Spinal Cord InjurySafetyClinical TrialsEfficacyInduced Pluripotent Stem CellsHuman Motor Neuron ProgenitorTransplantation
Investigational drug(s) / intervention(s)
Allogeneic Human Induced Pluripotent Stem Cell (iPSC)-Derived Motor Neuron Progenitor Cells
Allogeneic Human Induced Pluripotent Stem Cell (iPSC)-Derived Motor Neuron Progenitor Cells: Description:
The Single Ascending Dose (SAD) and Muliple Ascending Dose (MAD) stages were built up in the study. XS228 in SAD and MAD following intrathecal injection through lumbar puncture in subacute spinal cord Injury participants.For SAD,the participants will single intrathecal injection with the dose level as 5×10\^7 cells 、1.5×10\^8 cells. For MAD, the participants will intrathecal injection of XS228 in Day 1, Day15, Day 29, Day 43 under the dose level of 5×10\^7 cells、1.5×10\^8 cells.Dose escalation followed a rule-based 3+3 design.
XS228 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 subacute spinal cord injury and represents a novel approach in regenerative medicine.
Study summary
This Phase I clinical trial is designed to evaluate the safety, tolerability of XS228 ( iPSC-Derived Motor Neuron Progenitor Cells) in patients with Subacute Spinal Cord Injury
Eligibility
Sex
ALL
Min age
18 Years
Max age
65 Years
Healthy volunteers
No
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.
Primary outcome measure(s)
The incidence of adverse events (AEs) and serious adverse events (SAEs) — 28 days after administration in the SAD treatment and 28 days after the final (fourth) administration in the MAD treatment To evaluate the safety and tolerability of XS228 in A single dose and the last dose of MAD treatment of Subacute Spinal Cord Injury through Adverse events (AE) related to XS228 ,incidence of SAE(serious adverse events).The severity of AEs observed during the trial will be graded according to the National Cancer Institute Common Terminology Criteria for Adverse Events (NCI-CTCAE) version 5.0.
DLT(Dose-limiting toxicity) — 28 days after administration in the SAD treatment and 28 days after the final (fourth) administration in the MAD treatment To evaluate the safety and tolerability of XS228 in A single dose and the last dose of MAD treatment of Subacute Spinal Cord Injury through DLT(Dose-limiting toxicity).A DLT is defined as any Grade 3 or higher adverse event (based on NCI-CTCAE Version 5.0) that occurs within 28 days following single-dose administration in the SAD treatment or the final dose in the MAD treatment , which is assessed as related to XS228, or any other significant adverse event as determined by the Safety Review Committee (SRC) .
RP2D(Recommended Phase 2 Dose) — After the last participant in the MAD treatment of the Phase I trial completes the 28-day DLT observation period After the last participant in the MAD treatment of the Phase I trial completes the 28-day DLT observation period following their final dose, the Safety Review Committee (SRC) and the sponsor will jointly determine the recommended dose for Phase II based on safety and preliminary efficacy data from Phase I.
Trial sites (1)
Facility
City
Region
Status
The Third Affiliated Hospital of Sun Yat-sen University
Guangzhou
Guangdong
Recruiting
More XellSmart Bio-Pharmaceutical (Suzhou) Co., Ltd. trials in China
This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
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