Safety of Intrathecal Riluzole in Patients With Amyotrophic Lateral Sclerosis

NCT07093268 | Not Yet Recruiting Phase 1 DMC

• 1 other identifier: ITR2023
• Study Type: interventional
• Target: 10
• Locations: 1 country
• Sites: 2

Brief Summary

The purpose of this study is to investigate the safety and tolerability of intrathecal riluzole in adults with amyotrophic lateral sclerosis.

Conditions

Amyotrophic Lateral Sclerosis; Motor Neuron Disease

Keywords

Neurodegenerative Diseases; Nervous System Diseases; Neuromuscular Diseases

Outcome Measures

Primary Outcomes (6)

• Safety and tolerability of 0.2mg/hr intrathecal infusion of intrathecal riluzole, Number of participants with abnormal vital signs

  Serial assessment of adverse events, vital signs (blood pressure, pulse, respiratory rate and temp)

  6 weeks

• Safety and tolerability of 0.2mg/hr intrathecal infusion of intrathecal riluzole, Number of participants with with abnormal physical examination findings

  Serial assessment of abnormal findings in a neurological examination for ALS patients

  6 weeks

• Safety and tolerability of 0.2mg/hr intrathecal infusion of intrathecal riluzole, Number of participants with abnormal neurological exam findings

  Serial assessment all adverse events, neurological examination

  6 weeks

• Safety and tolerability of 0.2mg/hr intrathecal, infusion of intrathecal riluzole, Number of participants with with abnormal laboratory tests results

  Serial assessment all abnormal findings in clinical laboratory test results

  6 weeks

• Safety and tolerability of 0.2mg/hr intrathecal infusion of intrathecal riluzole, Number of participants with abnormal findings in the Neuropathic Pain Scale (NPS)

  Serial assessment all abnormal findings in the Neuropathic Pain Scale (NPS)

  6 weeks

• Safety and tolerability of 0.2mg/hr intrathecal infusion of intrathecal riluzole, Number of participants with adverse events, Columbia-Suicide Severity Rating Scale (C-SSRS).

  Serial assessment all adverse events in the Columbia-Suicide Severity Rating Scale (C-SSRS).

  6 weeks

Secondary Outcomes (6)

• Safety and tolerability of 0.2mg/hr intrathecal infusion of intrathecal riluzole, Number of participants with abnormal vital signs

  6 months

• Safety and tolerability of 0.2mg/hr intrathecal infusion of intrathecal riluzole, Number of participants with abnormal physical examination findings

  6 months

• Safety and tolerability of 0.2mg/hr intrathecal infusion of intrathecal riluzole, Number of participants with abnormal neurological exam findings

  6 months

• Safety and tolerability of 0.2mg/hr intrathecal, infusion of intrathecal riluzole, Number of participants with with abnormal laboratory tests results

  6 months

• Safety and tolerability of 0.2mg/hr intrathecal infusion of intrathecal riluzole, Number of participants with abnormal findings in the Neuropathic Pain Scale (NPS)

  6 months

Other Outcomes (7)

• Exploratory - Measure of changes in blood and cerebrospinal fluid biomarkers and urinary p75

  9 months

• Exploratory - Measure of clinical outcome of Handheld dynamometry (HHD) during dosing and post treatment periods.

  9 months

• Exploratory - Tracheostomy free survival

  9 months

Study Arms (1)

Single Arm

EXPERIMENTAL

Intrathecal riluzole continuous infusion up to 0.2 mg / hr.

Drug: Intrathecal Riluzole

Interventions

Intrathecal Riluzole DRUG

Intrathecal infusion of riluzole for 6 weeks followed by 6 months treatment with safety committee approval.

Single Arm

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Men and women aged 18 years or older.
• Participants are ambulatory with or without an assistive device.
• Sporadic or familial ALS diagnosis with possible, laboratory-supported probable, probable, or definite as defined by revised El Escorial criteria.
• Slow vital capacity (SVC) measure ≥70% of predicted for gender, height, and age.
• Medically able to undergo implantation of the SynchroMed II Infusion Pump according to the judgment of the investigator, or the presence of a previously implanted IT pump (not to be used for concurrent IT infusion of another IT agent).
• Capable of reading and providing informed consent and following study procedures.
• Geographic accessibility to the study center and the ability to travel to the clinic for study visits by ground transportation.
• Women must not be able to become pregnant (eg, post menopausal, surgically sterile, or using adequate birth control methods) for the duration of the study and 3 months after study completion. Adequate contraception includes: abstinence, hormonal contraception (oral contraception, implanted contraception, injected contraception or other hormonal (patch or contraceptive ring, for example) contraception), intrauterine device in place for ≥3 months, barrier method in conjunction with spermicide, or another adequate method.
• Taking and tolerating oral riluzole 50 mg twice a day for at least 30 days prior screening and willingness to continue oral riluzole throughout duration of the study.
• Patients may take other drugs approved for treatment of ALS at the dose prescribed by their neurologist.

You may not qualify if:

• Participants with bulbar-onset ALS
• Participants at risk of increased bleeding or uncontrolled bleeding during the SynchroMed II Infusion Pump implantation or following explant. This includes but is not limited to:
• Anatomical factors at or near the site of implantation;
• Underlying disorders of the coagulation cascade or platelet function (eg, hemophilia, Von Willebrand's disease, liver disease);
• Administration of antiplatelet or anticoagulant medication within 7 days before or after pump implantation (eg, aspirin, clopidogrel bisulfate, rivaroxaban, nonsteroidal anti-inflammatory agents \[NSAIDs\]), or
• Use of nutritional supplements (eg, St John's Wort) within 7 days before or after pump implantation.
• Presence of infection including but not limited to: meningitis, ventriculitis, skin infection, bacteremia, or septicemia.
• Testing positive for HIV (anti-HIV antibody), HBV (HBV surface antigen) or HCV (anti-HCV antibody; HCV RNA if anti-HCV antibody is positive) at screening.
• Inability to have the infusion pump implanted ≤ 2.5 cm below the skin surface.
• Body weight and size unable to accept the infusion pump bulk and weight.
• Spinal anomalies which would complicate the implantation and fixation of the catheter for IP delivery.
• Serum aspartate aminotransferase (AST) and alanine aminotransferase (ALT) value \> 2.0 times the upper normal.
• A life expectancy of less than 6 months, based on the judgment of the investigator.
• Presence of tracheostomy.
• The presence of unstable psychiatric disease, cognitive impairment, or dementia that would impair the ability of the participant to provide informed consent, per investigator judgment.

Sponsors & Collaborators

• Brain Trust Bio: lead

Study Sites (2)

Sunshine Coast University Hospital

Birtinya, Queensland, 4575, Australia

Location

Flinders Medical Centre

Bedford Park, South Australia, 5042, Australia

Location

MeSH Terms

Conditions

Amyotrophic Lateral Sclerosis; Motor Neuron Disease; Neurodegenerative Diseases; Nervous System Diseases; Neuromuscular Diseases

Condition Hierarchy (Ancestors)

Spinal Cord Diseases; Central Nervous System Diseases; TDP-43 Proteinopathies; Proteostasis Deficiencies; Metabolic Diseases; Nutritional and Metabolic Diseases

Study Officials

• David Schultz, Dr

  Flinders Medical Centre

  PRINCIPAL INVESTIGATOR

• Antony Winkel, Dr

  Sunshine Coast University Hospital

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Chen Benkler, PhD

CONTACT

+1 857 285 8300; chen.benkler@gmail.com

Tina Soulis, PhD

CONTACT

+61 429 300 705; tina.soulis@alithialifesciences.com

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Masking Details: Open Label
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: Open label, competitive recruitment.

Study Record Dates

• First Submitted: May 8, 2024
• First Posted: July 30, 2025
• Study Start: August 15, 2025
• Primary Completion (Estimated): June 15, 2026
• Study Completion (Estimated): January 15, 2027
• Last Updated: July 30, 2025

Record last verified: 2025-07

Data Sharing

• IPD Sharing: Will not share

Locations

AU (2)