An Evaluation of Treatments for Sustained Clinical Response (18 Months) in Symptomatic Alzheimer's Disease (AD-SMART)
Alzheimer's Disease- Systematic Multi-Arm Adaptive Randomised Trial
調査の概要
状態
詳細な説明
AD-SMART is a platform trial using a multi-arm multi-stage (MAMS) adaptive design.
Participants are randomised (1:1:1) to standard of care plus placebo, atomoxetine, or metformin. Interim analyses are conducted in stages (Stage 1, Stage 2, and Stage 3) to determine whether treatment arms should continue or stop early for lack of activity.
The primary objective is to assess therapeutic benefit by measuring change in cognitive function and activities of daily living over 18 months. Secondary outcomes include neuropsychiatric symptoms, quality of life, caregiver burden, safety, and health economic outcomes.
Exploratory analyses include blood biomarkers and MRI imaging to investigate disease progression and treatment response.
研究の種類
入学 (推定)
段階
- フェーズ 3
連絡先と場所
研究連絡先
- 名前:Trial Management Team UCL InCTU
- 電話番号:+44 (0)20 7670 4700
- メール:mrcctu.adsmart@ucl.ac.uk
研究場所
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Cambridge、イギリス、CB21 5EF
- 募集
- Windsor Research Unit
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Chertsey、イギリス、KT16 9AU
- 募集
- Two Bridges Research & Development Clinic
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参加基準
適格基準
就学可能な年齢
- 大人
- 高齢者
健康ボランティアの受け入れ
説明
Inclusion Criteria
Patient meets all inclusion criteria:
1. Adults aged ≥55 years on the day of screening, no upper age limit 2. Either:
- Confirmed clinical diagnosis of Alzheimer's Disease (AD)
- Confirmed clinical diagnosis of Mixed Dementia consisting of Alzheimer's Disease and Vascular Dementia 3. Mini Mental State Examination score of ≥17 4. Confirmatory blood biomarker testing (pTau-217) (positive or intermediate via validated assays) ≤ 365 days prior to screening or between screening and randomisation or a positive amyloid PET scan (if available) or a positive amyloid CSF test (if available) 5. Randomisation should ideally take place within 4 weeks of the screening visit but no later than 8 weeks after the screening visit 6. Must be able and willing to comply with the treatment and assessment schedule and requirements including being able to start trial treatment ≤ 2 weeks after randomisation 7. Willing and able to have MRI scans in accordance with the assessment schedule unless participant is clinically contraindicated due to:
- Pacemakers or defibrillators (unless MRI-conditional models)
- Aneurysm clips, stents or metal implants (unless MRI safe)
- Cochlear implants (unless MRI-conditional models)
- Metal fragments in the body
- Severe claustrophobia 8. Negative pregnancy test ≤4 weeks prior to randomisation for women of child-bearing potential 9. Normal liver function at screening consisting of all the following:
- Total serum bilirubin <1.5 x ULN (except for participants with Gilbert's disease, for whom the upper limit of total serum bilirubin is 51.3 μmol/l or 3mg/dl)
- Alanine aminotransferase (ALT) <3 x ULN;
- Alkaline phosphatase <3 x ULN 10. Documented participant and study partner informed consent 11. If a participant is being re-randomised into the trial, additional timing of entry requirements must also be met:
- For participants being re-randomised after completing 18 months' follow-up and the arm was not closed due to lack of activity, a 12-week washout period from last dose of IMP must be completed before their screening visit. If the efficacy analysis indicates that the IMP was ineffective then this washout period can be reduced to 6 weeks.
For participants being re-randomised following treatment arm termination due to lack of activity, a 6-week washout period from their last dose of IMP must be completed prior to screening assessment
Study Partner inclusion criteria:
- Participant that meets the AD-SMART eligibility criteria has consented to participation in the trial
- Has at least twice-weekly contact with participant
- Be 18 years or older at the time of providing consent
- Willing to complete study partner questionnaires as outlined in visit schedule
- Willing to attend remote and in-person study visits with participant
- Documented informed consent
Exclusion Criteria:
Patient meets none of the exclusion criteria:
- Fazekas Score =3 reported from an MRI taken at any time prior to randomisation, if an MRI is not clinically contraindicated (reasons specified in Inclusion Criteria 7) A. MRI does not need to be repeated if Fazekas score = 0, 1 or 2 reported from an MRI performed ≤365 days to screening visit (if Fazekas score has not been reported, refer to Section 7.4) b. MRI should be conducted if participant is not clinically contraindicated to MRIs and previous MRI where Fazekas score = 0, 1 or 2 was >365 days or previous MRI scan is not available for Fazekas score reporting or participant has never had an MRI
- Clinical diagnosis of Dementia with Lewy bodies
- Clinical diagnosis of Parkinson's disease
- Clinical diagnosis of Frontotemporal Dementia
- Cardiac failure (American Heart Association Stage C or D)
- Significant respiratory comorbidity (hospitalisation within the previous ≤6 months due to respiratory comorbidity)
- Renal failure (CKD IV or eGFR ≤45 mL/min/1.73m²) at any time point prior to randomisation
- Malignancy (except if in complete remission) e.g. solid organ or haematological or melanoma
- Score of ≥1 on C-SSRS at screening visit
- Individuals without an identified study partner (refer to Section 4 for further details on study partners)
- Individuals who have an Alzheimer's Disease or Central Nervous System medication (e.g. antidepressant) change or dose change ≤28 days prior to screening
- Use of an Investigational Medicinal Product (IMP) or Investigational Medical Device (IMD) ≤26 weeks prior to randomisation (except for AD-SMART participants that are being re-randomised. See Section 5.4 for further information).
- Receiving antibody-based amyloid clearing treatment for Alzheimer's Disease within 26 weeks prior to randomisation
- Unable or unwilling to comply with study procedures
- Unable to swallow whole capsules
- Individuals who are living in the same household as an AD-SMART participant who is actively taking trial medication
- Female participants that are pregnant or breastfeeding
- Women of child-bearing potential (WOCBP) who are unwilling or unable to use an acceptable method of contraception (see acceptable methods of contraception) whilst on trial treatment and up to 12 weeks after the last dose of study drug
- Male participants with a partner of child-bearing potential unwilling or unable to use an acceptable method of contraception whilst on trial treatment and up to 12 weeks after the last dose of the study drug.
- Male participants unwilling to desist from sperm donation during the trial and for 12 weeks after the last dose of trial treatment
- Current or previous exposure to any of the currently recruiting AD-SMART IMPs ≤26 weeks before randomisation.
- History of alcohol and/or drug abuse and/or dependence within the 5 years prior to screening visit.
- Any concurrent medical condition, abnormal laboratory tests or uncontrolled, clinically significant systemic disease that, in the opinion of the Investigator, could cause study participation to be detrimental to the participant.
- Participants who are not eligible for any of the trial IMPs, according to the eligibility criteria listed in the individual drug appendices. Please note that participants can enter the trial if they are eligible for at least one of the trial treatment arms, but do not need to be eligible for all.
Arm-specific eligibility criteria:
Atomoxetine-specific exclusion eligibility criteria:
In addition to the core inclusion and exclusion criteria, refer to section 2.2 of AD-SMART Appendix 1 Atomoxetine' for the arm-specific exclusion eligibility criteria for the Atomoxetine arm which must also be met.
Metformin-specific exclusion eligibility criteria:
In addition to the core inclusion and exclusion criteria, refer to section 2.2 of AD-SMART Appendix 2 Metformin' for the arm-specific exclusion eligibility criteria for the metformin arm which must also be met.
研究計画
研究はどのように設計されていますか?
デザインの詳細
- 主な目的:処理
- 割り当て:ランダム化
- 介入モデル:並列代入
- マスキング:トリプル
武器と介入
参加者グループ / アーム |
介入・治療 |
|---|---|
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プラセボコンパレーター:Standard of Care + Placebo
Placebo arm (Oral capsule matching active treatments)
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Oral capsule matching active treatments
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アクティブコンパレータ:Standard of Care + Atomoxetine
Atomoxetine (Oral, up to 100 mg/day)
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Atomoxetine (Oral, up to 100 mg/day)
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アクティブコンパレータ:Standard of Care + Metformin
Metformin (immediate release) Oral, up to 2000 mg/day
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Metformin IR Oral, up to 2000 mg/day
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この研究は何を測定していますか?
主要な結果の測定
結果測定 |
メジャーの説明 |
時間枠 |
|---|---|---|
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Change in Alzheimer's Disease Assessment Scale-Cognitive Subscale (ADAS-Cog) score
時間枠:Baseline to 18 months
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The Alzheimer's Disease Assessment Scale-Cognitive Subscale (ADAS-Cog) measures the severity of cognitive impairment in dementia.
Scores range from 0 to 70, with higher scores indicating worse cognitive performance (Unit of Measure: ADAS-Cog score (0-70).
ADAS-Cog will be administered at baseline, month 6, month 12, and month 18 (end of study), and change over time will be assessed.
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Baseline to 18 months
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Change in Amsterdam Instrumental Activities of Daily Living Questionnaire - Short Version (A-IADL-Q-SV) score
時間枠:Baseline to 18 months
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The Amsterdam Instrumental Activities of Daily Living Questionnaire - Short Version (A-IADL-Q-SV) assesses functional impairment in instrumental activities of daily living.
Scores range from 0 to 100, with higher scores indicating better functional performance (Unit of Measure: A-IADL-Q-SV score (0-100).
The A-IADL-Q-SV will be administered at baseline, month 6, month 12, and month 18 (end of study), and change over time will be evaluated.
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Baseline to 18 months
|
二次結果の測定
結果測定 |
メジャーの説明 |
時間枠 |
|---|---|---|
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Change in Neuropsychiatric Inventory (NPI) total score
時間枠:Baseline to 18 months
|
The Neuropsychiatric Inventory (NPI) is a structured, informant-based interview assessing neuropsychiatric and behavioural symptoms across 12 domains.
Total scores range from 0 to 144 (Unit of Measure: NPI total score (0-144)), with higher scores indicating more severe neuropsychiatric symptoms.
The NPI will be administered at baseline, month 6, month 12, and month 18 (end of study), and change over time will be evaluated.
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Baseline to 18 months
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Change in EQ-5D-5L health-related quality-of-life score (participant-reported)
時間枠:Baseline to 18 months
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The EQ-5D-5L (EuroQol 5-Dimension 5-Level) questionnaire measures participant-reported health-related quality of life across five domains: mobility, self-care, usual activities, pain/discomfort, and anxiety/depression.
Responses are converted into a health utility index score ranging from less than 0 (health states worse than death) to 1 (full health), with higher scores indicating better health-related quality of life (Unit of Measure: EQ-5D-5L utility score (<0 to 1)).
The EQ-5D-5L will be administered at baseline, month 3, month 6, month 12, month 15, and month 18 (end of study), and change over time will be assessed.
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Baseline to 18 months
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Change in EQ-5D-5L health-related quality-of-life score (study partner-reported)
時間枠:Baseline to 18 months
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The EQ-5D-5L (EuroQol 5-Dimension 5-Level) questionnaire measures study partner-reported health-related quality of life across five domains: mobility, self-care, usual activities, pain/discomfort, and anxiety/depression.
Responses are converted into a health utility index score ranging from less than 0 (health states worse than death) to 1 (full health), with higher scores indicating better health-related quality of life (Unit of Measure: EQ-5D-5L utility score (<0 to 1)).
The EQ-5D-5L will be administered at baseline, month 6, month 12, and month 18 (end of study), and change over time will be assessed.
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Baseline to 18 months
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Change in Zarit Burden Interview (ZBI) total score
時間枠:Baseline to 18 months
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The Zarit Burden Interview (ZBI) assesses subjective caregiver burden, including emotional, social, and physical strain associated with providing care.
Total scores range from 0 to 88, with higher scores indicating greater caregiver burden (Unit of Measure: ZBI total score (0-88)).
The ZBI will be administered at baseline, month 6, month 12, and month 18 (end of study), and change over time will be evaluated.
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Baseline to 18 months
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Incidence of treatment-emergent adverse events and serious adverse events
時間枠:Baseline to 18 months
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Safety profile of investigational medicinal products (IMPs), including all adverse events (AEs), serious adverse events (SAEs), adverse reactions (ARs), and suspected unexpected serious adverse reactions (SUSARs).
Events will be systematically collected, assessed, and analysed throughout the study from first dose until end of follow-up (Unit of Measure: Number of participants experiencing at least one AE or SAE)
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Baseline to 18 months
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Change in health and social care resource use and associated costs
時間枠:Baseline to 18 months
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Health and social care resource utilisation will be measured using a structured Resource Use Questionnaire (RUQ).
The RUQ captures participant use of primary care, secondary care, community services, urgent and emergency care, social care, and informal care.
It also collects information needed to estimate costs for economic evaluation.
Unit of Measure: Resource use counts and costs (GBP).
Resource use will be assessed at baseline (covering the preceding 6 months), month 6, month 12, and month 18 (end of study), and changes over time will be evaluated.
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Baseline to 18 months
|
その他の成果指標
結果測定 |
メジャーの説明 |
時間枠 |
|---|---|---|
|
Change in plasma pTau-217 concentration
時間枠:Baseline to 18 months
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Exploratory biomarker outcome assessing change in plasma pTau-217 levels (Unit of Measure: pg/mL)
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Baseline to 18 months
|
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MRI structural brain changes
時間枠:Baseline to 18 months
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Exploratory Outcome Measures
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Baseline to 18 months
|
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Change in plasma amyloid-β concentrations
時間枠:Baseline to 18 months
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Exploratory biomarker outcome assessing change in plasma amyloid-β isoforms (Unit of Measure: pg/mL)
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Baseline to 18 months
|
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Change in plasma neurofilament light chain (NfL) concentration
時間枠:Baseline to 18 months
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Exploratory biomarker outcome assessing change in plasma NfL levels (Unit of Measure: pg/mL)
|
Baseline to 18 months
|
協力者と研究者
研究記録日
主要日程の研究
研究開始 (推定)
一次修了 (推定)
研究の完了 (推定)
試験登録日
最初に提出
QC基準を満たした最初の提出物
最初の投稿 (実際)
学習記録の更新
投稿された最後の更新 (実際)
QC基準を満たした最後の更新が送信されました
最終確認日
詳しくは
本研究に関する用語
その他の研究ID番号
- ND003
- 70766 (その他の識別子:Central Portfolio Management System (CPMS))
- ISRCTN17108793 (レジストリ識別子:ISRCTN)
個々の参加者データ (IPD) の計画
個々の参加者データ (IPD) を共有する予定はありますか?
IPD プランの説明
Research teams may approach the UCL InCTU (mrcctu.adsmart@ucl.ac.uk) with a formal data-sharing request detailing the specific requirement, proposed research, qualification of researchers and publication plan if they are interested in using AD-SMART data. The request will be reviewed by the trial committees.
Data and/or samples will be available for sharing following the end of a trial arm and the unblinding of participants. Researchers wishing to access the AD-SMART Trial data should contact the Trial Management Group in the first instance. Following trial completion, requests for data and/ or sample sharing will be reviewed by an AD-SMART access committee, which will include the trial's Chief Investigators.
Data and/ or samples will be shared during the trial according to the CTU's controlled access approach.
IPD 共有時間枠
医薬品およびデバイス情報、研究文書
米国FDA規制医薬品の研究
米国FDA規制機器製品の研究
この情報は、Web サイト clinicaltrials.gov から変更なしで直接取得したものです。研究の詳細を変更、削除、または更新するリクエストがある場合は、register@clinicaltrials.gov。 までご連絡ください。 clinicaltrials.gov に変更が加えられるとすぐに、ウェブサイトでも自動的に更新されます。