Clinical Research Directory

Browse clinical research sites, groups, and studies.

Sites Groups Studies

Back to Studies

NOT YET RECRUITING

NCT07554872

PHASE1

Study of Neural Stem Cell-Derived Exosomes in Moderate-to-Severe Early-Onset Alzheimer's Disease

Sponsor: Shanghai Mental Health Center

View on ClinicalTrials.gov

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.

Official title: A Phase I, Open-Label, Single-Center, Frequency-Escalation Study of the Safety, Tolerability, and Preliminary Efficacy of Neural Stem Cell-Derived Exosomes in Patients With Moderate-to-Severe Early-Onset Alzheimer's Disease

Key Details

Gender

All

Age Range

50 Years - 75 Years

Study Type

INTERVENTIONAL

Enrollment

Start Date

2026-05

Completion Date

2027-12

Last Updated

2026-04-28

Healthy Volunteers

Conditions

Alzheimer Disease (AD)

Interventions

BIOLOGICAL

Neural Stem Cell-Derived Exosomes

The 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.

* 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 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 psychiatric disorder or symptoms. * Significant active physical illness, including severe cardiac disease, severe systemic infection, or severe liver/kidney dysfunction. * Elevated tumor markers or tumor history. * Immune-related disease. * Significant nasal obstruction. * Serious suicide risk. * Participation in another clinical trial or stem cell therapy within the past 6 months. * Any other condition judged inappropriate by the investigator. * Contraindications to MRI or inability to complete MRI examinations, including non-MRI-compatible metallic implants, certain stents, plates, pacemakers, or severe claustrophobia.

Locations (1)

Shanghai Mental Health Center

Shanghai, Shanghai Municipality, China