Phase I Study of XS228 iPSC-Derived Motor Neuron Progenitor Cells Evaluating Safety and Early Effects in Adults with Subacute Spinal Cord Injury

What this Trial Is About

Researchers are evaluating the safety and tolerability of XS228, an investigational cell therapy made from motor neuron progenitor cells derived from human induced pluripotent stem cells iPSCs, in patients with subacute spinal cord injury. This Phase I trial focuses on patients who have experienced spinal cord injury between 14 to 60 days prior to screening, with injuries ranging from cervical to lumbar regions and classified by the ASIA Impairment Scale. The study aims to explore early safety and preliminary efficacy in this population. The trial includes two treatment stages Single Ascending Dose SAD and Multiple Ascending Dose MAD. Participants receive XS228 through intrathecal injection via lumbar puncture. In the SAD stage, a single dose ranging from 5107 to 1.5108 cells is given. In the MAD stage, participants receive four doses on Days 1, 15, 29, and 43 at similar dose levels, with dose escalation following a 33 design. The therapy is administered to study its safety, tolerability, and early effects on spinal cord injury. Participants will be monitored closely for adverse events and dose-limiting toxicities up to 28 days after dosing. Researchers will assess neurological function and recovery using tools like the ASIA Impairment Scale, ASIA motor and sensory scores, and the Spinal Cord Independence Measure-III at multiple time points up to one year after treatment. The trial spans over a year of follow-up to evaluate safety and motor neuron function changes, with all care provided under medical supervision.

Safety and Early Efficacy of iPSC-Derived Motor Neuron Progenitor Cells (XS228) in Subacute Spinal Cord Injury: A Phase I Trial