Testing NB-4746 High dose against a placebo for amyotrophic lateral sclerosis (ALS)
Official title: A Clinical Trial to Evaluate NB-4746 in Participants With Amyotrophic Lateral Sclerosis.
A Phase 1b/2 Study to Evaluate NB-4746 in Participants With Amyotrophic Lateral Sclerosis.
Interventions
NB-4746 High dose
NB-4746 will be administered twice daily in 2 capsules (high dose)
NB-4746 Low dose
NB-4746 will be administered twice daily in 2 capsules (low dose)
Placebo
Two capsules of placebo will be administered twice daily.
NB-4746 (Dose TBD)
Two capsules of NB-4746 will be administered twice daily (dose will be defined after Ph 1b data is reviewed).
Canadian Sites (2)
Stan Cassidy Center for Rehabilitation
Fredericton, New Brunswick, Canada
Genge Partners
Montreal, Quebec, Canada
Eligibility Criteria
See who this study is looking for30 criteria
The trial’s own eligibility text, word for word from the registry. Only the trial site can say who takes part.
Inclusion
- +\- ALS Criteria for Phase 1b:
- +Participants in Phase 1b must have:
- +Diagnosis of ALS per Gold Coast Criteria; and
- +Symptom onset ≤48 months prior to randomizationRandomisedWhich group you go into is decided by chance, not by you or your doctor.Read more → on Day 1. Date of first ALS symptom (any ALS symptom) onset is defined as the onset of weakness in the limbs, bulbar region, or trunk. Weakness in the bulbar region includes dysarthria and dysphagia.
- +ALS Criteria for Phase 2Phase 2A middle-stage study looking at what a treatment does and watching for side effects.Read more →:
- +Participants in Phase 2 must have:
- +Diagnosis of ALS per Gold Coast Criteria; and
- +Symptom onset ≤24 months prior to randomization on Day 1. Date of first ALS symptom (any ALS symptom) onset is defined as the onset of weakness in the limbs, bulbar region, or trunk. Weakness in the bulbar region includes dysarthria and dysphagia.
- +Additional Inclusion CriteriaInclusion criteriaThe things you must have or be for a study to consider you.Read more → (All Participants):
- +Male or female participants aged ≥18 years and ≤80 years at the time of signing informed consentInformed consentThe process of being told what taking part involves, then choosing freely.Read more →.
- +Participants must demonstrate functional oral intake and be independent of enteral nutritional support (e.g., PEG, G-tube) at the time of screeningScreeningThe checks done before joining, to see whether a study fits.Read more → per Principal InvestigatorPrincipal investigatorThe doctor or researcher responsible for running the study at a site.Read more →'s judgement.
- +Slow vital capacity (SVC) ≥60% of predicted at Screening.
- +If taking riluzole, participant must be on a stable dose for ≥60 days prior to Screening lab blood draw.
- +If taking edaravone, participant must have completed at least 1 cycle of edaravone prior to screening labs.
- +Capable of giving signed informed consent, as described in Section 13.2.2, which includes compliance with the requirements and restrictions listed in the ICF and in this protocolProtocolThe detailed plan a study must follow.Read more →.
- +Willing to adhere to contraceptive requirements during the study period as described in Appendix 1.
Exclusion
- −Presence of tracheostomy or permanent assisted ventilation; defined as \> 22 hours daily of mechanical ventilation for more than 1 week (7 days).
- −History or presence of clinically significant uncontrolled medical conditions (other than ALS) that include cardiovascular, respiratory, hepatic, renal, gastrointestinal, endocrinological, hematological, or neurological disorders capable of significantly altering the absorption, metabolism, or elimination of drugs; constituting a risk when taking the study drugs; or interfering with the interpretation of data as determined by the InvestigatorPrincipal investigatorThe doctor or researcher responsible for running the study at a site.Read more →.
- −Lifetime history of cancer except for basal cell or squamous epithelial carcinomas of the skin that have been resected with no evidence of metastatic disease for 3 years. Fully resected basal cell carcinoma and squamous cell carcinoma with no evidence of recurrence for 1 year are permitted.
- −Has a spinal deformity or other condition that may prevent the performance of a lumbar puncture.
- −International Normalized Ratio (INR) \>1.4, platelet count \<50,000/μL, or use of warfarin, heparin, or direct oral anticoagulants.
- −History of Class III/IV heart failure (per New York Heart Association \[NYHA\]).
- −Inability to swallow or tolerate oral medications at ScreeningScreeningThe checks done before joining, to see whether a study fits.Read more →.
- −Current participation in any other investigational drug study or receipt of an investigational drug within 30 days or 5 half-lives (whichever is longer) before Screening.
- −Known sensitivity to the NB-4746 study drug or any of the formulation components.
- −Has received NB-4746 at any time prior to initial Screening.
- −Any other reason that, in the opinion of the Investigator, would confound the conduct of this study or the interpretation of the results or that could compromise the participant's safety.
- −Currently taking or planning to receive tofersen for the treatment of ALS.
- −Female participants who have a positive serum pregnancy test at Screening, positive urine pregnancy test on Day 1, or who are breastfeeding on Day 1.
- −If a participant does not transition to the OLE in ≤30 days following completion of the main study, the participant will need to be rescreened for laboratory criteria and contraception/pregnancy criteria.
In plain language
Assembled directly from this trial’s registry record. Every sentence traces to a field below — nothing here is generated or interpreted.
What this study is
This study is testing a treatment for a condition.
Phase 1Phase 1The earliest stage of human testing, in a small group, focused on safety.Read more →/2 — a combined study that starts with early safety and continues into what the treatment does.
What is being given or done in this study: NB-4746 High dose, NB-4746 Low dose, PlaceboPlaceboA dummy treatment with no active medicine, used for comparison.Read more →, NB-4746 (Dose TBD).
From the trial registry
Built from these fields:
- designModule.designInfo.primaryPurpose
- designModule.phases
- armsInterventionsModule.interventions[].name
How the study is run
Which group you would be placed in is decided by chance, like a coin flip — not by you and not by your doctor.
You, the study team, the people giving the treatment, and the people analysing the results would all be kept unaware of which group you are in until the study ends.
From the trial registry
Built from these fields:
- designModule.designInfo.allocation
- designModule.designInfo.maskingInfo.masking
Is there a placebo?
One group receives a placeboPlaceboA dummy treatment with no active medicine, used for comparison.Read more → — a dummy treatment with no active medicine in it.
There are 5 groups in this study.
From the trial registry
Built from this field:
- armsInterventionsModule.armGroups[].type
Who the study is looking for
The study lists an age range of 18 years to 80 years.
The study is open to people of any sex.
The study does not accept healthy volunteersHealthy volunteerSomeone without the condition being studied who takes part anyway.Read more →.
These are the criteria the study lists. Only the trial siteTrial siteA hospital or clinic where a study is actually run.Read more → can decide whether someone can take part.
From the trial registry
Built from these fields:
- eligibilityModule.minimumAge
- eligibilityModule.maximumAge
- eligibilityModule.sex
- eligibilityModule.healthyVolunteers
How big and how long
The study aims to enrol about 80 people.
The study is currently expected to finish around February 2028.
The main measurement is taken over: From enrollmentEnrolmentThe number of participants a study plans to include, or has included.Read more → to 28 days.
From the trial registry
Built from these fields:
- designModule.enrollmentInfo.count
- statusModule.completionDateStruct.date
- outcomesModule.primaryOutcomes[].timeFrame
What the study measures
Ph 1b - Number of TEAEs and SAEs — measured over From enrollmentEnrolmentThe number of participants a study plans to include, or has included.Read more → to 28 days.
Evaluate safety and tolerability of NB-4746 in adult participants with ALS — measured over From enrollment to Week 12.
From the trial registry
Built from these fields:
- outcomesModule.primaryOutcomes[].measure
- outcomesModule.primaryOutcomes[].timeFrame
Source: NCT07369076 on ClinicalTrials.gov. The full registry text is further down this page — this summary never replaces it.
Not medical advice. What do these terms mean?
What is being tested — in plain terms
About NB-4746 High doseDrug
NB-4746 will be administered twice daily in 2 capsules (high dose)
From the trial registry — its own words, unedited.
What a drug is here: A medicine — a chemical or small-molecule treatment — given as the thing being studied.
Read the full explanation → · in clinical review
About NB-4746 Low doseDrug
NB-4746 will be administered twice daily in 2 capsules (low dose)
From the trial registry — its own words, unedited.
What a drug is here: A medicine — a chemical or small-molecule treatment — given as the thing being studied.
Read the full explanation → · in clinical review
About PlaceboDrug
Two capsules of placebo will be administered twice daily.
From the trial registry — its own words, unedited.
What a drug is here: A medicine — a chemical or small-molecule treatment — given as the thing being studied.
Read the full explanation → · in clinical review
About NB-4746 (Dose TBD)Drug
Two capsules of NB-4746 will be administered twice daily (dose will be defined after Ph 1b data is reviewed).
From the trial registry — its own words, unedited.
What a drug is here: A medicine — a chemical or small-molecule treatment — given as the thing being studied.
Read the full explanation → · in clinical review
Common questions
Answered from this trial’s registry record. Where the record doesn’t say, these answers say so rather than filling the gap.
Am I eligible for this trial?
Only the trial siteTrial siteA hospital or clinic where a study is actually run.Read more → can decide whether someone can take part. Concord cannot make that determination, and neither can any tool that has not examined you.
What the study lists: an age range of 18 years to 80 years.
The full inclusionInclusion criteriaThe things you must have or be for a study to consider you.Read more → and exclusion criteriaExclusion criteriaThe things that would prevent someone from taking part.Read more → are published on this page, exactly as the study team wrote them.
The useful next step is to bring this trial to your doctor. Answering a few questions first gives them something concrete to review.
From the trial registry
- eligibilityModule.minimumAge
- eligibilityModule.maximumAge
- eligibilityModule.eligibilityCriteria
Is there a placebo?
Yes. This study includes a placeboPlaceboA dummy treatment with no active medicine, used for comparison.Read more → group — a dummy treatment with no active medicine in it.
From the trial registry
- armsInterventionsModule.armGroups[].type
Would I know which treatment I am getting?
You, the study team, the people giving the treatment, and the people analysing the results would all be kept unaware of which group you are in until the study ends.
Which group you would be placed in is decided by chance, like a coin flip — not by you and not by your doctor.
From the trial registry
- designModule.designInfo.maskingInfo.masking
- designModule.designInfo.allocation
Who can join?
The study lists an age range of 18 years to 80 years.
It is open to people of any sex.
It does not accept healthy volunteersHealthy volunteerSomeone without the condition being studied who takes part anyway.Read more →.
Only the trial siteTrial siteA hospital or clinic where a study is actually run.Read more → can confirm whether a particular person can take part.
From the trial registry
- eligibilityModule.minimumAge
- eligibilityModule.maximumAge
- eligibilityModule.sex
- eligibilityModule.healthyVolunteers
How long would this take?
The study's main measurement is taken over: From enrollmentEnrolmentThe number of participants a study plans to include, or has included.Read more → to 28 days.
The study as a whole is currently expected to finish around 2028-02.
How long any one person takes part can differ from the study length. The study team can tell you what the schedule looks like in practice.
From the trial registry
- outcomesModule.primaryOutcomes[].timeFrame
- statusModule.completionDateStruct.date
How many people are taking part?
The study aims to enrol about 80 people.
From the trial registry
- designModule.enrollmentInfo.count
Where is this happening?
This study lists 2 locations, including: Fredericton, New Brunswick, Canada; Montreal, Quebec, Canada.
Sites can open and close during a study, so confirm with the team before travelling.
From the trial registry
- trial_locations
About This Trial
The purpose of this trial is to learn about the effects of NB-4746 compared with placebo in people with amyotrophic lateral sclerosis. The questions this trial aims to answer in comparing NB-4746 to placebo are: * What adverse events associated with taking NB-4746 are reported during this trial? (An adverse event is any sign or symptom that participants have during a trial. Adverse events may or may not be caused by treatments in the trial.) * How does NB-4746 move into, through, and out of the body of the participants? * What is the change in the level of neurofilament light (NfL) in the participants' blood? (NfL is a marker used to measure the extent of damage to the nerve cells.) This trial has 2 parts. The trial doctors will start Part A before starting Part B of the trial. Participants have an option to enter the open label extension after completing Part A or Part B. Part A: Participants will be randomly placed into 1 of the 3 groups. There are equal chances to be assigned to either group. Group 1: Participants will receive NB-4746 capsules at a low dose to take by mouth twice daily for 1 month. Group 2: Participants will receive NB-4746 capsules at a high dose to take by mouth twice daily for 1 month. Group 3: Participants will receive placebo capsules to take twice daily for approximately 1 month. Part B: Participants will be randomly placed into 1 of the 2 groups. There are equal chances to be assigned to either group. Group 1: Participants will receive NB-4746 capsules at a dose determined by Part A to take by mouth twice daily for 12 weeks. Group 2: Participants will receive placebo capsules to take twice daily for approximately 12 weeks. None of the participants, trial doctors, or trial staff will know which treatment the participants will receive during Part A or B. Some trials are done this way because knowing what treatment the participants receive can affect the results of the trial. Doing a trial this way helps to make sure that the results are looked at with fairness across all treatments. Open-Label Extension: Upon the completion of Part A or Part B, the doctor will verify the participant's willingness to continue receiving study treatment. This open label extension continues until each participant completes up to 1 year of treatment. The trial doctors will check participants' ALS and general health throughout the trial.
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See if this trial could fit youThis page is for informational purposes only and does not constitute medical advice. Clinical trial eligibility can only be determined by the trial site after proper screening. Trial information is sourced from ClinicalTrials.gov and may not reflect the most current status. Always consult your healthcare provider before making decisions about clinical trial participation.