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Starting soon Phase 1

Safety of Intrathecal Riluzole in Patients With Amyotrophic Lateral Sclerosis

NCT07093268 · tracked via the Priya Life Science Australia tracker
Sponsor
Brain Trust Bio
Phase
Phase 1
Started
2025-08-15
Last updated
2025-07-30

Condition(s) studied

Amyotrophic Lateral SclerosisMotor Neuron Disease

Investigational drug(s) / intervention(s)

Intrathecal Riluzole →

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

Study summary

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

Eligibility

Sex
ALL
Min age
18 Years
Max age
—
Healthy volunteers
No
Inclusion Criteria: * 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. Exclusion Criteria: * 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: 1. Anatomical factors at or near the site of implantation; 2. Underlying disorders of the coagulation cascade or platelet function (eg, hemophilia, Von Willebrand's disease, liver disease); 3. 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 4. 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. * History of active substance abuse within the prior year. * At risk for committing suicide per investigator judgment. * Clinically significant history of unstable or severe cardiac, oncologic, hepatic, or renal disease, or other medically significant illness. * Pregnant women or women currently breastfeeding. * Exposure to any investigational drug, device, or biologic within 30 days of screening.

Primary outcome measure(s)

  • Safety and tolerability of 0.2mg/hr intrathecal infusion of intrathecal riluzole, Number of participants with abnormal vital signs — 6 weeks
    Serial assessment of adverse events, vital signs (blood pressure, pulse, respiratory rate and temp)
  • Safety and tolerability of 0.2mg/hr intrathecal infusion of intrathecal riluzole, Number of participants with with abnormal physical examination findings — 6 weeks
    Serial assessment of abnormal findings in a neurological examination for ALS patients
  • Safety and tolerability of 0.2mg/hr intrathecal infusion of intrathecal riluzole, Number of participants with abnormal neurological exam findings — 6 weeks
    Serial assessment all adverse events, neurological examination
  • Safety and tolerability of 0.2mg/hr intrathecal, infusion of intrathecal riluzole, Number of participants with with abnormal laboratory tests results — 6 weeks
    Serial assessment all abnormal findings in clinical laboratory test results
  • 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 weeks
    Serial assessment all abnormal findings in the Neuropathic Pain Scale (NPS)
  • 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). — 6 weeks
    Serial assessment all adverse events in the Columbia-Suicide Severity Rating Scale (C-SSRS).

Trial sites (2)

FacilityCityRegionStatus
Sunshine Coast University Hospital Birtinya Queensland
Flinders Medical Centre Bedford Park South Australia
Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT07093268 on ClinicalTrials.gov ↗ ← All trials in Australia