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Gene therapy for Frontotemporal Dementia with Progranulin Mutations

A Phase 1/2 Open-Label, Ascending Dose, Multicenter Study to Evaluate the Safety and Preliminary Efficacy of AVB-101 Administered by Bilateral Intrathalamic Infusion in Subjects With Frontotemporal Dementia With Progranulin Mutations (FTD-GRN)

Phase1; Phase2 Interventional AviadoBio Ltd · NCT06064890

This study is testing a one-time gene therapy to see if it can safely help people with Frontotemporal Dementia caused by a specific gene mutation by restoring a protein in the brain.

Quick facts

PhasePhase1; Phase2
Study typeInterventional
Enrollment18 (estimated)
Ages30 Years to 75 Years
SexAll
SponsorAviadoBio Ltd Industry-sponsored
Locations19 sites (Columbus, Ohio and 18 other locations)
Trial IDNCT06064890 on ClinicalTrials.gov

What this trial studies

This clinical study evaluates the safety and effectiveness of AVB-101, a gene therapy product, in treating Frontotemporal Dementia with Progranulin Mutations (FTD-GRN). The study aims to determine if a one-time treatment can safely restore normal levels of the protein progranulin in the brain and potentially slow or stop the progression of this early-onset dementia. Participants will receive the treatment directly without a placebo, allowing researchers to assess the therapy's impact on symptoms and brain function. The study includes patients aged 30 to 75 who carry a pathogenic GRN mutation and meet specific diagnostic criteria.

Who should consider this trial

Good fit: Ideal candidates are individuals aged 30 to 75 with a pathogenic GRN mutation and diagnosed with Frontotemporal Dementia.

Not a fit: Patients with severe dementia or those unable to comply with study procedures may not benefit from this study.

Why it matters

Potential benefit: If successful, this therapy could significantly improve the quality of life for patients with FTD-GRN by slowing disease progression.

How similar studies have performed: While gene therapy for dementia is a novel approach, similar studies targeting other genetic forms of dementia have shown promising results.

Eligibility criteria

Show full inclusion / exclusion criteria 
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

Where this trial is running

Columbus, Ohio and 18 other locations

Study contacts

How to participate

  1. Review the eligibility criteria above with your treating physician.
  2. Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
  3. Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.
View on ClinicalTrials.gov → Find more trials like this →
Conditions Frontotemporal DementiaFTDFTD-GRNDementia, FrontotemporalGene TherapyAAVIntrathalamicIntraparenchymal
Last reviewed 2026-06-13 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.