iPSC-derived motor neuron progenitor cell therapy for subacute spinal cord injury
A Phase I Clinical Study Evaluating the Safety, Tolerability, and 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
This trial will test whether XS228 (iPSC-derived motor neuron progenitor cells) is safe and tolerable for adults 18–65 with recent (14–60 day) cervical to lumbar traumatic spinal cord injuries classified AIS A–C.
Quick facts
| Phase | Phase 1 |
|---|---|
| Study type | Interventional |
| Enrollment | 12 (estimated) |
| Ages | 18 Years to 65 Years |
| Sex | All |
| Sponsor | XellSmart Bio-Pharmaceutical (Suzhou) Co., Ltd. Industry-sponsored |
| Locations | 1 site (Guangzhou, Guangdong) |
| Trial ID | NCT06976229 on ClinicalTrials.gov |
What this trial studies
This Phase I interventional trial administers allogeneic iPSC-derived motor neuron progenitor cells (XS228) to adults in the subacute phase of traumatic spinal cord injury. The primary goal is to monitor safety and tolerability after cell administration, with regular neurological exams, imaging, and adverse event surveillance. Exploratory measures will look for early signals of efficacy such as motor or sensory changes. Participants are enrolled based on defined AIS grades and timing since injury and must be able to comply with study procedures.
Who should consider this trial
Good fit: Adults 18–65 with traumatic cervical (C4) to lumbar (L2) spinal cord injury occurring 14–60 days earlier, classified as AIS A, B, or C, who can provide informed consent and comply with visits are the intended candidates.
Not a fit: People with chronic injuries beyond 60 days, severe traumatic brain injury that prevents neurological assessment, unstable respiratory or circulatory status, or inability to use effective contraception are unlikely to benefit from participation.
Why it matters
Potential benefit: If successful, XS228 could help protect or replace damaged motor neurons and improve motor recovery after subacute spinal cord injury.
How similar studies have performed: Preclinical animal studies of iPSC-derived motor neuron and other stem-cell progenitors have shown functional improvements, but clinical experience with iPSC-derived motor neuron progenitors in humans remains very limited.
Eligibility criteria
Show full inclusion / exclusion criteria
Inclusion Criteria: Age: 18 to 65 years (inclusive), regardless of gender. 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: * Neurological Inability 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.
Where this trial is running
Guangzhou, Guangdong
- The Third Affiliated Hospital of Sun Yat-sen University — Guangzhou, Guangdong, China (Recruiting)
Study contacts
- Principal investigator: Limin Rong, prof and M.D — Third Affiliated Hospital, Sun Yat-Sen University
- Study coordinator: Michael LEE
- Email: CEO@xellsmart.com
- Phone: +86 21 64027719
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.