Efficacy and Safety of Tazbentetol in ALS Participants

Spinogenix

Status and phase: Not yet enrolling; Phase 3; Phase 2

Conditions: Amyotrophic Lateral Sclerosis

Treatments: Drug: Tazbentetol; Drug: placebo

Study type: Interventional

Funder types: Industry

Identifiers

NCT07325591

SPG302-ALS-003 (SPARK)

Details and patient eligibility

About

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.

Full 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.

Enrollment: 430 estimated patients

Sex: All

Ages: 18 to 80 years old

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Quadruple Blind

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430 participants in 2 patient groups, including a placebo group

tazbentetol

Experimental group

Description:

synthetic small molecule

Treatment:

Drug: Tazbentetol

Placebo

Placebo Comparator group

Description:

placebo

Treatment:

Drug: placebo