Amyotrophic Lateral SclerosisPhase 1/2REALS-1NCT04840823
Enoxacin for Amyotrophic Lateral Sclerosis (ALS)
A Phase 1/2 study testing Enoxacin in people with Amyotrophic Lateral Sclerosis, run by McGill University at 1 site in Canada.
Trial summary
The study will assess the safety of the drug enoxacin at specific dose levels in adults with ALS. Read more about this study
- Who is it for?
- Ages 18 to 85 years
- Conditions the study is for
- Amyotrophic Lateral Sclerosis
- What is being tested?
- Enoxacin
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 8 participants
- Where
- 1 siteCanada
- Study dates
- 26 Mar 2021to 15 Nov 2023
- Phase
- Phase 1/2
- Are there results?
- None posted in the registry
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 to 85 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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
Who cannot join
- 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 locations and contacts
1 site in 1 country.
Canada1 site
Montreal Neurological Institute-Hospital
Montreal, Quebec, H3A 2B4
About this study
Official title: 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
The study will assess the safety of the drug enoxacin at specific dose levels in adults with ALS.
Read the detailed 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.
- Conditions
- Amyotrophic Lateral Sclerosis
- Being tested
- Enoxacin, Placebo
- Keywords
- Enoxacin
- Medical subject headings
- Amyotrophic Lateral Sclerosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
EnoxacinDrug
Oral 200mg tablet
PlaceboDrug
Oral tablet
| Study group | Role | What they receive |
|---|---|---|
| Enoxacin 200mg twice daily | Experimental | 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. Receives: Drug: Enoxacin; Drug: Placebo |
| Enoxacin 400mg twice daily | Experimental | 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. Receives: Drug: Enoxacin; Drug: Placebo |
| Enoxacin 600mg twice daily | Experimental | 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. Receives: Drug: Enoxacin |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence of adverse events (AEs) and serious adverse events (SAEs)
The incidence of adverse events (new or worsened from baseline (where baseline refers to those AEs recorded prior to dosing on day 1 of dosing)) will be summarized by primary system organ class and preferred term as frequency count and percentage of participants with AEs.
Measured: From baseline (prior to dosing on day 1 of dosing) to 14 day +/- 2 day follow up visit
Incidence of abnormalities in clinical laboratory assessments
Clinical laboratory data will be characterized by abnormalities in values and in changes from baseline values, where baseline refers to measurements taken prior to dosing on day 1 of dosing.
Measured: From baseline (prior to dosing on day 1 of dosing) to 14 day +/- 2 day follow up visit
Incidence of abnormalities in vital signs
Vital sign data will be characterized by abnormalities in values and in changes from baseline values, where baseline refers to measurements taken prior to dosing on day 1 of dosing.
Measured: From baseline (prior to dosing on day 1 of dosing) to 14 day +/- 2 day follow up visit
Incidence of abnormalities in physical and neurological examinations
Physical and neurological examinations will be characterized by abnormalities and in changes from baseline, where baseline refers to measurements taken prior to dosing on day 1 of dosing.
Measured: From baseline (prior to dosing on day 1 of dosing) to 14 day +/- 2 day follow up visit
Incidence of abnormalities in electrocardiograms (ECGs)
ECG data will be characterized by abnormalities in values and in changes from baseline values, where baseline refers to measurements taken prior to dosing on day 1 of dosing.
Measured: From baseline (prior to dosing on day 1 of dosing) to 14 day +/- 2 day follow up visit
Ability of participants to remain on their assigned dose for the full 30 day treatment period
The ability of participants to remain on each dose level will be measured by the mean number of missed doses.
Measured: From the beginning (day 1) to the end (day 30) of the 30 day treatment period
Secondary outcomes (10)
Maximum plasma concentration (Cmax) of enoxacin after administration on day 1 and 30
Enoxacin plasma concentrations measured in each individual participant prior to dosing and at 1, 2, 4, 6, 8 and 24 hours post morning dosing on days 1 and 30 of dosing will be used to derive the Cmax.
Measured: Prior to dosing and at 1, 2, 4, 6, 8 and 24 hours post morning dosing on days 1 and 30 of dosing.
Time of maximum plasma concentration (Tmax) of enoxacin after administration on day 1 and 30
Enoxacin plasma concentrations measured in each individual participant prior to dosing and at 1, 2, 4, 6, 8 and 24 hours post morning dosing on days 1 and 30 of dosing will be used to derive the Tmax.
Measured: Prior to dosing and at 1, 2, 4, 6, 8 and 24 hours post morning dosing on days 1 and 30 of dosing.
Area under the plasma concentration-time curve from time zero until the time corresponding with the last observed quantifiable concentration (AUC 0-last) of enoxacin after administration on day 1 and 30
Enoxacin plasma concentrations measured in each individual participant prior to dosing and at 1, 2, 4, 6, 8 and 24 hours post morning dosing on days 1 and 30 of dosing will be used to derive the (AUC) 0-last.
Measured: Prior to dosing and at 1, 2, 4, 6, 8 and 24 hours post morning dosing on days 1 and 30 of dosing.
Area under the plasma concentration-time curve extrapolated to infinity (AUC 0-inf) of enoxacin after administration on day 1 and 30
Enoxacin plasma concentrations measured in each individual participant prior to dosing and at 1, 2, 4, 6, 8 and 24 hours post morning dosing on days 1 and 30 of dosing will be used to derive the AUC 0-inf.
Measured: Prior to dosing and at 1, 2, 4, 6, 8 and 24 hours post morning dosing on days 1 and 30 of dosing.
Terminal half-life (t1/2) of enoxacin after administration on day 1 and 30
Enoxacin plasma concentrations measured in each individual participant prior to dosing and at 1, 2, 4, 6, 8 and 24 hours post morning dosing on days 1 and 30 of dosing will be used to derive the t1/2.
Measured: Prior to dosing and at 1, 2, 4, 6, 8 and 24 hours post morning dosing on days 1 and 30 of dosing.
Accumulation ratio (R) of enoxacin after administration on day 1 and 30
Enoxacin plasma concentrations measured in each individual participant prior to dosing and at 1, 2, 4, 6, 8 and 24 hours post morning dosing on days 1 and 30 of dosing will be used to derive the R.
Measured: Prior to dosing and at 1, 2, 4, 6, 8 and 24 hours post morning dosing on days 1 and 30 of dosing.
Trough plasma concentration at pre-dose of enoxacin on day 7, 14, 21, and 30
Enoxacin plasma concentrations measured in each individual participant prior to morning dosing on days 7, 14, 21, and 30 will be used to derive the trough plasma concentration at pre-dose.
Measured: Prior to morning dosing on days 7, 14, 21, and 30.
Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised (ALSFRS-R) score at baseline and at the end of the follow-up period
The ALSFRS-R will be used to measure activities of daily living (ADL) and global function across four domains (respiratory, bulbar function, gross motor skills, and fine motor skills) and consists of 12 questions, each scored from 0 to 4, for a total possible score of 48, with higher scores representing better function.
Measured: At baseline (prior to dosing on day 1 of dosing) and at the 14 day +/- 2 day follow-up visit
King's College (KINGS) stage at baseline and at the end of the follow-up period
The KINGS staging system for ALS will be used to assess the course of the disease and is based on the number of involved regions (where the three possible regions are bulbar, upper limb or lower limb) for the first three stages and the need for gastrostomy and non-invasive ventilation for the subsequent stages. The possible stages in the KINGS staging system are as follows: Stage 1: First Region Involved; Stage 2: Second Region Involved; Stage 3: Third Region Involved; Stage 4a: Nutritional Failure (need for gastrostomy); Stage 4b: Respiratory Failure (need for non-invasive ventilation); and Stage 5: Death.
Measured: At baseline (prior to dosing on day 1 of dosing) and at the 14 day +/- 2 day follow-up visit
Forced Vital Capacity (FVC) measurements at baseline and at the end of the follow-up period
Measured: At baseline (prior to dosing on day 1 of dosing) and at the 14 day +/- 2 day follow-up visit
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted12 Apr 2021
- Study start26 Mar 2021
- Primary completion15 Nov 2023
- Study completion15 Nov 2023
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (4)Papers the sponsor has linked to this study.
- 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 1/2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 8 participants (actual)
Sponsor and investigators
- Lead sponsor
- McGill University
- Sponsor type
- University, hospital or other
- Collaborators
- Weizmann Institute of Science, Apotex Inc.
- Responsible party
- Principal investigator, Angela Genge, McGill University
- Investigators
- Angela Genge, MD, FRCP, Principal investigator, McGill University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04840823
- Sponsor's protocol ID
- REALS-1
- Acronym
- REALS-1
- Status last verified
- Jan 2024
- First submitted
- 29 Mar 2021
- Last updated
- 26 Jan 2024
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Haramati S, Chapnik E, Sztainberg Y, Eilam R, Zwang R, Gershoni N, McGlinn E, Heiser PW, Wills AM, Wirguin I, Rubin LL, Misawa H, Tabin CJ, Brown R Jr, Chen A, Hornstein E. miRNA malfunction causes spinal motor neuron disease. Proc Natl Acad Sci U S A. 2010 Jul 20;107(29):13111-6. doi: 10.1073/pnas.1006151107. Epub 2010 Jun 29.
PubMed 20616011Emde A, Eitan C, Liou LL, Libby RT, Rivkin N, Magen I, Reichenstein I, Oppenheim H, Eilam R, Silvestroni A, Alajajian B, Ben-Dov IZ, Aebischer J, Savidor A, Levin Y, Sons R, Hammond SM, Ravits JM, Moller T, Hornstein E. Dysregulated miRNA biogenesis downstream of cellular stress and ALS-causing mutations: a new mechanism for ALS. EMBO J. 2015 Nov 3;34(21):2633-51. doi: 10.15252/embj.201490493. Epub 2015 Sep 1.
PubMed 26330466Reichenstein I, Eitan C, Diaz-Garcia S, Haim G, Magen I, Siany A, Hoye ML, Rivkin N, Olender T, Toth B, Ravid R, Mandelbaum AD, Yanowski E, Liang J, Rymer JK, Levy R, Beck G, Ainbinder E, Farhan SMK, Lennox KA, Bode NM, Behlke MA, Moller T, Saxena S, Moreno CAM, Costaguta G, van Eijk KR, Phatnani H, Al-Chalabi A, Basak AN, van den Berg LH, Hardiman O, Landers JE, Mora JS, Morrison KE, Shaw PJ, Veldink JH, Pfaff SL, Yizhar O, Gross C, Brown RH Jr, Ravits JM, Harms MB, Miller TM, Hornstein E. Human genetics and neuropathology suggest a link between miR-218 and amyotrophic lateral sclerosis pathophysiology. Sci Transl Med. 2019 Dec 18;11(523):eaav5264. doi: 10.1126/scitranslmed.aav5264.
PubMed 31852800Magen I, Kaneb HM, Masnata M, Pulimood N, Emde A, Genge A, Hornstein E. Cell-free miRNAs are pharmacodynamic biomarkers for enhanced DICER activity by enoxacin in human patients with ALS. Mol Ther. 2026 Jun 3;34(6):3224-3230. doi: 10.1016/j.ymthe.2026.03.002. Epub 2026 Mar 6.
PubMed 41792996
Study at a glance
From the registry record, last updated by the sponsor on 26 Jan 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- 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
- ClinicalTrials.gov ID
- NCT04840823
- Acronym / sponsor ID
- REALS-1 / REALS-1
- Conditions
- Amyotrophic Lateral Sclerosis
- Keywords
- Enoxacin
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 8 participants (actual)
- Lead sponsor
- McGill UniversityUniversity, hospital or other
- Collaborators
- Weizmann Institute of Science, Apotex Inc.
- Study start
- 26 Mar 2021 (actual)
- Primary completion
- 15 Nov 2023 (actual)
- Study completion
- 15 Nov 2023 (actual)
- First posted
- 12 Apr 2021 (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 study will assess the safety of the drug enoxacin at specific dose levels in adults with ALS.
Detailed 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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| Enoxacin 200mg twice daily | Experimental | Drug: Enoxacin, Drug: PlaceboEnoxacin 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. |
| Enoxacin 400mg twice daily | Experimental | Drug: Enoxacin, Drug: PlaceboEnoxacin 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. |
| Enoxacin 600mg twice daily | Experimental | Drug: EnoxacinEnoxacin 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. |
Who may qualify
- Ages
- 18 to 85 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (8 listed)
- 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
Key exclusion criteria (6 listed)
- 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
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 |
|---|---|---|
| EnoxacinOral 200mg tablet | Drug | Enoxacin 200mg twice daily, Enoxacin 400mg twice daily, Enoxacin 600mg twice daily |
| PlaceboOral tablet | Drug | Enoxacin 200mg twice daily, Enoxacin 400mg twice daily |
Primary outcome
Incidence of adverse events (AEs) and serious adverse events (SAEs)
Time frame: From baseline (prior to dosing on day 1 of dosing) to 14 day +/- 2 day follow up visit
The incidence of adverse events (new or worsened from baseline (where baseline refers to those AEs recorded prior to dosing on day 1 of dosing)) will be summarized by primary system organ class and preferred term as frequency count and percentage of participants with AEs.
Incidence of abnormalities in clinical laboratory assessments
Time frame: From baseline (prior to dosing on day 1 of dosing) to 14 day +/- 2 day follow up visit
Clinical laboratory data will be characterized by abnormalities in values and in changes from baseline values, where baseline refers to measurements taken prior to dosing on day 1 of dosing.
Incidence of abnormalities in vital signs
Time frame: From baseline (prior to dosing on day 1 of dosing) to 14 day +/- 2 day follow up visit
Vital sign data will be characterized by abnormalities in values and in changes from baseline values, where baseline refers to measurements taken prior to dosing on day 1 of dosing.
Incidence of abnormalities in physical and neurological examinations
Time frame: From baseline (prior to dosing on day 1 of dosing) to 14 day +/- 2 day follow up visit
Physical and neurological examinations will be characterized by abnormalities and in changes from baseline, where baseline refers to measurements taken prior to dosing on day 1 of dosing.
Incidence of abnormalities in electrocardiograms (ECGs)
Time frame: From baseline (prior to dosing on day 1 of dosing) to 14 day +/- 2 day follow up visit
ECG data will be characterized by abnormalities in values and in changes from baseline values, where baseline refers to measurements taken prior to dosing on day 1 of dosing.
Ability of participants to remain on their assigned dose for the full 30 day treatment period
Time frame: From the beginning (day 1) to the end (day 30) of the 30 day treatment period
The ability of participants to remain on each dose level will be measured by the mean number of missed doses.
10 secondary outcomes
Maximum plasma concentration (Cmax) of enoxacin after administration on day 1 and 30
Time frame: Prior to dosing and at 1, 2, 4, 6, 8 and 24 hours post morning dosing on days 1 and 30 of dosing.
Time of maximum plasma concentration (Tmax) of enoxacin after administration on day 1 and 30
Time frame: Prior to dosing and at 1, 2, 4, 6, 8 and 24 hours post morning dosing on days 1 and 30 of dosing.
Area under the plasma concentration-time curve from time zero until the time corresponding with the last observed quantifiable concentration (AUC 0-last) of enoxacin after administration on day 1 and 30
Time frame: Prior to dosing and at 1, 2, 4, 6, 8 and 24 hours post morning dosing on days 1 and 30 of dosing.
Area under the plasma concentration-time curve extrapolated to infinity (AUC 0-inf) of enoxacin after administration on day 1 and 30
Time frame: Prior to dosing and at 1, 2, 4, 6, 8 and 24 hours post morning dosing on days 1 and 30 of dosing.
Terminal half-life (t1/2) of enoxacin after administration on day 1 and 30
Time frame: Prior to dosing and at 1, 2, 4, 6, 8 and 24 hours post morning dosing on days 1 and 30 of dosing.
Accumulation ratio (R) of enoxacin after administration on day 1 and 30
Time frame: Prior to dosing and at 1, 2, 4, 6, 8 and 24 hours post morning dosing on days 1 and 30 of dosing.
Trough plasma concentration at pre-dose of enoxacin on day 7, 14, 21, and 30
Time frame: Prior to morning dosing on days 7, 14, 21, and 30.
Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised (ALSFRS-R) score at baseline and at the end of the follow-up period
Time frame: At baseline (prior to dosing on day 1 of dosing) and at the 14 day +/- 2 day follow-up visit
King's College (KINGS) stage at baseline and at the end of the follow-up period
Time frame: At baseline (prior to dosing on day 1 of dosing) and at the 14 day +/- 2 day follow-up visit
Forced Vital Capacity (FVC) measurements at baseline and at the end of the follow-up period
Time frame: At baseline (prior to dosing on day 1 of dosing) and at the 14 day +/- 2 day follow-up visit
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 26 Jan 2024.
- Background: PubMed 20616011Haramati S, Chapnik E, Sztainberg Y, Eilam R, Zwang R, Gershoni N, McGlinn E, Heiser PW, Wills AM, Wirguin I, Rubin LL, Misawa H, Tabin CJ, Brown R Jr, Chen A, Hornstein E. miRNA malfunction causes spinal motor neuron disease. Proc Natl Acad Sci U S A. 2010 Jul 20;107(29):13111-6. doi: 10.1073/pnas.1006151107. Epub 2010 Jun 29.
- Background: PubMed 26330466Emde A, Eitan C, Liou LL, Libby RT, Rivkin N, Magen I, Reichenstein I, Oppenheim H, Eilam R, Silvestroni A, Alajajian B, Ben-Dov IZ, Aebischer J, Savidor A, Levin Y, Sons R, Hammond SM, Ravits JM, Moller T, Hornstein E. Dysregulated miRNA biogenesis downstream of cellular stress and ALS-causing mutations: a new mechanism for ALS. EMBO J. 2015 Nov 3;34(21):2633-51. doi: 10.15252/embj.201490493. Epub 2015 Sep 1.
- Background: PubMed 31852800Reichenstein I, Eitan C, Diaz-Garcia S, Haim G, Magen I, Siany A, Hoye ML, Rivkin N, Olender T, Toth B, Ravid R, Mandelbaum AD, Yanowski E, Liang J, Rymer JK, Levy R, Beck G, Ainbinder E, Farhan SMK, Lennox KA, Bode NM, Behlke MA, Moller T, Saxena S, Moreno CAM, Costaguta G, van Eijk KR, Phatnani H, Al-Chalabi A, Basak AN, van den Berg LH, Hardiman O, Landers JE, Mora JS, Morrison KE, Shaw PJ, Veldink JH, Pfaff SL, Yizhar O, Gross C, Brown RH Jr, Ravits JM, Harms MB, Miller TM, Hornstein E. Human genetics and neuropathology suggest a link between miR-218 and amyotrophic lateral sclerosis pathophysiology. Sci Transl Med. 2019 Dec 18;11(523):eaav5264. doi: 10.1126/scitranslmed.aav5264.
- Derived from this study: PubMed 41792996Magen I, Kaneb HM, Masnata M, Pulimood N, Emde A, Genge A, Hornstein E. Cell-free miRNAs are pharmacodynamic biomarkers for enhanced DICER activity by enoxacin in human patients with ALS. Mol Ther. 2026 Jun 3;34(6):3224-3230. doi: 10.1016/j.ymthe.2026.03.002. Epub 2026 Mar 6.
- Clinical Research Unit at The Montreal Neurological Institute-Hospital, McGill UniversityLink provided by the sponsor.
- 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. This study is not recruiting at the moment.
Angela Genge, MD, FRCP principal investigator
McGill University
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing Enoxacin in people with Amyotrophic Lateral Sclerosis, run by McGill University at 1 site in Canada.
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 8 inclusion and 6 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: finished, as of the sponsor's last update on 26 Jan 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Canada. 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 combined 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.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Incidence of adverse events (AEs) and serious adverse events (SAEs) (time frame: From baseline (prior to dosing on day 1 of dosing) to 14 day +/- 2 day follow up visit). 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?
On the ClinicalTrials.gov record for NCT04840823, which also lists 4 linked publications.
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 26 Jan 2024.