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NCT Number: NCT06883019

Lecanemab for Early Onset Familial Alzheimer's Disease

The goal of this observational study is to learn about the efficacy of Lecanemab treatment for early-onset familial Alzheimer's disease (AD) in patients under 65 years of age with a family history of AD. Participants will receive Lecanemab at a dosage of 10 mg/kg every two weeks for a total of 18 months and will undergo cognitive assessments, PET and MRI scans, blood/fluid tests and whole genome sequencing. The study will explore the effects of genetic and hereditary factors on the efficacy of Lecanemab treatment in early-onset familial AD patients.

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Key information

Age range

18 year–65 year

Sex eligibility

All sexes

Study type

Observational

Primary location

Xuanwu Hospital, Capital Medical University, Beijing, China

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Who can participate

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • Age at onset ≤ 65 years, with a minimum age of 18 years; no restriction on gender.
  • Diagnosis of Alzheimer's Disease (AD) and Mild Cognitive Impairment (MCI): Must meet the clinical diagnostic criteria for AD-related MCI and mild AD as defined by the National Institute on Aging and the Alzheimer's Association (NIA-AA) (2011); confirmed Aβ positivity through Aβ-PET/CT, Aβ-PET/MRI, or cerebrospinal fluid testing.
  • MMSE ≥ 21 or MoCA ≥ 17 or CDR = 0.5
  • No significant signs found in the neurological examination
  • Participants must be capable of completing cognitive assessments and other tests.
  • Informed consent must be obtained from the participants and their legal guardians, with a dated signature, prior to any operations or tests related to the protocol, committing to comply with the research procedures and cooperate throughout the study process.

Exclusion criteria

  • Cognitive decline caused by other reasons: cerebrovascular disease, central nervous system infections, Creutzfeldt-Jakob disease, Huntington's disease, Parkinson's disease, Lewy body dementia, traumatic dementia, other physical and chemical factors (drugs, alcohol, CO, etc.), significant systemic diseases (hepatic encephalopathy, pulmonary encephalopathy, etc.), intracranial space-occupying lesions (subdural hematoma, brain tumor), endocrine system disorders (thyroid disease, parathyroid disease), and dementia caused by vitamin deficiencies or any other reasons.
  • Patients with other unstable diseases, or those who have had a stroke or transient ischemic attack, bleeding disorders, or seizures within the previous 12 months.
  • Patients with psychiatric disorders who meet DSM-IV criteria for schizophrenia or other mental illnesses, bipolar disorder, major depression, or delirium.
  • Patients with unstable or severe heart, lung, liver, kidney, hematological diseases; those with known malignancies or other serious prognoses.
  • Exclusion of cerebral amyloid angiopathy-related inflammation/β-amyloid-related cerebral vasculitis (CAAri/ABRA).
  • Presence of uncorrectable visual or auditory impairments that prevent completion of relevant assessments or scales.
  • Patients who cannot undergo MRI due to claustrophobia, pacemakers, defibrillators, or metal implants.
  • MRI findings showing more than four microhemorrhages (diameter < 10 mm), evidence of surface iron deposition, vascular edema, diffuse white matter disease, multiple lacunar strokes, or any strokes involving major vascular regions. Presence of evidence of cerebral contusions, brain softening, cerebral aneurysms, or other vascular malformations, central nervous system (CNS) infections, as well as brain tumors other than meningiomas or arachnoid cysts.
  • Patients taking warfarin, vitamin K antagonists, or direct oral anticoagulants (dabigatran, rivaroxaban, edoxaban, apixaban, betrixaban) or heparin; patients receiving thrombolysis; patients with coagulation disorders.
  • Pregnant or lactating women.
  • Patients deemed unsuitable for use by clinicians apart from the exclusion criteria listed above.
  • Patients with severe allergies to lecanemab or any excipients of this product.

Treatment and study plan

Primary outcomes

  1. Chang of CDR-SB score

    Time frame: baseline, 9 month, 18 month

    CDR-SB, clinical dementia rating-sum of boxes

Secondary outcomes

  1. Change of amyloid burden

    Time frame: baseline, 9 month, 18 month

    AV45-PET

  2. Change of ADAS-cog score

    Time frame: baseline, 9 month, 18 month

    ADAS-cog, the Alzheimer's Disease Assessment Scale-Cognitive Subscale

  3. Change of ADCS-ADL score

    Time frame: baseline, 9 month, 18 month

    ADCS-ADL, the Alzheimer's Disease Cooperative Study - Activities of Daily Living Scale (ADCS-ADL)

  4. Change of MoCA score

    Time frame: baseline, 9 month, 18 month

    MoCA, the Montreal Cognitive Assessment

  5. Change of BNT score

    Time frame: baseline, 9 month, 18 month

    BNT, the Boston Naming Test

  6. Change of TMT score

    Time frame: baseline, 9 month, 18 month

    TMT, the trail making test

  7. Change of HAMA and HAMD score

    Time frame: baseline, 9 month, 18 month

    HAMA, Hamilton Anxiety Scale; HAMD, Hamilton Depression Scale

  8. Change of biomarkers

    Time frame: baseline, 9 month, 18 month

    p-tau181, p-tau217, Aβ40, Aβ42 , pEAβ3-42, GFAP, NFL, TRPC6 ( blood, urine, feces, gingival crevicular fluid, cerebrospinal fluid)

  9. Positron emission tomography (PET)

    Time frame: baseline, 9 month, 18 month

    Other PET including FDG-PET, Tau-PET, TSPO-PET, SV2A-PET, exendin-4 (GLP-1R)-PET, colinergic receptor probe (ASEM) PET

  10. Change of structural MRI

    Time frame: baseline, 9 month, 18 month

    3D T1-weighted, 3D T2-weighted and Diffusion Tensor Imaging (DTI)

  11. Change of functional MRI

    Time frame: baseline, 9 month, 18 month

    Blood oxygenation level dependent (BOLD) imaging

  12. Change of magnetic susceptibility

    Time frame: baseline, 9 month, 18 month

    Quantitative susceptibility mapping (QSM) imaging

  13. Change of perfusion imaging

    Time frame: baseline, 9 month, 18 month

    Arterial spin labeling (ASL) imaging

  14. Whole Genome Sequencing

    Time frame: baseline

  15. Change of speech information

    Time frame: baseline, 9 month, 18 month

    Speech recording of Cookie-theft description task, using self-developed ASR speech analysis software (China software copyright number: 2016RS164680) for speech analysis

Study contacts

Contact information is provided by the study sponsor or research team.

Jinwen Xiao

CONTACT

[email protected]

+86 13917310784

Sponsors and collaborators

Lead sponsor

RenJi Hospital

Other

Registry information

Official study title

A Real World Study of Lecanemab Treatment in Participants with Early Onset Familial Alzheimer's Disease

Important dates

Study start
2025
Primary completion
2027
Study completion
2027
First posted
Mar 19, 2025
Registry last updated
Mar 19, 2025

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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