The Effect of GD-iExo-003 in Acute Ischemic Stroke (ExoCURE)

The Effect of Exosomes Derived From Human Induced Pluripotent Stem Cell (GD-iExo-003) in Acute Ischemic Stroke: an Exploratory Study.

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.

Study Overview

• Status: Completed
• Conditions: Acute Ischemic Stroke
• Intervention / Treatment: Drug: a placebo of exosomes derived from human induced pluripotent stem cell for injection; Drug: exosomes derived from human induced pluripotent stem cell for injection

Detailed Description

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
• Enrollment (Actual): 29
• Phase: Phase 2; Phase 1

Contacts and Locations

Study Locations

• China
  • Beijing, China, 100053
    • Xuanwu Hospital, Capital Medical University

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

• Clinical diagnosis of acute ischemic stroke
• Age 18-70 years, inclusion of both genders
• Modified Rankin Scale score before stroke of 0-1
• NIHSS score 6-20 at inclusion that did not change by ≥4 points from screening to baseline assessment.
• Time of stroke onset is known and treatment can be started between day 1 and 7 of onset.
• Confirmation of hemispheric cortical infarct with magnetic resonance imaging or computed tomography
• Subjects who received intravenous thrombolysis or underwent mechanical reperfusion are eligible if they meet all other eligibility criteria.
• Adequate hepatic and renal function: serum aspartate aminotransferase ≤2.5× upper limit of normal; serum alanine aminotransferase ≤2.5× upper limit of normal; blood urea nitrogen ≤1.25× upper limit of normal; serum creatinine ≤1.25× upper limit of normal
• Adequate cardiac function.
• Subjects or legal representative can sign the informed consent and must be willing and able to comply with all aspects of treatment and follow-up schedule.

Exclusion Criteria:

• Presence of intracranial hemorrhage on CT including hemorrhagic stroke, epidural hematoma, subdural hematoma, intraventricular hemorrhage, intraventricular hemorrhage, subarachnoid hemorrhage or hemorrhagic transformation, etc.
• Presence of a lacunar or a brainstem infarct as the etiology of current symptoms.
• Evidence of brain tumor or history of epilepsy or traumatic brain injury.
• Subjects with present malignant disease.
• Subjects with severe comorbidities including immunodeficiency or coagulation disorders.
• Subjects with Alzheimer's disease, Parkinson's disease or other degenerative neurological disease.
• Ongoing systemic infection, severe local infection or taking immunosuppressants.
• Subjects with positive hepatitis B surface antibody (HBsAg) and positive hepatitis B core antibody (HBcAb), or HBsAg-positive virus carriers, positive hepatitis C antibody, positive syphilis antibody or HIV
• Allergy to the study products.
• Documented allergies
• Participation in any clinical trial in the last 3 months
• Inability or unwillingness to comply with the study schedule
• Pregnancy, childbearing potential (unless it is certain that pregnancy is not possible), oe breast feeding
• Other serious medical or psychiatric illness that is not adequately controlled
• Other circumstances that the investigator considers inappropriate for participation in the trial.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Sequential Assignment
• Masking: Quadruple

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Exosomes group; Patients in this arm will be given exosomes derived from human induced pluripotent stem cell for injection once a day for 7 days.	Drug: exosomes derived from human induced pluripotent stem cell for injection; Exosomes derived from human induced pluripotent stem cell for injection (3.0ml, 1×10^11particles/ml).; Other Names: GD-iExo-003
Placebo Comparator: Exosomes placebo group; Patients in this arm will be given a placebo of exosomes derived from human induced pluripotent stem cell for injection once a day for 7 days.	Drug: a placebo of exosomes derived from human induced pluripotent stem cell for injection; Exosomes placebo, 3.0ml; Other Names: GD-iExo-003 placebo

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Incidence of severe adverse events	The proportion of patients who experienced severe adverse events.	90±7 days

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
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.	90±7 days
Functional outcome	The range of mRS scores by shift analysis.	90±7 days
NIHSS score change	The change of National Institutes of Health Stroke Scale (NIHSS) score of day 14 to baseline.	14±2 days
Quality of Life (EQ-5D-5L)	The value of EQ-5D-5L score.	90±7 days
Barthel Index (BI)	The value of BI	90±7 days
NIHSS score change	The change of National Institutes of Health Stroke Scale (NIHSS) score of day 7 to baseline.	90±7 days
MoCA	The value of MoCA	90±7 days

Other Outcome Measures

Outcome Measure	Measure Description	Time Frame
Blood marker changes from baseline to discharge	A number of blood markers will be examined including C-reactive protein, glial fibrillary acidic protein, IL-1β, IL-6, IL-8, IL-10, Tumor Necrosis Factor-alpha.	at discharge, an average of 14 days
Degree of cerebral edema changes from baseline to 48 hours	Degree of cerebral edema changes from baseline to 48 hours.	48 hours
Single-cell RNA sequencing of peripheral blood	Single-cell RNA sequencing of peripheral blood from baseline to day 7.	7±1 days
Infarct volume	The infarct volume is measured by CT or MRI at 14 days	14±2 days

Collaborators and Investigators

• Sponsor: Xuanwu Hospital, Beijing
• Collaborators: Guidon Pharmaceutics Ltd.
• Investigators: Principal Investigator: Junwei Hao, MD; PhD, Xuanwu Hospital, Beijing

Study record dates

Study Major Dates

• Study Start (Actual): June 5, 2024
• Primary Completion (Actual): May 6, 2026
• Study Completion (Actual): May 6, 2026

Study Registration Dates

• First Submitted: November 12, 2023
• First Submitted That Met QC Criteria: November 15, 2023
• First Posted (Actual): November 18, 2023

Study Record Updates

• Last Update Posted (Actual): August 24, 2026
• Last Update Submitted That Met QC Criteria: August 21, 2026
• Last Verified: August 1, 2026

More Information

Terms related to this study

• Keywords: exosome
• Additional Relevant MeSH Terms: Cerebrovascular Disorders; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Vascular Diseases; Cardiovascular Diseases; Stroke; Ischemic Stroke; Therapeutics; Drug Administration Routes; Drug Therapy; Injections
• Other Study ID Numbers: XMEC-2023-004

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: UNDECIDED
• IPD Plan Description: The individual participant data underlying the trial results will be made available upon reasonable request to the corresponding author.

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No