Safety of Intrathecal Riluzole in Patients With Amyotrophic Lateral Sclerosis

ConditionsAmyotrophic Lateral Sclerosis Motor Neuron Disease

Trial statusNot yet recruiting

Trial phasePhase 1

Trial typeInterventional

Biological sexAll

Age18+

SponsorBrain Trust Bio

About this trial

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

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

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.

Disqualifiers

Participants with bulbar-onset ALS

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

Trial design

Design model

Single group

Treatments tested in this trial

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

Treatment groups

10 Participants

are divided into 1 treatment group

Group A: Single ArmExperimental treatment 1 intervention

Trial outcomes

Primary outcomes

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)

Time frame

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

Time frame

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

Time frame

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

Time frame

6 weeks

Secondary outcomes

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)

Time frame

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

Time frame

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

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

Serial assessment all abnormal clinical laboratory test results

Time frame

6 months

Other outcomes

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

Measure of BDNF, pNFH and NfL) and cerebrospinal fluid (pNFH, NfL, Chit-1, CHI3L1, MCP-1, IL-6, IL-8, IL-17, IL-18, MIP-1α, TNF-α, CRP, 8-oxodG, Ferritin, BDNF, NGF, p75ECD biomarkers

Time frame

9 months

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

Measure of Handheld dynamometry (HHD)

Time frame

9 months

Exploratory - Tracheostomy free survival

To compare tracheostomy free survival for all participants with data from historical controls

Time frame

9 months

Exploratory - Measure of clinical outcome measures during dosing and post treatment periods.

Measure of the ALS Functional Rating Scale - Revised (ALSFRS-R),

Time frame

9 months

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

Brain Trust Bio

Lead sponsor

Source ClinicalTrials.gov

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