The Safety and Preliminary Efficacy of Exosomes Derived From Umbilical Cord Mesenchymal Stem Cell (CB-Exo-A600) in Mild to Moderate Alzheimer's Disease
This study is divided into two phases: a dose-escalation phase and an expansion cohort phase. The dose-escalation phase is a single-center study, while the expansion cohort phase is a multicenter, prospective, randomized, double-blind, placebo-controlled study.
• Age ≥ 50 years, male or female.
• Clinical diagnosis of AD (mild to moderate stage, corresponding to clinical stage 4-5 at screening according to the 2024 National Institute on Aging/Alzheimer's Association \[NIA/AA\] criteria).
• Patients with cerebrospinal fluid biomarker data supporting an AD diagnosis within the past 3 years, or a positive amyloid Positron Emission Tomography (PET) scan result within the past 3 years, or a plasma p-tau217 test result indicating brain amyloid positivity.
• Magnetic Resonance Imaging (MRI) or Computed Tomography (CT) scan performed within 6 months prior to screening shows findings consistent with the clinical diagnosis of mild to moderate AD and no other clinically significant comorbid pathologies, particularly cerebrovascular disease. If an MRI or CT scan is not available within the 6 months prior to screening, an MRI must be completed and results confirmed before the subject initiates treatment.
• Modified Hachinski Ischemic Score (mHIS) ≤ 4.
• Mini-Mental Status Examination (MMSE) score between 10 and 24 (inclusive).
• The subject has a clearly identified and reliable caregiver who meets the following criteria: able to independently read and understand relevant study documents at the study site and communicate necessary information with the investigator; willing to comply with clinical study procedures and ensure the provision of accurate information regarding the subject's status throughout the study; resides with the subject or provides care for the subject for no less than 2 hours per day on at least 3 days per week.
• Female subjects of childbearing potential (including women of reproductive age and those less than 1 year postmenopausal) must use effective contraceptive methods throughout the study.
• Patients have been on stable doses of cholinesterase inhibitors (e.g., donepezil, rivastigmine, galantamine, huperzine A), excitatory amino acid receptor antagonists (e.g., memantine), or other cognitive-enhancing medications (e.g., sodium oligomannate) for at least 60 days prior to enrollment and must continue on the same doses throughout the study period.
• The subject or their legal guardian voluntarily signs a written informed consent form and is able to comply with the study requirements for dosing and follow-up.