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
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
12
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.
The Third Affiliated Hospital of Sun Yat-sen University
Guangzhou, Guangdong, China
RECRUITINGThe incidence of adverse events (AEs) and serious adverse events (SAEs)
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.
Time frame: 28 days after administration in the SAD treatment and 28 days after the final (fourth) administration in the MAD treatment
DLT(Dose-limiting toxicity)
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) .
Time frame: 28 days after administration in the SAD treatment and 28 days after the final (fourth) administration in the MAD treatment
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 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.
Time frame: After the last participant in the MAD treatment of the Phase I trial completes the 28-day DLT observation period
Improvement in ASIA Impairment Scale (AIS) grade
To evaluate the Improvement in ASIA Impairment Scale (AIS) grade after the administration 0f XS228(ASIA Impairment Scale (AIS) grade)
Time frame: Improvement in ASIA Impairment Scale (AIS) grade from baseline at Day 29, Day 90, Day 180, Day 270, and Day 360 after the first dose administration.
Changes in American Spinal Injury Association (ASIA) Motor Score
Changes in motor scores assessed by the ASIA score scale (total score range from 0 to 100, higher values represent a better outcome)
Time frame: From baseline at Day 29, Day 90, Day 180, Day 270, and Day 360 after the first dose administration.
Changes in American Spinal Injury Association (ASIA) Sensory Score
Changes in sensory scores assessed by the ASIA score scale (total score range from 0 to 224, higher values represent a better outcome)
Time frame: From baseline at Day 29, Day 90, Day 180, Day 270, and Day 360 after the first dose administration.
Changes in Spinal Cord Independence Measure-III (SCIM-III)
SCIM-III(range: 0-100, with lower score indicating greater disability)
Time frame: From baseline at Day 29, Day 90, Day 180, Day 270, and Day 360 after the first dose administration.
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