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.
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
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 psychiatric disorder or symptoms.
Sequential
Neural Stem Cell-Derived Exosomes
Biological/VaccineThe investigational product is a neural stem cell-derived exosome preparation administered intranasally. The product specification is 6 × 10\^9 particles per 2 mL vial. The product is thawed to room temperature before administration and delivered into both nostrils. The same investigational product is used in all study groups; the groups differ only in dosing frequency.
Number of Participants With Treatment-Related Adverse Events
Number of participants experiencing treatment-related adverse events assessed according to Common Terminology Criteria for Adverse Events (CTCAE) version 4.0.
Number of Participants With Treatment-Related Clinical Laboratory Abnormalities
Number of participants with treatment-related clinical laboratory abnormalities during the treatment period.
Change in Chinese Mini-Mental Status (CMMS) Score
Cognitive function will be assessed using the Chinese Mini-Mental Status (CMMS), a Chinese-language screening instrument for global cognitive impairment. Total scores range from 0 to 30, with higher scores indicating better cognitive performance.
Change in Severe Impairment Battery (SIB) Score
Cognitive function will be assessed using the Severe Impairment Battery (SIB), a validated instrument for cognitive function in moderate-to-severe dementia. Total scores range from 0 to 100, with higher scores indicating better cognitive performance.
Change in Neuropsychiatric Inventory (NPI) Score
Neuropsychiatric symptoms will be assessed using the Neuropsychiatric Inventory (NPI), a standardized instrument for behavioral and psychological symptoms in dementia. Total scores range from 0 to 144, with higher scores indicating greater neuropsychiatric symptom burden.
Change in Geriatric Depression Scale (GDS) Score
Depressive symptoms will be assessed using the 30-item Geriatric Depression Scale (GDS-30), a questionnaire designed to assess depressive symptoms in older adults. Total scores range from 0 to 30, with higher scores indicating more severe depressive symptoms.
Click on the lead sponsor to view all of their trials.
Shanghai Mental Health Center
Lead sponsor
Shanghai Angecon Biotechnology Co., Ltd.
Collaborator
This trial is not recruiting at the moment. You can still explore other options:
