A Study of PBFT02 in Participants With FTD and Mutations in the Granulin Precursor (GRN) or C9ORF72 Genes (NCT04747431)
A Phase 1b Open-Label, Multicenter, Dose-Escalation Study to Assess the Safety, Tolerability, and Pharmacodynamic Effects of a Single Dose of PBFT02 Delivered Into the ICM of Adults With FTD and Mutations in the GRN or C9ORF72 Genes
🩺 Plain-English Summary (8th-Grade Level)
PBFT02 is a gene therapy for frontotemporal dementia intended to deliver a functional copy of the GRN gene to the brain. This study will assess the safety, tolerability and efficacy of this treatment in patients with frontotemporal dementia and mutations in the granulin precursor (GRN) or chromosome 9 open reading frame 72 (C9ORF72) genes
Who can join: Review the eligibility criteria below.
Location: Michigan Alzheimer's Disease Center — Ann Arbor, Michigan
Age range: 35 Years to 75 Years
🧪 Interventions in This Study
📋 "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 (10 Sites)
- Michigan Alzheimer's Disease Center — Ann Arbor, Michigan
- University of Pennsylvania — Philadelphia, Pennsylvania
- Vanderbilt University Medical Center — Nashville, Tennessee
- University of Texas at Houston — Houston, Texas
- Eastern Health-Box Hill Hospital — Melbourne, Victoria
- Hospital das Clinicas da Universidade Federal de Minas Gerais (UFMG) — Minas Gerais,
- Hospital das Clinicas da Faculdade de Medicina da Universidade de Sao Paulo (HCFMUSP) — São Paulo,
- University of Toronto, Toronto Western Hospital — Toronto, Ontario
- Montreal Neurological Institute-Hospital — Montreal, Quebec
- Centro Hospitalar e Universitário de Coimbra — Coimbra,
💰 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
- 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.
- Review the official NIH record below to find the coordinating site.
- Ask about the visit schedule, what is covered at no cost, and any travel support.
- Confirm with your own doctor before making a decision.