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

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.

Conditions

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

Interventions

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

Sponsors & Collaborators

• Third Affiliated Hospital, Sun Yat-Sen University

  collaborator OTHER

• XellSmart Bio-Pharmaceutical (Suzhou) Co., Ltd.

  lead INDUSTRY

Principal Investigators

• Limin Rong, prof and M.D · Third Affiliated Hospital, Sun Yat-Sen University

Study Design

Allocation

RANDOMIZED

Purpose

TREATMENT

Masking

DOUBLE

Model

PARALLEL

Eligibility

Min Age

18 Years

Max Age

65 Years

Sex

ALL

Healthy Volunteers

No

Timeline & Regulatory

Start

2028-05-25

Primary Completion

2031-05-25

Completion

2031-05-26

Countries

• China

Study Locations