Amyotrophic Lateral Sclerosis (ALS)Phase 1/2NCT07369076
A Clinical Trial to Evaluate NB-4746 in Participants With Amyotrophic Lateral Sclerosis.
A Phase 1/2 study testing NB-4746 High dose, NB-4746 Low dose and NB-4746 (Dose TBD) in people with Amyotrophic Lateral Sclerosis (ALS), run by Nura Bio at 12 sites in 4 countries.
Trial summary
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… Read more about this study
- Who is it for?
- Ages 18 to 80 years
- Conditions the study is for
- Amyotrophic Lateral Sclerosis (ALS)
- What is being tested?
- NB-4746 High dose, NB-4746 Low dose and NB-4746 (Dose TBD)
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 80 participants
- Where
- 12 sitesin 4 countries
- Study dates
- 4 Mar 2026to Feb 2028
- Phase
- Phase 1/2
- Are there results?
- Not yet: the study is under way
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 80 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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 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.
- ALS Criteria for Phase 2:
- Participants in Phase 2 must have:
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- 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 Criteria (All Participants):
- Male or female participants aged ≥18 years and ≤80 years at the time of signing informed consent.
- Participants must demonstrate functional oral intake and be independent of enteral nutritional support (e.g., PEG, G-tube) at the time of screening per Principal Investigator'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.
- Willing to adhere to contraceptive requirements during the study period as described in Appendix 1.
- 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 protocol.
Who cannot join
- 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 Investigator.
- 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.
- 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.
- 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.
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- History of Class III/IV heart failure (per New York Heart Association [NYHA]).
- Inability to swallow or tolerate oral medications at Screening.
- 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.
- 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.
Trial locations and contacts
12 sites in 4 countries. Contact the study team or a site near you to ask about taking part.
12 sites in 4 countries
Australia6 sites, 6 recruiting
- Recruiting
Flinders Medical Centre
Bedford Park, South Australia, 5042
Karalyn Ernst, +62 08 8204 5712, karalyn.ernst@sa.gov.au
- Recruiting
Royal Brisbane and Women's Hospital
Brisbane, Queensland, 4029
Susan Heggie, (07) 3646 0016, Susan.Heggie@health.qld.gov.au
- Recruiting
Calvery Health care Bethlehem
Caulfield South, Victoria, 3162
Yana Wu, 6195953294, yana.wu@calvarycare.org.au
- Recruiting
Concord Repatriation General Hospital
Concord, New South Wales, 2139
Eunji Lee, +61 02 9767 6820, Eunji.lee2@health.nsw.gov.au
- Recruiting
Perron Institute
Nedlands, Western Australia, 6009
Ruby Shehatha, 61864570213, ruby.shehatha@perron.uwa.edu.au
- Recruiting
Neuroscience Research Australia
Randwick, New South Wales, 2031
Eleanor Ramsey, +62 02 9399 1146, clinicaneurology@neura.edu.au
Canada3 sites, 3 recruiting
- Recruiting
Stan Cassidy Center for Rehabilitation
Fredericton, New Brunswick, E3B 0C7
Shane McCullum, (506) 447-4379, Shane.a.mccullum@horizonnb.ca
- Recruiting
Genge Partners
Montreal, Quebec, H4A 3T2
Kevin Munoz, +1 514-906-3876 ext. 6026, kmunoz@gpresearchinc.com
- Recruiting
Sunnybrook Health Sciences Centre
Toronto, Ontario, 3M5
Eunice Lam, (416) 480-6100 ext. 687561, eunice2.lam@sri.utoronto.ca
Poland2 sites, 2 recruiting
- Recruiting
Linden Sp. z o.o. sp.k.
Krakow
Anna Sołtysiak, 48 602 827 838, anna.soltysiak@cmlinden.com
- Recruiting
CityClinic Research Sp. z o.o.
Warsaw
Dorota Jablonska, 48 788637220, jablonskapresite@gmail.com
Netherlands1 site, 1 recruiting
- Recruiting
UMC Utrect
Utrecht, CX
Willy Kool, 31 205663186, w.kool@umcutrecht.nl
About this study
Official title: A Phase 1b/2 Study to Evaluate NB-4746 in Participants With Amyotrophic Lateral Sclerosis.
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:
Read more
- 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.
- Conditions
- Amyotrophic Lateral Sclerosis (ALS)
- Being tested
- NB-4746 High dose, NB-4746 Low dose, Placebo, NB-4746 (Dose TBD)
- Keywords
- Amyotrophic Lateral Sclerosis, ALS
- 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.
NB-4746 High doseDrug
NB-4746 will be administered twice daily in 2 capsules (high dose)
NB-4746 Low doseDrug
NB-4746 will be administered twice daily in 2 capsules (low dose)
PlaceboDrug
Two capsules of placebo will be administered twice daily.
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).
| Study group | Role | What they receive |
|---|---|---|
| Phase 1b - NB-4746, High dose | Experimental | Participants will receive two NB-4746 capsules (high dose), BID Receives: Drug: NB-4746 High dose |
| Phase 1b - NB-4746, Low dose | Experimental | Participants will receive two NB-4746 capsules (low dose), BID Receives: Drug: NB-4746 Low dose |
| Ph 1b - Placebo | Placebo comparator | Participants will receive two capsules of placebo per dose, BID. Receives: Drug: Placebo |
| Ph 2 - Active | Experimental | Participants will receive two capsules of NB-4746 (dose selected after review of Ph 1b data), BID Receives: Drug: NB-4746 (Dose TBD) |
| Ph 2 - Placebo | Placebo comparator | Participants will receive two capsules of placebo, BID Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Ph 1b - Number of TEAEs and SAEs
Number of treatment emergent adverse events (TEAEs) and number of serious adverse events (SAEs).
This will be assessed in the Ph 1b part of the study.
Measured: From enrollment to 28 days
Evaluate safety and tolerability of NB-4746 in adult participants with ALS.
Incidence of treatment emergent adverse events (TEAEs) and abnormalities in vital signs, clinical laboratory assessments, and electrocardiograms (ECGs).
This will be assessed in the Ph 2 part of the study
Measured: From enrollment to Week 12
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted27 Jan 2026
- Study start4 Mar 2026
- Primary completionApr 2027 (estimated)
- Study completionFeb 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Amyotrophic Lateral Sclerosis (ALS)Studies 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
- 80 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Nura Bio
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07369076
- Sponsor's protocol ID
- NB-4746-201
- Status last verified
- Apr 2026
- First submitted
- 21 Jan 2026
- Last updated
- 17 Sep 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Undecided
Study at a glance
From the registry record, last updated by the sponsor on 17 Sep 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 1b/2 Study to Evaluate NB-4746 in Participants With Amyotrophic Lateral Sclerosis.
- ClinicalTrials.gov ID
- NCT07369076
- Acronym / sponsor ID
- NB-4746-201
- Conditions
- Amyotrophic Lateral Sclerosis (ALS)
- Keywords
- Amyotrophic Lateral Sclerosis, ALS
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 80 participants (estimated)
- Lead sponsor
- Nura BioIndustry (pharma, biotech, devices)
- Study start
- 4 Mar 2026 (actual)
- Primary completion
- Apr 2027 (estimated)
- Study completion
- Feb 2028 (estimated)
- First posted
- 27 Jan 2026 (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 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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 5
| Arm | Type | Receives |
|---|---|---|
| Phase 1b - NB-4746, High dose | Experimental | Drug: NB-4746 High doseParticipants will receive two NB-4746 capsules (high dose), BID |
| Phase 1b - NB-4746, Low dose | Experimental | Drug: NB-4746 Low doseParticipants will receive two NB-4746 capsules (low dose), BID |
| Ph 1b - Placebo | Placebo comparator | Drug: PlaceboParticipants will receive two capsules of placebo per dose, BID. |
| Ph 2 - Active | Experimental | Drug: NB-4746 (Dose TBD)Participants will receive two capsules of NB-4746 (dose selected after review of Ph 1b data), BID |
| Ph 2 - Placebo | Placebo comparator | Drug: PlaceboParticipants will receive two capsules of placebo, BID |
Who may qualify
- Ages
- 18 to 80 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (16 listed)
- ALS Criteria for Phase 1b:
- Participants in Phase 1b must have:
- Participants in Phase 2 must have:
- Male or female participants aged ≥18 years and ≤80 years at the time of signing informed consent.
- Participants must demonstrate functional oral intake and be independent of enteral nutritional support (e.g., PEG, G-tube) at the time of screening per Principal Investigator's judgement.
Key exclusion criteria (14 listed)
- 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 Investigator.
- 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.
- 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.
- Has a spinal deformity or other condition that may prevent the performance of a lumbar puncture.
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 |
|---|---|---|
| NB-4746 High doseNB-4746 will be administered twice daily in 2 capsules (high dose) | Drug | Phase 1b - NB-4746, High dose |
| NB-4746 Low doseNB-4746 will be administered twice daily in 2 capsules (low dose) | Drug | Phase 1b - NB-4746, Low dose |
| PlaceboTwo capsules of placebo will be administered twice daily. | Drug | Ph 1b - Placebo, Ph 2 - Placebo |
| NB-4746 (Dose TBD)Two capsules of NB-4746 will be administered twice daily (dose will be defined after Ph 1b data is reviewed). | Drug | Ph 2 - Active |
Primary outcome
Ph 1b - Number of TEAEs and SAEs
Time frame: From enrollment to 28 days
Number of treatment emergent adverse events (TEAEs) and number of serious adverse events (SAEs). This will be assessed in the Ph 1b part of the study.
Evaluate safety and tolerability of NB-4746 in adult participants with ALS.
Time frame: From enrollment to Week 12
Incidence of treatment emergent adverse events (TEAEs) and abnormalities in vital signs, clinical laboratory assessments, and electrocardiograms (ECGs). This will be assessed in the Ph 2 part of the study
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 17 Sep 2026.
- Individual participant dataSharing planned: Undecided
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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.
12 sites in 4 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing NB-4746 High dose, NB-4746 Low dose and NB-4746 (Dose TBD) in people with Amyotrophic Lateral Sclerosis (ALS), run by Nura Bio at 12 sites in 4 countries.
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 16 inclusion and 14 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: looking for participants now, as of the sponsor's last update on 17 Sep 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 12 sites in Australia, Canada, Poland, Netherlands. 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. Some participants in this study may receive a placebo. 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?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Lahar Mehta, MD (801-918-7637, lmehta@nurabio.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Ph 1b - Number of TEAEs and SAEs (time frame: From enrollment to 28 days). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 17 Sep 2026.