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Gene therapy infused directly into brain aims to halt rare dementia

NCT ID NCT06064890

Disease control Sponsor: AviadoBio Ltd Source: ClinicalTrials.gov ↗

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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 Jun 25, 2026 · Last updated Aug 14, 2026 · Updated 3 times

Summary

This early-phase trial tests a gene therapy called AVB-101 for people with frontotemporal dementia caused by GRN gene mutations. The therapy delivers a working copy of the GRN gene directly into the brain during a one-time procedure. The study will enroll 18 adults aged 30-75 to check safety and whether it can restore normal levels of a key protein, potentially slowing the disease.

What this could mean

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

Active substance
AVB-101 (a gene therapy that delivers a working copy of the GRN gene into brain cells)
What this could lead to
If it works, this could slow or stop the progression of a devastating early-onset dementia by restoring a missing protein in the brain.
What could go wrong
This is a very early, small trial (18 people) testing safety first. Gene therapy delivered directly to the brain carries risks like brain swelling or bleeding, and it may not improve symptoms.

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 1/2

Runs two stages together: safety and dose first, then whether the treatment works.

Participants

About 18 people

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

Started

Aug 2023

Expected to finish

Mar 2030

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 75 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, 30 to 75 years of age * Carriers of a pathogenic GRN mutation * FTD as evidenced by CDR + NACC FTLD global score of 0.5, 1.0, or 2.0 * Presence of 1 or more of the criteria for diagnosis of possible bvFTD or PPA * Able and willing to comply with all procedures and the study visit schedule * Able and willing to give written informed consent prior to study participation, and agree to designate a legal representative to act on their wishes to continue participation should they lose capacity to consent at some point during the study OR If, in the Investigator's opinion, the subject lacks capacity to consent, written informed consent of their legal representative must be obtained in accordance with local laws, regulations, and/or customs. In countries where local laws, regulations, and/or customs do not permit subjects who lack capacity to consent to participate in this study, these subjects will not be enrolled * An identified, informed study partner who is able and willing to support the participant in the study and to provide assessments of the participant during the study Exclusion Criteria: * Severe dementia, defined as CDR + NACC FTLD global score of 3.0, or other symptoms that preclude the ability to comply with study procedures and/or pose unacceptable safety risk to the subject * Any concurrent disease that may cause cognitive impairment unrelated to mutations in the GRN gene, such as other causes of dementia, neurosyphilis, hydrocephalus, stroke, small vessel ischemic disease, uncontrolled hypothyroidism, or vitamin B12 deficiency * Clinically significant abnormality on MRI at Screening considered to be a contraindication to Intrathalamic infusion * Surgically significant pattern of brain atrophy on MRI at Screening that interferes with planned neurosurgical trajectory * Previous treatment with any gene or cell therapy * Previous treatment with any investigational medicinal product (IMP) within 60 days or 5 half-lives (whichever is longer) prior to study drug treatment * Concomitant disease, any clinically significant laboratory abnormality, or treatment which, in the opinion of the Investigator, may pose an unacceptable safety risk to the participant or interfere with study conduct or the participant's ability to comply with study procedures including neurosurgical administration under anesthesia

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Conditions

The condition(s) this trial relates to.

dementia frontotemporal dementia GRN-related frontotemporal lobar degeneration with Tdp43 inclusions primary progressive aphasia semantic dementia

As listed by the trial registrant

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

Dementia, frontotemporal Frontotemporal dementia FTD FTD-GRN

How to take part

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

  1. The places running it

    19 sites in 9 countries. The list below names each one and where it is.

  2. 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 ↗

  3. 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

Locations

  • Amsterdam UMC

    RECRUITING

    Amsterdam, Netherlands

  • Cambridge University Hospitals NHS Foundation Trust

    RECRUITING

    Cambridge, CB2 0QQ, United Kingdom

  • Centrum Medyczne NeuroProtect Sp z o.o.

    RECRUITING

    Warsaw, Poland

  • Euromedis Sp. z o.o.

    RECRUITING

    Szczecin, 70-111, Poland

  • Fondazione IRCCS Istituto Neurologico Carlo Besta

    RECRUITING

    Milan, Italy

  • Hospital Clinic Barcelona

    RECRUITING

    Barcelona, 08036, Spain

  • Hospital Universitari i Politecnic La Fe

    RECRUITING

    Valencia, 46026, Spain

  • Houston Methodist Hospital

    RECRUITING

    Houston, Texas, 77030, United States

  • Mazowiecki Szpital Brodnowski Sp. z o. o.

    RECRUITING

    Warsaw, Poland

  • NEURO-CARE Sp. z o.o. Sp. Komandytowa

    RECRUITING

    Katowice, Poland

  • Neurologia Slaska Centrum Medyczne

    RECRUITING

    Katowice, Poland

  • Skåne University Hospital

    RECRUITING

    Lund, Sweden

  • Sunnybrook Health Sciences Centre

    RECRUITING

    Toronto, Ontario, Canada

  • The Ohio State University (OSU) Wexner Medical Center

    RECRUITING

    Columbus, Ohio, 43210, United States

  • UZ Leuven

    RECRUITING

    Leuven, Belgium

  • University College London Hospitals

    RECRUITING

    London, United Kingdom

  • University Hospital of Wales

    RECRUITING

    Cardiff, CF14 4XW, United Kingdom

  • Uniwersyteckie Centrum Kliniczne, SUM w Katowicach

    RECRUITING

    Katowice, Poland

  • Vanderbilt University Medical Centre

    RECRUITING

    Nashville, Tennessee, 37232, United States

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