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Gene Therapy Trial Report

Summary

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)


NCTID NCT06064890 (View at clinicaltrials.gov)
Description
Development Status Active
Indication Frontotemporal Dementia, FTD, FTD-GRN
Disease Ontology Term DOID:0060672
Compound Name AVB-101
Compound Description AAV.PGRN
Sponsor AviadoBio Ltd
Funder Type Industry
Recruitment Status
Enrollment Count 9 (ESTIMATED)
Results Posted Not Available

Therapy Information


Target Gene/Variant GRN
Therapy Type Gene transfer
Therapy Route In-vivo
Mechanism of Action Functional gene replacement
Route of Administration Intrathalamic
Drug Product Type Viral vector
Delivery System Viral transduction
Vector Type AAV9
Editor Type none
Dose 1 Undisclosed low dose
Dose 2 Undisclosed high dose

Study Record Dates


Current Stage Phase1, Phase2
Submit Date 2023-09-12
Completion Date 2030-03-21
Last Update 2026-02-27

Participation Criteria


Eligible Age 30 Years - 75 Years
Standard Ages Adult, Older adult
Sexes Eligible for Study 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 * 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
View Inclusion and Exclusion Criteria at ClinicalTrials.gov

Locations


No.of Trial Sites 19
Locations Canada,Netherlands,Sweden,Belgium,United States,Poland,Italy,United Kingdom,Spain

Regulatory Information


Has US IND True
FDA Designations Fast Track, Orphan Drug Designation
Recent Updates Second dose cohort is complete, company intends to initiate a third dose cohort in Q3 2025, early biomarker data expected 2026

Resources/Links