Evaluation of the Safety and Preliminary Efficacy of Intranasal Administration of Human Umbilical Cord Mesenchymal Stem Cell-Derived Small Extracellular Vesicles (hUC-MSC-sEV-001) in Patients for Ischemic Stroke
Based on adhering to the GCP principles of national clinical trial research and the standardized processes of CROs, this study aims to evaluate the safety and preliminary efficacy of hUC-MSC-sEV-001 in treating ischemic stroke by recruiting a small sample population. Additionally, the study will seek to determine an appropriate dosage, laying the foundation for subsequent research.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
9
Experimental drug: hUC-MSC-SEV-001 nasal drops derived from small extracellular vesicles of human umbilical cord mesenchymal stem cells Treatment plan: On the basis of conventional treatment, hUC-MSC-SEV-001 nasal drops will be administered: hUC-MSC-SEV-001 will be administered via nasal drip for a total of 4 times, with a dosage of 1 × 10 \* 11articles per dose. The patient will receive one dose on the day of enrollment, and another dose of hUC-MSC-SEV-001 will be administered on the 2nd, 3rd, and 4th days after enrollment.
253 Gongye Blvd Middle, Guangzhou, Guangdong, China 510280
Guangzhou, Guangdong, China
Dose-Limiting Toxicity (DLT) Related to hUC-MSC-sEV-001
Dose-Limiting Toxicity (DLT) Related to hUC-MSC-sEV-001 Includes: 1. Grade 3-4 severe adverse events occurring on day 14 (+-2), graded according to Common Terminology Criteria for Adverse Events (CTCAE) V5.0; 2. NIHSS score increase of ≥4 from baseline on day 14 (+-2); NIHSS score assessed by a professional rehabilitation physician; 3. Symptomatic intracranial hemorrhage occurring on day 14 (+-2); detected via CT scan and patient symptom presentation.
Time frame: Day 14 (+-2) post-enrollment
Serious adverse events related to the investigational drug within 90 (+-7) days
The occurrence of serious adverse events related to the investigational drug will be assessed according to CTCAE v5.0, based on monitoring of the patient's vital signs (pulse, blood pressure, respiration, and body temperature), as well as laboratory tests including complete blood count, urinalysis, liver and kidney function, glucose and lipid metabolism, coagulation profile, serum electrolytes, tumor markers, and electrocardiogram.
Time frame: At baseline, Day 7 (+-1), Day 14 (+-2), and Day 90 (+-7)post-enrollment
Distribution of Modified Rankin Scale (mRS) Scores within 90 (+-7) days
Measured using the Modified Rankin Scale (mRS) scale. The mRS ranges from 0 to 6, with higher numbers indicating greater disability.
Time frame: baseline, Day 7 (+-1), Day 14 (+-2), and Day 90 (+-7)post-enrollment
Barthel Index within 90 (±7) days
Measured using the Barthel Index. The total score of the Barthel Index ranges from 0 to 100 points. A higher Barthel Index score indicates a better (more positive) outcome, while a lower score indicates a poorer (more challenging) outcome.
Time frame: baseline, Day 7 (+-1), Day 14 (+-2), and Day 90 (+-7)post-enrollment
Five-level, five-dimensional EuroQol (EQ-5D-5L)
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Measured using the Five-level, five-dimensional EuroQol (EQ-5D-5L). It has two main scores with distinct ranges. The Index Score (Utility Score) comes from mapping responses on 5 health dimensions to a standardized scale. Its minimum is typically -0.594 to -0.101 (varies by region; negatives mean health worse than death for some), and maximum is 1.0 (full health, no limitations). The Visual Analog Scale (VAS) Score is a subjective self-rating on a 100mm vertical line, with 0 ("Worst imaginable health") as minimum and 100 ("Best imaginable health") as maximum. For both scores, higher means better HRQoL.
Time frame: baseline, Day 7 (+-1), Day 14 (+-2), and Day 90 (+-7)post-enrollment
Proportion of Modified Rankin Scale (mRS) Scores of 0-2 within 90 (+-7) days
Measured using the mRS scale
Time frame: baseline, Day 7 (+-1), Day 14 (+-2), and Day 90 (+-7)post-enrollment
All-cause mortality at 90 (+-7) days
Contact the patient or their family to confirm survival status
Time frame: Day 90 (+-7)post-enrollment
Changes in infarct volume at 14 (+-2) days compared to baseline
Using CTA、 CTP and MRI to measure changes in infarct volume in patients
Time frame: baseline, Day 14 (+-2) post-enrollment
Change from Baseline in National Institutes of Health Stroke Scale (NIHSS) Score
Measured using the National Institutes of Health Stroke Scale (NIHSS). Its total score range spans from a minimum of 0 points (indicating no detectable neurological deficits, suggesting minimal or no stroke-related impairment) to a maximum of 42 points (representing severe, widespread neurological dysfunction, often associated with profound stroke-related disability); regarding the scoring standard, a higher score means a worse outcome.
Time frame: baseline, Day 7 (+-1) and Day 14 (+-2)post-enrollment