NCT06974968

Efficacy of iPSC-Derived Motor Neuron Cells (XS228) in Subacute Spinal Cord Injury: A Phase II Randomized Controlled Trial

A Phase II Clinical Study Evaluating the Preliminary Efficacy of Human Allogeneic Induced Pluripotent Stem Cell (iPSC)-Derived Motor Neuron Progenitor Cells (XS228 Cell Injection) in Patients With Subacute Spinal Cord Injury

• Status: Not Yet Recruiting
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 60 (estimated)
• Sponsor: XellSmart Bio-Pharmaceutical (Suzhou) Co., Ltd. · Industry
• Sex: All
• Age: 18 Years – 65 Years
• Healthy volunteers: Not accepted

Summary

Purpose: This clinical trial is studying an investigational cell therapy called XS228-a lab-made stem cell product designed to help repair damaged nerves in the spinal cord. The goal is to see if XS228 is safe and can improve movement, sensation, and function in people with recent spinal cord injuries. Study Treatment: XS228 contains specialized nerve-supporting cells derived from human stem cells. These cells are injected into the spinal fluid (intrathecal administration) in a single dose. Who Can Join? Adults aged 18-65 with a spinal cord injury (thoracic or lumbar level) that occurred 2-12 weeks before enrollment. Participants must have severe but incomplete paralysis (ASIA Impairment Scale Grade A , B or C). Study Plan: Phase II (Main Study): About 60 participants will be randomly assigned to receive either XS228 or a placebo (inactive solution) in a 2:1 ratio. Follow-up: Patients will be monitored for 1 year, with regular check-ups to assess safety, nerve function, and recovery progress. What Researchers Are Looking For: Primary Goal: Measure changes in leg and arm function using the ASIA Motor Score at 6 months. Secondary Goals: Improvement in ASIA Impairment Scale (AIS) grade (e.g., from "complete" to "incomplete" paralysis). Recovery of sensation and bladder/bowel control. Safety (monitoring for side effects like infections or immune reactions). Exploratory Tests: MRI scans and biomarker tests in spinal fluid to see if the treatment helps nerve regrowth. Why This Study Matters: If successful, XS228 could become the first stem cell therapy to promote meaningful recovery in spinal cord injury patients. Currently, no treatments exist to repair nerve damage-this trial aims to change that.

Detailed description

Title: A Randomized, Double-Blind, Placebo-Controlled Phase II Trial to Evaluate the Efficacy and Safety of XS228 Cell Injection in Patients with Subacute Thoracic or Lumbar SCI Study Type: Interventional (Clinical Trial) Phase: Phase II Allocation: Randomized (2:1, XS228 vs. Placebo) Masking: Double-blind (Participant, Investigator) Primary Purpose: Treatment Hypothesis: XS228 (allogeneic iPSC-derived motor neuron progenitor cells) will demonstrate statistically significant improvement in motor function (ASIA Motor Score) compared to placebo at 6 months post-treatment. The therapy will exhibit an acceptable safety profile with no dose-limiting toxicities (DLTs). Intervention Details Investigational Product: XS228 Cell Injection: Cryopreserved suspension of optimal dose from Phase I. Placebo: Normal saline with identical packaging/labeling. Administration: 4 intrathecal injection via lumbar puncture (Day 1, Day 15, Day 29 and Day 43). Concomitant Therapies: Standard rehabilitation protocols (uniform across sites). Prohibited: Other experimental therapies or stem cell treatments during the study.

Conditions

• Spinal Cord Injury
• Safety
• Efficacy
• Clinical Trial
• Induced Pluripotent Stem Cells
• Human Motor Neuron Progenitor
• Transplantation
• RCT

Interventions

Type	Name	Description
BIOLOGICAL	Allogeneic Human Induced Pluripotent Stem Cell (iPSC)-Derived Motor Neuron Progenitor Cells	Description: XS228 Cell Injection is an investigational, allogeneic cell therapy product composed of motor neuron progenitor cells (MNPCs) derived from human induced pluripotent stem cells (iPSCs). This advanced therapy medicinal product (ATMP) is being developed for the treatment of spinal cord injury (SCI) and represents a novel approach in regenerative medicine.
OTHER	Placebo	Standard rehabilitation protocols

Timeline

• Start date: 2028-05-25
• Primary completion: 2031-05-25
• Completion: 2031-05-26
• First posted: 2025-05-16
• Last updated: 2025-11-25

Locations

1 site across 1 country: China