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) (NCT06064890)
A Phase 1/2 Open-Label, Ascending Dose, Multicenter Study to Evaluate the Safety and Preliminary Efficacy of AVB-101 Administered by Bilateral Intrathalamic Infusion in Subjects With Frontotemporal Dementia With Progranulin Mutations (FTD-GRN)
🩺 Plain-English Summary (8th-Grade Level)
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 (FTD-GRN). FTD-GRN is an early-onset form of dementia, a progressive brain disorder that affects behavior, language and movement. These symptoms result from below normal levels of a protein called progranulin (PGRN) in the brain, which leads to the death of nerve cells (neurons), affecting the brain's ability to function. The main questions that the study aims to answer are: 1. Is a one-time treatment with AVB-101 safe for patients with FTD-GRN? 2. Does a one-time treatment with AVB-101 restore PGRN levels to at least normal levels? 3. Could AVB-101 work as a treatment to slow down or stop progression of FTD-GRN? In this study there is no placebo (a dummy pill or treatment used for comparison purposes), so all participants will receive a one-time treatment of AVB-101 delivered directly to the brain, with follow-up assessments for 5 years.
Who can join: Review the eligibility criteria below.
Location: The Ohio State University (OSU) Wexner Medical Center — Columbus, Ohio
Age range: 30 Years to 75 Years
🧪 Interventions in This Study
- Intrathalamic AAV.PGRN administration (PROCEDURE)
- Intrathalamic AVB-101 (GENETIC)
📋 "Do I Qualify?" — 1-Minute Self Screener
Instant ChecklistFull eligibility criteria are available on the official NIH protocol. View the criteria ↗
📍 All Participating Trial Locations (19 Sites)
- The Ohio State University (OSU) Wexner Medical Center — Columbus, Ohio
- Vanderbilt University Medical Centre — Nashville, Tennessee
- Houston Methodist Hospital — Houston, Texas
- UZ Leuven — Leuven,
- Sunnybrook Health Sciences Centre — Toronto, Ontario
- Fondazione IRCCS Istituto Neurologico Carlo Besta — Milan,
- Amsterdam UMC — Amsterdam,
- NEURO-CARE Sp. z o.o. Sp. Komandytowa — Katowice,
- Neurologia Slaska Centrum Medyczne — Katowice,
- Uniwersyteckie Centrum Kliniczne, SUM w Katowicach — Katowice,
- Euromedis Sp. z o.o. — Szczecin,
- Centrum Medyczne NeuroProtect Sp z o.o. — Warsaw,
- Mazowiecki Szpital Brodnowski Sp. z o. o. — Warsaw,
- Hospital Clinic Barcelona — Barcelona,
- Hospital Universitari i Politecnic La Fe — Valencia,
- Skåne University Hospital — Lund,
- Cambridge University Hospitals NHS Foundation Trust — Cambridge,
- University Hospital of Wales — Cardiff,
- University College London Hospitals — London,
📞 Contact the Study Coordinator
Phone: +44 203-089-7917
Email: clinicaltrials@aviadobio.com
Coordinator details come directly from the registered NIH protocol. Confirm the study is still recruiting before traveling.
💰 Cost, Insurance & Patient Rights
- 100% Free Items: The investigational treatment and protocol-required procedures are supplied at zero cost by the sponsor.
- Routine Patient Care: Under federal law (ACA § 2709) and many state statutes, routine care costs are covered during participation.
- Voluntary Participation: You may withdraw at any time without affecting your standard medical care.
More studies in Frontotemporal Dementia
- Neurofilament Surveillance Project (NSP) — San Francisco, California
- A Study of PBFT02 in Participants With FTD and Mutations in the Granulin Precursor (GRN) or C9ORF72 Genes — Ann Arbor, Michigan
- Music, Social Attention, and Dementia — San Francisco, California
- Phase 1/2 Clinical Trial of LY3884963 in Patients With Frontotemporal Dementia With Progranulin Mutations (FTD-GRN) — Maitland, Florida
- A Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of DNL593 in Healthy Participants and Participants With Frontotemporal Dementia (FTD-GRN) — San Francisco, California
- Investigating Complex Neurodegenerative Disorders Related to Amyotrophic Lateral Sclerosis and Frontotemporal Dementia — Bethesda, Maryland
Explore by condition: All Frontotemporal Dementia clinical trials
💡 How to Participate
- Check the checklist above to see if you may qualify.
- Contact the study coordinator using the phone/email above.
- Ask about the visit schedule, what is covered at no cost, and any travel support.
- Confirm with your own doctor before making a decision.