Efficacy of iPSC-Derived Motor Neuron Cells (XS228) in Subacute Spinal Cord Injury: A Phase II Randomized Controlled Trial
NCT ID: NCT06974968
Last Updated: 2025-11-25
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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NOT_YET_RECRUITING
PHASE2
60 participants
INTERVENTIONAL
2028-05-25
2031-05-26
Brief Summary
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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.
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Detailed Description
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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
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Study Design
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RANDOMIZED
PARALLEL
TREATMENT
DOUBLE
Due to the need for cell-based product preparation based on randomization results, an unblinded team will be established to handle drug preparation and packaging. Unblinded team members will not perform any clinical assessments. Further details are provided in the Drug Management Manual.
Study Groups
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XS228 Interventional Group
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, Day 43).
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.
Placebo Control Group
Standard rehabilitation protocols
Placebo
Standard rehabilitation protocols
Interventions
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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.
Placebo
Standard rehabilitation protocols
Other Intervention Names
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Eligibility Criteria
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Inclusion Criteria
Etiology: Cervical (C4) to lumbar (L2) spinal cord injury (SCI) caused by traumatic injury or surgery-related factors.
Severity:
Classified as ASIA Impairment Scale (AIS) Grades A, B, or C. MRI-confirmed evidence of spinal cord injury.
Disease Stage:
Primary SCI occurring 14 to 60 days prior to screening (subacute phase).
Contraception:
Participants of childbearing potential (male and female) must agree to use effective non-hormonal contraceptive methods during the trial and for 6 months after trial completion.
Compliance:
Voluntarily participate in the clinical study. Ability to understand and comply with study procedures. Participant or legal guardian can provide written informed consent.
Exclusion Criteria
Primary spinal cord injury (SCI) during screening with concomitant severe traumatic brain injury precluding neurological function assessment.
Respiratory/Circulatory Instability
High cervical SCI (C1-C3) causing respiratory/circulatory compromise requiring endotracheal intubation or tracheostomy.
Life-Threatening Multiorgan Dysfunction
Concurrent severe injuries to other organ systems with life-threatening dysfunction.
Unstable Thoracoabdominal Injuries
Injuries to lungs, liver, kidneys, spleen, etc., deemed unstable by the investigator.
Prior Spinal Pathology
History of SCI or coexisting spinal disorders (e.g., ankylosing spondylitis, spinal deformities, primary/metastatic spinal tumors, spinal vascular malformations, syringomyelia).
Local Infection/Increased ICP
Active infection at the lumbar puncture site or intracranial hypertension during screening.
Severe Infections
Sepsis, septic shock, or severe pneumonia (per IDSA/ATS 2007 diagnostic criteria).
Confounding Neurological/Psychiatric Conditions
Parkinson's disease, severe dementia, myasthenia gravis, stroke, Guillain-Barré syndrome, diabetic neuropathy, or other conditions interfering with study assessments.
Cardiac Abnormalities (any of the following):
Congestive heart failure (NYHA Class III/IV). Severe uncontrolled arrhythmias (e.g., sick sinus syndrome, third-degree AV block).
Unstable angina or acute myocardial infarction within 3 months prior. Pulmonary Complications
Pulmonary hypertension, pulmonary embolism, or suspected embolism during screening.
Uncontrolled Hypertension/Hypotension
Systolic BP \>160 mmHg or diastolic BP \>100 mmHg; or systolic BP \<90 mmHg or diastolic BP \<60 mmHg.
Active Autoimmune Diseases
Requiring immunosuppressants (e.g., uncontrolled hyperthyroidism, systemic lupus erythematosus).
Immunosuppressant Non-Compliance
Unwillingness or inability to use immunosuppressants per protocol.
Laboratory Abnormalities (any of the following):
ALT/AST \>2×ULN or total bilirubin \>2×ULN. eGFR \<60 mL/min/1.73m² (CKD-EPI 2021 formula). APTT/PT \>2.5×ULN (without anticoagulants). Platelets \<100×10⁹/L or hemoglobin \<90 g/L. Allergy
History of severe allergies or hypersensitivity to trial drug/excipients (human albumin, lactated Ringer's solution).
Infectious Diseases
HBsAg+ with HBV DNA \>1000 IU/mL; HCV-Ab+; HIV-Ab+; or TP-Ab+. Lumbar Puncture Refusal
Unwillingness to undergo intrathecal administration procedures. Pregnancy/Lactation
Females who are pregnant or breastfeeding. Malignancy
Active malignancy or anticancer therapy within 5 years prior. Recent Clinical Trial Participation
Enrollment in another drug trial within 3 months prior. Investigator Discretion
Any condition deemed unsuitable for participation by the investigator
18 Years
65 Years
ALL
No
Sponsors
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Third Affiliated Hospital, Sun Yat-Sen University
OTHER
XellSmart Bio-Pharmaceutical (Suzhou) Co., Ltd.
INDUSTRY
Responsible Party
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Principal Investigators
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Limin Rong, prof and M.D
Role: PRINCIPAL_INVESTIGATOR
Third Affiliated Hospital, Sun Yat-Sen University
Locations
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The Third Affiliated Hospital of Sun Yat-sen University
Guangzhou, Guangdong, China
Countries
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Central Contacts
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Facility Contacts
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Other Identifiers
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XS228-Allo-SCI-CN2-P01
Identifier Type: -
Identifier Source: org_study_id
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