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NCT04200911: CARPE_DIEM

Cognition, Age, and RaPamycin Effectiveness - DownregulatIon of thE mTor Pathway

Completed EARLY_PHASE1 Results posted Last updated 8 January 2025
What this trial tests

EARLY_PHASE1 trial testing Rapamune in Cognitive Impairment, Mild in 10 participants. Completed in 13 January 2022.

Timeline
1 June 2020
Primary endpoint
13 January 2022
13 January 2022

Quick facts

Lead sponsorThe University of Texas Health Science Center at San Antonio
PhaseEARLY_PHASE1
StatusCompleted
Study typeINTERVENTIONAL
Allocationna
Designsingle group
Maskingnone
Primary purposetreatment
Enrollment10
Start date1 June 2020
Primary completion13 January 2022
Estimated completion13 January 2022
Sites1 location across United States

Drugs / interventions tested

Conditions studied

Sponsor

The University of Texas Health Science Center at San Antonio

Who can join

Adults 55 to 85, any sex, with Cognitive Impairment, Mild or Alzheimer Disease. Patients with the condition only — healthy volunteers not accepted.

Results — posted to ClinicalTrials.gov

Per-arm endpoint measurements with 95% confidence intervals where reported. Source: trial results section.

Blood Brain Barrier Penetration of RAPA Primary · 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. Change is calculated as value at 8 weeks minus the value at baseline.

GroupValue95% CI
RAPA Intervention0± 0
Adverse Events Secondary · Baseline to 8 weeks

Number of adverse events experienced across all 10 subjects after they were enrolled and randomized to treatment, regardless of relatedness to intervention.

GroupValue95% CI
RAPA Intervention20
Change in Vitals From Baseline to 8 Weeks Secondary · Baseline to 8 weeks

Evaluation of vitals. Change is calculated as value at 8 weeks minus the value at baseline.

systolic blood pressure
GroupValue95% CI
RAPA Intervention10.45± 7.82
diastolic blood pressure
GroupValue95% CI
RAPA Intervention1.7± 5.87
Percentage of Study Drug Pills Taken Secondary · Baseline to 8 weeks

Average percentage of study drug pills taken across all 10 subjects after they were enrolled and randomized to treatment. The percentage of study drug pills taken was evaluated by having participants return any leftover study drug pills at each visit during the active treatment period.

GroupValue95% CI
RAPA Intervention92± 5
Change in CSF AD Biomarkers From Baseline to 8 Weeks Secondary · Baseline to 8 weeks

Evaluation of CSF AD biomarkers. Change is calculated as value at 8 weeks minus the value at baseline.

CSF ptau-181
GroupValue95% CI
RAPA Intervention2.64± 2.72
CSF abeta 40
GroupValue95% CI
RAPA Intervention615.57± 1210.06
CSF Abeta42
GroupValue95% CI
RAPA Intervention21.52± 62.12
CSF GFAP
GroupValue95% CI
RAPA Intervention6262.21± 4928.96
CSF NFL
GroupValue95% CI
RAPA Intervention367.18± 310.71
Change in Plasma AD Biomarkers From Baseline to 8 Weeks Secondary · Baseline to 8 weeks

Evaluation of plasma AD biomarkers. Change is calculated as value at 8 weeks minus the value at baseline.

plasma ptau-181
GroupValue95% CI
RAPA Intervention-0.22± 0.47
plasma abeta 40
GroupValue95% CI
RAPA Intervention-8.86± 12.28
Plasma Abeta42
GroupValue95% CI
RAPA Intervention-0.67± 0.69
Plasma GFAP
GroupValue95% CI
RAPA Intervention-27.70± 64.78
Plasma NFL
GroupValue95% CI
RAPA Intervention0.84± 4.64
Change in CSF Inflammatory Markers From Baseline to 8 Weeks Secondary · Baseline to 8 weeks

Evaluation of CSF inflammatory markers. Change is calculated as value at 8 weeks minus the value at baseline.

IL-10
GroupValue95% CI
RAPA Intervention0.02± 0.02
IL-6
GroupValue95% CI
RAPA Intervention-0.02± 0.25
IL-8
GroupValue95% CI
RAPA Intervention5.76± 9.10
TNF-α
GroupValue95% CI
RAPA Intervention0.03± 0.07
IL-12
GroupValue95% CI
RAPA Intervention0.35± 0.53
IL-15
GroupValue95% CI
RAPA Intervention0.29± 0.28
IL-16
GroupValue95% CI
RAPA Intervention0.63± 3.40
IL-17a
GroupValue95% CI
RAPA Intervention0.03± 0.09
Change in Plasma Inflammatory Markers From Baseline to 8 Weeks Secondary · Baseline to 8 weeks

Evaluation of plasma inflammatory markers. Change is calculated as value at 8 weeks minus the value at baseline.

IL-10
GroupValue95% CI
RAPA Intervention-0.18± 0.53
IL-6
GroupValue95% CI
RAPA Intervention0.29± 0.54
IL-8
GroupValue95% CI
RAPA Intervention0.16± 1.57
TNF-α
GroupValue95% CI
RAPA Intervention0.06± 0.44
IL-12
GroupValue95% CI
RAPA Intervention-16.56± 38.52
IL-15
GroupValue95% CI
RAPA Intervention0.12± 0.31
IL-16
GroupValue95% CI
RAPA Intervention16.18± 42.70
IL-17a
GroupValue95% CI
RAPA Intervention0.36± 1.68
Safety Labs - Change in White Blood Cell and Platelet Counts From Baseline to 8 Weeks Secondary · Baseline to 8 weeks

Evaluation of safety labs - white blood cell and platelet counts. Change is calculated as value at 8 weeks minus the value at baseline.

White Blood Cell Count
GroupValue95% CI
RAPA Intervention0.42± 1.92
Immature Granulocytes
GroupValue95% CI
RAPA Intervention-0.21± 1.19
Eosinophils
GroupValue95% CI
RAPA Intervention0.10± 2.5
Basophils
GroupValue95% CI
RAPA Intervention-0.19± 0.40
Platelets
GroupValue95% CI
RAPA Intervention20.60± 35.19
Safety Labs - Change in Red Blood Cell Count Secondary · Baseline to 8 weeks

Evaluation of safety labs - red blood cell counts. Change in red blood cell count calculated as value at 8 weeks minus the value at baseline.

GroupValue95% CI
RAPA Intervention-0.01± 0.27
Safety Labs - Change in Mean Corpuscular Volume Secondary · 8 weeks

Evaluation of safety labs - Change in Mean Corpuscular volume. Change is calculated as value at 8 weeks minus the value at baseline

GroupValue95% CI
RAPA Intervention-2.70± 2.16
Safety Labs - Change in Mean Corpuscular Hemoglobin Secondary · 8-weeks

safety labs - Mean Corpuscular Hemoglobin. Change is calculated as value at 8 weeks minus the value at baseline.

GroupValue95% CI
RAPA Intervention-0.81± 0.69

Adverse events — posted to ClinicalTrials.gov

Time frame: 8 weeks. Reporting threshold: 0%. Adverse-event reports describe events observed during the trial — not all are caused by the drug.

RAPA Intervention
Serious: 1/10 (10%)
Deaths: 0/10

Serious adverse events (1 terms)

ReactionSystemRAPA Intervention
Altered Mental StatusNervous system disorders—
Other adverse events (13 terms — click to expand)

ReactionSystemRAPA Intervention
Leg edemaBlood and lymphatic system disorders—
FallGeneral disorders—
Intermittent RashSkin and subcutaneous tissue disorders—
HeadacheNervous system disorders—
Urinary retentionRenal and urinary disorders—
HyperkalemiaBlood and lymphatic system disorders—
Upper respiratory infectionRespiratory, thoracic and mediastinal disorders—
Skin infectionSkin and subcutaneous tissue disorders—
EmesisGastrointestinal disorders—
CoughRespiratory, thoracic and mediastinal disorders—
DiarrheaGastrointestinal disorders—
Urinary incontinenceRenal and urinary disorders—
Urinary urgencyRenal and urinary disorders—

Most-reported serious reactions: Altered Mental Status.

Data from ClinicalTrials.gov NCT04200911 adverse events section.

Sponsor's own description

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

Publications & conference data

8 peer-reviewed publications reference this trial (live from Europe PMC):

  1. Epigenetic regulation of aging: implications for interventions of aging and diseases.
    Wang K, Liu H, Hu Q, Wang L, et al · · 2022 · cited 406× · PMID 36336680 · DOI 10.1038/s41392-022-01211-8
  2. Alzheimer's disease drug development pipeline: 2023.
    Cummings J, Zhou Y, Lee G, Zhong K, et al · · 2023 · cited 326× · PMID 37251912 · DOI 10.1002/trc2.12385
  3. Role of lysosomes in physiological activities, diseases, and therapy.
    Zhang Z, Yue P, Lu T, Wang Y, et al · · 2021 · cited 238× · PMID 33990205 · DOI 10.1186/s13045-021-01087-1
  4. Tauopathies: new perspectives and challenges.
    Zhang Y, Wu KM, Yang L, Dong Q, et al · · 2022 · cited 212× · PMID 35392986 · DOI 10.1186/s13024-022-00533-z
  5. Biological aging processes underlying cognitive decline and neurodegenerative disease.
    Gonzales MM, Garbarino VR, Pollet E, Palavicini JP, et al · · 2022 · cited 211× · PMID 35575089 · DOI 10.1172/jci158453
  6. Targeting lysosomes in human disease: from basic research to clinical applications.
    Cao M, Luo X, Wu K, He X. · · 2021 · cited 162× · PMID 34744168 · DOI 10.1038/s41392-021-00778-y
  7. The PI3K/Akt signaling axis in Alzheimer's disease: a valuable target to stimulate or suppress?
    Razani E, Pourbagheri-Sigaroodi A, Safaroghli-Azar A, Zoghi A, et al · · 2021 · cited 159× · PMID 34386944 · DOI 10.1007/s12192-021-01231-3
  8. Neurogenesis in aging and age-related neurodegenerative diseases.
    Culig L, Chu X, Bohr VA. · · 2022 · cited 150× · PMID 35490966 · DOI 10.1016/j.arr.2022.101636

Verify or expand the search:

Other trials of Rapamune

Trials testing the same drug.

Other recruiting trials for Cognitive Impairment, Mild

Currently open trials in the same condition.

Other The University of Texas Health Science Center at San Antonio trials

Trials by the same sponsor.

Verify against primary sources

Data sources for this page

Drug Landscape aggregates and links these public records for informational use only. Always verify against the primary source before clinical or regulatory decisions. Canonical URL: https://druglandscape.com/trial/NCT04200911.

Primary sources · FDA · ClinicalTrials.gov · EMA · SEC EDGAR · ChEMBL · Wikidata · full sourcing