Moyamoya DiseasePhase 1NCT07065409
Treatment of Moyamoya Disease With iPSC-derived Exosomes
A Phase 1 study testing iPSC-EVs in people with Moyamoya Disease, run by Huaqiu Zhang at 1 site in China.
Trial 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… Read more about this study
- Who is it for?
- Ages 18 to 75 years
- Conditions the study is for
- Moyamoya Disease
- What is being tested?
- iPSC-EVs
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 9 participants
- Where
- 1 siteChina
- Study dates
- 26 May 2025to 31 Oct 2026
- Phase
- Phase 1
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 75 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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.
Who cannot join
- 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;
Show all 10Show fewer
- 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.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
China1 site, 1 recruiting
- Recruiting
Tongji hospital
Wuhan, Hubei, 430030
Huaqiu Zhang, Clinical Professor, 13419632963, lyc@tjh.tjmu.edu.cn
About this study
Official title: Phase I Clinical Trial of Induced Pluripotent Stem Cell-derived Extracellular Vesicles Combined With Temporal Muscle Flap Application in Promoting Intracranial and Extracranial Blood Flow Reconstruction in Patients With Moyamoya Disease
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.
Read the detailed description
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. Because this abnormal vascular network at the base of the skull looks like wisps of smoke on cerebral angiography, the Japanese Suzuki first named it "Moyamoya Disease" in the 1960s. 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. Epidemiological studies have found that Moyamoya disease is mainly distributed among the population in East Asia, including Japan, South Korea and China, and its incidence rate in China has shown a significant upward trend in recent years.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.
In recent years, with the popularity of IPscs, the emerging therapeutic approach of extracellular vesicles derived from IPscs (iPSC-EVs) has gradually come into the public view. Extracellular vesicles derived from IPscs have specific bioactive substances related to iPSC functions. Their main advantages are manifested as follows: (1) They have a strong ability to promote regeneration and can promote vascular regeneration; (2) It has a powerful function of inhibiting inflammatory responses and can significantly inhibit the release of inflammatory factors by microglia, astrocytes and oligodendrocytes. (3) The homogeneity and stability of IPscs enable extracellular vesicles derived from IPscs to have high drugability; (4) IPscs have good potential for gene editing and can support the engineering of extracellular vesicles and drug delivery in extracellular vesicles. (5) The homing effect of IPSC-differentiated cells can endow the corresponding extracellular vesicles with better tissue-targeting characteristics.
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
- Being tested
- iPSC-EVs
- Medical subject headings
- Moyamoya Disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
iPSC-EVsBiological
iPSC-EVs，The drug number is NouvSoma002-01. It is from iRegene Therapeutics Co., Ltd.
| Study group | Role | What they receive |
|---|---|---|
| iPSC-EVs | Experimental | Not described Receives: Biological: iPSC-EVs |
What is being measured
The primary outcome is the main question the study is designed to answer.
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].
Measured: 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.
Measured: 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
Measured: 1 month
Secondary outcomes (2)
Imaging examinations
Imaging examinations for post-treatment imaging assessment of blood flow reconstruction function.
Imaging examinations: DSA, MRI, CTP, TCD, HR-VWI, and CT.
Measured: 3 month, 6 month, 12 month, and 24 month
Neuropsychological tests
Neuropsychological tests for post-treatment imaging assessment of blood flow reconstruction function.
neuropsychological tests: mRS, MMSE and MoCA
Measured: 3 month, 6 month, 12 month, and 24 month
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted15 Jul 2025
- Study start26 May 2025
- Primary completion31 May 2026 (estimated)
- Study completion31 Oct 2026 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Moyamoya DiseaseStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 9 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Huaqiu Zhang
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor-investigator, Huaqiu Zhang, Tongji Hospital
- Investigators
- Huaqiu Zhang, Principal investigator, Tongji Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07065409
- Sponsor's protocol ID
- TJ-IRB202503034
- Status last verified
- Sep 2025
- First submitted
- 9 May 2025
- Last updated
- 1 Oct 2025
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 1 Oct 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Phase I Clinical Trial of Induced Pluripotent Stem Cell-derived Extracellular Vesicles Combined With Temporal Muscle Flap Application in Promoting Intracranial and Extracranial Blood Flow Reconstruction in Patients With Moyamoya Disease
- ClinicalTrials.gov ID
- NCT07065409
- Acronym / sponsor ID
- TJ-IRB202503034
- Conditions
- Moyamoya Disease
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 9 participants (estimated)
- Lead sponsor
- Huaqiu ZhangUniversity, hospital or other
- Study start
- 26 May 2025 (actual)
- Primary completion
- 31 May 2026 (estimated)
- Study completion
- 31 Oct 2026 (estimated)
- First posted
- 15 Jul 2025 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Detailed description
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. Because this abnormal vascular network at the base of the skull looks like wisps of smoke on cerebral angiography, the Japanese Suzuki first named it "Moyamoya Disease" in the 1960s. 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. Epidemiological studies have found that Moyamoya disease is mainly distributed among the population in East Asia, including Japan, South Korea and China, and its incidence rate in China has shown a significant upward trend in recent years.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.
In recent years, with the popularity of IPscs, the emerging therapeutic approach of extracellular vesicles derived from IPscs (iPSC-EVs) has gradually come into the public view. Extracellular vesicles derived from IPscs have specific bioactive substances related to iPSC functions. Their main advantages are manifested as follows: (1) They have a strong ability to promote regeneration and can promote vascular regeneration; (2) It has a powerful function of inhibiting inflammatory responses and can significantly inhibit the release of inflammatory factors by microglia, astrocytes and oligodendrocytes. (3) The homogeneity and stability of IPscs enable extracellular vesicles derived from IPscs to have high drugability; (4) IPscs have good potential for gene editing and can support the engineering of extracellular vesicles and drug delivery in extracellular vesicles. (5) The homing effect of IPSC-differentiated cells can endow the corresponding extracellular vesicles with better tissue-targeting characteristics.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| iPSC-EVs | Experimental | Biological: iPSC-EVs |
Who may qualify
- Ages
- 18 to 75 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (8 listed)
- 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.
Key exclusion criteria (10 listed)
- 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;
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| iPSC-EVsiPSC-EVs，The drug number is NouvSoma002-01. It is from iRegene Therapeutics Co., Ltd. | Biological | iPSC-EVs |
Primary outcome
Incidence of Treatment-Emergent Adverse Events [Safety and Tolerability]
Time frame: 3 Day、2 week、1 month
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].
Imaging examinations
Time frame: 1 month
Imaging examinations were conducted on the patients before iPSC-EVs treatment, and at 1 month after treatment. Imaging examinations: MRI and TCD.
Neuropsychological tests
Time frame: 1 month
Neuropsychological tests were conducted on the patients before iPSC-EVs treatment, and at 1 month after treatment. neuropsychological tests: mRS, MMSE and MoCA
2 secondary outcomes
Imaging examinations
Time frame: 3 month, 6 month, 12 month, and 24 month
Neuropsychological tests
Time frame: 3 month, 6 month, 12 month, and 24 month
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 1 Oct 2025.
- Individual participant dataSharing planned: No
Related trials for Moyamoya Disease
RecruitingObservational study
Development and Validation of Risk Prediction Models for Stroke and Cognitive Impairment in Moyamoya Disease and Moyamoya Syndrome
Moyamoya Disease, Moyamoya Syndrome
Enrolling by invitationEarly Phase 1
Clinical Safety and Efficacy Evaluation of the Blood-cooling and Stasis-removing Formula as an Adjunct to Atorvastatin in the Treatment of Hemorrhagic Moyamoya Disease.
Moyamoya Disease
Recruiting
A Multicenter, Prospective, Double-blind, Randomized Controlled Study on the Efficacy and Safety of Ischemic Preconditioning Therapy Versus Optimal Medical Therapy in Reducing the Recurrence of Ischemic Events in Symptomatic Moyamoya Disease
Moya Moya Disease, Moyamoya Syndrome
Enrolling by invitation
Fufang Tongluo Decoction as an Adjunct to Aspirin and Atorvastatin for Ischemic Moyamoya Disease
Moyomoya Disease
Active, not recruitingObservational study
Florida Cerebrovascular Disease Biorepository and Genomics Center
Cerebrovascular Disease, Ischemic Stroke
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Huaqiu Zhang principal investigator
Tongji Hospital
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing iPSC-EVs in people with Moyamoya Disease, run by Huaqiu Zhang at 1 site in China.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 8 inclusion and 10 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 1 Oct 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in China. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Huaqiu Zhang (13419632963, lyc@tjh.tjmu.edu.cn), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Incidence of Treatment-Emergent Adverse Events [Safety and Tolerability] (time frame: 3 Day、2 week、1 month). The study lists 3 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 1 Oct 2025.