Gene therapy for Frontotemporal Dementia with Progranulin Mutations

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

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.

Why it matters

Who can join: the full rules

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 it's running

• The Ohio State University (OSU) Wexner Medical Center
  Columbus, Ohio, United States Recruiting
• Vanderbilt University Medical Centre
  Nashville, Tennessee, United States Recruiting
• Houston Methodist Hospital
  Houston, Texas, United States Recruiting
• UZ Leuven
  Leuven, Belgium Recruiting
• Sunnybrook Health Sciences Centre
  Toronto, Ontario, Canada Recruiting
• Fondazione IRCCS Istituto Neurologico Carlo Besta
  Milan, Italy Recruiting

Study contacts

• Study coordinator: AviadoBio Clinical Trials
• Email: clinicaltrials@aviadobio.com
• Phone: +44 203-089-7917

Similar studies

Other studies for the same condition or treatment. Recruiting studies are listed first.

• Temporal Interference Stimulation for cognitive impairment

  This study tests a non-invasive brain stimulation method called temporal interference stimulation to see if it can improve thinking and memory in people with mild cognitive impairment, Alzheimer's disease, or frontotemporal dementia.

  Recruiting Tianjin Huanhu Hospital · Tianjin, Tianjin Municipality · NCT07643363

• Hyperspectral retinal imaging to detect Alzheimer’s and other neurodegenerative changes

  This project will test whether a special retinal camera that scans many wavelengths of light can detect signs of Alzheimer’s and other neurodegenerative diseases in people with dementia or at risk.

  Recruiting Center for Eye Research Australia · Melbourne, Victoria · NCT07545473

• Magnetic brain stimulation for frontotemporal lobar degeneration

  This trial will try repetitive transcranial magnetic stimulation (rTMS) to see if it is safe and helps people with frontotemporal lobar degeneration or asymptomatic people at risk.

  Recruiting Università degli Studi di Brescia · Brescia, Italy · NCT07316413

• Omics-based profiling to find disease mechanisms and biomarkers in Alzheimer’s, Parkinson’s, FTD, and related dementias

  This project will use advanced omics tests (genetics, gene expression, proteins, and metabolites) to try to find biological markers and disease mechanisms in people with Alzheimer’s, Parkinson’s, frontotemporal and young-onset dementias, or mild cognitive impairment.

  Recruiting Ospedale Policlinico San Martino · Genoa, Genoa · NCT07235111

• ARO-MAPT-SC in healthy adults and people with early Alzheimer's disease

  This trial will test whether ARO-MAPT-SC is safe, tolerable, and produces the expected drug levels and biomarker effects in healthy adults and people with early Alzheimer's disease.

  Recruiting at 4 sites Phase 1/2 Arrowhead Pharmaceuticals · East York, Ontario and 3 other locations · NCT07221344

• STELLA-FTD: Telehealth support program for family caregivers of frontotemporal degeneration

  This program will see if eight weekly live video group sessions help family caregivers of people with frontotemporal degeneration manage challenging behaviors and connect with community and peer resources.

  Recruiting Oregon Health and Science University · Portland, Oregon · NCT06613204

How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.