Actively Recruiting
Evaluation of NB-4746 Medication Compared to Placebo in Adults With Amyotrophic Lateral Sclerosis to Assess Safety and Effects
Led by Nura Bio · Updated on 2026-06-22
80
Participants Needed
8
Research Sites
43 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are evaluating the effects of the drug NB-4746 compared with a placebo in adults with amyotrophic lateral sclerosis ALS. This trial aims to understand the safety of NB-4746, how the drug moves through the body, and changes in a blood marker called neurofilament light NfL that reflects nerve cell damage. The study is conducted in two parts and includes an option for participants to join an open-label extension phase. In Part A, participants are randomly assigned to one of three groups low-dose NB-4746 capsules taken twice daily, high-dose NB-4746 capsules taken twice daily, or placebo capsules taken twice daily, all for about one month. In Part B, participants are randomly assigned to either NB-4746 at a dose determined from Part A or placebo, both taken twice daily for approximately 12 weeks. After completing Part A or B, participants may choose to enter an open-label extension to continue treatment for up to one year. During the trial, participants will have their ALS symptoms and overall health monitored regularly. The study team will assess safety by recording treatment-emergent adverse events and serious adverse events. Blood samples will be collected to measure NfL levels and evaluate drug movement in the body. Participants will be followed throughout the study and during the extension phase to track health status and treatment effects up to one year.
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...
- Diagnosis of ALS according to Gold Coast Criteria
- Symptom onset within 48 months prior to randomization for Phase 1b or within 24 months for Phase 2
- Age 18 to 80 years at time of consent
- Able to eat normally by mouth without enteral nutritional support
- Slow vital capacity of at least 60% predicted at screening
- Stable dose of riluzole for 60 days if taking
- Completed at least one cycle of edaravone prior to screening if taking
- Willing to follow contraceptive requirements during the study
- Capable of giving signed informed consent
You will not qualify if you...
- Use 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 or squamous cell carcinoma with no recurrence as specified
- Positive pregnancy or breastfeeding at screening or Day 1 if female
- Spinal deformity or condition preventing lumbar puncture
- Blood clotting abnormalities or use of certain blood thinners
- History of severe heart failure (Class III/IV NYHA)
- Inability to swallow or tolerate oral medications
- Participation in another investigational drug study within 30 days or 5 half-lives
- Sensitivity to NB-4746 or its components
- Prior receipt of NB-4746 before screening
- Use or planned use of tofersen for ALS treatment
- Any other condition that could affect study conduct or safety
- Failure to transition to the open-label extension within 30 days requires rescreening
Research Team
L
Lahar Mehta, MD
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