Safety and Efficacy of Human Adipose-Derived Stem Cell Exosomes in Acute Ischemic Stroke

NCT07398612 | Not Yet Recruiting Phase 1 DMC

• 1 other identifier: IIT-ATMP-2025011
• Study Type: interventional
• Target: 60
• Locations: 0 countries
• Sites: N/A

Brief 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). 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.

Conditions

Acute Ischemic Stroke

Keywords

acute ischemic stroke; human adipose-derived stem cell; exosome; NIHSS; mRS

Outcome Measures

Primary Outcomes (9)

• 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.

  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.

  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

  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

  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

  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.

  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

  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.

  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.

  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)

  Baseline (Day 1, pre-dose) to Day 14 post-dose.

• Change in NIHSS Score after 14-Day Treatment (For Multiple-Dose Study)

  Baseline (Day 1, pre-dose) to Day 14 (end of treatment).

• Change in Disability & Daily Function (For Multiple-Dose Study)

  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)

  Baseline MRI (within screening period, Day -7 to -1) to Day 90 MRI.

• Change in Neurological Function (For Single-Dose Study)

  Baseline (Day 1, pre-dose) to Day 14 post-dose.

Study Arms (7)

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.

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.

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.

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.

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.

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.

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.

Other: Placebo

Interventions

Human Adipose-Derived Stem Cell Exosomes BIOLOGICAL

This intervention involves the use of allogeneic Human Adipose-Derived Stem Cell Exosomes (ADSC-exo), provided as a sterile nasal spray (STX11102).

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

Placebo OTHER

This intervention serves as the placebo control. It is a sterile 0.9% sodium chloride (normal saline) solution formulated as a nasal spray

Multiple Dose Study High-Dose Placebo; Multiple Dose Study Low-Dose Placebo

Eligibility Criteria

• Age: 18 Years - 80 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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.
• 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.

Sponsors & Collaborators

• Shanghai General Hospital, Shanghai Jiao Tong University School of Medicine: lead
• Xinhua Hospital, Shanghai Jiao Tong University School of Medicine: collaborator
• Shanghai 10th People's Hospital: collaborator
• First Affiliated Hospital of the Chinese People's Liberation Army Naval Medical University: collaborator
• Shanghai Jiao Tong University Affiliated Sixth People's Hospital: collaborator

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MeSH Terms

Conditions

Ischemic Stroke

Condition Hierarchy (Ancestors)

Stroke; Cerebrovascular Disorders; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Vascular Diseases; Cardiovascular Diseases

Central Study Contacts

Xia Liu, M.D., Ph.D.

CONTACT

86+19121579288; liuxia6700@163.com

Ming Zhang

CONTACT

86+17826520869; zhangming@stexo.com

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Masking Details: This study employs a double-blind design. The following parties are masked (blinded): Subjects: Participants will not know whether they are receiving ADSC-exo or placebo (saline). Investigators and Site Staff: All personnel directly involved in treating subjects, evaluating clinical outcomes (including efficacy scale assessments), and managing subject care during the trial will be blinded to treatment assignment. This includes the Principal Investigator, sub-investigators, and study nurses. Sponsor's Clinical Team: Personnel from the sponsor involved in the day-to-day monitoring and clinical operations of the trial will remain blinded. To maintain the blind, the active drug (ADSC-exo solution) and the placebo (saline) are prepared to be identical in appearance, packaging, and labeling. An interactive web response system (IWRS) will manage randomization and drug kit assignment. The sponsor will designate a limited number of unblinded personnel (e.g., an unblinded statistician respon
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Professor

Model Details: 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

Study Record Dates

• First Submitted: December 30, 2025
• First Posted: February 10, 2026
• Study Start: January 31, 2026
• Primary Completion (Estimated): May 31, 2028
• Study Completion (Estimated): August 31, 2028
• Last Updated: February 10, 2026

Record last verified: 2025-12

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL, SAP, ICF
• Time Frame: The de-identified IPD, study protocol, SAP, and ICF will become available 12 months after the publication of the primary study results (anticipated around September 2029, based on the Study Completion date of August 31, 2028). The data will be accessible for a period of 5 years thereafter (anticipated until September 2034). Requests can be submitted during this 5-year window.
• Access Criteria: Access will be granted to qualified researchers from accredited scientific or medical institutions for the purpose of conducting analyses to achieve pre-specified research goals in the approved proposal. Examples include independent validation of results, meta-analysis, or exploration of novel scientific questions. Requestors must submit a methodologically sound research proposal through the designated access portal.