Acute Ischemic StrokePhase 1/2ExoCURENCT06138210
The Effect of GD-iExo-003 in Acute Ischemic Stroke
A Phase 1/2 study testing exosomes derived from human induced pluripotent stem cell for injection in people with Acute Ischemic Stroke, run by Xuanwu Hospital, Beijing at 1 site in China.
Trial summary
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. Read more about this study
- Who is it for?
- Ages 18 to 70 years
- Conditions the study is for
- Acute Ischemic Stroke
- What is being tested?
- exosomes derived from human induced pluripotent stem cell for injection
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 29 participants
- Where
- 1 siteChina
- Study dates
- 5 Jun 2024to 6 May 2026
- Phase
- Phase 1/2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 70 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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
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- 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.
Who cannot join
- 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.
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- 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.
Trial locations and contacts
1 site in 1 country.
About this study
Official title: 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.
Read the 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.
- Conditions
- Acute Ischemic Stroke
- Being tested
- exosomes derived from human induced pluripotent stem cell for injection, a placebo of exosomes derived from human induced pluripotent stem cell for injection
- Keywords
- exosome
- Medical subject headings
- Ischemic Stroke
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
exosomes derived from human induced pluripotent stem cell for injectionDrug
Also called GD-iExo-003
Exosomes derived from human induced pluripotent stem cell for injection (3.0ml, 1×10^11particles/ml).
a placebo of exosomes derived from human induced pluripotent stem cell for injectionDrug
Also called GD-iExo-003 placebo
Exosomes placebo, 3.0ml
| Study group | Role | What they receive |
|---|---|---|
| Exosomes group | Experimental | Patients in this arm will be given exosomes derived from human induced pluripotent stem cell for injection once a day for 7 days. Receives: Drug: exosomes derived from human induced pluripotent stem cell for injection |
| Exosomes placebo group | Placebo comparator | 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. Receives: Drug: a placebo of exosomes derived from human induced pluripotent stem cell for injection |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence of serious adverse events
The proportion of patients who experienced serious adverse events.
Measured: 90±7 days
Secondary outcomes (7)
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.
Measured: 90±7 days
Functional outcome
The range of mRS scores by shift analysis.
Measured: 90±7 days
NIHSS score change
The change of National Institutes of Health Stroke Scale (NIHSS) score of day 14 to baseline.
Measured: 14±2 days
NIHSS score change
The change of National Institutes of Health Stroke Scale (NIHSS) score of day 7 to baseline.
Measured: 90±7 days
Quality of Life (EQ-5D-5L)
The value of EQ-5D-5L score.
Measured: 90±7 days
Barthel Index (BI)
The value of BI
Measured: 90±7 days
MoCA
The value of MoCA
Measured: 90±7 days
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted18 Nov 2023
- Study start5 Jun 2024
- Primary completion6 May 2026
- Study completion6 May 2026
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Acute Ischemic StrokeStudies of the same condition, open and under way.
- More trials run by Xuanwu Hospital, BeijingThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Sequential
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 29 participants (actual)
Sponsor and investigators
- Lead sponsor
- Xuanwu Hospital, Beijing
- Sponsor type
- University, hospital or other
- Collaborators
- Guidon Pharmaceutics Ltd.
- Responsible party
- Principal investigator, Junwei Hao, MD, Xuanwu Hospital, Beijing
- Investigators
- Junwei Hao, MD; PhD, Principal investigator, Xuanwu Hospital, Beijing
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06138210
- Sponsor's protocol ID
- XMEC-2023-004
- Acronym
- ExoCURE
- Status last verified
- Aug 2026
- First submitted
- 12 Nov 2023
- Last updated
- 10 Sep 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Undecided: The individual participant data underlying the trial results will be made available upon reasonable request to the corresponding author.
Publications
Ma G, Zuo Y, Li N, Bian W, Mu J, Yao X, Feng G, Mo R, Meng S, Wu Y, Wu J, Chen X, Ma Q, Gao D, Zhong L, Zhang J, Hao J. Clinical safety and preliminary efficacy of allogeneic human induced pluripotent stem cell-derived exosomes in patients with acute ischaemic stroke: protocol of a phase I/II clinical trial in China. BMJ Open. 2026 Sep 16;16(9):e115350. doi: 10.1136/bmjopen-2025-115350.
PubMed 42749369
Study at a glance
From the registry record, last updated by the sponsor on 10 Sep 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- The Effect of Exosomes Derived From Human Induced Pluripotent Stem Cell (GD-iExo-003) in Acute Ischemic Stroke: an Exploratory Study.
- ClinicalTrials.gov ID
- NCT06138210
- Acronym / sponsor ID
- ExoCURE / XMEC-2023-004
- Conditions
- Acute Ischemic Stroke
- Keywords
- exosome
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 29 participants (actual)
- Lead sponsor
- Xuanwu Hospital, BeijingUniversity, hospital or other
- Collaborators
- Guidon Pharmaceutics Ltd.
- Study start
- 5 Jun 2024 (actual)
- Primary completion
- 6 May 2026 (actual)
- Study completion
- 6 May 2026 (actual)
- First posted
- 18 Nov 2023 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
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 design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Sequential
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Exosomes group | Experimental | Drug: exosomes derived from human induced pluripotent stem cell for injectionPatients in this arm will be given exosomes derived from human induced pluripotent stem cell for injection once a day for 7 days. |
| Exosomes placebo group | Placebo comparator | Drug: a placebo of exosomes derived from human induced pluripotent stem cell for injectionPatients 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. |
Who may qualify
- Ages
- 18 to 70 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (10 listed)
- 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.
Key exclusion criteria (15 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| exosomes derived from human induced pluripotent stem cell for injectionAlso: GD-iExo-003Exosomes derived from human induced pluripotent stem cell for injection (3.0ml, 1×10^11particles/ml). | Drug | Exosomes group |
| a placebo of exosomes derived from human induced pluripotent stem cell for injectionAlso: GD-iExo-003 placeboExosomes placebo, 3.0ml | Drug | Exosomes placebo group |
Primary outcome
Incidence of serious adverse events
Time frame: 90±7 days
The proportion of patients who experienced serious adverse events.
7 secondary outcomes
Favorable functional outcome
Time frame: 90±7 days
Functional outcome
Time frame: 90±7 days
NIHSS score change
Time frame: 14±2 days
NIHSS score change
Time frame: 90±7 days
Quality of Life (EQ-5D-5L)
Time frame: 90±7 days
Barthel Index (BI)
Time frame: 90±7 days
MoCA
Time frame: 90±7 days
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 10 Sep 2026.
- Derived from this study: PubMed 42749369Ma G, Zuo Y, Li N, Bian W, Mu J, Yao X, Feng G, Mo R, Meng S, Wu Y, Wu J, Chen X, Ma Q, Gao D, Zhong L, Zhang J, Hao J. Clinical safety and preliminary efficacy of allogeneic human induced pluripotent stem cell-derived exosomes in patients with acute ischaemic stroke: protocol of a phase I/II clinical trial in China. BMJ Open. 2026 Sep 16;16(9):e115350. doi: 10.1136/bmjopen-2025-115350.
- Individual participant dataSharing planned: Undecided. The individual participant data underlying the trial results will be made available upon reasonable request to the corresponding author.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Junwei Hao, MD; PhD principal investigator
Xuanwu Hospital, Beijing
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing exosomes derived from human induced pluripotent stem cell for injection in people with Acute Ischemic Stroke, run by Xuanwu Hospital, Beijing at 1 site in China.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 10 inclusion and 15 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 10 Sep 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in China. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Incidence of serious adverse events (time frame: 90±7 days). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT06138210, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 10 Sep 2026.