Alzheimer's Disease (AD)ROOT-ADNCT07923812
Effectiveness and Safety of Disease-Modifying Therapy for Alzheimer's Disease: A Multicenter Retrospective Study
An observational study of Alzheimer's Disease (AD), run by Fudan University at 1 site in China.
Trial summary
The goal of this multicenter retrospective observational study is to learn about the real-world safety and clinical effectiveness of disease-modifying therapy (DMT) in adults with mild cognitive impairment (MCI) due to Alzheimer's disease (AD) or AD dementia. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Alzheimer's Disease (AD)
- What is being tested?
- Disease-Modifying Therapy
- How many people
- 3,000 participants
- Where
- 1 siteChina
- Study dates
- 26 Oct 2026to 31 Dec 2027
- Phase
- Observational
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Study population: Patients with Alzheimer's disease (AD)-related mild cognitive impairment (MCI) and AD-related dementia.
Who may be able to join
None listed.
Who cannot join
- The patient, when cognitive capacity permits, explicitly refuses to provide informed consent, or the patient's legally authorized representative or guardian refuses to provide consent on the patient's behalf, either in written or electronic form.
- Amyloid-beta (Aβ) pathological evidence is missing or does not meet the study requirements.
- Presence of a major neurological disorder that may confound the assessment of cognitive outcomes.
- Previous treatment with other anti-amyloid monoclonal antibodies, or participation in an interventional clinical trial that may affect the assessment of study outcomes.
- Serious inconsistencies or errors in key participant information.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
China1 site, 1 recruiting
- Recruiting
Huashan Hospital, Fudan University, No. 12 Wulumuqi Middle Road, Jing'an District, Shanghai
Shanghai, Jingan, 200040
Jintai Yu, MD, +86 21 52888163, jintai_yu@fudan.edu.cn
About this study
The goal of this multicenter retrospective observational study is to learn about the real-world safety and clinical effectiveness of disease-modifying therapy (DMT) in adults with mild cognitive impairment (MCI) due to Alzheimer's disease (AD) or AD dementia. The main questions it aims to answer are:
What are the safety outcomes of DMT, including amyloid-related imaging abnormalities (ARIA), adverse events, and infusion reactions (IRRs)? How do Clinical Dementia Rating (CDR) measures change in patients who receive DMT compared with those who do not?
Read more
Researchers will compare patients who receive DMT as part of their routine clinical care with patients who do not receive DMT to evaluate differences in clinical outcomes and safety.
Participants will not receive any study-assigned intervention or additional treatment as part of this study. Researchers will retrospectively collect existing clinical data, including cognitive and neuropsychiatric assessments, imaging findings, biomarker results, DMT use, and safety information.
- Conditions
- Alzheimer's Disease (AD)
- Being tested
- Disease-Modifying Therapy
- Medical subject headings
- Alzheimer Disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Disease-Modifying TherapyDrug
Participants received disease-modifying therapy as part of routine clinical care. Treatment information, including treatment type, dosage, duration, and discontinuation status, will be retrospectively collected from medical records.
| Study group | Role | What they receive |
|---|---|---|
| Disease-Modifying Therapy Group | The disease-modifying therapy group includes patients with MCI-AD or AD dementia who received disease-modifying therapy during routine clinical practice. Patients were diagnosed based on clinical criteria and available biomarker evidence according to real-world clinical practice. Baseline characteristics, treatment information, clinical outcomes, and safety data will be retrospectively collected and analyzed. Receives: Drug: Disease-Modifying Therapy | |
| Non-Disease-Modifying Therapy Group | The non-disease-modifying therapy group includes patients with MCI-AD or AD dementia who did not receive disease-modifying therapy. Patients may have received conventional symptomatic treatments or other non-disease-modifying treatments according to routine clinical practice. Clinical characteristics, treatment information, clinical outcomes, and safety data will be retrospectively collected and analyzed. |
What is being measured
The primary outcome is the main question the study is designed to answer.
Safety of Disease-Modifying Therapy
To evaluate the safety of anti-amyloid disease-modifying therapies (DMTs; lecanemab or donanemab) in patients with Alzheimer's disease (AD)-related mild cognitive impairment (MCI) or AD dementia. Safety outcomes include the occurrence of amyloid-related imaging abnormalities (ARIA), and the number of patients experiencing, incidence proportion (%), and severity of treatment-related adverse events and infusion-related reactions (IRRs).
Measured: From treatment initiation through up to 30 months of follow-up
Clinical Effectiveness of Disease-Modifying Therapy
To evaluate changes in Clinical Dementia Rating (CDR)-related measures in patients receiving anti-amyloid disease-modifying therapies (DMTs; lecanemab or donanemab) and those receiving usual care, and to compare these changes between the two groups.
Measured: Baseline and follow-up assessments up to 30 months
Secondary outcomes (9)
Change from baseline in Mini-Mental State Examination total score
The Mini-Mental State Examination (MMSE) assesses global cognitive function. Total scores range from 0 to 30, with higher scores indicating better cognitive function. Change is calculated as the score at each available follow-up assessment minus the baseline score. Changes will be compared between the anti-amyloid treatment and usual-care cohorts.
Measured: Baseline and follow-up assessments up to 30 months
Change from baseline in Montreal Cognitive Assessment total score
The Montreal Cognitive Assessment (MoCA) assesses global cognitive function. For the standard 30-point version, total scores range from 0 to 30, with higher scores indicating better cognitive function. Change is calculated as the score at each available follow-up assessment minus the baseline score. Changes will be compared between the anti-amyloid treatment and usual-care cohorts. The instrument version and education-adjustment rule will be recorded and applied consistently.
Measured: Baseline and follow-up assessments up to 30 months
Change From Baseline in Amyloid Positron Emission Tomography (PET) Burden Measured in Centiloids
Amyloid burden is quantified using Centiloid (CL) values. Higher values indicate greater amyloid burden. Change is calculated as the Centiloid value at each available follow-up examination minus the baseline value. Changes will be compared between the anti-amyloid treatment and usual-care cohorts.
Measured: Baseline and follow-up assessments up to 30 months
Duration of Disease-Modifying Therapy
Treatment duration will be calculated from the date of the first administration of lecanemab or donanemab to the date of the last administration within the observation period and expressed in months.
Measured: From treatment initiation through up to 30 months of follow-up
Permanent Discontinuation of Disease-Modifying Therapy
The proportion of patients who permanently discontinue lecanemab or donanemab during follow-up will be assessed. Reasons for permanent treatment discontinuation will be recorded and summarized descriptively.
Measured: From treatment initiation through up to 30 months of follow-up
Change from baseline in the 20-Item Activities of Daily Living Scale Score
The 20-item Activities of Daily Living (ADL) Scale assesses functional independence in daily activities. Total scores range from 20 to 80, with higher scores indicating greater functional impairment. Change is calculated as the score at each available follow-up assessment minus the baseline score. Changes will be compared between the anti-amyloid treatment and usual-care cohorts.
Measured: Baseline and follow-up assessments up to 30 months
Change from baseline in Hamilton Anxiety Rating Scale total score
The 14-item Hamilton Anxiety Rating Scale (HAMA) assesses anxiety symptom severity. Each item is scored from 0 to 4, yielding a total score from 0 to 56. Higher scores indicate more severe anxiety symptoms. Change is calculated as the score at each available follow-up assessment minus the baseline score. Changes will be compared between the anti-amyloid treatment and usual-care cohorts.
Measured: Baseline and follow-up assessments up to 30 months
Change from baseline in the 17-Item Hamilton Depression Rating ScaleTotal Score
The 17-item Hamilton Depression Rating Scale (HAMD-17) assesses depressive symptom severity. Total scores range from 0 to 52, with higher scores indicating more severe depressive symptoms. Change is calculated as the score at each available follow-up assessment minus the baseline score. Changes will be compared between the anti-amyloid treatment and usual-care cohorts.
Measured: Baseline and follow-up assessments up to 30 months
Change from baseline in the 12-Item Neuropsychiatric Inventory Total Score
The 12-item Neuropsychiatric Inventory (NPI-12) assesses neuropsychiatric symptom severity across 12 domains. Each domain score is calculated by multiplying symptom frequency (1-4) by severity (1-3), with a score of 0 assigned when symptoms are absent. The total score is the sum of the 12 domain scores and ranges from 0 to 144, with higher scores indicating greater neuropsychiatric symptom burden. Caregiver distress scores are not included in the total score. Change is calculated as the score at each available follow-up assessment minus the baseline score. Changes will be compared between the anti-amyloid treatment and usual-care cohorts.
Measured: Baseline and follow-up assessments up to 30 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted6 Nov 2026
- Study start26 Oct 2026
- Primary completion30 Jun 2027 (estimated)
- Study completion31 Dec 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Alzheimer's Disease (AD)Studies of the same condition, open and under way.
- More trials run by Fudan UniversityThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Retrospective
- Enrolment
- 3,000 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Fudan University
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Jintai Yu, Huashan Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07923812
- Sponsor's protocol ID
- KY2025-1559
- Acronym
- ROOT-AD
- Status last verified
- Nov 2026
- First submitted
- 26 Oct 2026
- Last updated
- 6 Nov 2026
- Results posted
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 6 Nov 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Effectiveness and Safety of Disease-Modifying Therapy for Alzheimer's Disease: A Multicenter Retrospective Study
- ClinicalTrials.gov ID
- NCT07923812
- Acronym / sponsor ID
- ROOT-AD / KY2025-1559
- Conditions
- Alzheimer's Disease (AD)
- Study type
- Observational
- Recruitment status
- Recruiting
- Enrolment
- 3,000 participants (estimated)
- Lead sponsor
- Fudan UniversityUniversity, hospital or other
- Study start
- 26 Oct 2026 (actual)
- Primary completion
- 30 Jun 2027 (estimated)
- Study completion
- 31 Dec 2027 (estimated)
- First posted
- 6 Nov 2026 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The goal of this multicenter retrospective observational study is to learn about the real-world safety and clinical effectiveness of disease-modifying therapy (DMT) in adults with mild cognitive impairment (MCI) due to Alzheimer's disease (AD) or AD dementia. The main questions it aims to answer are:
What are the safety outcomes of DMT, including amyloid-related imaging abnormalities (ARIA), adverse events, and infusion reactions (IRRs)? How do Clinical Dementia Rating (CDR) measures change in patients who receive DMT compared with those who do not?
Researchers will compare patients who receive DMT as part of their routine clinical care with patients who do not receive DMT to evaluate differences in clinical outcomes and safety.
Participants will not receive any study-assigned intervention or additional treatment as part of this study. Researchers will retrospectively collect existing clinical data, including cognitive and neuropsychiatric assessments, imaging findings, biomarker results, DMT use, and safety information.
Study design
- Observational model
- Cohort
- Time perspective
- Retrospective
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Disease-Modifying Therapy Group | – | Drug: Disease-Modifying TherapyThe disease-modifying therapy group includes patients with MCI-AD or AD dementia who received disease-modifying therapy during routine clinical practice. Patients were diagnosed based on clinical criteria and available biomarker evidence according to real-world clinical practice. Baseline characteristics, treatment information, clinical outcomes, and safety data will be retrospectively collected and analyzed. |
| Non-Disease-Modifying Therapy Group | – | –The non-disease-modifying therapy group includes patients with MCI-AD or AD dementia who did not receive disease-modifying therapy. Patients may have received conventional symptomatic treatments or other non-disease-modifying treatments according to routine clinical practice. Clinical characteristics, treatment information, clinical outcomes, and safety data will be retrospectively collected and analyzed. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
- Study population
- Patients with Alzheimer's disease (AD)-related mild cognitive impairment (MCI) and AD-related dementia.
Key inclusion criteria (0 listed)
Key exclusion criteria (5 listed)
- The patient, when cognitive capacity permits, explicitly refuses to provide informed consent, or the patient's legally authorized representative or guardian refuses to provide consent on the patient's behalf, either in written or electronic form.
- Amyloid-beta (Aβ) pathological evidence is missing or does not meet the study requirements.
- Presence of a major neurological disorder that may confound the assessment of cognitive outcomes.
- Previous treatment with other anti-amyloid monoclonal antibodies, or participation in an interventional clinical trial that may affect the assessment of study outcomes.
- Serious inconsistencies or errors in key participant information.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Disease-Modifying TherapyParticipants received disease-modifying therapy as part of routine clinical care. Treatment information, including treatment type, dosage, duration, and discontinuation status, will be retrospectively collected from medical records. | Drug | Disease-Modifying Therapy Group |
Primary outcome
Safety of Disease-Modifying Therapy
Time frame: From treatment initiation through up to 30 months of follow-up
To evaluate the safety of anti-amyloid disease-modifying therapies (DMTs; lecanemab or donanemab) in patients with Alzheimer's disease (AD)-related mild cognitive impairment (MCI) or AD dementia. Safety outcomes include the occurrence of amyloid-related imaging abnormalities (ARIA), and the number of patients experiencing, incidence proportion (%), and severity of treatment-related adverse events and infusion-related reactions (IRRs).
Clinical Effectiveness of Disease-Modifying Therapy
Time frame: Baseline and follow-up assessments up to 30 months
To evaluate changes in Clinical Dementia Rating (CDR)-related measures in patients receiving anti-amyloid disease-modifying therapies (DMTs; lecanemab or donanemab) and those receiving usual care, and to compare these changes between the two groups.
9 secondary outcomes
Change from baseline in Mini-Mental State Examination total score
Time frame: Baseline and follow-up assessments up to 30 months
Change from baseline in Montreal Cognitive Assessment total score
Time frame: Baseline and follow-up assessments up to 30 months
Change From Baseline in Amyloid Positron Emission Tomography (PET) Burden Measured in Centiloids
Time frame: Baseline and follow-up assessments up to 30 months
Duration of Disease-Modifying Therapy
Time frame: From treatment initiation through up to 30 months of follow-up
Permanent Discontinuation of Disease-Modifying Therapy
Time frame: From treatment initiation through up to 30 months of follow-up
Change from baseline in the 20-Item Activities of Daily Living Scale Score
Time frame: Baseline and follow-up assessments up to 30 months
Change from baseline in Hamilton Anxiety Rating Scale total score
Time frame: Baseline and follow-up assessments up to 30 months
Change from baseline in the 17-Item Hamilton Depression Rating ScaleTotal Score
Time frame: Baseline and follow-up assessments up to 30 months
Change from baseline in the 12-Item Neuropsychiatric Inventory Total Score
Time frame: Baseline and follow-up assessments up to 30 months
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 6 Nov 2026.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Alzheimer's Disease (AD), run by Fudan University at 1 site in China.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 0 inclusion and 5 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 6 Nov 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in China. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Jintai Yu, MD (+86 21 52888163, jintai_yu@fudan.edu.cn), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Safety of Disease-Modifying Therapy (time frame: From treatment initiation through up to 30 months of follow-up). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 6 Nov 2026.