Treatment of Moyamoya Disease With iPSC-derived Exosomes

NCT07065409 | Recruiting Phase 1

• 1 other identifier: TJ-IRB202503034
• Study Type: interventional
• Target: 9
• Locations: 1 country
• Sites: 1

Brief Summary

Moyamoya disease is a cerebrovascular disease clinically characterized by chronic progressive stenosis or occlusion at the ends of bilateral internal carotid arteries and the origin of anterior cerebral arteries and middle cerebral arteries, followed by the formation of abnormal vascular networks at the base of the skull. Clinically, patients with Moyamoya disease mainly present with ischemic or hemorrhagic stroke, and there are two peaks of incidence in children aged 3-5 and middle-aged people aged 40-50. Moreover, as the pathogenesis and treatment evaluation of Moyamoya disease are still in the research trough at present, new discoveries are prone to occur and thus attract a great deal of attention. It not only has a beneficial promoting effect on the treatment and diagnosis of patients, but also makes it easier for research topics to be reported in top journals. This study intends to combine iPSC-EVs local skin transplantation with temporal muscle application to promote muscle angiogenesis and the establishment of extracranial and intracranial collateral circulation after temporal muscle application. The above-mentioned design features high efficiency, safety and convenience, and is an innovative exploration both at home and abroad. We hope to screen out safe, efficient and simple preparation methods and transplantation methods of iPSC-EVs through systematic experiments, establish an effective clinical evaluation system, and provide auxiliary means for intracranial and extracranial blood flow reconstruction surgery in the treatment of Moyamoya disease. Moreover, in terms of topic selection, iPSC is currently one of the most promising directions for innovative treatment worldwide.

Conditions

Moyamoya Disease

Outcome Measures

Primary Outcomes (3)

• Incidence of Treatment-Emergent Adverse Events [Safety and Tolerability]

  Evaluate the tolerance and adverse reactions of patients after 3 days, 2 weeks and 1 month of iPSC-EVs treatment. Incidence of Treatment-Emergent Adverse Events \[Safety and Tolerability\].

  3 Day、2 week、1 month

• Imaging examinations

  Imaging examinations were conducted on the patients before iPSC-EVs treatment, and at 1 month after treatment. Imaging examinations: MRI and TCD.

  1 month

• Neuropsychological tests

  Neuropsychological tests were conducted on the patients before iPSC-EVs treatment, and at 1 month after treatment. neuropsychological tests: mRS, MMSE and MoCA

  1 month

Secondary Outcomes (2)

• Imaging examinations

  3 month, 6 month, 12 month, and 24 month

• Neuropsychological tests

  3 month, 6 month, 12 month, and 24 month

Study Arms (1)

iPSC-EVs

EXPERIMENTAL

Biological: iPSC-EVs

Interventions

iPSC-EVs BIOLOGICAL

iPSC-EVs，The drug number is NouvSoma002-01. It is from iRegene Therapeutics Co., Ltd.

iPSC-EVs

Eligibility Criteria

• Age: 18 Years - 75 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Diagnosed adult patients with MMD aged 18-75 years (inclusive), with bilateral terminal occlusion of the internal carotid arteries (ICA) and stenosis or occlusion of the anterior cerebral artery (ACA) and middle cerebral artery (MCA) at the origin, accompanied by the formation of abnormal vascular networks at the base of the skull, as indicated by head DSA or MRA. Unilateral or bilateral lesions are acceptable. Suzuki score ≥ 3.
• Relevant bone marrow, liver, kidney, and heart function indicators meet the following standards (based on the normal values of the clinical trial center): absolute neutrophil count (ANC) ≥ 1.5×109/L, platelets ≥ 100×109/L, total serum bilirubin ≤ 1.5 times the upper limit of normal (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.
• Patients have undergone temporal muscle patch surgery and have not achieved satisfactory improvement in symptoms.
• Patients have been followed up for ≥ 3 months since the surgery.
• Vascular DSA performed 3 months after the surgery indicates poor blood flow reconstruction.
• CTP or ASL performed 3 months after the surgery shows ischemia.
• Patients still have clinical manifestations of cerebral ischemia or cerebral infarction due to MMD 3 months after the surgery.
• Patients or their legal representatives have given informed consent and signed the informed consent form.

You may not qualify if:

• Patients with severe liver or kidney dysfunction or other complications;
• Patients with a history of mental disorders or mental diseases;
• Patients with coagulation disorders;
• Patients with extensive cerebral infarction or in a coma;
• Patients who only undergo direct bypass surgery;
• Patients who have not signed the surgical consent form;
• Patients with allergic constitutions or a clear history of allergies;
• Pregnant women, lactating women, and patients with plans to conceive during the trial period;
• Patients who have participated in other clinical trials in the past three months;
• Patients deemed unsuitable for the trial by the researcher.

Sponsors & Collaborators

• Huaqiu Zhang: lead

Study Sites (1)

Tongji hospital

Wuhan, Hubei, 430030, China

RECRUITING

MeSH Terms

Conditions

Moyamoya Disease

Condition Hierarchy (Ancestors)

Carotid Artery Diseases; Cerebrovascular Disorders; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Cerebral Arterial Diseases; Intracranial Arterial Diseases; Arterial Occlusive Diseases; Vascular Diseases; Cardiovascular Diseases

Study Officials

• Huaqiu Zhang

  Tongji Hospital

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Huaqiu Zhang

CONTACT

13419632963; lyc@tjh.tjmu.edu.cn

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR INVESTIGATOR
• PI Title: Clinical Professor

Study Record Dates

• First Submitted: May 9, 2025
• First Posted: July 15, 2025
• Study Start: May 26, 2025
• Primary Completion (Estimated): May 31, 2026
• Study Completion (Estimated): October 31, 2026
• Last Updated: October 1, 2025

Record last verified: 2025-09

Data Sharing

• IPD Sharing: Will not share

Locations

CN (1)