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 Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Enrollment
80
NB-4746 will be administered twice daily in 2 capsules (high dose)
NB-4746 will be administered twice daily in 2 capsules (low dose)
Two capsules of placebo will be administered twice daily.
Two capsules of NB-4746 will be administered twice daily (dose will be defined after Ph 1b data is reviewed).
Concord Repatriation General Hospital
Concord, New South Wales, Australia
RECRUITINGNeuroscience Research Australia
Randwick, New South Wales, Australia
RECRUITINGRoyal Brisbane and Women's Hospital
Brisbane, Queensland, Australia
RECRUITINGFlinders Medical Centre
Bedford Park, South Australia, Australia
RECRUITINGCalvery Health care Bethlehem
Caulfield South, Victoria, Australia
RECRUITINGPerron Institute
Nedlands, Western Australia, Australia
RECRUITINGStan Cassidy Center for Rehabilitation
Fredericton, New Brunswick, Canada
RECRUITINGSunnybrook Health Sciences Centre
Toronto, Ontario, Canada
RECRUITINGGenge Partners
Montreal, Quebec, Canada
RECRUITINGUMC Utrect
Utrecht, CX, Netherlands
RECRUITING...and 2 more locations
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.
Time frame: 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
Time frame: From enrollment to Week 12
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