NCT07093268

Safety of Intrathecal Riluzole in Patients With Amyotrophic Lateral Sclerosis

A Phase 1 Study to Determine the Safety and Tolerability of Continuous Intrathecal Riluzole in Patients With Progressive Ambulatory Amyotrophic Lateral Sclerosis

• Status: Not Yet Recruiting
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 10 (estimated)
• Sponsor: Brain Trust Bio · Industry
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

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

Detailed description

ALS/MND is a progressive motor neurone disease. Oral riluzole is approved for the treatment of ALS/MND in Australia as well as other countries. Unfortunately, in pill form, the drug's efficacy is limited by erratic absorption from the gut, dosing is limited by liver toxicity, and patients often experience brain fog as a troubling side effect. Evidence from dogs suggests that receiving the same drug through an implanted pump that infuses it directly into the cerebrospinal fluid around the cells that support breathing, allows these cells to get 3 to 10 times as much riluzole , and guarantees that it is always there at the desired amount. In addition, this delivery approach should not contribute to increased brain fog. Patients who have demonstrated the ability to tolerate oral riluzole will have a SynchroMed® II Infusion Pump and Ascenda ® Intrathecal Catheter implanted. Once deemed medically appropriate, 6-weeks of continuous IT riluzole will be administered. Safety and tolerability will be assessed by a safety committee for the first 3 participants enrolled. With Safety Committee approval, these participants will continue on 6-months of treatment and a further 7 participants will be enrolled for a total of 32 weeks treatment.

Conditions

• Amyotrophic Lateral Sclerosis
• Motor Neuron Disease

Interventions

Type	Name	Description
DRUG	Intrathecal Riluzole	Intrathecal infusion of riluzole for 6 weeks followed by 6 months treatment with safety committee approval.

Timeline

• Start date: 2025-08-15
• Primary completion: 2026-06-15
• Completion: 2027-01-15
• First posted: 2025-07-30
• Last updated: 2025-07-30

Locations

2 sites across 1 country: Australia