- ICH GCP
- Registro degli studi clinici negli Stati Uniti
- Sperimentazione clinica NCT07724132
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
Panoramica dello studio
Stato
Condizioni
Intervento / Trattamento
Descrizione dettagliata
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.
Tipo di studio
Iscrizione (Stimato)
Fase
- Fase 3
Contatti e Sedi
Contatto studio
- Nome: Trial Management Team UCL InCTU
- Numero di telefono: +44 (0)20 7670 4700
- Email: mrcctu.adsmart@ucl.ac.uk
Luoghi di studio
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Cambridge, Regno Unito, CB21 5EF
- Reclutamento
- Windsor Research Unit
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Chertsey, Regno Unito, KT16 9AU
- Reclutamento
- Two Bridges Research & Development Clinic
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Criteri di partecipazione
Criteri di ammissibilità
Età idonea allo studio
- Adulto
- Adulto più anziano
Accetta volontari sani
Descrizione
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.
Piano di studio
Come è strutturato lo studio?
Dettagli di progettazione
- Scopo principale: Trattamento
- Assegnazione: Randomizzato
- Modello interventistico: Assegnazione parallela
- Mascheramento: Triplicare
Armi e interventi
Gruppo di partecipanti / Arm |
Intervento / Trattamento |
|---|---|
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Comparatore placebo: Standard of Care + Placebo
Placebo arm (Oral capsule matching active treatments)
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Oral capsule matching active treatments
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Comparatore attivo: 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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Comparatore attivo: 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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Cosa sta misurando lo studio?
Misure di risultato primarie
Misura del risultato |
Misura Descrizione |
Lasso di tempo |
|---|---|---|
|
Change in Alzheimer's Disease Assessment Scale-Cognitive Subscale (ADAS-Cog) score
Lasso di tempo: Baseline to 18 months
|
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
Lasso di tempo: Baseline to 18 months
|
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
|
Misure di risultato secondarie
Misura del risultato |
Misura Descrizione |
Lasso di tempo |
|---|---|---|
|
Change in Neuropsychiatric Inventory (NPI) total score
Lasso di tempo: 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)
Lasso di tempo: Baseline to 18 months
|
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)
Lasso di tempo: Baseline to 18 months
|
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
Lasso di tempo: Baseline to 18 months
|
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
Lasso di tempo: 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
Lasso di tempo: Baseline to 18 months
|
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
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Altre misure di risultato
Misura del risultato |
Misura Descrizione |
Lasso di tempo |
|---|---|---|
|
Change in plasma pTau-217 concentration
Lasso di tempo: Baseline to 18 months
|
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
Lasso di tempo: Baseline to 18 months
|
Exploratory Outcome Measures
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Baseline to 18 months
|
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Change in plasma amyloid-β concentrations
Lasso di tempo: Baseline to 18 months
|
Exploratory biomarker outcome assessing change in plasma amyloid-β isoforms (Unit of Measure: pg/mL)
|
Baseline to 18 months
|
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Change in plasma neurofilament light chain (NfL) concentration
Lasso di tempo: Baseline to 18 months
|
Exploratory biomarker outcome assessing change in plasma NfL levels (Unit of Measure: pg/mL)
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Baseline to 18 months
|
Collaboratori e investigatori
Studiare le date dei record
Studia le date principali
Inizio studio (Stimato)
Completamento primario (Stimato)
Completamento dello studio (Stimato)
Date di iscrizione allo studio
Primo inviato
Primo inviato che soddisfa i criteri di controllo qualità
Primo Inserito (Effettivo)
Aggiornamenti dei record di studio
Ultimo aggiornamento pubblicato (Effettivo)
Ultimo aggiornamento inviato che soddisfa i criteri QC
Ultimo verificato
Maggiori informazioni
Termini relativi a questo studio
Termini MeSH pertinenti aggiuntivi
Altri numeri di identificazione dello studio
- ND003
- 70766 (Altro identificatore: Central Portfolio Management System (CPMS))
- ISRCTN17108793 (Identificatore di registro: ISRCTN)
Piano per i dati dei singoli partecipanti (IPD)
Hai intenzione di condividere i dati dei singoli partecipanti (IPD)?
Descrizione del piano IPD
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.
Periodo di condivisione IPD
Informazioni su farmaci e dispositivi, documenti di studio
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Queste informazioni sono state recuperate direttamente dal sito web clinicaltrials.gov senza alcuna modifica. In caso di richieste di modifica, rimozione o aggiornamento dei dettagli dello studio, contattare register@clinicaltrials.gov. Non appena verrà implementata una modifica su clinicaltrials.gov, questa verrà aggiornata automaticamente anche sul nostro sito web .