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)

PHASE1/PHASE2RecruitingINTERVENTIONAL
Enrollment

9

Participants

Timeline

Start Date

August 30, 2023

Primary Completion Date

April 1, 2026

Study Completion Date

October 31, 2030

Conditions
Frontotemporal DementiaFTDFTD-GRNDementia, Frontotemporal
Interventions
PROCEDURE

Intrathalamic AAV.PGRN administration

One-time MRI-guided stereotaxic infusion of AAV.PGRN into the brain

GENETIC

Intrathalamic AVB-101

AVB-101 is made from an adeno-associated virus, serotype 9 (AAV9). AAVs are small viruses that are naturally occurring and do not cause illness or infection on their own. AVB-101 has been modified to contain a copy of the correct (non-mutated) GRN gene, plus some other genetic material to enable the GRN gene to function inside neurons (cells within the brain). AVB-101 has also been modified so that it cannot divide and make new copies of itself (known as 'replication'), which means that it cannot cause disease or a large immune response in your body.

Trial Locations (17)

37232

RECRUITING

Vanderbilt University Medical Centre, Nashville

43210

RECRUITING

The Ohio State University (OSU) Wexner Medical Center, Columbus

46026

RECRUITING

Hospital Universitari i Politecnic La Fe, Valencia

77030

RECRUITING

Houston Methodist Hospital, Houston

Unknown

RECRUITING

Sunnybrook Health Sciences Centre, Toronto

RECRUITING

Amsterdam UMC, Amsterdam

RECRUITING

NEURO-CARE Sp. z o.o. Sp. Komandytowa, Katowice

RECRUITING

Neurologia Slaska Centrum Medyczne, Katowice

RECRUITING

Uniwersyteckie Centrum Kliniczne, SUM w Katowicach, Katowice

RECRUITING

Centrum Medyczne NeuroProtect Sp z o.o., Warsaw

RECRUITING

Mazowiecki Szpital Brodnowski Sp. z o. o., Warsaw

RECRUITING

Skåne University Hospital, Lund

RECRUITING

University College London Hospitals, London

70-111

RECRUITING

Euromedis Sp. z o.o., Szczecin

08036

RECRUITING

Hospital Clinic Barcelona, Barcelona

CB2 0QQ

RECRUITING

Cambridge University Hospitals NHS Foundation Trust, Cambridge

CF14 4XW

RECRUITING

University Hospital of Wales, Cardiff

Sponsors

Lead Sponsor

All Listed Sponsors
lead

AviadoBio Ltd

INDUSTRY

NCT06064890 - 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) | Biotech Hunter | Biotech Hunter