Actively Recruiting
Safety, Tolerability, and Preliminary Efficacy of iPSC-Derived Motor Neuron Progenitor Cells (XS228) in Subacute Spinal Cord Injury: A Phase I Clinical Study
Led by XellSmart Bio-Pharmaceutical (Suzhou) Co., Ltd. · Updated on 2025-11-25
12
Participants Needed
1
Research Sites
14 weeks
Total Duration
On this page
Sponsors
X
XellSmart Bio-Pharmaceutical (Suzhou) Co., Ltd.
Lead Sponsor
T
Third Affiliated Hospital, Sun Yat-Sen University
Collaborating Sponsor
AI-Summary
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 5×10^7 to 1.5×10^8 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 3+3 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.
CONDITIONS
Brief Title
Safety and Early Efficacy of iPSC-Derived Motor Neuron Progenitor Cells (XS228) in Subacute Spinal Cord Injury: A Phase I Trial
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Age between 18 and 65 years, any gender
- Cervical (C4) to lumbar (L2) spinal cord injury caused by trauma or surgery
- ASIA Impairment Scale Grade A, B, or C with MRI-confirmed spinal cord injury
- Primary spinal cord injury occurred 14 to 60 days before screening
- Agreement to use effective non-hormonal contraception during and for 6 months after the trial if of childbearing potential
- Ability to understand, comply with study procedures, and provide written informed consent
You will not qualify if you...
- Severe traumatic brain injury preventing neurological assessment
- High cervical spinal cord injury (C1-C3) causing respiratory or circulatory failure requiring intubation or tracheostomy
- Severe life-threatening organ injuries
- Unstable injuries to lungs, liver, kidneys, or spleen
- History of spinal cord injury or spinal disorders such as ankylosing spondylitis or spinal tumors
- Active infection at lumbar puncture site or intracranial hypertension
- Severe infections including sepsis or pneumonia
- Neurological or psychiatric conditions interfering with assessments
- Serious heart conditions or recent acute heart events
- Pulmonary hypertension or embolism
- Uncontrolled high or low blood pressure
- Active autoimmune diseases needing immunosuppressants
- Non-compliance with immunosuppressant use
- Significant abnormal lab results including liver, kidney, blood clotting, or blood cell counts
- History of severe allergies to trial drug or components
- Positive tests for hepatitis B, C, HIV, or syphilis
- Refusal of lumbar puncture
- Pregnant or breastfeeding females
- Active cancer or recent cancer therapy within 5 years
- Participation in another drug trial within 3 months
- Any condition the investigator considers unsuitable for participation
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - Up to 43 days
Participants receive one of two dosing regimens of XS228 through intrathecal injection via lumbar puncture to treat subacute spinal cord injury.
1 to 4 intrathecal injection visits on Day 1, Day 15, Day 29, and Day 43 depending on dosing regimen
Duration - Up to 360 days after first dose
Participants are monitored for safety and efficacy through assessments up to one year after the first dose administration.
Visits at Day 29, Day 90, Day 180, Day 270, and Day 360 post first dose
Trial Site Locations
Total: 1 location
1
The Third Affiliated Hospital of Sun Yat-sen University
Guangzhou, Guangdong, China, 510630
Actively Recruiting
Research Team
M
Michael LEE
M
Mao Pang, prof and M.D
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SEQUENTIAL
Primary Purpose
TREATMENT
Number of Arms
1
Similar Trials
Frequently Asked Questions
Have more questions? Get in touch with our team for quick support
Not the Right Trial for You?
Explore thousands of other clinical trials that might be a better match.
Sign up to get personalized trial recommendations delivered to your inbox.
Already have an account? Log in here