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    Clinical Trial Results:
    Evaluating rapamycin treatment in Alzheimer’s disease using positron emission tomography (ERAP)

    Summary
    EudraCT number
    		 2023-000127-36
    Trial protocol
    		 SE  
    Global end of trial date
    11 Dec 2024

    Results information
    Results version number
    		 v1(current)
    This version publication date
    24 Oct 2025
    First version publication date
    24 Oct 2025
    Other versions

    Trial information

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    Trial identification
    Sponsor protocol code
    KIH22001
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 NCT06022068
    WHO universal trial number (UTN)
    		 -
    Sponsors
    Sponsor organisation name
    Karolinska Institutet
    Sponsor organisation address
    Nobels väg 15a, Stockholm, Sweden, 17177
    Public contact
    Brain Molecular Imaging Centre, Karolinska Institutet, jonas.svensson@ki.se
    Scientific contact
    Brain Molecular Imaging Centre, Karolinska Institutet, 46 702753831, jonas.svensson@ki.se
    Paediatric regulatory details
    Is trial part of an agreed paediatric investigation plan (PIP)
    No
    Does article 45 of REGULATION (EC) No 1901/2006 apply to this trial?
    No
    Does article 46 of REGULATION (EC) No 1901/2006 apply to this trial?
    No
    Results analysis stage
    Analysis stage
    Final
    Date of interim/final analysis
    20 Aug 2025
    Is this the analysis of the primary completion data?
    Yes
    Primary completion date
    11 Dec 2024
    Global end of trial reached?
    Yes
    Global end of trial date
    11 Dec 2024
    Was the trial ended prematurely?
    No
    General information about the trial
    Main objective of the trial
    To evaluate the efficacy of sirolimus in patients suffering from MCI or early-stage AD.
    Protection of trial subjects
    The study was approved by the Swedish Medical Products Agency (5.1‐2023‐8283) and the Swedish Ethical Review Authority (2023‐03075‐02 and 2023‐00611‐01). Written informed consent was obtained from all participants and their designated study partners before any study procedures were initiated.
    Background therapy
    		 -
    Evidence for comparator
    		 -
    Actual start date of recruitment
    01 Sep 2023
    Long term follow-up planned
    No
    Independent data monitoring committee (IDMC) involvement?
    No
    Population of trial subjects
    Number of subjects enrolled per country
    Country: Number of subjects enrolled
    Sweden: 14
    Worldwide total number of subjects
    14
    EEA total number of subjects
    14
    Number of subjects enrolled per age group
    In utero
    0
    Preterm newborn - gestational age < 37 wk
    0
    Newborns (0-27 days)
    0
    Infants and toddlers (28 days-23 months)
    0
    Children (2-11 years)
    0
    Adolescents (12-17 years)
    0
    Adults (18-64 years)
    12
    From 65 to 84 years
    2
    85 years and over
    0

    Subject disposition

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    Recruitment
    Recruitment details
    		 Participants were recruited from the Memory Clinic patient population. Some patients had previously consented to be contacted about clinical trial opportunities, while others expressed interest after being informed about the trial by their treating physician.

    Pre-assignment
    Screening details
    		 Inclusion criteria: amyloid-positivity, age between 50 and 89 years of age, has amyloid-positivity with a clinical diagnosis of mild cognitive impairment (MCI) or mild dementia of Alzheimer's type according to the NIA-AA 2018 criteria.

    Period 1
    Period 1 title
    Overall trial (overall period)
    Is this the baseline period?
    		 Yes
    Allocation method
    Not applicable
    Blinding used
    		 Not blinded
    Blinding implementation details
    Not applicable

    Arms
    Arm title
    		 Rapamycin
    Arm description
    		 -
    Arm type
    		 Experimental

    Investigational medicinal product name
    Rapamycin
    Investigational medicinal product code
    Other name
    Rapamune
    Pharmaceutical forms
    Tablet
    Routes of administration
    Oral use
    Dosage and administration details
    The first dose was 3 mg, which was increased to the target dose of 7 mg/week from the second week onward if well tolerated.

    Number of subjects in period 1
    		Rapamycin
    Started
    14
    Completed
    13
    Not completed
    1
         Adverse event, non-fatal
    1

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    Overall trial
    Reporting group description
    		 -

    Reporting group values
    		 Overall trial Total
    Number of subjects
    		 14 14
    Age categorical
    Units: Subjects
        In utero
    		 0
        Preterm newborn infants (gestational age < 37 wks)
    		 0
        Newborns (0-27 days)
    		 0
        Infants and toddlers (28 days-23 months)
    		 0
        Children (2-11 years)
    		 0
        Adolescents (12-17 years)
    		 0
        Adults (18-64 years)
    		 0
        From 65-84 years
    		 0
        85 years and over
    		 0
    Age continuous
    Units: years
        arithmetic mean (standard deviation)
    		 60.9 ( 4.2 ) -
    Gender categorical
    Units: Subjects
        Female
    		 8 8
        Male
    		 6 6

    End points

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    End points reporting groups
    Reporting group title
    Rapamycin
    Reporting group description
    		 -

    Subject analysis set title
    Baseline
    Subject analysis set type
    		 Full analysis
    Subject analysis set description
    15 days prior to first dose of rapamycin

    Subject analysis set title
    Follow-up
    Subject analysis set type
    		 Full analysis
    Subject analysis set description
    14 days post last dose of rapamycin

    Primary: Change in brain FDG-PET uptake

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    End point title
    		 Change in brain FDG-PET uptake
    End point description
    % change from baseline in FDG SUVR (temporoparietal lobe)
    End point type
    Primary
    End point timeframe
    Pre and post treatment
    End point values
    		 Baseline Follow-up
    Number of subjects analysed
    13
    13
    Units: unitless ratio
        arithmetic mean (standard deviation)
    1.04 ( 0.10 )
    1.04 ( 0.11 )
    Statistical analysis title
    		 Change in brain FDG-PET uptake
    Comparison groups
    Baseline v Follow-up
    Number of subjects included in analysis
    26
    Analysis specification
    Pre-specified
    Analysis type
    		 other [1]
    P-value
    		 = 0.991
    Method
    		 t-test, 2-sided
    Confidence interval
    Notes
    [1] - mean comparison

    Secondary: Change in CSF markers - Ab42

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    End point title
    		 Change in CSF markers - Ab42
    End point description
    Ab42 at baseline and at follow-up
    End point type
    Secondary
    End point timeframe
    pre and post treatment
    End point values
    		 Baseline Follow-up
    Number of subjects analysed
    10
    10
    Units: pg/mL
        arithmetic mean (standard deviation)
    614 ( 191 )
    653 ( 135 )
    Statistical analysis title
    		 Change in CSF markers protein concentration levels
    Comparison groups
    Baseline v Follow-up
    Number of subjects included in analysis
    20
    Analysis specification
    Pre-specified
    Analysis type
    		 other [2]
    P-value
    		 = 0.18
    Method
    		 t-test, 2-sided
    Confidence interval
    Notes
    [2] - mean comparison

    Secondary: Change in CSF markers - p-tau

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    End point title
    		 Change in CSF markers - p-tau
    End point description
    p-tau at baseline and at follow-up
    End point type
    Secondary
    End point timeframe
    pre and post treatment
    End point values
    		 Baseline Follow-up
    Number of subjects analysed
    10
    10
    Units: pg/mL
        arithmetic mean (standard deviation)
    107.10 ( 34.37 )
    110.10 ( 33.30 )
    Statistical analysis title
    		 Change in CSF markers protein concentration levels
    Comparison groups
    Baseline v Follow-up
    Number of subjects included in analysis
    20
    Analysis specification
    Pre-specified
    Analysis type
    		 other [3]
    P-value
    		 = 0.59
    Method
    		 t-test, 2-sided
    Confidence interval
    Notes
    [3] - mean comparison

    Secondary: Change in CSF markers - total-tau

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    End point title
    		 Change in CSF markers - total-tau
    End point description
    Total-tau at baseline and at follow-up
    End point type
    Secondary
    End point timeframe
    pre and post treatment
    End point values
    		 Baseline Follow-up
    Number of subjects analysed
    10
    10
    Units: pg/mL
        arithmetic mean (standard deviation)
    616 ( 150 )
    751 ( 206 )
    Statistical analysis title
    		 Change in CSF markers protein concentration levels
    Comparison groups
    Baseline v Follow-up
    Number of subjects included in analysis
    20
    Analysis specification
    Pre-specified
    Analysis type
    		 other [4]
    P-value
    		 = 0.003
    Method
    		 t-test, 2-sided
    Confidence interval
    Notes
    [4] - mean comparison

    Secondary: Change in cognition

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    End point title
    		 Change in cognition
    End point description
    Total Montreal Cognitive Assessment (MoCA) score at baseline and at follow-up
    End point type
    Secondary
    End point timeframe
    pre and post treatment
    End point values
    		 Baseline Follow-up
    Number of subjects analysed
    12
    12
    Units: MoCA sum score
        arithmetic mean (standard deviation)
    24.33 ( 2.93 )
    24.75 ( 4.61 )
    Statistical analysis title
    		 change in total MoCA score
    Comparison groups
    Baseline v Follow-up
    Number of subjects included in analysis
    24
    Analysis specification
    Pre-specified
    Analysis type
    		 other [5]
    P-value
    		 = 0.54
    Method
    		 t-test, 2-sided
    Confidence interval
    Notes
    [5] - mean comparison

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    1st Sep 2023 - 11th Dec 2024
    Assessment type
    		 Non-systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 NA
    Dictionary version
    NA
    Reporting groups
    Reporting group title
    Overall study
    Reporting group description
    		 -

    Serious adverse events
    		 Overall study
    Total subjects affected by serious adverse events
         subjects affected / exposed
    0 / 14 (0.00%)
         number of deaths (all causes)
    0
         number of deaths resulting from adverse events
    0
    Frequency threshold for reporting non-serious adverse events: 5%
    Non-serious adverse events
    		 Overall study
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    12 / 14 (85.71%)
    Injury, poisoning and procedural complications
    Wisdom teeth removal
         subjects affected / exposed
    1 / 14 (7.14%)
         occurrences all number
    1
    Erythrocyte sedimentation rate increased
    Additional description: increased erythrocyte sedimentation rate (ESR)
         subjects affected / exposed
    1 / 14 (7.14%)
         occurrences all number
    1
    creatine kinase increased
         subjects affected / exposed
    1 / 14 (7.14%)
         occurrences all number
    1
    Nervous system disorders
    Vertigo
         subjects affected / exposed
    1 / 14 (7.14%)
         occurrences all number
    2
    Paraesthesia
         subjects affected / exposed
    1 / 14 (7.14%)
         occurrences all number
    1
    Restless legs syndrome
    Additional description: crawling / tingling feeling in legs
         subjects affected / exposed
    1 / 14 (7.14%)
         occurrences all number
    1
    Ear and labyrinth disorders
    Tinnitus
    Additional description: worsening of pre-existing tinnitus
         subjects affected / exposed
    1 / 14 (7.14%)
         occurrences all number
    1
    Hearing disability
    Additional description: Trouble hearing
         subjects affected / exposed
    1 / 14 (7.14%)
         occurrences all number
    1
    Eye disorders
    Cataract
         subjects affected / exposed
    1 / 14 (7.14%)
         occurrences all number
    1
    Gastrointestinal disorders
    Diarrhoea
         subjects affected / exposed
    2 / 14 (14.29%)
         occurrences all number
    6
    mouth sore
         subjects affected / exposed
    4 / 14 (28.57%)
         occurrences all number
    9
    Dysgeusia
         subjects affected / exposed
    1 / 14 (7.14%)
         occurrences all number
    1
    Nausea
         subjects affected / exposed
    1 / 14 (7.14%)
         occurrences all number
    2
    Flatulence
         subjects affected / exposed
    1 / 14 (7.14%)
         occurrences all number
    1
    stomach pain
         subjects affected / exposed
    1 / 14 (7.14%)
         occurrences all number
    1
    Respiratory, thoracic and mediastinal disorders
    Cough
    Additional description: Dry cough
         subjects affected / exposed
    1 / 14 (7.14%)
         occurrences all number
    2
    Throat irritation
    Additional description: sore throat
         subjects affected / exposed
    1 / 14 (7.14%)
         occurrences all number
    1
    Skin and subcutaneous tissue disorders
    Hair growth abnormal
    Additional description: Regrowth of scalp hair
         subjects affected / exposed
    1 / 14 (7.14%)
         occurrences all number
    1
    Rash
    Additional description: skin rash
         subjects affected / exposed
    1 / 14 (7.14%)
         occurrences all number
    1
    Skin tightness
    Additional description: reduced facial wrinkles
         subjects affected / exposed
    1 / 14 (7.14%)
         occurrences all number
    1
    Psychiatric disorders
    feeling low
         subjects affected / exposed
    1 / 14 (7.14%)
         occurrences all number
    1
    Endocrine disorders
    Glucose tolerance impaired
    Additional description: increased fasting glucose
         subjects affected / exposed
    1 / 14 (7.14%)
         occurrences all number
    1
    Hot flush
         subjects affected / exposed
    1 / 14 (7.14%)
         occurrences all number
    1
    Thyroid hormones decreased
    Additional description: Increased levotyroxin dose
         subjects affected / exposed
    1 / 14 (7.14%)
         occurrences all number
    1
    Musculoskeletal and connective tissue disorders
    Skin discolouration
    Additional description: swelling / discoloration of hand
         subjects affected / exposed
    1 / 14 (7.14%)
         occurrences all number
    1
    Swelling
    Additional description: swelling of legs
         subjects affected / exposed
    1 / 14 (7.14%)
         occurrences all number
    2
    Infections and infestations
    Bacterial infection
         subjects affected / exposed
    2 / 14 (14.29%)
         occurrences all number
    2
    Upper respiratory tract infection
    Additional description: viral infections
         subjects affected / exposed
    2 / 14 (14.29%)
         occurrences all number
    2
    Metabolism and nutrition disorders
    increased serum lipids
         subjects affected / exposed
    1 / 14 (7.14%)
         occurrences all number
    1

    More information

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    Substantial protocol amendments (globally)
    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    08 Jun 2023
    Added magnetic resonance tomography measurements procedures as exploratory outcomes.

    Interruptions (globally)
    Were there any global interruptions to the trial? No

    Limitations and caveats
    Limitations of the trial such as small numbers of subjects analysed or technical problems leading to unreliable data.
    None reported
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