Intervention Type: DRUG

participants in double blind placebo controlled phase will be randomized to received study drug tazbentetol at 300 mg once daily and placebo tablets once daily. Participants in the open-label extension phase will receive the dose determined from DBPC.

placebo

Intervention Type: DRUG

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

Placebo

placebo

Group Type: PLACEBO_COMPARATOR

placebo

Intervention Type: DRUG

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

tazbentetol 300 mg twice daily

synthetic small molecule

Group Type: EXPERIMENTAL

Tazbentetol 300 mg twice daily

Intervention Type: DRUG

participants in double blind placebo controlled phase will be randomized to received study drug tazbenetol at 300 mg twice daily. Participants in the open-label extension phase will receive the dose determined from DBPC.

Interventions

Tazbentetol 300 mg once daily

participants in double blind placebo controlled phase will be randomized to received study drug tazbentetol at 300 mg once daily and placebo tablets once daily. Participants in the open-label extension phase will receive the dose determined from DBPC.

Intervention Type: DRUG

placebo

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

Intervention Type: DRUG

Tazbentetol 300 mg twice daily

participants in double blind placebo controlled phase will be randomized to received study drug tazbenetol at 300 mg twice daily. Participants in the open-label extension phase will receive the dose determined from DBPC.

Intervention Type: DRUG

Other Intervention Names

SPG302; SPG302

Eligibility Criteria

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

• Minimum Eligible Age: 18 Years
• Maximum Eligible Age: 80 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Spinogenix

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Central Contacts

Study Contact

Role: CONTACT

contact@spinogenix.com

503 915 1400

Other Identifiers

SPG302-ALS-003 (SPARK)

Identifier Type: -

Identifier Source: org_study_id