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].
Parallel
Gut-X-001 + Placebo capsule
DrugParticipants will receive Gut-X-001 orally at a dose of 2 active capsules (10 mg of active ingredient per capsule) plus 2 placebo capsules (0 mg of active ingredient per capsule) per administration, 3 times daily (at 8:00 AM, 12:00 PM, and 10:00 PM), administered before meals and before bedtime. Total active ingredient per administration: 20 mg; total daily dose: 60 mg.
Gut-X-001
DrugParticipants will receive Gut-X-001 orally at a dose of 4 active capsules (10 mg of active ingredient per capsule) per administration, 3 times daily (at 8:00 AM, 12:00 PM, and 10:00 PM), administered before meals and before bedtime. Total active ingredient per administration: 40 mg; total daily dose: 120 mg.
Placebo
DrugParticipants will receive 4 placebo capsules (0 mg of active ingredient per capsule) orally per administration, 3 times daily (at 8:00 AM, 12:00 PM, and 10:00 PM), administered before meals and before bedtime. Placebo capsules are identical in appearance to the active Gut-X-001 capsules to maintain blinding.
Change from baseline in ADAS-Cog13 score at Month 6
Change from baseline in the Alzheimer's Disease Assessment Scale - Cognitive Subscale 13-item version (ADAS-Cog13) score. Higher scores indicate greater cognitive impairment.
Change from baseline in ADAS-Cog13 score at Month 12 (OLE)
Change from baseline and from Month 6 in ADAS-Cog13 score during the open-label extension (OLE) phase. Higher scores indicate greater cognitive impairment.
Change from baseline in ADCS-ADL score at Month 6 and Month 12 (OLE)
Change from baseline in the Alzheimer's Disease Cooperative Study - Activities of Daily Living (ADCS-ADL) scale score. Lower scores indicate greater functional impairment.
Change from baseline in PSQI score at Month 6 and Month 12 (OLE)
Change from baseline in the Pittsburgh Sleep Quality Index (PSQI) score. Higher scores indicate poorer sleep quality.
Change from baseline in NPI score at Month 6 and Month 12 (OLE)
Change from baseline in the Neuropsychiatric Inventory (NPI) score. Higher scores indicate more severe neuropsychiatric symptoms.
Rate of ≥90% medication adherence at Month 6
Proportion of participants who complete at least 90% of the prescribed dosing regimen during the double-blind phase.
Incidence of clinically significant abnormal CBC findings at Day 7, Month 3, Month 6, and Month 12 (OLE)
Proportion of participants meeting any of the following criteria: (i) anemia (hemoglobin \<90 g/L in males or \<85 g/L in females, or requiring transfusion); (ii) thrombocytopenia (platelets \<50.0×10⁹/L or requiring transfusion); (iii) leukopenia (WBC \<1.5×10⁹/L, lymphocytes \<0.5×10⁹/L, or neutrophils \<0.60×10⁹/L); or (iv) any CBC abnormality deemed clinically significant by the investigator.
Incidence of clinically significant hepatic biochemical abnormalities at Day 7, Month 3, Month 6, and Month 12 (OLE)
Proportion of participants meeting any of the following criteria: (i) ALT or AST \>5× ULN; (ii) ALT or AST \>3× ULN with total bilirubin \>2× ULN (Hy's Law); or (iii) ALT or AST \>3× ULN with symptoms of hepatic injury (e.g., nausea, vomiting, jaundice).
Incidence of acute kidney injury or significant renal function deterioration at Day 7, Month 3, Month 6, and Month 12 (OLE)
Proportion of participants meeting any of the following criteria: (i) acute: serum creatinine increase ≥1.5-fold from baseline (confirmed or presumed to have occurred within 7 days) or increase ≥26.5 µmol/L (0.3 mg/dL) within 48 hours; or (ii) chronic: eGFR decrease ≥30% from baseline.
Click on the lead sponsor to view all of their trials.
Beijing Tiantan Hospital
Lead sponsor
Beijing University of Chemical Technology
Collaborator
Jilin University
Collaborator
This trial is not recruiting at the moment. You can still explore other options:
