Cell-Based Therapy for White Matter Repair in Periventricular Leukomalacia
brief summary
The goal of this study is to explore a new treatment that may help repair brain damage in individuals with periventricular leukomalacia (PVL), a condition that affects white matter in the brain. Researchers are testing whether a combination of a novel cell therapy and specific molecular agents can support brain repair. The main questions the study aims to answer are: Can the treatment help regrow white matter and improve myelin repair? Does the treatment reduce scarring in the brain? Is the treatment safe and well-tolerated? The study uses several components, including: A specific type of neural progenitor cell to form the basis of the therapy. A small molecule compound to support cell function and survival. An agent designed to promote the repair of the myelin sheath. An enzyme intended to break down scar tissue in the brain. Researchers will study how these components work together to protect and repair the brain by influencing key pathways involved in damage and recovery.
official title
Phase 1 Study Evaluating the Safety and Preliminary Efficacy of a Novel Cell-Based and Small Molecule Combination Therapy for White Matter Injury in Patients With Periventricular Leukomalacia