Purpose

Evaluation of central nervous system penetration of orally administered Rapamune (RAPA) in older adults with Mild Cognitive Impairment (MCI) or early Alzheimer's disease (AD) and investigate associated safety, tolerability, target engagement, cognition, and functional status as initial proof-of-concept study

Conditions

Eligibility

Eligible Ages
Between 55 Years and 85 Years
Eligible Genders
All
Accepts Healthy Volunteers
Yes

Inclusion Criteria

  • Diagnosis of Mild Cognitive Impairment (MCI), Clinical Dementia Rating Scale (CDR)=0.5-1; Hopkins Verbal Learning Test-Revised (HVLT-R) Delayed Recall ≤5% based on age-adjusted normal values, clinician approved
  • Normal blood cell counts without clinically significant excursions
  • A Legally Authorized Representative (LAR) if necessary for consent
  • An LAR or study partner to accompany participant to all visits
  • Availability for all study visits
  • Stable dose of AD medications) Donepezil, rivastigmine, memantine, galantamine) for at least 3 months

Exclusion Criteria

  • Diabetes (HbA1c≥6.5% or anti-diabetic medications)
  • History of skin ulcers or poor wound healing
  • Current tobacco or illicit drug use or alcohol abuse
  • Use of anti-platelet or anti-coagulant medications other than aspirin
  • Current medications that affect cytochrome P450 3A4
  • Immunosuppressant therapy within the last year
  • Chemotherapy or radiation treatment within the last year
  • Current or chronic history of liver disease or known hepatic or biliary abnormalities
  • Current or chronic history of pulmonary disease or abnormal pulse oximetry (<90%)
  • Chronic heart failure
  • Pregnancy
  • Recent history (past 6 months) of myocardial infarction, active coronary artery disease, intestinal disorders, stroke, or transient ischemic attack
  • significant neurological conditions other than AD
  • Poorly controlled blood pressure (systolic BP>160, diastolic BP>90mmHg)
  • Active inflammatory, autoimmune, infectious, hepatic, gastrointestinal, malignant, and/or psychiatric disease
  • History of, or Magnetic Resonance Imaging (MRI) positive for any space occupying lesion, including mass effect and/or abnormal intracranial pressure, which would indicate contraindication to lumbar puncture

Study Design

Phase
Early Phase 1
Study Type
Interventional
Allocation
N/A
Intervention Model
Single Group Assignment
Primary Purpose
Treatment
Masking
None (Open Label)

Arm Groups

ArmDescriptionAssigned Intervention
Experimental
RAPA intervention
Sirolimus 1mg orally once a day for 8 weeks
  • Drug: Rapamune
    Sirolimus 1mg capsules
    Other names:
    • Sirolimus

Recruiting Locations

UTHSA McDermott Clinical Sciences Building
San Antonio, Texas 78229
Contact:
Mitzi Gonzales, PhD
210-450-9047
gonzalesM20@uthscsa.edu

More Details

Status
Recruiting
Sponsor
The University of Texas Health Science Center at San Antonio

Study Contact

Mitzi Gonzales, PhD
210-450-9047
gonzalesM20@uthscsa.edu

Detailed Description

This study is an open-label pilot study of orally administered RAPA to measure its target engagement in Cerebrospinal Fluid (CSF) and blood, and to establish the feasibility and safety of RAPA treatment in older adults with MCI and early stage AD as initial proof-of-concept for a larger Phase 2 clinical trial.

Notice

Study information shown on this site is derived from ClinicalTrials.gov (a public registry operated by the National Institutes of Health). The listing of studies provided is not certain to be all studies for which you might be eligible. Furthermore, study eligibility requirements can be difficult to understand and may change over time, so it is wise to speak with your medical care provider and individual research study teams when making decisions related to participation.