Extension Study of Participants From SPG302-ALS-001

An Open-label Extension of SPG302-ALS-001 Study to Evaluate the Long-term Safety and Efficacy of Daily Oral SPG302 Treatment in Participants With Amyotrophic Lateral Sclerosis

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

Study Overview

• Status: Terminated
• Conditions: Amyotrophic Lateral Sclerosis (ALS)
• Intervention / Treatment: Drug: SPG302

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 Type: Interventional
• Enrollment (Actual): 16
• Phase: Phase 2

Contacts and Locations

Study Locations

• Australia
  • New South Wales
    • North Ryde, New South Wales, Australia, 2109
      • Macquarie University
  • Queensland
    • Herston, Queensland, Australia, 4029
      • Royal Brisbane and Women's Hospital
  • South Australia
    • Adelaide, South Australia, Australia, 5042
      • Flinders Medical Center

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: No

Description

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 Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Experimental: Open Label Extension; 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.	Drug: SPG302; Open label SPG302 to be self-administered daily by eligible participants for 52 weeks.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Treatment emergent adverse events and serious adverse events	Incidence, nature, and severity of treatment emergent adverse events (TEAEs) and serious adverse events (SAEs)	Up to 52 weeks
C-SSRS (Columbia Suicide Severity Rating Scale)	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	Up to 52 weeks

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Change in the Amyotrophic Lateral Sclerosis Functional Rating Scale-revised (ALSFRS-R) scores	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	up to 52 weeks
Change in Edinburgh Cognitive and Behavioural ALS Screen (ECAS)	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.	up to 52 weeks
Changes from baseline in neurofilament light biomarker (NfL)	To assess the effect of SGP302 on NfL, a biomarker of neurodegeneration. A higher level of this biomarker indicates a progression of this disease.	up to 52 weeks.

Collaborators and Investigators

• Sponsor: Spinogenix

Study record dates

Study Major Dates

• Study Start (Actual): May 29, 2025
• Primary Completion (Actual): June 30, 2025
• Study Completion (Actual): August 31, 2025

Study Registration Dates

• First Submitted: March 24, 2025
• First Submitted That Met QC Criteria: March 24, 2025
• First Posted (Actual): March 30, 2025

Study Record Updates

• Last Update Posted (Actual): December 17, 2025
• Last Update Submitted That Met QC Criteria: December 10, 2025
• Last Verified: December 1, 2025

More Information

Terms related to this study

• Keywords: Amyotrophic Lateral Sclerosis; regenerative; synapse
• Additional Relevant MeSH Terms: Central Nervous System Diseases; Nervous System Diseases; Neuromuscular Diseases; Metabolic Diseases; Neurodegenerative Diseases; Spinal Cord Diseases; TDP-43 Proteinopathies; Proteostasis Deficiencies; Motor Neuron Disease; Nutritional and Metabolic Diseases; Amyotrophic Lateral Sclerosis
• Other Study ID Numbers: SPG302-ALS-002 OLE

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No