NCT07728175 · University of Missouri-Columbia
Effects of Sirolimus on Asymptomatic ApoE4 Carriers
What this study is about
Alzheimer's disease is a devastating neurodegenerative disease characterized by accumulation of clumps (also called plaques) and bundles of fibers (also called tangles) in the brain, for which there is currently no cure. Sirolimus (Rapamycin) is an FDA-approved medication which may improve the blood flow to the brain.
View original scientific description
Alzheimer's disease is a devastating neurodegenerative disease characterized by accumulation of clumps (also called plaques) and bundles of fibers (also called tangles) in the brain, for which there is currently no cure. Sirolimus (Rapamycin) is an FDA-approved medication which may improve the blood flow to the brain. The purpose of the clinical trial is to find out whether sirolimus can help improve blood flow and energy use in the brain in women ages 45 to 65 who have the ApoE4 gene, a gene which increases the risk of developing Alzheimer's disease later in life. There will be two arms in this trail, a sirolimus arm and a placebo arm. A placebo is a pill that looks like the study drug, but it does not have any real medicine in it. Participants will be randomized to one arm or the other, but not to both arms. Three study visits over a 12-week period are required.
Interventions
DRUG
Sirolimus (rapamycin)
Participants in Sirolimus arm will receive Sirolimus.
DRUG
Placebo
Participants in the Placebo arm will receive Placebo
Primary outcome measures
Change in Cerebral Blood Flow as measured by MRI
Time frame: Baseline to 4 weeks
Rate of blood perfusion expressed as mL/100g/min globally and regionally
Who can participate
This study lists these criteria on ClinicalTrials.gov. A study coordinator reviews eligibility during screening — this page does not determine whether you qualify.
Inclusion criteria
- Willing and able to provide informed consent
- Sex assigned at birth: female
- 45-65 years old
- Able to perform self-care and activities of daily living with no or minimal assistance
- Post-menopausal status or use of highly effective contraception. Post-menopausal is defined as either
- 12 months of spontaneous amenorrhea with an appropriate clinical profile (e.g., age-appropriate, history of vasomotor symptoms)
- surgical bilateral oophorectomy (with or without hysterectomy), total hysterectomy, or tubal ligation at least 6 weeks prior to screening. For oophorectomy alone, post-menopausal status must be confirmed by follow-up hormone level assessment
- Highly effective contraception includes intrauterine devices (IUD), oral contraceptives, or other hormonal contraceptives including injectables, transdermal patches, implants or vaginal rings, or refraining from heterosexual intercourse during the 4 weeks of taking the study drug and for 2 weeks after no longer taking the study drug
- Body weight of ≥ 40 kg
- Ability to effectively communicate with the investigator and comply with study requirements
Exclusion criteria
- Diagnosis of mild cognitive impairment (MCI), dementia, or Alzheimer's disease
- BMI ≥ 35 (based on MRI feasibility)
- Type 1 diabetes or poorly controlled type 2 diabetes (HbA1c ≥ 6.5%)
- History of skin ulcers or poor wound healing
- Current tobacco or illicit drug use or alcohol abuse (defined as ≥ 3 per day or ≥ 7 per week for women) (Per NIAAA guidelines)
- Use of anti-platelet or anti-coagulant medications other than aspirin
- Required use of medications that affect cytochrome P450 3A4 (CYP3A4) or alter cerebral blood flow (see Appendix 1 for tables of excluded medications)
- Immunosuppressant therapy within the last year
- Chemotherapy or radiation treatment within the last year
- Current or chronic history of liver or kidney disease or known hepatic or biliary abnormalities
- Untreated hypertriglyceridemia (fasting triglycerides \< 300 mg/dl)
- Current or chronic significant history of pulmonary disease
- Chronic heart failure
- Pregnancy or lactation
- Recent history (past six months) of myocardial infarction, active coronary artery disease, intestinal disorders, stroke, or transient ischemic attack
- Poorly controlled blood pressure (systolic BP \> 160 or diastolic BP \> 100 mmHg)
- Active inflammatory, COVID-19, autoimmune, infectious, hepatic, gastrointestinal, malignant, and/or severe mental illness
- History of, or MRI, or CT positive for, any space occupying brain lesion, including mass effect or abnormal intracranial pressure
- Organ transplant recipients
- History of Stroke
- History of ruptured intracranial aneurysm
- History of malignancy within the past 2 years, with the following exceptions:
- Localized basal cell or squamous cell carcinoma of the skin
- Prostate cancer confined to the gland (AJCC stage T2N0M0 or better)
- Cervical carcinoma in situ
- Breast cancer localized to the breast
- History of epilepsy
Where
- Columbia, Missouri
Related conditions & keywords
Frequently asked questions
What is a clinical trial?
A clinical trial is a research study that tests new medical treatments, drugs, devices, or procedures to determine their safety and effectiveness. Trials are carefully designed and monitored to protect participants while advancing medical knowledge.
Is it safe to participate?
Clinical trials follow strict safety guidelines and ethical standards. Trials must be reviewed and approved, and participants are closely monitored by medical professionals throughout the study. You can withdraw at any time if you choose.
Will I be compensated?
Many clinical trials offer compensation for your time, travel expenses, and inconvenience. The specific compensation varies by study and will be discussed during the screening process. All study-related medical care is typically provided at no cost to participants.
Will I receive a placebo instead of treatment?
When effective treatment exists, participants typically receive either the standard treatment plus the study intervention, or the standard treatment plus placebo. You would not be denied effective care. Placebos are primarily used when no proven treatment is available, or in addition to standard care. Your trial consent form will clearly explain what treatments you may receive.
Can I leave a trial if I change my mind?
Absolutely. Participation in clinical trials is completely voluntary. You have the right to withdraw from the study at any time, for any reason, without penalty or loss of benefits to which you are otherwise entitled.
How long does a clinical trial last?
Trial duration varies widely depending on the study design and purpose. Some trials last just a few weeks, while others may continue for months or years. The study coordinator will provide specific timeline information during your screening call.
Data: ClinicalTrials.gov · synced Oct 5, 2026 · Source of record for eligibility and locations