This is a multicenter, randomized, double-blinded, placebo-controlled, dose-escalation trial. The objective of this study is evaluating safety and preliminary efficacy of intravenous exosomes derived from human induced pluripotent stem cell (GD-iExo-003) in acute ischemic stroke.
This is a multicenter, randomized, double-blinded, placebo-controlled, dose-escalation trial. This study will consist of 2 parts, with part 1 being a dose-escalation study and part 2 being an expanded safety study based on part 1 findings. A traditional 3+3 dose escalation design will be implemented in part 1. Cohort 1: receive 2×10\^9 particles/kg; cohort 2: 4×10\^9 particles/kg and cohort 3: 8×10\^9 particles/kg. If no dose-limiting toxicities (DLTs) are observed for 2 weeks after administration of the first injection, a new cohort will be enrolled at the next planned dose level. If DLTs are observed in 1 participant in the cohort, another 3 participants will be treated in the same dose level. Dose escalation will be stopped until DLTs are observed in \>33% of the participants. In part 2, 20 subjects will be randomized in a 1:1 ratio \[exosome (n=10) or exosome placebo (n=10)\]. The dose level will be determined by Data Safety Monitoring Board based on part 1.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Enrollment
29
Exosomes derived from human induced pluripotent stem cell for injection (3.0ml, 1×10\^11particles/ml).
Exosomes placebo, 3.0ml
Xuanwu Hospital, Capital Medical University
Beijing, China
RECRUITINGIncidence of severe adverse events
The proportion of patients who experienced severe adverse events.
Time frame: 90±7 days
Favorable functional outcome
Rate of favorable functional outcome defined as a modified Rankin Scale (mRS, scores range from 0 to 6, with 0 to 2 indicating favorable outcome and 3 to 6 indicating unfavorable outcome including 6 as death) score of 0-2.
Time frame: 90±7 days
Functional outcome
The range of mRS scores by shift analysis.
Time frame: 90±7 days
NIHSS score change
The change of National Institutes of Health Stroke Scale (NIHSS) score of day 14 to baseline.
Time frame: 14±2 days
NIHSS score change
The change of National Institutes of Health Stroke Scale (NIHSS) score of day 7 to baseline.
Time frame: 90±7 days
Quality of Life (EQ-5D-5L)
The value of EQ-5D-5L score.
Time frame: 90±7 days
Barthel Index (BI)
The value of BI
Time frame: 90±7 days
MoCA
The value of MoCA
Time frame: 90±7 days
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