Extension Study of Participants From SPG302-ALS-001

Phase 2

Not yet recruiting

• Conditions: Amyotrophic Lateral Sclerosis (ALS)
• Interventions: Drug: SPG302

• Registration Number: NCT06903286
• Lead Sponsor: Spinogenix

Brief Summary

This study will evaluate the long-term safety and efficacy of participants enrolled in SPG302-ALS-101 with Amyotrophic Lateral Sclerosis (ALS)

Detailed Description

This is an open-label extension study of SPG302-ALS-001 to investigate the long-term safety, tolerability, and efficacy of SPG302 administered orally in participants with Amyotrophic Lateral Sclerosis (ALS). This study will allow participants in the parent study to continue dosing. Enrolled participants will continue at the dose they received at the end of the first trial, and will self-administer SPG302 orally every day for up to 52 weeks. Participants will have an in-person clinic visit every 3 months (± 3 days) and a monthly phone visit. An end of treatment visit will occur within 7 days of the last dose of SPG302. A final visit to collect safety data will be conducted up to 1 month (± 3 days) post last dose.

Study Timeline

• Study First Submitted: 2025-03-24
• Study First Submitted QC: 2025-03-24
• Study First Posted: 2025-03-30
• Status Verified: 2025-05
• Last Update Submitted: 2025-05-13
• Last Update Posted: 2025-05-14
• Study Start: 2025-05-29
• Primary Completion: 2026-05-30
• Study Completion: 2026-08-31

Recruitment & Eligibility

• Status: NOT_YET_RECRUITING
• Sex: All
• Target Recruitment: 16

Inclusion Criteria

• Must have participated in all study activities of SPG302-ALS-001, the parent study

Exclusion Criteria

• Unable to reliably and regularly swallow whole oral medications on a daily basis.
• Medical conditions that investigator or sponsor determine would interfere with participation in clinical trial

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Experimental: Open Label Extension	SPG302	Active SPG302 to be administered to adult participants with ALS who completed initial study. Dose to be administered to be dose received during previous study.

Primary Outcome Measures

Name	Time	Method
Treatment emergent adverse events and serious adverse events	Up to 52 weeks	Incidence, nature, and severity of treatment emergent adverse events (TEAEs) and serious adverse events (SAEs)
C-SSRS (Columbia Suicide Severity Rating Scale)	Up to 52 weeks	Prospective suicidality assessment is performed using the Columbia-Suicide Severity Rating Scale (C-SSRS), a questionnaire to evaluate suicidal ideation and behavior. Answer "yes" on item 4 or 5 of the Suicidal Ideation section or "yes" on any item of the Suicidal Behavior section is considered positive. The suicidal behavior lethality sub-scale evaluates the level of actual or potential medical damage

Secondary Outcome Measures

Name	Time	Method
Changes from baseline in neurofilament light biomarker (NfL)	up to 52 weeks.	To assess the effect of SGP302 on NfL, a biomarker of neurodegeneration. A higher level of this biomarker indicates a progression of this disease.
Change in the Amyotrophic Lateral Sclerosis Functional Rating Scale-revised (ALSFRS-R) scores	up to 52 weeks	Questionnaire administered by a clinician that includes a series of questions about 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. This outcome would evaluate these scores based on patient demographics including clinical presentation at outset of study, presence of ventilation assistance, and patient demographics. Results will be compared to matched historical controls
Change in Edinburgh Cognitive and Behavioural ALS Screen (ECAS)	up to 52 weeks	The Edinburgh Cognitive and Behavioural ALS Screen (ECAS) 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.

Trial Locations

Locations (3)

Macquarie University; 🇦🇺 North Ryde, New South Wales, Australia

Royal Brisbane and Women's Hospital; 🇦🇺 Herston, Queensland, Australia

Flinders Medical Center; 🇦🇺 Adelaide, South Australia, Australia