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Can a gene therapy clear the toxic tau tangle in Alzheimer's?

NCT ID NCT07764146

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now This study
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Aug 13, 2026 · Last updated Sep 04, 2026 · Updated 2 times

Summary

This early-stage trial tests an experimental gene therapy called VY1706 in people with early Alzheimer's disease. The goal is to see if it is safe and tolerable, and whether it can reduce tau protein buildup in the brain, a hallmark of the disease. Participants receive one of three dose levels, and researchers monitor safety and measure changes in tau biomarkers.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance
VY1706, an experimental gene therapy given in low, mid, or high doses
What this could lead to
If successful, VY1706 could offer a new way to slow or halt Alzheimer's progression by targeting tau protein buildup in the brain.
What could go wrong
This is an early, small dose-escalation study focused on safety, so it may not prove effective. Gene therapies carry risks like immune reactions or unintended effects.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 1

The first testing in people. Mainly checks safety and dose, usually in a small group.

Participants

About 18 people

The number the study aims to enrol. It can still change while the study runs.

Started

Sep 2026

Expected to finish

Apr 2029

An estimate. End dates often move.

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

30 to 80 years

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

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..

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Conditions

The condition(s) this trial relates to.

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

How to take part

Only the study team decides who joins. These are the ways to reach them.

  1. The study's own enquiry address

    This study publishes an address for enquiries. See it below .

  2. The places running it

    1 site. The list below names each one and where it is.

  3. The official record

    ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.

    Open the record ↗

  4. A doctor treating you

    A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.

Contacts and locations

Study contacts

  • Contact

    Email: •••••@•••••

Locations

  • K2 Medical Research, LLC

    RECRUITING

    Maitland, Florida, 32751, United States

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