Sirolimus for Genetic Predisposition to Alzheimer's Disease

KR
NB
Overseen ByNathan Bresette
Age: 18+
Sex: Female
Trial Phase: Phase 1
Sponsor: University of Missouri-Columbia
Breakthrough TherapyThis drug has been fast-tracked for approval by the FDA given its high promise

What You Need to Know Before You Apply

What is the purpose of this trial?

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.

Participants will: (i) Complete some questionnaires about how well you think; (ii) Complete genetic testing for the ApoE4 gene; (iii) Have blood work, blood pressure and height and weight collected; (iv) Take either Sirolimus or a placebo daily, by mouth, for approximately 4 weeks; (v) Keep a diary of when the sirolimus or placebo is taken; (vi) Complete 2 Magnetic Resonance Imaging (MRI) exams

Who Is on the Research Team?

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Ai-Ling Lin, PhD

Principal Investigator

University of Missouri-Columbia

Are You a Good Fit for This Trial?

This trial is for women aged 45 to 65 who are healthy but carry the ApoE4 gene, which increases Alzheimer's risk. Participants must not have symptoms of Alzheimer's or other major health issues.

Inclusion Criteria

Willing and able to provide informed consent
I am between 45 and 65 years old.
I am post-menopausal due to natural or surgical reasons.
See 5 more

Exclusion Criteria

Current tobacco or illicit drug use or alcohol abuse defined as ≥ 3 drinks per day or ≥ 7 drinks per week for women
BMI ≥ 35 based on MRI feasibility
I have been diagnosed with mild cognitive impairment, dementia, or Alzheimer's disease.
See 20 more

What Are the Treatments Tested in This Trial?

Interventions

  • Sirolimus (rapamycin)

Trial Overview

The study tests whether taking sirolimus (rapamycin) daily for about 4 weeks can improve brain blood flow and energy use in ApoE4 carriers, compared to a placebo. Participants are randomly assigned to either group.

How Is the Trial Designed?

2

Treatment groups

Active Control

Placebo Group

Group I: SirolimusActive Control1 Intervention
Group II: PlaceboPlacebo Group1 Intervention

Find a Clinic Near You

Who Is Running the Clinical Trial?

University of Missouri-Columbia

Lead Sponsor

Trials
387
Recruited
629,000+