Human Induced Neural Stem Cell-derived Exosomes for Treating Acute Ischemic Stroke

NCT07143786 | Not Yet Recruiting Phase 1 DMC

• 1 other identifier: iNSC-Exo-AIS2025
• Study Type: interventional
• Target: 38
• Locations: 0 countries
• Sites: N/A

Brief 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

Conditions

Ischemic Stroke, Acute

Keywords

induced neural stem cell; exosome; acute ischemic stroke; safety; efficacy

Outcome Measures

Primary Outcomes (2)

• Dose limiting toxicity

  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.

  90（±7）days

Secondary Outcomes (7)

• AEs and SAEs

  Within 90 days

• mRS

  14（±1）days

• Percentage of mRS 0-2

  14 (±1), 90 (±7) days

• NIHSS score change compared to baseline

  14 (±1), 90 (±7) days

• Barthel Index change compared to baseline

  14 (±1), 90 (±7) days

Study Arms (2)

Exosome group

EXPERIMENTAL

Acute ischemic stroke patients that receive intravenous iNSC-Exo (5.0×10\^11 \~ 2.0×10\^12 particles) administration

Drug: iNSC-EV01

Control group

PLACEBO COMPARATOR

Acute ischemic stroke patients that receive intravenous placebo administration

Drug: Placebo

Interventions

iNSC-EV01 DRUG

Intravenous induced neural stem cell-derived exosome

Exosome group

Placebo DRUG

iNSC-EV01 mimics

Control group

Eligibility Criteria

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

You may qualify if:

• 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.

You may not qualify if:

• 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:
• 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;

Sponsors & Collaborators

• Xuanwu Hospital, Beijing: lead
• Wiseheart Co.Ltd: collaborator

MeSH Terms

Conditions

Ischemic Stroke

Condition Hierarchy (Ancestors)

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

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Professor

Study Record Dates

• First Submitted: August 7, 2025
• First Posted: August 27, 2025
• Study Start: October 1, 2025
• Primary Completion (Estimated): July 31, 2027
• Study Completion (Estimated): December 31, 2027
• Last Updated: August 27, 2025

Record last verified: 2025-08

Data Sharing

• IPD Sharing: Will not share