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

November 26, 2025 updated by: Junwei Hao, MD, Xuanwu Hospital, Beijing

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

Recruiting

Conditions

Intervention / Treatment

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 (Estimated)

29

Phase

  • Phase 2
  • Phase 1

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Contact Backup

Study Locations

  • China
      • Beijing, China, 100053
        • Recruiting
        • Xuanwu Hospital, Capital Medical University
        • Contact:

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

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

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

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

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
Time Frame: 90±7 days
The proportion of patients who experienced severe adverse events.
90±7 days

Secondary Outcome Measures

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

Other Outcome Measures

Outcome Measure
Measure Description
Time Frame
Change of infarct volume
Time Frame: 14±2 days
The infarct volume is measured by CT or MRI from the baseline to 14 days
14±2 days
Blood marker changes from baseline to discharge
Time Frame: at discharge, an average of 14 days
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
Time Frame: 48 hours
Degree of cerebral edema changes from baseline to 48 hours.
48 hours
Single-cell RNA sequencing of peripheral blood
Time Frame: 7±1 days
Single-cell RNA sequencing of peripheral blood from baseline to day 7.
7±1 days

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Xuanwu Hospital, Beijing

Collaborators

Guidon Pharmaceutics Ltd.

Investigators

  • Principal Investigator: Junwei Hao, MD; PhD, Xuanwu Hospital, Beijing

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

June 5, 2024

Primary Completion (Estimated)

March 30, 2026

Study Completion (Estimated)

March 30, 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 (Estimated)

December 4, 2025

Last Update Submitted That Met QC Criteria

November 26, 2025

Last Verified

November 1, 2025

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • XMEC-2023-004

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

IPD Plan Description

The study will not share individual participant data to other researchers.

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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