Amyotrophic Lateral SclerosisPhase 1NCT07093268
Safety of Intrathecal Riluzole in Patients With Amyotrophic Lateral Sclerosis
A Phase 1 study testing Intrathecal Riluzole in people with Amyotrophic Lateral Sclerosis and Motor Neuron Disease, run by Brain Trust Bio at 2 sites in Australia.
Trial summary
The purpose of this study is to investigate the safety and tolerability of intrathecal riluzole in adults with amyotrophic lateral sclerosis. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Amyotrophic Lateral Sclerosis, Motor Neuron Disease
- What is being tested?
- Intrathecal Riluzole
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 10 participants
- Where
- 2 sitesAustralia
- Study dates
- 15 Aug 2025to 15 Jan 2027
- Phase
- Phase 1
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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.
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- 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.
Who cannot join
- 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.
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- 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.
Trial locations and contacts
2 sites in 1 country. Contact the study team or a site near you to ask about taking part.
Australia2 sites
Flinders Medical Centre
Bedford Park, South Australia, 5042
David Shcults, Dr, +618 8204 4187, david.schultz@sa.gov.au
Karalyn Ernst, +618 8204 5172, Karalyn.Ernst@sa.gov.au
Sunshine Coast University Hospital
Birtinya, Queensland, 4575
Antony Winkel, Dr, antony.winkel@health.qld.gov.au
Helen Rodgers, helen.rodgers2@health.qld.gov.au
About this study
Official title: A Phase 1 Study to Determine the Safety and Tolerability of Continuous Intrathecal Riluzole in Patients With Progressive Ambulatory Amyotrophic Lateral Sclerosis
The purpose of this study is to investigate the safety and tolerability of intrathecal riluzole in adults with amyotrophic lateral sclerosis.
Read the 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.
- Being tested
- Intrathecal Riluzole
- Keywords
- Neurodegenerative Diseases, Nervous System Diseases, Neuromuscular Diseases
- Medical subject headings
- Amyotrophic Lateral Sclerosis, Motor Neuron Disease, Neurodegenerative Diseases, Nervous System Diseases, Neuromuscular Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Intrathecal RiluzoleDrug
Intrathecal infusion of riluzole for 6 weeks followed by 6 months treatment with safety committee approval.
| Study group | Role | What they receive |
|---|---|---|
| Single Arm | Experimental | Intrathecal riluzole continuous infusion up to 0.2 mg / hr. Receives: Drug: Intrathecal Riluzole |
What is being measured
The primary outcome is the main question the study is designed to answer.
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)
Measured: 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
Measured: 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
Measured: 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
Measured: 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)
Measured: 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).
Measured: 6 weeks
Secondary 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)
Measured: 6 months
Safety and tolerability of 0.2mg/hr intrathecal infusion of intrathecal riluzole, Number of participants with abnormal physical examination findings
Serial assessment of abnormal findings in a neurological examination for ALS patients
Measured: 6 months
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
Measured: 6 months
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 clinical laboratory test results
Measured: 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)
Serial assessment all abnormal findings in the Neuropathic Pain Scale (NPS)
Measured: 6 months
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).
Measured: 6 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted30 Jul 2025
- Study start15 Aug 2025 (estimated)
- Primary completion15 Jun 2026 (estimated)
- Study completion15 Jan 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Amyotrophic Lateral SclerosisStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Model description
- Open label, competitive recruitment.
- Masking
- None (open label)
- Enrolment
- 10 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Brain Trust Bio
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- David Schultz, Dr, Principal investigator, Flinders Medical CentreAntony Winkel, Dr, Principal investigator, Sunshine Coast University Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07093268
- Sponsor's protocol ID
- ITR2023
- Status last verified
- Jul 2025
- First submitted
- 8 May 2024
- Last updated
- 30 Jul 2025
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 30 Jul 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 1 Study to Determine the Safety and Tolerability of Continuous Intrathecal Riluzole in Patients With Progressive Ambulatory Amyotrophic Lateral Sclerosis
- ClinicalTrials.gov ID
- NCT07093268
- Acronym / sponsor ID
- ITR2023
- Keywords
- Neurodegenerative Diseases, Nervous System Diseases, Neuromuscular Diseases
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Not yet recruiting
- Enrolment
- 10 participants (estimated)
- Lead sponsor
- Brain Trust BioIndustry (pharma, biotech, devices)
- Study start
- 15 Aug 2025 (estimated)
- Primary completion
- 15 Jun 2026 (estimated)
- Study completion
- 15 Jan 2027 (estimated)
- First posted
- 30 Jul 2025 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
- Model description
- Open label, competitive recruitment.
| Arm | Type | Receives |
|---|---|---|
| Single Arm | Experimental | Drug: Intrathecal RiluzoleIntrathecal riluzole continuous infusion up to 0.2 mg / hr. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (10 listed)
- 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).
Key exclusion criteria (20 listed)
- 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:
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Intrathecal RiluzoleIntrathecal infusion of riluzole for 6 weeks followed by 6 months treatment with safety committee approval. | Drug | Single Arm |
Primary outcome
Safety and tolerability of 0.2mg/hr intrathecal infusion of intrathecal riluzole, Number of participants with abnormal vital signs
Time frame: 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
Time frame: 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
Time frame: 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
Time frame: 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)
Time frame: 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).
Time frame: 6 weeks
Serial assessment all adverse events in the Columbia-Suicide Severity Rating Scale (C-SSRS).
6 secondary outcomes
Safety and tolerability of 0.2mg/hr intrathecal infusion of intrathecal riluzole, Number of participants with abnormal vital signs
Time frame: 6 months
Safety and tolerability of 0.2mg/hr intrathecal infusion of intrathecal riluzole, Number of participants with abnormal physical examination findings
Time frame: 6 months
Safety and tolerability of 0.2mg/hr intrathecal infusion of intrathecal riluzole, Number of participants with abnormal neurological exam findings
Time frame: 6 months
Safety and tolerability of 0.2mg/hr intrathecal, infusion of intrathecal riluzole, Number of participants with with abnormal laboratory tests results
Time frame: 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)
Time frame: 6 months
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).
Time frame: 6 months
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 30 Jul 2025.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
David Schultz, Dr principal investigator
Flinders Medical Centre
Antony Winkel, Dr principal investigator
Sunshine Coast University Hospital
2 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Intrathecal Riluzole in people with Amyotrophic Lateral Sclerosis and Motor Neuron Disease, run by Brain Trust Bio at 2 sites in Australia.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 10 inclusion and 20 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: opening soon, as of the sponsor's last update on 30 Jul 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites in Australia. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Chen Benkler, PhD (+1 857 285 8300, chen.benkler@gmail.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Safety and tolerability of 0.2mg/hr intrathecal infusion of intrathecal riluzole, Number of participants with abnormal vital signs (time frame: 6 weeks). The study lists 6 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 30 Jul 2025.