Actively Recruiting
A Phase 1b/2 Study to Evaluate NB-4746 in Participants With Amyotrophic Lateral Sclerosis
Led by Nura Bio · Updated on 2026-05-22
80
Participants Needed
6
Research Sites
43 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are studying the effects of NB-4746 compared with a placebo in adults with amyotrophic lateral sclerosis (ALS). The trial aims to evaluate reported side effects, how the drug is processed in the body, and changes in a blood marker called neurofilament light, which indicates nerve damage. The study includes two parts and offers an optional open-label extension for continued treatment up to one year. In Part A, participants are randomly assigned to one of three groups, receiving either a low dose or high dose of NB-4746 capsules or a placebo, taken twice daily for about one month. In Part B, participants are again randomized to receive NB-4746 at a dose determined from Part A or placebo, taken twice daily for 12 weeks. After completing Part A or B, participants may join an open-label extension to continue NB-4746 treatment for up to one year. Throughout the study, participants will have their ALS status and overall health monitored. Researchers will track side effects, safety, and tolerability from enrollment through the treatment periods. Blood tests will measure the neurofilament light levels to assess nerve damage. The trial includes regular check-ins and assessments to understand how NB-4746 affects participants over time.
CONDITIONS
Brief Title
A Clinical Trial to Evaluate NB-4746 in Participants With Amyotrophic Lateral Sclerosis.
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Adults aged 18 to 80 years at the time of informed consent
- Diagnosis of ALS according to Gold Coast Criteria
- Symptom onset within 48 months prior to randomization for Phase 1b
- Symptom onset within 24 months prior to randomization for Phase 2
- Score of at least 2 on the swallowing function of the ALSFRS-R
- Slow vital capacity (SVC) of at least 60% of predicted at screening
- Stable dose of riluzole for at least 60 days prior to Day 1, if taking
- Completed at least one cycle of edaravone prior to Day 1, if taking
- Screening laboratory test values within normal ranges
- Willing to follow contraceptive requirements during the study
- Ability to provide signed informed consent and comply with study requirements
You will not qualify if you...
- Presence of tracheostomy or permanent assisted ventilation exceeding 22 hours daily for more than 7 days
- Clinically significant uncontrolled medical conditions that affect drug metabolism or pose risk
- History of cancer except fully resected basal cell or squamous cell carcinoma with no recurrence as specified
- Positive pregnancy test or breastfeeding at screening or Day 1 (for females)
- Spinal deformity or condition preventing lumbar puncture
- INR greater than 1.4, platelet count less than 50,000/µL, or use of certain anticoagulants
- History of Class III or IV heart failure
- Inability to swallow or tolerate oral medications at screening
- Participation in another investigational drug study within 30 days or 5 half-lives prior to screening
- Known sensitivity to NB-4746 or its components
- Prior receipt of NB-4746 before initial screening
- Use or planned use of tofersen for ALS treatment
- Any other condition that may interfere with study conduct or safety as judged by the investigator
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey: What to Expect
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - Up to 12 weeks
Participants receive NB-4746 or placebo capsules twice daily as part of the study treatment.
Multiple visits during the treatment period
Trial Site Locations
Total: 6 locations
1
Concord Repatriation General Hospital
Concord, New South Wales, Australia, 2139
Actively Recruiting
2
Neuroscience Research Australia
Randwick, New South Wales, Australia, 2031
Actively Recruiting
3
Royal Brisbane and Women's Hospital
Brisbane, Queensland, Australia, 4029
Actively Recruiting
4
Flinders Medical Centre
Bedford Park, South Australia, Australia, 5042
Actively Recruiting
5
Stan Cassidy Center for Rehabilitation
Fredericton, New Brunswick, Canada, E3B 0C7
Actively Recruiting
6
Genge Partners
Montreal, Quebec, Canada, H4A 3T2
Actively Recruiting
Research Team
L
Lahar Mehta, MD
How is the study designed?
Study Type
INTERVENTIONAL
Masking
QUADRUPLE
Allocation
RANDOMIZED
Model
PARALLEL
Primary Purpose
TREATMENT
Number of Arms
5
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