Enoxacin for Amyotrophic Lateral Sclerosis (ALS) (REALS-1)

McGill University

Status and phase: Completed; Phase 2; Phase 1

Conditions: Amyotrophic Lateral Sclerosis

Treatments: Drug: Placebo; Drug: Enoxacin

Study type: Interventional

Funder types: Other; Industry

Identifiers

NCT04840823

REALS-1

Details and patient eligibility

About

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

Full description

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.

Enrollment: 8 patients

Sex: All

Ages: 18 to 85 years old

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Quadruple Blind

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8 participants in 3 patient groups

Enoxacin 200mg twice daily

Experimental group

Description:

Enoxacin 200mg twice daily (one dose in the morning and one dose in the evening) for 30 days, except day 1 and day 30 when only the morning dose will be taken. Participants will take 1 active 200mg enoxacin tablet and 2 placebo tablets per dose.

Treatment:

Drug: Placebo

Drug: Enoxacin

Enoxacin 400mg twice daily

Experimental group

Description:

Enoxacin 400mg twice daily (one dose in the morning and one dose in the evening) for 30 days, except day 1 and day 30 when only the morning dose will be taken. Participants will take 2 active 200mg enoxacin tablets and 1 placebo tablet per dose.

Treatment:

Drug: Placebo

Drug: Enoxacin

Enoxacin 600mg twice daily

Experimental group

Description:

Enoxacin 600mg twice daily (one dose in the morning and one dose in the evening) for 30 days, except day 1 and day 30 when only the morning dose will be taken. Participants will take 3 active 200mg enoxacin tablets per dose.

Treatment:

Drug: Enoxacin