Take an experimental medication being tested for amyotrophic lateral sclerosis

Back to Amyotrophic Lateral Sclerosis (ALS) trials

Recruiting

What is this trial?

This trial is testing whether a new medication called NB-4746 is safe and effective for people with ALS (a disease that causes progressive nerve damage and muscle weakness). The study will compare NB-4746 at different doses against placebo (a dummy pill with no active ingredient) to see if the medication can reduce markers of nerve damage in the blood.

ALS is a serious disease with limited treatment options, and there is an urgent need to find new medicines that can slow the damage to nerve cells. This trial is designed to see whether this new treatment could offer hope to newly diagnosed patients.

Do you qualify?

You likely qualify if…

you have been diagnosed with ALS within the past 2–4 years (depending on which part of the study you join)
you are between 18 and 80 years old
you can still eat and drink normally without feeding tubes
your lung function is at least 60% of predicted
you have not had a tracheostomy or been on mechanical ventilation for more than 22 hours a day

You likely don't qualify if…

you are pregnant, trying to become pregnant, or breastfeeding
you have a history of cancer (except certain skin cancers that have been fully treated)
you have a serious medical condition affecting your heart, lungs, liver, or kidneys that could interfere with the study
you are taking blood thinners like warfarin or heparin, or have a bleeding disorder
you have a spinal condition that would prevent a spinal tap (lumbar puncture)

What participation looks like

If you join, you'll take capsules by mouth twice a day. Part A lasts about 1 month, during which you'll be randomly assigned to receive either a low dose, high dose, or placebo. Then Part B lasts 12 weeks, where you'll again be randomly assigned to the medication (at a dose chosen based on Part A results) or placebo. Neither you nor the doctors will know which treatment you're receiving. You'll have blood tests to check for side effects and to measure a marker of nerve damage, and you'll have the option to continue in an open-label extension after the study ends.

Testing a New Drug to Slow Nerve Damage in ALS

What is this trial?

Do you qualify?

What participation looks like

Trial locations(8 sites)