Ischemic Stroke, AcutePhase 1/2NSAISNCT07143786
Human Induced Neural Stem Cell-derived Exosomes for Treating Acute Ischemic Stroke
A Phase 1/2 study testing iNSC-EV01 in people with Ischemic Stroke, Acute, run by Xuanwu Hospital, Beijing.
Trial summary
A phase I/IIa clinical trial investigating the safety and preliminary efficacy of intravenous administration of human induced neural stem cell-derived exosomes for acute ischemic stroke Read more about this study
- Who is it for?
- Ages 18 to 80 years
- Conditions the study is for
- Ischemic Stroke, Acute
- What is being tested?
- iNSC-EV01
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 38 participants
- Where
- No sites listed yet
- Study dates
- 1 Oct 2025to 31 Dec 2027
- Phase
- Phase 1/2
- Are there results?
- Not yet: the study is under way
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 80 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Aged 18-80 years (inclusive), regardless of gender;
- Clinically diagnosed with an anterior circulation ischemic stroke in the current episode, confirmed by head MRI/CT, and able to receive the investigational product within 1 week after symptom onset;
- National Institutes of Health Stroke Scale (NIHSS) score of 6-20 (inclusive) at randomization, with NIHSS item Ia score <2, and a change in NIHSS score from baseline to randomization of <4 points;
- Female subjects of childbearing potential, or male subjects with partners of childbearing potential, must have no plans for pregnancy during the study and voluntarily use effective contraception;
- Modified Rankin Scale (mRS) score of 0 or 1 before the onset of the current stroke symptoms, as self-reported or reported by family members.
- All subjects, or their legal guardians, must provide written informed consent after receiving full information about the study and voluntarily participate in this clinical trial.
Who cannot join
- Those with epilepsy, Alzheimer's disease, Parkinson's disease, severe depression, or other neurological disorders or psychiatric illnesses that the investigator deems would impair their ability to participate in the trial or affect the assessment of the study;
- Patients who have experienced hemorrhagic transformation after the current ischemic stroke and are deemed unsuitable for participation in the clinical trial by the investigator;
- Patients with malignant tumors, except for those with low-grade malignant tumors such as basal cell carcinoma, papillary thyroid carcinoma, and localized prostate cancer in situ, who have received radical treatment for more than five years;
- Patients with severe infections, including sepsis, septic shock, severe pneumonia (refer to the 2007 criteria for severe pneumonia in adults by the Infectious Diseases Society of America/American Thoracic Society for the diagnosis of severe pneumonia);
- Patients with respiratory failure, or those with current evidence of pulmonary embolism or suspected pulmonary embolism;
- Patients whose organ function meets any one or more of the following criteria:
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- Absolute Neutrophil Count (ANC) < 1.5 × 10⁹/L, Platelets (PLT) < 100 × 10⁹/L;
- Hemoglobin (Hb) < 90 g/L;
- Aspartate Aminotransferase (AST) > 2.5 × Upper Limit of Normal (ULN) and/or Alanine Aminotransferase (ALT) > 2.5 × ULN, Total Serum Bilirubin (TBIL) > 1.5 × ULN;
- Creatinine > 1.5 × ULN;
- For patients not receiving anticoagulant or antithrombotic therapy: International Normalized Ratio (INR) > 1.7 or Activated Partial Thromboplastin Time (APTT) > 1.25 × ULN; for patients receiving anticoagulant or antithrombotic therapy: INR > 3.0 or APTT > 1.5 × ULN;
- Patients with a history of or current severe cardiovascular diseases:
- Those with myocardial ischemia, myocardial infarction, or unstable angina pectoris graded above CTCAE (Common Terminology Criteria for Adverse Events) Grade II;
- Severe arrhythmias deemed clinically significant by the investigator;
- Cardiac insufficiency of NYHA (New York Heart Association) Class III-IV;
- Those with other acute severe life-threatening complications;
- Patients with poorly controlled hypertension (defined as persistent systolic blood pressure > 220 mmHg or diastolic blood pressure > 120 mmHg despite antihypertensive treatment);
- Patients with poorly controlled diabetes mellitus (defined as blood glucose remaining > 16.8 mmol/L despite treatment) or hypoglycemia (blood glucose < 2.8 mmol/L);
- Patients with a history of immunodeficiency, including: HIV-positive status, other acquired or congenital immunodeficiency diseases, idiopathic IgA deficiency, or those who have received systemic corticosteroid therapy (≥ 10 mg/day prednisone equivalent) or immunosuppressive drug therapy within 14 days prior to receiving the study drug, or who are expected to require such therapy during the trial;
- Patients positive for Hepatitis B surface antigen (HBsAg) or Hepatitis B core antibody (HBcAb) with positive HBV-DNA, positive Hepatitis C antibody (HCV), or positive Treponema pallidum antibody (TPAb/RPR);
- Patients who have participated in other drug clinical trials within 3 months prior to screening;
- Patients with known allergies to the study drug or any of its components (e.g., human serum albumin);
- Patients unable to undergo cranial CT/MRI examinations for any reason;
- Patients who have undergone major surgery, suffered severe trauma within 3 months prior to the first dose, or plan to undergo surgery that may affect neurological function assessment during the trial;
- Pregnant or lactating patients;
- Patients with other severe systemic diseases, or a history of any diseases or laboratory abnormalities that may confound study results, interfere with the subject's participation in study procedures, or are not in the subject's best interest to participate, and who are deemed unsuitable for enrollment by the investigator.
About this study
Official title: Human Induced Neural Stem Cell-derived Exosomes for Treating Acute Ischemic Stroke: an Exploratory Clinical Trial
A phase I/IIa clinical trial investigating the safety and preliminary efficacy of intravenous administration of human induced neural stem cell-derived exosomes for acute ischemic stroke
- Conditions
- Ischemic Stroke, Acute
- Being tested
- iNSC-EV01, Placebo
- Keywords
- induced neural stem cell, exosome, acute ischemic stroke, safety, efficacy
- Medical subject headings
- Ischemic Stroke
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
iNSC-EV01Drug
Intravenous induced neural stem cell-derived exosome
PlaceboDrug
iNSC-EV01 mimics
| Study group | Role | What they receive |
|---|---|---|
| Exosome group | Experimental | Acute ischemic stroke patients that receive intravenous iNSC-Exo (5.0×10^11 ~ 2.0×10^12 particles) administration Receives: Drug: iNSC-EV01 |
| Control group | Placebo comparator | Acute ischemic stroke patients that receive intravenous placebo administration Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Dose limiting toxicity
Measured: Within 90 days
mRS
modified Ranking Scale (mRS), ranging from 0 to 6, with higher scores indicating worse outcome, and a score of 6 representing death.
Measured: 90（±7）days
Secondary outcomes (7)
AEs and SAEs
Adverse events and severe adverse events
Measured: Within 90 days
mRS
modified Ranking Scale (mRS), which ranges from 0 to 6, with higher scores indicating worse outcome, and a score of 6 representing death.
Measured: 14（±1）days
Percentage of mRS 0-2
modified Ranking Scale (mRS), which ranges from 0 to 6, with higher scores indicating worse outcome, and a score of 6 representing death.
Measured: 14 (±1), 90 (±7) days
NIHSS score change compared to baseline
National Institutes of Health Stroke Scale (NIHSS), ranging from 0 to42, with higher score indicating more severe disabilities
Measured: 14 (±1), 90 (±7) days
Barthel Index change compared to baseline
Barthel Index ranges from 0 to 100, with higher score indicating better daily living abilities
Measured: 14 (±1), 90 (±7) days
EQ5D-5L change compared to baseline
EuroQol Five Dimensions Questionnaire (EQ5D) - Five levels, ranging from 5 to 25, with higher score indicating better outcome
Measured: 14 (±1), 90 (±7) days
Infarct volume change compared to baseline
Infarct volume (cm3) measured by MRI
Measured: 14 (±1), 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 posted27 Aug 2025
- Study start1 Oct 2025 (estimated)
- Primary completion31 Jul 2027 (estimated)
- Study completion31 Dec 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Ischemic Stroke, AcuteStudies of the same condition, open and under way.
- 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
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 38 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Xuanwu Hospital, Beijing
- Sponsor type
- University, hospital or other
- Collaborators
- Wiseheart Co.Ltd
- Responsible party
- Principal investigator, Junwei Hao, MD, Xuanwu Hospital, Beijing
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07143786
- Sponsor's protocol ID
- iNSC-Exo-AIS2025
- Acronym
- NSAIS
- Status last verified
- Aug 2025
- First submitted
- 7 Aug 2025
- Last updated
- 27 Aug 2025
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 27 Aug 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Human Induced Neural Stem Cell-derived Exosomes for Treating Acute Ischemic Stroke: an Exploratory Clinical Trial
- ClinicalTrials.gov ID
- NCT07143786
- Acronym / sponsor ID
- NSAIS / iNSC-Exo-AIS2025
- Conditions
- Ischemic Stroke, Acute
- Keywords
- induced neural stem cell, exosome, acute ischemic stroke, safety, efficacy
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Not yet recruiting
- Enrolment
- 38 participants (estimated)
- Lead sponsor
- Xuanwu Hospital, BeijingUniversity, hospital or other
- Collaborators
- Wiseheart Co.Ltd
- Study start
- 1 Oct 2025 (estimated)
- Primary completion
- 31 Jul 2027 (estimated)
- Study completion
- 31 Dec 2027 (estimated)
- First posted
- 27 Aug 2025 (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.
A phase I/IIa clinical trial investigating the safety and preliminary efficacy of intravenous administration of human induced neural stem cell-derived exosomes for acute ischemic stroke
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Exosome group | Experimental | Drug: iNSC-EV01Acute ischemic stroke patients that receive intravenous iNSC-Exo (5.0×10^11 ~ 2.0×10^12 particles) administration |
| Control group | Placebo comparator | Drug: PlaceboAcute ischemic stroke patients that receive intravenous placebo administration |
Who may qualify
- Ages
- 18 to 80 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (6 listed)
- Aged 18-80 years (inclusive), regardless of gender;
- Clinically diagnosed with an anterior circulation ischemic stroke in the current episode, confirmed by head MRI/CT, and able to receive the investigational product within 1 week after symptom onset;
- National Institutes of Health Stroke Scale (NIHSS) score of 6-20 (inclusive) at randomization, with NIHSS item Ia score <2, and a change in NIHSS score from baseline to randomization of <4 points;
- Female subjects of childbearing potential, or male subjects with partners of childbearing potential, must have no plans for pregnancy during the study and voluntarily use effective contraception;
- Modified Rankin Scale (mRS) score of 0 or 1 before the onset of the current stroke symptoms, as self-reported or reported by family members.
Key exclusion criteria (26 listed)
- Those with epilepsy, Alzheimer's disease, Parkinson's disease, severe depression, or other neurological disorders or psychiatric illnesses that the investigator deems would impair their ability to participate in the trial or affect the assessment of the study;
- Patients who have experienced hemorrhagic transformation after the current ischemic stroke and are deemed unsuitable for participation in the clinical trial by the investigator;
- Patients with malignant tumors, except for those with low-grade malignant tumors such as basal cell carcinoma, papillary thyroid carcinoma, and localized prostate cancer in situ, who have received radical treatment for more than five years;
- Patients with severe infections, including sepsis, septic shock, severe pneumonia (refer to the 2007 criteria for severe pneumonia in adults by the Infectious Diseases Society of America/American Thoracic Society for the diagnosis of severe pneumonia);
- Patients with respiratory failure, or those with current evidence of pulmonary embolism or suspected pulmonary embolism;
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 |
|---|---|---|
| iNSC-EV01Intravenous induced neural stem cell-derived exosome | Drug | Exosome group |
| PlaceboiNSC-EV01 mimics | Drug | Control group |
Primary outcome
Dose limiting toxicity
Time frame: Within 90 days
mRS
Time frame: 90（±7）days
modified Ranking Scale (mRS), ranging from 0 to 6, with higher scores indicating worse outcome, and a score of 6 representing death.
7 secondary outcomes
AEs and SAEs
Time frame: Within 90 days
mRS
Time frame: 14（±1）days
Percentage of mRS 0-2
Time frame: 14 (±1), 90 (±7) days
NIHSS score change compared to baseline
Time frame: 14 (±1), 90 (±7) days
Barthel Index change compared to baseline
Time frame: 14 (±1), 90 (±7) days
EQ5D-5L change compared to baseline
Time frame: 14 (±1), 90 (±7) days
Infarct volume change compared to baseline
Time frame: 14 (±1), 90 (±7) days
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 27 Aug 2025.
- Individual participant dataSharing planned: No
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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.
The record lists no contact. Use the official record to reach the sponsor.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing iNSC-EV01 in people with Ischemic Stroke, Acute, run by Xuanwu Hospital, Beijing.
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 6 inclusion and 26 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: opening soon, as of the sponsor's last update on 27 Aug 2025. Status can change between updates, so confirm with the study contact before making plans.
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?
This study is still under way. Results are not available in the source record yet.
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?
Dose limiting toxicity (time frame: Within 90 days). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 27 Aug 2025; AzTrials last refreshed its copy on 30 Oct 2026.