Treatment of Moyamoya Disease With iPSC-derived Exosomes

Launched by HUAQIU ZHANG · Jul 3, 2025

Keywords

ClinConnect Summary

This clinical trial is studying a new treatment for Moyamoya disease, a condition where certain blood vessels in the brain become narrowed or blocked, leading to strokes or other brain problems. The trial is testing whether a special therapy using tiny particles called exosomes, made from lab-grown stem cells (called iPSC-derived exosomes), can help improve blood flow in the brain after surgery. These exosomes are applied near the temporal muscle (a muscle on the side of the head) to encourage new blood vessel growth and improve circulation between areas inside and outside the brain.

This study is for adults aged 35 to 65 who have been diagnosed with Moyamoya disease and have already had surgery to help with blood flow but still have symptoms or poor blood flow three months after surgery. Participants need to be generally healthy in terms of liver, kidney, heart, and blood function. If eligible, patients will receive this new therapy as an additional treatment to help rebuild blood flow in the brain. The goal is to find a safe, effective, and easy way to support brain blood circulation in Moyamoya disease, potentially improving symptoms and quality of life. If you or a loved one fits these criteria and are interested, you can discuss this option with your doctor or the study team.

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Eligibility criteria

• Inclusion Criteria:
• • 1. For adult patients with a clear diagnosis of Moyamoya disease aged between 35 and 65 years (inclusive), cranial DSA angiography or magnetic resonance angiography (MRA) suggests bilateral terminal occlusion of the internal carotid artery (ICA) accompanied by stenosis or occlusion at the origin of the anterior cerebral artery (ACA) and the middle cerebral artery (MCA). And it is accompanied by the formation of abnormal vascular networks at the base of the skull; Unilateral or bilateral lesions are both acceptable. Suzuki score ≥3 points.
• • 2. Have sufficient bone marrow, liver, kidney and heart functions (based on the normal values of the clinical trial center) : Absolute neutrophil count (ANC) ≥ 1.5×109/L, platelet count ≥ 100×109/L, hemoglobin ≥ 90 g/L, serum total bilirubin ≤ 1.5 times the upper limit of normal value (ULN), ALT, AST or ALP ≤ 3 times ULN; Serum creatinine ≤ 1.5 times ULN, International Normalized Ratio (INR) ≤ 1.5 times ULN, APTT ≤ 1.5 times ULN; Left ventricular ejection fraction ˃50%;
• • 3. The patient has undergone temporal muscle application surgery and feels that the symptoms have not improved well.
• • 4. The patient has been followed up for ≥3 months since the date of the surgery;
• • 5. Vascular DSA of the patient 3 months after the operation indicated poor blood flow reconstruction effect;
• • 6. Cerebral perfusion (CTP or ASL) of the patient 3 months after the operation showed ischemia;
• • 7. The patient still had clinical manifestations of Moyamoya disease such as cerebral ischemia or cerebral infarction 3 months after the operation;
• • 8. The patient or their legal representative gives informed consent and signs the informed consent form.
• Exclusion Criteria:
• • 1. Severe complications such as liver and kidney dysfunction, etc.
• • 2. Patients with a history of mental disorders or mental illnesses;
• • 3. Patients with coagulation disorders or those requiring long-term oral anticoagulant drugs;
• • 4. Patients with an mRS Score of ≥4 or those with large-area cerebral infarction or in a coma;
• • 5. Patients with Moyamoya syndrome complicated with atherosclerosis, autoimmune diseases, brain tumors, those who have received radiotherapy or sickle cell anemia, etc.
• • 6. Surgical methods include patients undergoing direct bypass surgery;
• • 7. Those who have not signed the surgical consent form;
• • 8. Patients with allergic constitutions or a clear history of allergies;
• • 9. Pregnant women, lactating women and patients with fertility plans during the trial period;
• • 10. Those who have participated in other clinical trials in the past three months;
• • 11. Patients whom the researchers consider unsuitable to participate in the trial.