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)

Launched by AVIADOBIO LTD · Sep 26, 2023

Keywords

ClinConnect Summary

This clinical trial is exploring a new gene therapy called AVB-101, aimed at treating a specific type of dementia known as Frontotemporal Dementia with Progranulin Mutations (FTD-GRN). This condition affects younger adults and leads to problems with behavior, language, and movement due to low levels of a protein called progranulin in the brain. The study will help researchers understand if a one-time treatment with AVB-101 is safe, if it can restore normal levels of progranulin, and if it can slow down or stop the progression of the disease.

To participate in this study, individuals must be between 30 and 75 years old and have a specific genetic mutation related to FTD-GRN. Participants will receive the AVB-101 treatment directly into their brains and will be monitored for five years to assess its effects. Importantly, there is no placebo involved, so everyone in the study will receive the actual treatment. Those interested should also have a supportive study partner who can help with assessments during the trial. This trial provides an opportunity to contribute to research that could lead to new options for managing this challenging condition.

Gender

ALL

Eligibility 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
• • 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 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