This is an exploratory clinical trial aimed at preliminarily evaluating the efficacy, safety, and feasibility of orally administered Gut-X-001 in patients with Alzheimer's disease (AD). An open-label extension (OLE) study will also be conducted to further investigate the effects of Gut-X-001. The study will assess the effects of Gut-X-001 on cognitive function, activities of daily living, neuroimaging indicators, and AD-related plasma biomarkers in AD patients. Safety will be systematically monitored, including the incidence of adverse events and changes in hematological and organ function parameters. Furthermore, the study will explore the regulatory effects of Gut-X-001 versus placebo on venous blood redox-related indicators and gut microbiota metabolite levels at different time points, providing a basis for multi-target intervention strategies and offering systematic evidence for the scientific rationale, feasibility, and safety of Gut-X-001 in the clinical management of AD.
Age ≥50 and ≤85 years.
Diagnosis of Alzheimer's disease confirmed by a qualified neurologist based on the 2024 National Institute on Aging - Alzheimer's Association (NIA-AA) diagnostic criteria, with at least one abnormal core biomarker, including amyloid PET, CSF Aβ42/40, phosphorylated tau181 (p-tau181)/Aβ42, total tau (t-tau)/Aβ42, or plasma p-tau217.
Clinical Dementia Rating (CDR) global score of 0.5 (for MCI due to AD) or 1.0 (for mild AD dementia).
If receiving acetylcholinesterase inhibitor (AChEI) and/or memantine therapy, the dose must have been stable for at least 3 months prior to screening.
Presence of other conditions that may contribute to cognitive impairment, including neurological disorders (e.g., vascular cognitive impairment, Parkinson's disease, frontotemporal dementia, Lewy body dementia) or psychiatric and affective disorders (e.g., severe anxiety/depression, schizophrenia).
Diagnosis of acute cerebral infarction, cerebral hemorrhage, subarachnoid hemorrhage, myocardial infarction, or heart failure within the 3 months prior to screening.
Presence of other active or significant neurological conditions, including recurrent epileptic seizures, intracranial space-occupying tumors, vascular malformations (including arteriovenous malformations, arterial malformations, or cavernous malformations), or untreated aneurysms with a diameter >3 mm.
Severe hepatic impairment [ALT or AST >3× upper limit of normal (ULN), or concurrent acute hepatitis, chronic active hepatitis, or liver cirrhosis], renal impairment [estimated glomerular filtration rate (eGFR) <45 mL/min/1.73 m²], active malignancy, severe anemia, chronic obstructive pulmonary disease (COPD), immune system disorders, uncontrolled diabetes, or uncontrolled hypertension [systolic blood pressure ≥160 mmHg or diastolic blood pressure ≥100 mmHg].
Beijing Tiantan Hospital
Lead sponsor
Beijing University of Chemical Technology
Collaborator
Jilin University
Collaborator