Status:
RECRUITING
A Study to Evaluate the Safety and Effect of AVB-101, a Gene Therapy Product, in Subjects With a Genetic Sub-type of Frontotemporal Dementia (FTD-GRN)
Lead Sponsor:
AviadoBio Ltd
Conditions:
Frontotemporal Dementia
FTD
Eligibility:
All Genders
30-75 years
Phase:
PHASE1
PHASE2
Brief Summary
The goal of this clinical study is to learn about an investigational gene therapy product called AVB-101, which is designed to treat a disease called Frontotemporal Dementia with Progranulin Mutations...
Eligibility Criteria
Inclusion
- 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
- A protocol defined minimum thalamic volume on each side on Screening MRI
- 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
- 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
- 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
Key Trial Info
Start Date :
August 30 2023
Trial Type :
INTERVENTIONAL
Allocation :
ESTIMATED
End Date :
October 31 2030
Estimated Enrollment :
9 Patients enrolled
Trial Details
Trial ID
NCT06064890
Start Date
August 30 2023
End Date
October 31 2030
Last Update
December 17 2025
Active Locations (18)
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1
The Ohio State University (OSU) Wexner Medical Center
Columbus, Ohio, United States, 43210
2
Vanderbilt University Medical Centre
Nashville, Tennessee, United States, 37232
3
Houston Methodist Hospital
Houston, Texas, United States, 77030
4
Sunnybrook Health Sciences Centre
Toronto, Ontario, Canada