A Randomized, Double-blind, Parallel Group, Single Centre, Phase 1b/2 Study to Assess the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Three Orally Administered Doses of Enoxacin (200mg Twice Daily, 400mg Twice Daily and 600mg Twice Daily) in Adults With Amyotrophic Lateral Sclerosis

Study Purpose:

The study will assess the safety of the drug enoxacin at specific dose levels in adults with ALS.

Study Status:

Not recruiting


Amyotrophic Lateral Sclerosis

Study Type:


Type of Intervention:


Intervention Name:

Enoxacin, Placebo




Phase 1/Phase 2

Study Chair(s)/Principal Investigator(s):

Angela Genge, MD, FRCP, McGill University

Clinicaltrials.gov ID:


Neals Affiliated?


Coordinating Center Contact Information

Full Study Summary:

Participants will be randomized to one of three doses of enoxacin (200, 400, or 600mg twice daily) for 30 days. On day 1, 7, 14, 21, and 30 of treatment and at a follow-up visit 14 days after the last dose, participants will be assessed for safety measures and blood will be collected to assist with the determination of enoxacin pharmacokinetics (PK) and pharmacodynamics (PD). On day 1 and day 30 of dosing, participants will only take one dose of study medication (the morning dose) to assist with determination of enoxacin single dose PK over a 24-hour period. A lumbar puncture (LP) to collect cerebrospinal fluid (CSF) for PD assessments will occur on day 1 and day 30.

Study Sponsor:

McGill University

Estimated Enrollment:


Estimated Study Start Date:

03 / 26 / 2021

Estimated Study Completion Date:

05 / 01 / 2023

Posting Last Modified Date:

03 / 23 / 2023

Date Study Added to neals.org:

04 / 12 / 2021

Minimum Age:

18 Years

Maximum Age:

85 Years

Inclusion Criteria:

- Diagnosis of familial or sporadic ALS

- FVC of ≥ 50 percent predicted

- If female, is not breastfeeding and is not pregnant

- Has been on a stable dose of riluzole, or has not taken riluzole, for at least 30 days prior to screening

- If taking concomitant edaravone at study entry, must have completed at least one cycle of edaravone therapy prior to screening

- Not currently taking and has not taken for at least 30 days prior to screening any Theophylline containing medications, clozapine, or duloxetine

- No active infection in the 30 days prior to randomization

- Has not taken any fluoroquinolone antibiotics for at least 30 days prior to screening

Exclusion Criteria:

- Hypersensitivity/allergy to fluoroquinolones

- Diagnosed with another neurodegenerative disease

- Significant pulmonary disorder not attributed to ALS, central nervous system disorder associated with seizures, myasthenia gravis, active rheumatologic disease, tendinopathy, or any severe uncontrolled medical condition (other than ALS)

- Severe renal impairment or impaired liver function

- Baseline prolongation of QT interval/corrected QT interval (QTc) at screening, treatment with any agent that may prolong Qt/QTc interval, or history of any other at-risk other cardiac condition

- Currently enrolled in another clinical trial involving an experimental drug or device

Montreal Neurological Institute-Hospital

Montreal, Quebec H3A 2B4