IV infusions of human umbilical cord mesenchymal stem cells for acute ischemic stroke (Phase IIa)

A Phase IIa Randomized, Blinded, Placebo-Controlled Clinical Trial to Evaluate the Efficacy and Safety of Intravenous Infusion of Human Umbilical Cord Mesenchymal Stem Cells (hUC-MSCs) in the Treatment of Acute Ischemic Stroke (AIS)

This Phase IIa test will see if one or multiple IV infusions of human umbilical cord mesenchymal stem cells help adults with recent acute ischemic stroke recover better and are safe.

Quick facts

• Phase: Phase 2
• Study type: Interventional
• Enrollment: 60 (estimated)
• Ages: 18 Years to 75 Years
• Sex: All
• Sponsor: Shenzhen Wingor Biotechnology Co., Ltd. (Industry-sponsored)
• Drugs / interventions: prednisone
• Locations: 1 site (Beijing, Beijing Municipality)
• Trial ID: NCT07084012 on ClinicalTrials.gov

What this trial studies

This randomized, double-blind, placebo-controlled Phase IIa trial uses a three-arm design comparing high-dose hUC-MSCs, low-dose hUC-MSCs, and placebo given by intravenous infusion. Patients with anterior circulation acute ischemic stroke within 72 hours of onset and baseline NIHSS 8–20 are enrolled and randomized to single or multiple infusions. The primary focus is on efficacy of the dosing regimens, with secondary endpoints monitoring safety and tolerability and exploratory analyses of pharmacokinetic and pharmacodynamic markers. Treatments and follow-up are conducted at Beijing Tiantan Hospital affiliated with Capital Medical University.

Who should consider this trial

Good fit: Adults aged 18–75 with anterior circulation acute ischemic stroke within 72 hours of onset, pre-stroke mRS ≤1, NIHSS 8–20, and who have not undergone or are not planned for thrombolysis or thrombectomy are the intended candidates.

Not a fit: Patients with intracranial hemorrhage, those who have had or will have thrombolysis or thrombectomy, major neurodegenerative or uncontrolled comorbid conditions, or strokes outside the 72-hour window are unlikely to benefit from this trial.

Why it matters

Potential benefit: If successful, this approach could reduce brain injury and improve functional recovery after acute ischemic stroke beyond current acute treatments.

How similar studies have performed: Previous small studies of mesenchymal stem cells in stroke have shown acceptable safety and some early signals of functional improvement, but definitive benefit has not yet been established in large trials.

Eligibility criteria

Inclusion Criteria:

1. Age 18 to 75 years, inclusive, regardless of gender.
2. Diagnosis of acute ischemic stroke (AIS).
3. Onset time ≤ 72 hours.
4. Anterior circulation cerebral infarction.
5. Modified Rankin Scale (mRS) score ≤ 1 before the onset of this stroke.
6. National Institutes of Health Stroke Scale (NIHSS) score between 8 and 20 (inclusive) at screening, and NIHSS item 1a (level of consciousness) score ≤ 1.
7. The subject or their legal guardian has signed the informed consent form.

Exclusion Criteria:

1. Planned or already undergone thrombolysis or thrombectomy for this stroke.
2. History of epilepsy (excluding secondary epilepsy that does not currently require medication), Parkinson's disease, Alzheimer's disease, severe depression, or other diseases that the investigator deems would affect the subject's participation in the trial or the assessment of efficacy.
3. Presence of intracranial hemorrhagic disease (e.g., intracerebral hemorrhage, intraventricular hemorrhage, subarachnoid hemorrhage, subdural/epidural hematoma, etc.). If only petechial bleeding is present, the investigator may determine whether the subject is suitable for inclusion in the study.
4. Computed tomography (CT) or magnetic resonance imaging (MRI) of the head showing a large ischemic area in the middle cerebral artery territory or midline shift greater than 1 cm on head CT/MRI, and the investigator assesses a high likelihood of surgical intervention or poor prognosis.
5. Presence of brain tumor or history of malignancy.
6. Liver or kidney insufficiency during the screening period: Aspartate aminotransferase (AST) \> 2.5 × upper limit of normal, alanine aminotransferase (ALT) \> 2.5 × upper limit of normal, estimated glomerular filtration rate (eGFR) \< 60 mL/min/1.73 m².
7. History of severe cardiovascular disease, which the investigator deems unsuitable for participation in this clinical trial.
8. Severe infection, including sepsis, septic shock, severe pneumonia, etc.
9. Systemic corticosteroid ( \> 10 mg/day prednisone equivalent) or immunosuppressive drug treatment within 14 days before receiving the investigational drug or during the trial.
10. History of alcohol abuse within the past year (defined as an average of more than 2 units per day (1 unit = 10 mL ethanol, i.e., 1 unit = 200 mL of 5% alcohol beer or 25 mL of 40% alcohol spirits or 85 mL of 12% alcohol wine).
11. Pregnant or breastfeeding women; or unwillingness to use reliable contraception (e.g., condoms) throughout the study period, or plans to donate sperm or eggs, or have plans for pregnancy.
12. Participation in an interventional clinical trial within the past 3 months, or receipt of other cell therapy (excluding blood transfusion).
13. Other situations in which the investigator deems the patient unsuitable for participation in this study (including but not limited to non-compliance with the principle of patient benefit, poor patient compliance, unacceptable laboratory abnormalities, etc.).

Where this trial is running

Beijing, Beijing Municipality

• Beijing Tiantan Hospital, Capital Medical University — Beijing, Beijing Municipality, China (Recruiting)

Study contacts

• Study coordinator: XiaohongWang,MD

Conditions Acute Ischemic Stroke AIS; hUC-MSCs; AIS,; Acute Ischemic Stroke