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Cognition, Age, and RaPamycin Effectiveness - DownregulatIon of thE mTor Pathway ((CARPE DIEM))

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ClinicalTrials.gov Identifier: NCT04200911
Recruitment Status : Active, not recruiting
First Posted : December 16, 2019
Last Update Posted : February 4, 2022
Information provided by (Responsible Party):
The University of Texas Health Science Center at San Antonio

Brief Summary:
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

Condition or disease Intervention/treatment Phase
Cognitive Impairment, Mild Alzheimer Disease Drug: Rapamune Early Phase 1

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.

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 10 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Cognition, Age, and RaPamycin Effectiveness - DownregulatIon of thE mTor Pathway (CARPE DIEM)
Actual Study Start Date : June 1, 2020
Actual Primary Completion Date : January 13, 2022
Estimated Study Completion Date : January 2023

Arm Intervention/treatment
Experimental: RAPA intervention
Sirolimus 1mg orally once a day for 8 weeks
Drug: Rapamune
Sirolimus 1mg capsules
Other Name: Sirolimus

Primary Outcome Measures :
  1. Blood brain barrier penetration of RAPA [ Time Frame: Change from Baseline to 8 weeks ]
    Lumbar punctures will be performed at baseline and after the final RAPA dose, to assess CSF levels of the drug.

Secondary Outcome Measures :
  1. Adverse events [ Time Frame: Baseline to 8 weeks ]
    Number of adverse events experienced

  2. Target engagement of RAPA treatment in Alzheimer's Disease (AD) [ Time Frame: Baseline and 8 weeks ]
    Evaluation of relevant AD biomarkers

  3. Mini-Mental State Exam (MMSE) [ Time Frame: Change from Baseline to 8 weeks ]
    A 30 point questionnaire used to measure cognitive impairment

  4. Alzheimer's Disease Assessment Scale (ADAS-Cog) Word list Immediate and Delayed [ Time Frame: Change from Baseline to 8 weeks ]
    A scale used to assess progression of Alzheimer's Disease

  5. Electronic Gait Mapping [ Time Frame: Change from Baseline to 8 weeks ]
    Assessment of physical functioning under single and dual task conditions

  6. Grip Strength [ Time Frame: Change from Baseline to 8 weeks ]
    Assessment of physical function using grip strength

  7. Clinical Dementia Rating (CDR) [ Time Frame: Change from Baseline to 8 weeks ]
    A 5 point scale used to characcterize six domains of cognitive an functional performance

  8. Benson Figure Copy [ Time Frame: Change from Baseline to 8 weeks ]
    A scale used to test memory recall

  9. Tablet-Based Cognitive Assessment (TabCat) [ Time Frame: Change from Baseline to 8 weeks ]
    A scale used to score cognitive assessment based on a number of assigned tasks

  10. Global Deterioration Scale [ Time Frame: Change from Baseline to 8 weeks ]
    A caregiver assessment of the stage of AD progression from Stage 1-7

  11. Neuropsychiatric Inventory (NPI) [ Time Frame: Change from Baseline to 8 weeks ]
    A scale to assess dementia-related behavioral symptoms

  12. Functional activities questionnaire (FAQ) [ Time Frame: Change from Baseline to 8 weeks ]
    An informant rates the subject's ability using a scoring system

  13. Pittsburgh Sleep Quality Index (PSQI) [ Time Frame: Change from baseline to 8 weeks ]
    A questionnaire used to assess sleep quality

Information from the National Library of Medicine

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Ages Eligible for Study:   55 Years to 85 Years   (Adult, Older Adult)
Sexes Eligible for Study:   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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04200911

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United States, Texas
UTHSA McDermott Clinical Sciences Building
San Antonio, Texas, United States, 78229
Sponsors and Collaborators
The University of Texas Health Science Center at San Antonio
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Principal Investigator: Mitzi Gonzales, PhD UT Health San Antonio
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Responsible Party: The University of Texas Health Science Center at San Antonio
ClinicalTrials.gov Identifier: NCT04200911    
Other Study ID Numbers: HSC20190850H
First Posted: December 16, 2019    Key Record Dates
Last Update Posted: February 4, 2022
Last Verified: February 2022

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Product Manufactured in and Exported from the U.S.: Yes
Additional relevant MeSH terms:
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Alzheimer Disease
Cognitive Dysfunction
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Neurodegenerative Diseases
Neurocognitive Disorders
Mental Disorders
Cognition Disorders
Anti-Bacterial Agents
Anti-Infective Agents
Antibiotics, Antineoplastic
Antineoplastic Agents
Antifungal Agents
Immunosuppressive Agents
Immunologic Factors
Physiological Effects of Drugs