The goal of this clinical trial is to learn if drug CB03-154 works to treat ALS in adults. It will also learn about the safety of drug CB03-154.
The main questions it aims to answer are:
* Does drug CB03-154 have an effect on delaying disease progression, improving function, and prolonging survival in adult ALS patients? * What medical problems do patients have when taking drug CB03-154? Researchers will compare drug CB03-154 to a placebo (a look-alike substance that contains no drug) to see if drug CB03-154 works to treat ALS.
Participants (adult ALS patients) will:
* Take drug CB03-154 or a placebo every day for 39 weeks (an additional 39 weeks would be required if entering the open-label extension phase). * Visit the clinic approximately every 2-3 months for checkups and tests, and there is also telephone follow-up in between. * Keep a diary of daily medication (CB03-154 or other concomitant medications), and if there are any unplanned medications, the reason (disease or symptoms) also need be recorded.
Agree to follow the treatment plan and trial procedures of this study, and sign the written informed consent form.
Male or female, aged 18 to 65 years, inclusive.
The weight of subjects during the screening period must not be less than 45 kg, and the BMI must not be less than 18 kg/m2.
Diagnosed according to the Revised EI Escorial diagnostic criteria set by the World Federation of Neurology: definite ALS, probable ALS, lab supported probable ALS, or possible ALS.
Significant cognitive impairment, mental disorders (such as schizophrenia, bipolar disorder), other neurodegenerative diseases (such as Parkinson's disease, Alzheimer's disease, frontotemporal dementia, etc.), substance abuse or other causes leading to neuromuscular weakness (such as myasthenia gravis), or other conditions that may interfere with the participants' participation in clinical study or, in the investigator's judgment, may interfere with outcome assessment or affect the completion of the trial.
Serum aspartate aminotransferase (AST), alanine aminotransferase (ALT), or bilirubin>2.0× upper limit of normal at screening.
Estimated glomerular filtration rate <59 mL/min/1.73m2 at Screening (using the Cockcroft-Gault formula to calculate eGFR: eGFR (mL/min/1.73m2) = Ccr × 0.84 × 1.73 / BSA; Ccr (mL/min) = [(140 - age) × weight (kg)] / [72 × Scr (mg/dL)], females multiply the result by 0.85, and Scr is the serum creatinine; BSA (m2) = 0.007184 × weight (kg)^0.425 × height (cm)^0.725).
D-dimer>2.0× upper limit of normal or venous ultrasound of the lower limbs shows deep vein thrombosis at screening or a history of venous thrombosis.