Alzheimer s Disease
Conditions
Keywords
MCI due to AD, Mild AD
Brief summary
VY1706 first in human study in early Alzheimer's Disease is a multicenter dose escalation study
Interventions
DRUGVY1706 Low dose
Low dose
DRUGVY1706 Mid dose
Mid Dose
DRUGVY1706 High Dose
High dose
DEVICEAnti-AAV9 Total Antibody (TAb) Assay
Anti-AAV9 Total Antibody (TAb) Assay
Sponsors
Voyager Therapeutics
Study design
Allocation
NON_RANDOMIZED
Intervention model
SEQUENTIAL
Primary purpose
TREATMENT
Masking
NONE
Masking description
Open label multicenter dose escalation cohort study
Eligibility
Sex/Gender
ALL
Age
30 Years to 80 Years
Healthy volunteers
No
Inclusion criteria
* Male or female participants aged 55 to 80 years (inclusive) at Screening or aged 30 to 80 years (inclusive) if presence of a historically documented dominantly inherited mutation associated with monogenic AD. * Clinical diagnosis of mild cognitive impairment (MCI) due to AD or mild AD with MMSE 18-30 and CDR Global score of 0.5-1. * Evidence of amyloid and tau pathology consistent with AD diagnosis by both: * Apart from the clinical diagnosis of early AD, participant must be in good health as determined by the Investigator. * If the participant is receiving an approved symptomatic AD treatment, such as acetylcholinesterase or NMDA inhibitors, the participant must be on a stable dose for at least 8 weeks prior to Screening and until Day 1. * Stable doses of all other (non-AD-related) concomitant medications for at least 4 weeks prior to Screening and until Day 1. * Must have an identified reliable Study Partner.
Exclusion criteria
Any medical or neurological/neurodegenerative or psychiatric condition (other than AD) that may be a contributing cause to cognitive impairment or could confound interpretation of drug effect, affect study assessments, or affect participant's ability to participate and complete the study or lead to safety concerns. * Seropositive for anti-AAV9 antibodies at Screening. * History of transient ischemic attack or stroke or any unexplained loss of consciousness within 1 year prior to Screening. * History of seizures within 10 years prior to Screening or history of epileptic syndrome (except for history of febrile seizures in childhood). * Presence of a clinically significant uncontrolled medical disorder that may compromise the participant's safety or their ability to complete all of the study assessments. * History of significant cardiovascular disease. * Contraindications to lumbar puncture, MRI imaging, PET imaging or corticosteroids. * History of, or positive test result for human immunodeficiency virus (HIV), hepatitis C or current acute hepatitis B. * History within 1 year prior to screening of drug or alcohol abuse. * History of severe allergies, or history of an anaphylactic reaction (nonactive hay fever is acceptable). * Previous or current use of an approved AD disease-modifying therapies * Previous or current participation in a clinical study involving any cell or gene therapies (including but not limited to AAV-based gene therapies) or active immunotherapies targeting Tau or amyloid, or any anti-amyloid or anti-Tau therapies or any therapeutic mAb, protein derived from a mAb, immunoglobulin therapy, antisense oligonucleotides, small interfering ribonucleic acid, or any other agent with purported disease-modifying effect in AD unless it can be documented that the participant only received placebo..
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| To characterize the safety and tolerability in participants with AD by Incidence of treatment emergent adverse events, changes from baseline in vital signs, physical and neurological exams and other safety measures | 52 weeks | Incidence of treatment emergent adverse events, clinically significant changes from baseline in vital signs, physical and neurological exams, Columbia Suicide-Severity Rating Scale, Electrocardiogram, Clinical lab parameters and transthoracic echocardiogram |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| To evaluate the effect of VY1706 on CSF biomarkers of Tau | 52 weeks | Change in baseline in CSF biomarkers of Tau |
| To evaluate the effect of VY1706 on Tau pathology | 52 weeks | Change from baseline in the SUVR on Tau pathology |
Countries
United States
Contacts
CONTACTVoyager
Outcome results
None listed