Single and multiple ascending dose study of oral ZKN-0013 or placebo given randomly to healthy volunteers to evaluate the safety, tolerability and the amount of drug in the body after drug administration.

ZKN-0013 is a new experimental medicine developed by Eloxx Pharmaceuticals that works by targeting faulty genetic instructions inside cells — potentially helping the body produce normal proteins instead of defective ones caused by gene mutations. This is a Phase 1 first-in-human study, meaning it is the very first time this drug is being tested in people. This trial is testing whether ZKN-0013 is safe and well tolerated in healthy volunteers. Participants will take one or several doses of the drug (or a placebo) and researchers will carefully monitor their safety, any side effects, and how the drug moves through the body over time. You may be eligible if you are a healthy adult aged 18–55, a non-smoker for at least one month, have a BMI between 18 and 32, and are not on regular prescription medications. People with any significant health conditions (heart, liver, kidney, lung, or psychiatric conditions), HIV, hepatitis, or a history of alcohol or drug misuse are not eligible. Contraception is required during the trial.