Study of Neural Stem Cell-Derived Exosomes in Moderate-to-Severe Early-Onset Alzheimer's Disease (NCT07554872) | Clinical Trial Compass
Not Yet RecruitingPhase 1
Study of Neural Stem Cell-Derived Exosomes in Moderate-to-Severe Early-Onset Alzheimer's Disease
China9 participantsStarted 2026-05
Plain-language summary
This is an open-label, single-center, phase I clinical study in patients with moderate-to-severe early-onset Alzheimer's disease. The study aims to evaluate the safety, tolerability, and preliminary efficacy of neural stem cell-derived exosomes (NSC-EVs) administered by the intranasal route. A total of 9 participants will be enrolled in 3 frequency-escalation groups: once every 3 days, once every other day, and once daily, each for 28 days. Participants will undergo screening and baseline assessment, a 28-day treatment period, and follow-up visits at 4, 8, and 24 weeks after the end of treatment.
Who can participate
Age range
50 Years – 75 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
* Inclusion Criteria:
* Male or postmenopausal female, aged 50 to 75 years.
* Meets the 2011 NIA-AA criteria for probable Alzheimer's disease dementia.
* Age at onset ≤65 years.
* CMMS score 5-20
* Stable dose for at least 2 months before enrollment if receiving pro-cognitive or psychiatric medications.
* Primary school education or above and able to complete study-required cognitive assessments.
* Hachinski Ischemic Score ≤4.
* GDS-30 total score ≤10.
* Screening brain MRI+DWI+SWI meeting protocol-defined cerebrovascular exclusion thresholds and no major structural abnormalities inconsistent with Alzheimer's disease.
* Positive amyloid pathology confirmed by Aβ-PET at screening or before enrollment.
* Adequate vision and hearing to complete assessments.
* Has a reliable caregiver able to accompany the participant to study visits and provide information for assessments.
* Willing to participate and sign informed consent.
Exclusion Criteria:
* Dementia due to causes other than Alzheimer's disease.
* Brain MRI showing any of the following: Fazekas white matter hyperintensity score \>2; more than 2 lacunar infarcts \>1.5 cm; lacunar infarcts involving critical regions such as the thalamus, hippocampus, entorhinal cortex, or parahippocampal region; cerebral hemorrhage, subdural hematoma, aneurysm, arteriovenous malformation, intracranial mass lesion, or other clinically significant structural abnormalities.
* Allergy to stem cell-derived exosomes or PET examination.
* Severe ps…
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1This is a Phase 1 trial focused on safety, not effectiveness — does that mean we won't really know yet whether the neural stem cell-derived exosomes actually help slow or reverse Alzheimer's symptoms, and how should that affect our decision about whether to consider it?
2The trial is listed as 'not yet recruiting,' so when might it actually open, and is it worth waiting for this study or would starting an available treatment now make more sense given where I am in my disease progression?
3Since the trial is specifically for moderate-to-severe early-onset Alzheimer's, how do my current symptoms and age compare to the kind of patient this study is designed for, and could that affect whether this would even be worth discussing as an option?
4The primary outcomes are tracking adverse events and lab abnormalities rather than cognitive improvement — what kinds of side effects or safety concerns should I be aware of that might come with a treatment involving exosomes derived from neural stem cells?
5Are there existing approved treatments or other clinical trials that might offer a better-understood risk-benefit profile for someone at my stage of Alzheimer's, compared to joining a first-in-human type Phase 1 safety study like this one?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
Number of Participants With Treatment-Related Adverse Events
Timeframe: Baseline to Week 4
2
Number of Participants With Treatment-Related Clinical Laboratory Abnormalities