Acute Ischemic StrokePhase 1/2NCT07398612
Safety and Efficacy of Human Adipose-Derived Stem Cell Exosomes in Acute Ischemic Stroke
A Phase 1/2 study testing Human Adipose-Derived Stem Cell Exosomes in people with Acute Ischemic Stroke, run by Shanghai General Hospital, Shanghai Jiao Tong University School of Medicine.
Trial summary
This is a Phase I/II, randomized, double-blind, placebo-controlled, single/multiple ascending dose clinical study (Investigator-Initiated Trial, IIT) evaluating the safety and efficacy of Human Adipose-Derived Stem Cell Exosomes (ADSC-exo, STX11102 Nasal Spray) in treating acute ischemic stroke (AIS). Read more about this study
- Who is it for?
- Ages 18 to 80 years
- Conditions the study is for
- Acute Ischemic Stroke
- What is being tested?
- Human Adipose-Derived Stem Cell Exosomes
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 60 participants
- Where
- No sites listed yet
- Study dates
- 31 Jan 2026to 31 Aug 2028
- 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
- Age 18-80 years, male or female
- Patients with acute ischemic stroke within 72 hours of symptom onset
- Internal carotid artery system stroke
- For the single-dose study: mild stroke patients (NIHSS score 1-4, inclusive of 1 and 4)
- For the multiple-dose study: moderate stroke patients (NIHSS score 5-12, inclusive of 5 and 12)
- Pre-stroke mRS score ≤ 1
- Subjects or their guardians voluntarily sign the informed consent form
Who cannot join
- Moderate or severe stroke (NIHSS score > 12).
- Lacunar infarction, brainstem or cerebellar infarction (confirmed by DWI-MRI).
- Requirement for endovascular interventional treatment for the current episode.
- Intracranial hemorrhagic diseases (including parenchymal, intraventricular, subarachnoid hemorrhage, etc.).
- Patients with malignant tumors.
- Patients with severe traumatic brain injury.
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- Patients with primary or secondary immunodeficiency diseases or requiring immunosuppressant medication.
- Serum alanine aminotransferase (ALT) or aspartate aminotransferase (AST) exceeding 3 times the upper limit of normal.
- Chronic kidney disease or current serum creatinine exceeding 1.5 times the upper limit of normal or estimated glomerular filtration rate (eGFR) < 60 mL/min/1.73m².
- Presence of severe infection or fever; patients with severe respiratory diseases.
- Positive for hepatitis B virus surface antigen (HBsAg); or positive for hepatitis B core antibody (HBcAb) with positive HBV-DNA; or positive for hepatitis C virus antibody (HCVAb), Treponema pallidum antibody (TPAb/RPR), or human immunodeficiency virus antibody (HIV).
- Patients who are pregnant or lactating at screening, or wish to become pregnant during the study period. Patients allergic to the product
- Patients allergic to the product or with severe allergic constitution.
- Patients deemed unsuitable for participation by the investigator, or for whom participation may pose a greater risk.
- Patients who have participated in another clinical trial within the past 3 months.
- Patients with prior use of exosomes.
Trial locations and contacts
No sites are listed yet. Contact the study team or a site near you to ask about taking part.
About this study
Official title: Clinical Study on the Safety and Efficacy of Human Adipose-Derived Stem Cell Exosomes in the Treatment of Acute Ischemic Stroke
This is a Phase I/II, randomized, double-blind, placebo-controlled, single/multiple ascending dose clinical study (Investigator-Initiated Trial, IIT) evaluating the safety and efficacy of Human Adipose-Derived Stem Cell Exosomes (ADSC-exo, STX11102 Nasal Spray) in treating acute ischemic stroke (AIS). The study consists of two sequential parts: a Single-Ascending Dose (SAD) study and a Multiple-Ascending Dose (MAD) study.
The SAD part will enroll 12 subjects with mild stroke (NIHSS 1-4). They will be sequentially enrolled into three dose cohorts (4 subjects each: 2×10⁹, 4×10⁹, and 8×10⁹ particles/mL) to receive a single nasal spray dose alongside standard care, with safety monitoring for 14 days. Dose escalation is contingent upon the safety review of the preceding cohort.
Upon confirming safety, the study proceeds to the MAD part, which will enroll 48 subjects with moderate stroke (NIHSS 5-12). They will be randomized into two dose groups (Low and High Dose), each containing an active treatment arm and a placebo arm (saline) in a 2:1 ratio (16 active:8 placebo per group). Subjects will self-administer the nasal spray daily for 14 days, with follow-up until Day 90. The primary objective is to evaluate safety, with secondary objectives assessing efficacy via neurological function scales (NIHSS, mRS, BI) and infarct volume change on MRI.
Read the detailed description
This clinical trial is designed to investigate the novel therapeutic agent Human Adipose-Derived Stem Cell Exosomes (ADSC-exo, STX11102) delivered via nasal spray for patients with acute ischemic stroke (AIS). The rationale is based on promising preclinical data demonstrating that intranasally administered ADSC-exo can cross the blood-brain barrier, modulate neuroinflammation, reduce infarct volume, and promote functional recovery in animal stroke models.
Study Design and Phases:
The trial employs a two-part, early-phase exploratory design integrating dose-finding and preliminary efficacy assessment.
Part 1: Single-Ascending Dose (SAD) Study: This is an open-label, dose-escalation phase to assess initial safety and tolerability. Twelve subjects with mild AIS (NIHSS 1-4, onset within 72 hours) will be enrolled at a single center. Three dose levels will be tested sequentially: Dose Cohort 1 (2×10⁹ particles/mL), Cohort 2 (4×10⁹ particles/mL), and Cohort 3 (8×10⁹ particles/mL), with 4 subjects per cohort. All subjects will receive a single dose of ADSC-exo nasal spray plus standard care. Escalation to the next higher dose cohort requires completion of the 14-day safety observation for all subjects in the current cohort and a formal safety review by the Principal Investigator (PI) and the sponsor. Escalation is permitted only if the number of subjects experiencing Grade ≥3 adverse events (AEs) considered related to the study drug is ≤50% (i.e., ≤2 subjects) within the completed cohort.
Part 2: Multiple-Ascending Dose (MAD) Study: This is a randomized, double-blind, placebo-controlled, dose-expansion phase to further evaluate safety and explore efficacy. Forty-eight subjects with moderate AIS (NIHSS 5-12, onset within 72 hours) will be enrolled across approximately five centers. Entry into this part requires a favorable safety review of all data from the SAD part. Subjects will be randomized into two sequential dose groups (Low Dose and High Dose). The specific doses (X and Y ×10⁹ particles/mL) for the MAD part will be determined based on SAD results. Each dose group consists of 24 subjects randomized in a 2:1 ratio to receive either ADSC-exo nasal spray (n=16) or matching placebo (saline, n=8) daily for 14 days, in addition to standard care. The Low Dose group must complete enrollment and 14-day safety follow-up before the High Dose group begins enrollment, applying the same safety criteria (≤50% of subjects with related Grade ≥3 AEs) for progression.
Study Population: Key inclusion criteria: age 18-80, anterior circulation ischemic stroke, pre-stroke mRS ≤1. Key exclusion criteria: severe stroke (NIHSS >12), lacunar/brainstem/cerebellar infarct, need for endovascular intervention, intracranial hemorrhage, malignancy, immunodeficiency, significant hepatic/renal impairment, active severe infection, and positive serology for HIV, HBV (with positive DNA), HCV, or syphilis.
Endpoints:
Primary Safety Endpoints: Incidence, severity, and causality of Treatment-Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs); changes in nasal mucosa, pulmonary function, and immunoglobulins.
Secondary Efficacy Endpoints:
SAD: Change from baseline in NIHSS, mRS, and Barthel Index (BI) at Day 14.
MAD: Change in NIHSS at Day 14; change in mRS and BI at Days 7, 14, 30, and 90; change in cerebral infarct lesion volume on MRI at Day 90.
Exploratory Endpoints (MAD): Changes in inflammatory cytokines (IL-2, IL-6, IL-12, IL-1β, TNF-α, IFN-γ).
Study Procedures: The schedule includes a 7-day screening period, treatment period (1 day for SAD, 14 days for MAD), and a follow-up period (14 days for SAD, 76 days for MAD). Assessments include neurological scales (NIHSS, mRS, BI), laboratory tests, vital signs, ECGs, MRI, and pulmonary function tests.
Statistical Analysis: This is an exploratory study with a sample size based on clinical practice. Safety will be analyzed in the Safety Set (SS). Efficacy will be analyzed descriptively for the SAD part. For the MAD part, efficacy will be analyzed in the Full Analysis Set (FAS) and Intent-to-Treat (ITT) set using appropriate statistical models (e.g., ANCOVA for continuous endpoints, Cochran-Mantel-Haenszel test for
- Conditions
- Acute Ischemic Stroke
- Being tested
- Human Adipose-Derived Stem Cell Exosomes, Placebo
- Keywords
- acute ischemic stroke, human adipose-derived stem cell, exosome, NIHSS, mRS
- Medical subject headings
- Ischemic Stroke
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Human Adipose-Derived Stem Cell ExosomesBiological
This intervention involves the use of allogeneic Human Adipose-Derived Stem Cell Exosomes (ADSC-exo), provided as a sterile nasal spray (STX11102).
PlaceboOther
This intervention serves as the placebo control. It is a sterile 0.9% sodium chloride (normal saline) solution formulated as a nasal spray
| Study group | Role | What they receive |
|---|---|---|
| Single Ascending Dose Study Cohort 1 | Experimental | This is an open-label, single-dose cohort in the dose-escalation phase (Part 1). Four (4) subjects with acute ischemic stroke will receive a single dose of Human Adipose-Derived Stem Cell Exosomes (ADSC-exo) Nasal Spray at a concentration of 2×10^9 particles per mL (total volume 1 mL), administered intranasally once. Receives: Biological: Human Adipose-Derived Stem Cell Exosomes |
| Single Ascending Dose Study Cohort 2 | Experimental | This is an open-label, single-dose cohort in the dose-escalation phase (Part 1). Four (4) subjects with acute ischemic stroke will receive a single dose of Human Adipose-Derived Stem Cell Exosomes (ADSC-exo) Nasal Spray at a concentration of 4×10^9 particles per mL (total volume 1 mL), administered intranasally once. Receives: Biological: Human Adipose-Derived Stem Cell Exosomes |
| Single Ascending Dose Study Cohort 3 | Experimental | This is an open-label, single-dose cohort in the dose-escalation phase (Part 1). Four (4) subjects with acute ischemic stroke will receive a single dose of Human Adipose-Derived Stem Cell Exosomes (ADSC-exo) Nasal Spray at a concentration of 8×10^9 particles per mL (total volume 1 mL), administered intranasally once. Receives: Biological: Human Adipose-Derived Stem Cell Exosomes |
| Multiple Dose Study Low-Dose ADSC-exo (X) | Experimental | This is a double-blind, multiple-dose treatment arm in the dose-expansion phase (Part 2). Sixteen (16) subjects with acute ischemic stroke will receive Human Adipose-Derived Stem Cell Exosomes (ADSC-exo) Nasal Spray at a low concentration (designated as X ×10^9 particles per mL, to be determined based on SAD results), administered intranasally once daily (QD) for 14 consecutive days. This is administered in addition to standard of care (SOC). This arm is compared to a placebo control arm within the same low-dose group. Receives: Biological: Human Adipose-Derived Stem Cell Exosomes |
| Multiple Dose Study Low-Dose Placebo | Placebo comparator | This is a double-blind, multiple-dose control arm in the dose-expansion phase (Part 2). Eight (8) subjects with acute ischemic stroke will receive a matching Placebo Nasal Spray (0.9% physiological saline), administered intranasally once daily (QD) for 14 consecutive days. This is administered in addition to standard of care (SOC). This arm serves as the comparator for the active low-dose ADSC-exo arm. Receives: Other: Placebo |
| Multiple Dose Study High-Dose ADSC-exo (Y) | Experimental | This is a double-blind, multiple-dose treatment arm in the dose-expansion phase (Part 2). Sixteen (16) subjects with acute ischemic stroke will receive Human Adipose-Derived Stem Cell Exosomes (ADSC-exo) Nasal Spray at a high concentration (designated as Y ×10^9 particles per mL, to be determined based on SAD results), administered intranasally once daily (QD) for 14 consecutive days. This is administered in addition to standard of care (SOC). This arm is compared to a placebo control arm within the same high-dose group. Enrollment into this high-dose group is contingent upon a safety review of the low-dose group. Receives: Biological: Human Adipose-Derived Stem Cell Exosomes |
| Multiple Dose Study High-Dose Placebo | Placebo comparator | This is a double-blind, multiple-dose control arm in the dose-expansion phase (Part 2). Eight (8) subjects with acute ischemic stroke will receive a matching Placebo Nasal Spray (0.9% physiological saline), administered intranasally once daily (QD) for 14 consecutive days. This is administered in addition to standard of care (SOC). This arm serves as the comparator for the active high-dose ADSC-exo arm. Receives: Other: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Safety and Tolerability of ADSC-exo Nasal Spray in Acute Ischemic Stroke.
Treatment-Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs): The incidence, severity (graded per CTCAE v5.0), and the investigator-assessed causality (relationship to the study drug) of all AEs and SAEs occurring during the treatment and follow-up period.
Measured: For Single-Dose Study: From first dose on Day 1 through the end of safety follow-up at Day 14. For Multiple-Dose Study: From first dose on Day 1 through the end of safety follow-up, which extends up to 14 days after the last dose on Day 14.
Safety and Tolerability of ADSC-exo Nasal Spray in Acute Ischemic Stroke.
Specific Safety Assessments:
Nasal Mucosa: Changes from baseline based on physical examination of the nasal cavity.
Measured: For Single-Dose Study: From first dose on Day 1 through the end of safety follow-up at Day 14. For Multiple-Dose Study: From first dose on Day 1 through the end of safety follow-up, which extends up to 14 days after the last dose on Day 14.
Safety and Tolerability of ADSC-exo Nasal Spray in Acute Ischemic Stroke.
Specific Safety Assessments: pulmonary function: Changes in FVC versus baseline
Measured: For Single-Dose Study: From first dose on Day 1 through the end of safety follow-up at Day 14. For Multiple-Dose Study: From first dose on Day 1 through the end of safety follow-up, which extends up to 14 days after the last dose on Day 14.
Safety and Tolerability of ADSC-exo Nasal Spray in Acute Ischemic Stroke
Specific Safety Assessments: pulmonary function: Changes in FEV versus baseline
Measured: For Single-Dose Study: From first dose on Day 1 through the end of safety follow-up at Day 14. For Multiple-Dose Study: From first dose on Day 1 through the end of safety follow-up, which extends up to 14 days after the last dose on Day 14.
Safety and Tolerability of ADSC-exo Nasal Spray in Acute Ischemic Stroke
Specific Safety Assessments: pulmonary function: Changes in PEF versus baseline
Measured: For Single-Dose Study: From first dose on Day 1 through the end of safety follow-up at Day 14. For Multiple-Dose Study: From first dose on Day 1 through the end of safety follow-up, which extends up to 14 days after the last dose on Day 14.
Safety and Tolerability of ADSC-exo Nasal Spray in Acute Ischemic Stroke.
Immunoglobulins: Changes from baseline in serum levels of IgA.
Measured: For Single-Dose Study: From first dose on Day 1 through the end of safety follow-up at Day 14. For Multiple-Dose Study: From first dose on Day 1 through the end of safety follow-up, which extends up to 14 days after the last dose on Day 14.
Safety and Tolerability of ADSC-exo Nasal Spray in Acute Ischemic Stroke.
Immunoglobulins: Changes from baseline in serum levels of IgE
Measured: For Single-Dose Study: From first dose on Day 1 through the end of safety follow-up at Day 14. For Multiple-Dose Study: From first dose on Day 1 through the end of safety follow-up, which extends up to 14 days after the last dose on Day 14.
Safety and Tolerability of ADSC-exo Nasal Spray in Acute Ischemic Stroke
Immunoglobulins: Changes from baseline in serum levels of IgM.
Measured: For Single-Dose Study: From first dose on Day 1 through the end of safety follow-up at Day 14. For Multiple-Dose Study: From first dose on Day 1 through the end of safety follow-up, which extends up to 14 days after the last dose on Day 14.
Safety and Tolerability of ADSC-exo Nasal Spray in Acute Ischemic Stroke
Immunoglobulins: Changes from baseline in serum levels of IgG.
Measured: For Single-Dose Study: From first dose on Day 1 through the end of safety follow-up at Day 14. For Multiple-Dose Study: From first dose on Day 1 through the end of safety follow-up, which extends up to 14 days after the last dose on Day 14.
Secondary outcomes (6)
Change in Neurological Function (For Single-Dose Study)
This outcome measures the effect of a single dose of ADSC-exo on neurological recovery in patients with mild acute ischemic stroke (NIHSS 1-4). The change from baseline will be assessed using three validated clinical scales at Day 14 post-dose: the National Institutes of Health Stroke Scale (NIHSS) for stroke severity.The scoring range is 0-42 points, with higher scores indicating more severe nerve damage.
Measured: Baseline (Day 1, pre-dose) to Day 14 post-dose.
Change in NIHSS Score after 14-Day Treatment (For Multiple-Dose Study)
This outcome measures the change in stroke severity after 14 days of multiple-dose treatment with ADSC-exo in patients with moderate acute ischemic stroke (NIHSS 5-12). The National Institutes of Health Stroke Scale (NIHSS) score will be compared from baseline (Day 1) to the end of the 14-day treatment period (Day 14). A decrease in NIHSS score indicates an improvement in neurological deficit.The scoring range is 0-42 points, with higher scores indicating more severe nerve damage.
Measured: Baseline (Day 1, pre-dose) to Day 14 (end of treatment).
Change in Disability & Daily Function (For Multiple-Dose Study)
This outcome measures the sustained effect of multiple-dose ADSC-exo treatment on global disability and daily living function over time. The change from baseline will be assessed using the modified Rankin Scale (mRS) for disability and the Barthel Index (BI) for activities of daily living at multiple timepoints: Day 7, Day 14, Day 30, and Day 90. Lower mRS scores(The scoring range is 0-6 points) and higher BI scores(The scoring range is 0-100 points) indicate better functional outcomes.
Measured: Baseline (Day 1, pre-dose) to Day 7, Day 14, Day 30, and Day 90.
Change in Cerebral Infarct Volume on MRI (For Multiple-Dose Study)
This outcome is an imaging-based measure of treatment efficacy. It assesses the change in the volume of the cerebral infarct lesion from baseline (pre-treatment) to 90 days after the start of treatment (Day 90) using Magnetic Resonance Imaging (MRI). A reduction in infarct volume is hypothesized to correlate with neurological recovery and is a key structural endpoint.
Measured: Baseline MRI (within screening period, Day -7 to -1) to Day 90 MRI.
Change in Neurological Function (For Single-Dose Study)
This outcome measures the effect of a single dose of ADSC-exo on neurological recovery in patients with mild acute ischemic stroke (NIHSS 1-4). The change from baseline will be assessed using the validated clinical scales at Day 14 post-dose: the modified Rankin Scale (mRS) for global disability.The scoring range is 0-6 points, with higher scores indicating more severe nerve damage.
Measured: Baseline (Day 1, pre-dose) to Day 14 post-dose.
Change in Neurological Function (For Single-Dose Study)
This outcome measures the effect of a single dose of ADSC-exo on neurological recovery in patients with mild acute ischemic stroke (NIHSS 1-4). The change from baseline will be assessed using the validated clinical scales at Day 14 post-dose:the Barthel Index (BI) for activities of daily living. The scoring range is 0-100 points, with higher BI scores indicate better functional outcomes.
Measured: Baseline (Day 1, pre-dose) to Day 14 post-dose.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted10 Feb 2026
- Study start31 Jan 2026 (estimated)
- Primary completion31 May 2028 (estimated)
- Study completion31 Aug 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (30)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 Shanghai General Hospital, Shanghai Jiao Tong University School of MedicineThe 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
- Parallel groups
- Model description
- This is an interventional clinical trial model that sequentially combines two distinct designs within a single protocol: An open-label, sequential cohort, single-ascending dose (SAD) design for initial dose-finding and safety profiling. It uses a modified "3+3" type logic (4 subjects per cohort) with predefined safety stopping rules to guide dose escalation between cohorts. A randomized, double-blind, parallel-group, multiple-ascending dose (MAD) design with placebo control for preliminary efficacy evaluation and further safety assessment. It features two sequentially enrolled dose cohorts (low and high), each with an internal 2:1 randomization (active:placebo). Progression from the low-dose to the high-dose cohort is contingent upon a safety review, making it a sequential cohort expansion within the randomized phase. This hybrid model is typical for early-phase trials of novel biological therapies, aiming to efficiently establish a safe dose range (SAD) before exploring preliminary
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 60 participants (estimated)
Sponsor and investigators
- Sponsor type
- University, hospital or other
- Collaborators
- Xinhua Hospital, Shanghai Jiao Tong University School of Medicine, Shanghai 10th People's Hospital, First Affiliated Hospital of the Chinese People's Liberation Army Naval Medical University, Shanghai Jiao Tong University Affiliated Sixth People's Hospital
- Responsible party
- Principal investigator, Yuncheng Wu, Shanghai General Hospital, Shanghai Jiao Tong University School of Medicine
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07398612
- Sponsor's protocol ID
- IIT-ATMP-2025011
- Status last verified
- Dec 2025
- First submitted
- 30 Dec 2025
- Last updated
- 10 Feb 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Yes: This study will share de-identified individual participant data (IPD) that underlies the results reported in the primary and secondary outcome publications. This will include baseline characteristics (e.g., age, sex, NIHSS score at entry, pre-stroke mRS), treatment assignment (dose group), and outcome data for all primary and secondary endpoints. Specifically, shared data will encompass safety endpoints (all recorded Treatment-Emergent Adverse Events, Serious Adverse Events, and changes in nasal mucosa, pulmonary function, and immunoglobulins) and efficacy endpoints (serial assessments of NIHSS, mRS, Barthel Index scores, and cerebral infarct volume change on MRI at Day 90 for the MAD part). All shared data will be fully anonymized to protect participant confidentiality.
Publications
Xie X, Song Q, Dai C, Cui S, Tang R, Li S, Chang J, Li P, Wang J, Li J, Gao C, Chen H, Chen S, Ren R, Gao X, Wang G. Clinical safety and efficacy of allogenic human adipose mesenchymal stromal cells-derived exosomes in patients with mild to moderate Alzheimer's disease: a phase I/II clinical trial. Gen Psychiatr. 2023 Oct 11;36(5):e101143. doi: 10.1136/gpsych-2023-101143. eCollection 2023.
PubMed 37859748Li Y, Tang Y, Yang GY. Therapeutic application of exosomes in ischaemic stroke. Stroke Vasc Neurol. 2021 Sep;6(3):483-495. doi: 10.1136/svn-2020-000419. Epub 2021 Jan 11.
PubMed 33431513Sonoda T, Matsuzaki J, Yamamoto Y, Sakurai T, Aoki Y, Takizawa S, Niida S, Ochiya T. Serum MicroRNA-Based Risk Prediction for Stroke. Stroke. 2019 Jun;50(6):1510-1518. doi: 10.1161/STROKEAHA.118.023648. Epub 2019 Apr 15.
PubMed 31136284Dehghani L, Khojasteh A, Soleimani M, Oraee-Yazdani S, Keshel SH, Saadatnia M, Saboori M, Zali A, Hashemi SM, Soleimani R. Safety of Intraparenchymal Injection of Allogenic Placenta Mesenchymal Stem Cells Derived Exosome in Patients Undergoing Decompressive Craniectomy Following Malignant Middle Cerebral Artery Infarct, A Pilot Randomized Clinical Trial. Int J Prev Med. 2022 Jan 19;13:7. doi: 10.4103/ijpvm.ijpvm_441_21. eCollection 2022.
PubMed 35281985Yang Y, Cai Y, Zhang Y, Liu J, Xu Z. Exosomes Secreted by Adipose-Derived Stem Cells Contribute to Angiogenesis of Brain Microvascular Endothelial Cells Following Oxygen-Glucose Deprivation In Vitro Through MicroRNA-181b/TRPM7 Axis. J Mol Neurosci. 2018 May;65(1):74-83. doi: 10.1007/s12031-018-1071-9. Epub 2018 Apr 29.
PubMed 29705934Yang J, Zhang X, Chen X, Wang L, Yang G. Exosome Mediated Delivery of miR-124 Promotes Neurogenesis after Ischemia. Mol Ther Nucleic Acids. 2017 Jun 16;7:278-287. doi: 10.1016/j.omtn.2017.04.010. Epub 2017 Apr 13.
PubMed 28624203Xin H, Katakowski M, Wang F, Qian JY, Liu XS, Ali MM, Buller B, Zhang ZG, Chopp M. MicroRNA cluster miR-17-92 Cluster in Exosomes Enhance Neuroplasticity and Functional Recovery After Stroke in Rats. Stroke. 2017 Mar;48(3):747-753. doi: 10.1161/STROKEAHA.116.015204.
PubMed 28232590Altmann P, Mildner M, Haider T, Traxler D, Beer L, Ristl R, Golabi B, Gabriel C, Leutmezer F, Ankersmit HJ. Secretomes of apoptotic mononuclear cells ameliorate neurological damage in rats with focal ischemia. F1000Res. 2014 Jun 19;3:131. doi: 10.12688/f1000research.4219.2. eCollection 2014.
PubMed 25383184Hayon Y, Dashevsky O, Shai E, Brill A, Varon D, Leker RR. Platelet microparticles induce angiogenesis and neurogenesis after cerebral ischemia. Curr Neurovasc Res. 2012 Aug;9(3):185-92. doi: 10.2174/156720212801619018.
PubMed 22621230Chen KH, Chen CH, Wallace CG, Yuen CM, Kao GS, Chen YL, Shao PL, Chen YL, Chai HT, Lin KC, Liu CF, Chang HW, Lee MS, Yip HK. Intravenous administration of xenogenic adipose-derived mesenchymal stem cells (ADMSC) and ADMSC-derived exosomes markedly reduced brain infarct volume and preserved neurological function in rat after acute ischemic stroke. Oncotarget. 2016 Nov 15;7(46):74537-74556. doi: 10.18632/oncotarget.12902.
PubMed 27793019Doeppner TR, Herz J, Gorgens A, Schlechter J, Ludwig AK, Radtke S, de Miroschedji K, Horn PA, Giebel B, Hermann DM. Extracellular Vesicles Improve Post-Stroke Neuroregeneration and Prevent Postischemic Immunosuppression. Stem Cells Transl Med. 2015 Oct;4(10):1131-43. doi: 10.5966/sctm.2015-0078. Epub 2015 Sep 3.
PubMed 26339036Zhang Y, Chopp M, Liu XS, Katakowski M, Wang X, Tian X, Wu D, Zhang ZG. Exosomes Derived from Mesenchymal Stromal Cells Promote Axonal Growth of Cortical Neurons. Mol Neurobiol. 2017 May;54(4):2659-2673. doi: 10.1007/s12035-016-9851-0. Epub 2016 Mar 19.
PubMed 26993303Stonesifer C, Corey S, Ghanekar S, Diamandis Z, Acosta SA, Borlongan CV. Stem cell therapy for abrogating stroke-induced neuroinflammation and relevant secondary cell death mechanisms. Prog Neurobiol. 2017 Nov;158:94-131. doi: 10.1016/j.pneurobio.2017.07.004. Epub 2017 Jul 23.
PubMed 28743464Parekkadan B, Milwid JM. Mesenchymal stem cells as therapeutics. Annu Rev Biomed Eng. 2010 Aug 15;12:87-117. doi: 10.1146/annurev-bioeng-070909-105309.
PubMed 20415588Chen G, Luo X, Qadri MY, Berta T, Ji RR. Sex-Dependent Glial Signaling in Pathological Pain: Distinct Roles of Spinal Microglia and Astrocytes. Neurosci Bull. 2018 Feb;34(1):98-108. doi: 10.1007/s12264-017-0145-y. Epub 2017 Jun 5.
PubMed 28585113
Study at a glance
From the registry record, last updated by the sponsor on 10 Feb 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Clinical Study on the Safety and Efficacy of Human Adipose-Derived Stem Cell Exosomes in the Treatment of Acute Ischemic Stroke
- ClinicalTrials.gov ID
- NCT07398612
- Acronym / sponsor ID
- IIT-ATMP-2025011
- Conditions
- Acute Ischemic Stroke
- Keywords
- acute ischemic stroke, human adipose-derived stem cell, exosome, NIHSS, mRS
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Not yet recruiting
- Enrolment
- 60 participants (estimated)
- Lead sponsor
- Shanghai General Hospital, Shanghai Jiao Tong University School of MedicineUniversity, hospital or other
- Collaborators
- Xinhua Hospital, Shanghai Jiao Tong University School of Medicine, Shanghai 10th People's Hospital, First Affiliated Hospital of the Chinese People's Liberation Army Naval Medical University, Shanghai Jiao Tong University Affiliated Sixth People's Hospital
- Study start
- 31 Jan 2026 (estimated)
- Primary completion
- 31 May 2028 (estimated)
- Study completion
- 31 Aug 2028 (estimated)
- First posted
- 10 Feb 2026 (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 Phase I/II, randomized, double-blind, placebo-controlled, single/multiple ascending dose clinical study (Investigator-Initiated Trial, IIT) evaluating the safety and efficacy of Human Adipose-Derived Stem Cell Exosomes (ADSC-exo, STX11102 Nasal Spray) in treating acute ischemic stroke (AIS). The study consists of two sequential parts: a Single-Ascending Dose (SAD) study and a Multiple-Ascending Dose (MAD) study.
The SAD part will enroll 12 subjects with mild stroke (NIHSS 1-4). They will be sequentially enrolled into three dose cohorts (4 subjects each: 2×10⁹, 4×10⁹, and 8×10⁹ particles/mL) to receive a single nasal spray dose alongside standard care, with safety monitoring for 14 days. Dose escalation is contingent upon the safety review of the preceding cohort.
Upon confirming safety, the study proceeds to the MAD part, which will enroll 48 subjects with moderate stroke (NIHSS 5-12). They will be randomized into two dose groups (Low and High Dose), each containing an active treatment arm and a placebo arm (saline) in a 2:1 ratio (16 active:8 placebo per group). Subjects will self-administer the nasal spray daily for 14 days, with follow-up until Day 90. The primary objective is to evaluate safety, with secondary objectives assessing efficacy via neurological function scales (NIHSS, mRS, BI) and infarct volume change on MRI.
Detailed description
This clinical trial is designed to investigate the novel therapeutic agent Human Adipose-Derived Stem Cell Exosomes (ADSC-exo, STX11102) delivered via nasal spray for patients with acute ischemic stroke (AIS). The rationale is based on promising preclinical data demonstrating that intranasally administered ADSC-exo can cross the blood-brain barrier, modulate neuroinflammation, reduce infarct volume, and promote functional recovery in animal stroke models.
Study Design and Phases:
The trial employs a two-part, early-phase exploratory design integrating dose-finding and preliminary efficacy assessment.
Part 1: Single-Ascending Dose (SAD) Study: This is an open-label, dose-escalation phase to assess initial safety and tolerability. Twelve subjects with mild AIS (NIHSS 1-4, onset within 72 hours) will be enrolled at a single center. Three dose levels will be tested sequentially: Dose Cohort 1 (2×10⁹ particles/mL), Cohort 2 (4×10⁹ particles/mL), and Cohort 3 (8×10⁹ particles/mL), with 4 subjects per cohort. All subjects will receive a single dose of ADSC-exo nasal spray plus standard care. Escalation to the next higher dose cohort requires completion of the 14-day safety observation for all subjects in the current cohort and a formal safety review by the Principal Investigator (PI) and the sponsor. Escalation is permitted only if the number of subjects experiencing Grade ≥3 adverse events (AEs) considered related to the study drug is ≤50% (i.e., ≤2 subjects) within the completed cohort.
Part 2: Multiple-Ascending Dose (MAD) Study: This is a randomized, double-blind, placebo-controlled, dose-expansion phase to further evaluate safety and explore efficacy. Forty-eight subjects with moderate AIS (NIHSS 5-12, onset within 72 hours) will be enrolled across approximately five centers. Entry into this part requires a favorable safety review of all data from the SAD part. Subjects will be randomized into two sequential dose groups (Low Dose and High Dose). The specific doses (X and Y ×10⁹ particles/mL) for the MAD part will be determined based on SAD results. Each dose group consists of 24 subjects randomized in a 2:1 ratio to receive either ADSC-exo nasal spray (n=16) or matching placebo (saline, n=8) daily for 14 days, in addition to standard care. The Low Dose group must complete enrollment and 14-day safety follow-up before the High Dose group begins enrollment, applying the same safety criteria (≤50% of subjects with related Grade ≥3 AEs) for progression.
Study Population: Key inclusion criteria: age 18-80, anterior circulation ischemic stroke, pre-stroke mRS ≤1. Key exclusion criteria: severe stroke (NIHSS >12), lacunar/brainstem/cerebellar infarct, need for endovascular intervention, intracranial hemorrhage, malignancy, immunodeficiency, significant hepatic/renal impairment, active severe infection, and positive serology for HIV, HBV (with positive DNA), HCV, or syphilis.
Endpoints:
Primary Safety Endpoints: Incidence, severity, and causality of Treatment-Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs); changes in nasal mucosa, pulmonary function, and immunoglobulins.
Secondary Efficacy Endpoints:
SAD: Change from baseline in NIHSS, mRS, and Barthel Index (BI) at Day 14.
MAD: Change in NIHSS at Day 14; change in mRS and BI at Days 7, 14, 30, and 90; change in cerebral infarct lesion volume on MRI at Day 90.
Exploratory Endpoints (MAD): Changes in inflammatory cytokines (IL-2, IL-6, IL-12, IL-1β, TNF-α, IFN-γ).
Study Procedures: The schedule includes a 7-day screening period, treatment period (1 day for SAD, 14 days for MAD), and a follow-up period (14 days for SAD, 76 days for MAD). Assessments include neurological scales (NIHSS, mRS, BI), laboratory tests, vital signs, ECGs, MRI, and pulmonary function tests.
Statistical Analysis: This is an exploratory study with a sample size based on clinical practice. Safety will be analyzed in the Safety Set (SS). Efficacy will be analyzed descriptively for the SAD part. For the MAD part, efficacy will be analyzed in the Full Analysis Set (FAS) and Intent-to-Treat (ITT) set using appropriate statistical models (e.g., ANCOVA for continuous endpoints, Cochran-Mantel-Haenszel test for
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 7
- Model description
- This is an interventional clinical trial model that sequentially combines two distinct designs within a single protocol: An open-label, sequential cohort, single-ascending dose (SAD) design for initial dose-finding and safety profiling. It uses a modified "3+3" type logic (4 subjects per cohort) with predefined safety stopping rules to guide dose escalation between cohorts. A randomized, double-blind, parallel-group, multiple-ascending dose (MAD) design with placebo control for preliminary efficacy evaluation and further safety assessment. It features two sequentially enrolled dose cohorts (low and high), each with an internal 2:1 randomization (active:placebo). Progression from the low-dose to the high-dose cohort is contingent upon a safety review, making it a sequential cohort expansion within the randomized phase. This hybrid model is typical for early-phase trials of novel biological therapies, aiming to efficiently establish a safe dose range (SAD) before exploring preliminary
| Arm | Type | Receives |
|---|---|---|
| Single Ascending Dose Study Cohort 1 | Experimental | Biological: Human Adipose-Derived Stem Cell ExosomesThis is an open-label, single-dose cohort in the dose-escalation phase (Part 1). Four (4) subjects with acute ischemic stroke will receive a single dose of Human Adipose-Derived Stem Cell Exosomes (ADSC-exo) Nasal Spray at a concentration of 2×10^9 particles per mL (total volume 1 mL), administered intranasally once. |
| Single Ascending Dose Study Cohort 2 | Experimental | Biological: Human Adipose-Derived Stem Cell ExosomesThis is an open-label, single-dose cohort in the dose-escalation phase (Part 1). Four (4) subjects with acute ischemic stroke will receive a single dose of Human Adipose-Derived Stem Cell Exosomes (ADSC-exo) Nasal Spray at a concentration of 4×10^9 particles per mL (total volume 1 mL), administered intranasally once. |
| Single Ascending Dose Study Cohort 3 | Experimental | Biological: Human Adipose-Derived Stem Cell ExosomesThis is an open-label, single-dose cohort in the dose-escalation phase (Part 1). Four (4) subjects with acute ischemic stroke will receive a single dose of Human Adipose-Derived Stem Cell Exosomes (ADSC-exo) Nasal Spray at a concentration of 8×10^9 particles per mL (total volume 1 mL), administered intranasally once. |
| Multiple Dose Study Low-Dose ADSC-exo (X) | Experimental | Biological: Human Adipose-Derived Stem Cell ExosomesThis is a double-blind, multiple-dose treatment arm in the dose-expansion phase (Part 2). Sixteen (16) subjects with acute ischemic stroke will receive Human Adipose-Derived Stem Cell Exosomes (ADSC-exo) Nasal Spray at a low concentration (designated as X ×10^9 particles per mL, to be determined based on SAD results), administered intranasally once daily (QD) for 14 consecutive days. This is administered in addition to standard of care (SOC). This arm is compared to a placebo control arm within the same low-dose group. |
| Multiple Dose Study Low-Dose Placebo | Placebo comparator | Other: PlaceboThis is a double-blind, multiple-dose control arm in the dose-expansion phase (Part 2). Eight (8) subjects with acute ischemic stroke will receive a matching Placebo Nasal Spray (0.9% physiological saline), administered intranasally once daily (QD) for 14 consecutive days. This is administered in addition to standard of care (SOC). This arm serves as the comparator for the active low-dose ADSC-exo arm. |
| Multiple Dose Study High-Dose ADSC-exo (Y) | Experimental | Biological: Human Adipose-Derived Stem Cell ExosomesThis is a double-blind, multiple-dose treatment arm in the dose-expansion phase (Part 2). Sixteen (16) subjects with acute ischemic stroke will receive Human Adipose-Derived Stem Cell Exosomes (ADSC-exo) Nasal Spray at a high concentration (designated as Y ×10^9 particles per mL, to be determined based on SAD results), administered intranasally once daily (QD) for 14 consecutive days. This is administered in addition to standard of care (SOC). This arm is compared to a placebo control arm within the same high-dose group. Enrollment into this high-dose group is contingent upon a safety review of the low-dose group. |
| Multiple Dose Study High-Dose Placebo | Placebo comparator | Other: PlaceboThis is a double-blind, multiple-dose control arm in the dose-expansion phase (Part 2). Eight (8) subjects with acute ischemic stroke will receive a matching Placebo Nasal Spray (0.9% physiological saline), administered intranasally once daily (QD) for 14 consecutive days. This is administered in addition to standard of care (SOC). This arm serves as the comparator for the active high-dose ADSC-exo arm. |
Who may qualify
- Ages
- 18 to 80 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (7 listed)
- Age 18-80 years, male or female
- Patients with acute ischemic stroke within 72 hours of symptom onset
- Internal carotid artery system stroke
- For the single-dose study: mild stroke patients (NIHSS score 1-4, inclusive of 1 and 4)
- For the multiple-dose study: moderate stroke patients (NIHSS score 5-12, inclusive of 5 and 12)
Key exclusion criteria (16 listed)
- Moderate or severe stroke (NIHSS score > 12).
- Lacunar infarction, brainstem or cerebellar infarction (confirmed by DWI-MRI).
- Requirement for endovascular interventional treatment for the current episode.
- Intracranial hemorrhagic diseases (including parenchymal, intraventricular, subarachnoid hemorrhage, etc.).
- Patients with malignant tumors.
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 |
|---|---|---|
| Human Adipose-Derived Stem Cell ExosomesThis intervention involves the use of allogeneic Human Adipose-Derived Stem Cell Exosomes (ADSC-exo), provided as a sterile nasal spray (STX11102). | Biological | Multiple Dose Study High-Dose ADSC-exo (Y), Multiple Dose Study Low-Dose ADSC-exo (X), Single Ascending Dose Study Cohort 1, Single Ascending Dose Study Cohort 2, Single Ascending Dose Study Cohort 3 |
| PlaceboThis intervention serves as the placebo control. It is a sterile 0.9% sodium chloride (normal saline) solution formulated as a nasal spray | Other | Multiple Dose Study High-Dose Placebo, Multiple Dose Study Low-Dose Placebo |
Primary outcome
Safety and Tolerability of ADSC-exo Nasal Spray in Acute Ischemic Stroke.
Time frame: For Single-Dose Study: From first dose on Day 1 through the end of safety follow-up at Day 14. For Multiple-Dose Study: From first dose on Day 1 through the end of safety follow-up, which extends up to 14 days after the last dose on Day 14.
Treatment-Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs): The incidence, severity (graded per CTCAE v5.0), and the investigator-assessed causality (relationship to the study drug) of all AEs and SAEs occurring during the treatment and follow-up period.
Safety and Tolerability of ADSC-exo Nasal Spray in Acute Ischemic Stroke.
Time frame: For Single-Dose Study: From first dose on Day 1 through the end of safety follow-up at Day 14. For Multiple-Dose Study: From first dose on Day 1 through the end of safety follow-up, which extends up to 14 days after the last dose on Day 14.
Specific Safety Assessments: Nasal Mucosa: Changes from baseline based on physical examination of the nasal cavity.
Safety and Tolerability of ADSC-exo Nasal Spray in Acute Ischemic Stroke.
Time frame: For Single-Dose Study: From first dose on Day 1 through the end of safety follow-up at Day 14. For Multiple-Dose Study: From first dose on Day 1 through the end of safety follow-up, which extends up to 14 days after the last dose on Day 14.
Specific Safety Assessments: pulmonary function: Changes in FVC versus baseline
Safety and Tolerability of ADSC-exo Nasal Spray in Acute Ischemic Stroke
Time frame: For Single-Dose Study: From first dose on Day 1 through the end of safety follow-up at Day 14. For Multiple-Dose Study: From first dose on Day 1 through the end of safety follow-up, which extends up to 14 days after the last dose on Day 14.
Specific Safety Assessments: pulmonary function: Changes in FEV versus baseline
Safety and Tolerability of ADSC-exo Nasal Spray in Acute Ischemic Stroke
Time frame: For Single-Dose Study: From first dose on Day 1 through the end of safety follow-up at Day 14. For Multiple-Dose Study: From first dose on Day 1 through the end of safety follow-up, which extends up to 14 days after the last dose on Day 14.
Specific Safety Assessments: pulmonary function: Changes in PEF versus baseline
Safety and Tolerability of ADSC-exo Nasal Spray in Acute Ischemic Stroke.
Time frame: For Single-Dose Study: From first dose on Day 1 through the end of safety follow-up at Day 14. For Multiple-Dose Study: From first dose on Day 1 through the end of safety follow-up, which extends up to 14 days after the last dose on Day 14.
Immunoglobulins: Changes from baseline in serum levels of IgA.
Safety and Tolerability of ADSC-exo Nasal Spray in Acute Ischemic Stroke.
Time frame: For Single-Dose Study: From first dose on Day 1 through the end of safety follow-up at Day 14. For Multiple-Dose Study: From first dose on Day 1 through the end of safety follow-up, which extends up to 14 days after the last dose on Day 14.
Immunoglobulins: Changes from baseline in serum levels of IgE
Safety and Tolerability of ADSC-exo Nasal Spray in Acute Ischemic Stroke
Time frame: For Single-Dose Study: From first dose on Day 1 through the end of safety follow-up at Day 14. For Multiple-Dose Study: From first dose on Day 1 through the end of safety follow-up, which extends up to 14 days after the last dose on Day 14.
Immunoglobulins: Changes from baseline in serum levels of IgM.
Safety and Tolerability of ADSC-exo Nasal Spray in Acute Ischemic Stroke
Time frame: For Single-Dose Study: From first dose on Day 1 through the end of safety follow-up at Day 14. For Multiple-Dose Study: From first dose on Day 1 through the end of safety follow-up, which extends up to 14 days after the last dose on Day 14.
Immunoglobulins: Changes from baseline in serum levels of IgG.
6 secondary outcomes
Change in Neurological Function (For Single-Dose Study)
Time frame: Baseline (Day 1, pre-dose) to Day 14 post-dose.
Change in NIHSS Score after 14-Day Treatment (For Multiple-Dose Study)
Time frame: Baseline (Day 1, pre-dose) to Day 14 (end of treatment).
Change in Disability & Daily Function (For Multiple-Dose Study)
Time frame: Baseline (Day 1, pre-dose) to Day 7, Day 14, Day 30, and Day 90.
Change in Cerebral Infarct Volume on MRI (For Multiple-Dose Study)
Time frame: Baseline MRI (within screening period, Day -7 to -1) to Day 90 MRI.
Change in Neurological Function (For Single-Dose Study)
Time frame: Baseline (Day 1, pre-dose) to Day 14 post-dose.
Change in Neurological Function (For Single-Dose Study)
Time frame: Baseline (Day 1, pre-dose) to Day 14 post-dose.
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 10 Feb 2026.
- Background: PubMed 37859748Xie X, Song Q, Dai C, Cui S, Tang R, Li S, Chang J, Li P, Wang J, Li J, Gao C, Chen H, Chen S, Ren R, Gao X, Wang G. Clinical safety and efficacy of allogenic human adipose mesenchymal stromal cells-derived exosomes in patients with mild to moderate Alzheimer's disease: a phase I/II clinical trial. Gen Psychiatr. 2023 Oct 11;36(5):e101143. doi: 10.1136/gpsych-2023-101143. eCollection 2023.
- Background: PubMed 33431513Li Y, Tang Y, Yang GY. Therapeutic application of exosomes in ischaemic stroke. Stroke Vasc Neurol. 2021 Sep;6(3):483-495. doi: 10.1136/svn-2020-000419. Epub 2021 Jan 11.
- Background: PubMed 31136284Sonoda T, Matsuzaki J, Yamamoto Y, Sakurai T, Aoki Y, Takizawa S, Niida S, Ochiya T. Serum MicroRNA-Based Risk Prediction for Stroke. Stroke. 2019 Jun;50(6):1510-1518. doi: 10.1161/STROKEAHA.118.023648. Epub 2019 Apr 15.
- Background: PubMed 35281985Dehghani L, Khojasteh A, Soleimani M, Oraee-Yazdani S, Keshel SH, Saadatnia M, Saboori M, Zali A, Hashemi SM, Soleimani R. Safety of Intraparenchymal Injection of Allogenic Placenta Mesenchymal Stem Cells Derived Exosome in Patients Undergoing Decompressive Craniectomy Following Malignant Middle Cerebral Artery Infarct, A Pilot Randomized Clinical Trial. Int J Prev Med. 2022 Jan 19;13:7. doi: 10.4103/ijpvm.ijpvm_441_21. eCollection 2022.
- Background: PubMed 29705934Yang Y, Cai Y, Zhang Y, Liu J, Xu Z. Exosomes Secreted by Adipose-Derived Stem Cells Contribute to Angiogenesis of Brain Microvascular Endothelial Cells Following Oxygen-Glucose Deprivation In Vitro Through MicroRNA-181b/TRPM7 Axis. J Mol Neurosci. 2018 May;65(1):74-83. doi: 10.1007/s12031-018-1071-9. Epub 2018 Apr 29.
- Background: PubMed 28624203Yang J, Zhang X, Chen X, Wang L, Yang G. Exosome Mediated Delivery of miR-124 Promotes Neurogenesis after Ischemia. Mol Ther Nucleic Acids. 2017 Jun 16;7:278-287. doi: 10.1016/j.omtn.2017.04.010. Epub 2017 Apr 13.
- Background: PubMed 28232590Xin H, Katakowski M, Wang F, Qian JY, Liu XS, Ali MM, Buller B, Zhang ZG, Chopp M. MicroRNA cluster miR-17-92 Cluster in Exosomes Enhance Neuroplasticity and Functional Recovery After Stroke in Rats. Stroke. 2017 Mar;48(3):747-753. doi: 10.1161/STROKEAHA.116.015204.
- Background: PubMed 25383184Altmann P, Mildner M, Haider T, Traxler D, Beer L, Ristl R, Golabi B, Gabriel C, Leutmezer F, Ankersmit HJ. Secretomes of apoptotic mononuclear cells ameliorate neurological damage in rats with focal ischemia. F1000Res. 2014 Jun 19;3:131. doi: 10.12688/f1000research.4219.2. eCollection 2014.
- Background: PubMed 22621230Hayon Y, Dashevsky O, Shai E, Brill A, Varon D, Leker RR. Platelet microparticles induce angiogenesis and neurogenesis after cerebral ischemia. Curr Neurovasc Res. 2012 Aug;9(3):185-92. doi: 10.2174/156720212801619018.
- Background: PubMed 27793019Chen KH, Chen CH, Wallace CG, Yuen CM, Kao GS, Chen YL, Shao PL, Chen YL, Chai HT, Lin KC, Liu CF, Chang HW, Lee MS, Yip HK. Intravenous administration of xenogenic adipose-derived mesenchymal stem cells (ADMSC) and ADMSC-derived exosomes markedly reduced brain infarct volume and preserved neurological function in rat after acute ischemic stroke. Oncotarget. 2016 Nov 15;7(46):74537-74556. doi: 10.18632/oncotarget.12902.
- Background: PubMed 26339036Doeppner TR, Herz J, Gorgens A, Schlechter J, Ludwig AK, Radtke S, de Miroschedji K, Horn PA, Giebel B, Hermann DM. Extracellular Vesicles Improve Post-Stroke Neuroregeneration and Prevent Postischemic Immunosuppression. Stem Cells Transl Med. 2015 Oct;4(10):1131-43. doi: 10.5966/sctm.2015-0078. Epub 2015 Sep 3.
- Background: PubMed 26993303Zhang Y, Chopp M, Liu XS, Katakowski M, Wang X, Tian X, Wu D, Zhang ZG. Exosomes Derived from Mesenchymal Stromal Cells Promote Axonal Growth of Cortical Neurons. Mol Neurobiol. 2017 May;54(4):2659-2673. doi: 10.1007/s12035-016-9851-0. Epub 2016 Mar 19.
- Individual participant dataSharing planned: Yes. This study will share de-identified individual participant data (IPD) that underlies the results reported in the primary and secondary outcome publications. This will include baseline characteristics (e.g., age, sex, NIHSS score at entry, pre-stroke mRS), treatment assignment (dose group), and outcome data for all primary and secondary endpoints. Specifically, shared data will encompass safety endpoints (all recorded Treatment-Emergent Adverse Events, Serious Adverse Events, and changes in nasal mucosa, pulmonary function, and immunoglobulins) and efficacy endpoints (serial assessments of NIHSS, mRS, Barthel Index scores, and cerebral infarct volume change on MRI at Day 90 for the MAD part). All shared data will be fully anonymized to protect participant confidentiality.
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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.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing Human Adipose-Derived Stem Cell Exosomes in people with Acute Ischemic Stroke, run by Shanghai General Hospital, Shanghai Jiao Tong University School of Medicine.
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 7 inclusion and 16 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 10 Feb 2026. 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?
Contact Xia Liu, M.D., Ph.D. (86+19121579288, liuxia6700@163.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Safety and Tolerability of ADSC-exo Nasal Spray in Acute Ischemic Stroke. (time frame: For Single-Dose Study: From first dose on Day 1 through the end of safety follow-up at Day 14. For Multiple-Dose Study: From first dose on Day 1 through the end of safety follow-up, which extends up to 14 days after the last dose on Day 14.). The study lists 9 primary outcomes in all. 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 NCT07398612, which also lists 30 linked publications.
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 Feb 2026.