Phase 1/2 Clinical Trial of LY3884963 in Patients With Frontotemporal Dementia With Progranulin Mutations (FTD-GRN) (NCT04408625)
A Phase 1/2 Ascending Dose Study to Evaluate the Safety and Effects on Progranulin Levels of LY3884963 in Patients With Fronto-Temporal Dementia With Progranulin Mutations (FTD-GRN)
🩺 Plain-English Summary (8th-Grade Level)
Study J4B-MC-OKAA is a Phase 1/2, multi-center, open-label ascending dose, first-in-human study that will evaluate the safety and effect of intra-cisternal LY3884963 administration on progranulin protein (PGRN) levels in patients with frontotemporal dementia with progranulin mutations (FTD-GRN). Two escalating dose (low dose and medium dose) cohorts are planned, as well as one bridging cohort which will allocate patients to receive either low or medium dose. The duration of the study is 5 years. During the first year, patients will be evaluated for the effect of LY3884963 on safety, tolerability, immunogenicity, biomarkers, and efficacy. Patients will follow up for an additional 4 years to monitor safety and changes on selected biomarkers and clinical outcomes.
Who can join: Review the eligibility criteria below.
Location: k2 Medical Research-Maitland — Maitland, Florida
Age range: 30 Years to 85 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 (12 Sites)
- k2 Medical Research-Maitland — Maitland, Florida
- PPD Phase 1 Clinic, 100 West Gore Street, Suite 202 — Orlando, Florida
- Lahey Hospital & Medical Center, 41 Burlington Mall Road — Burlington, Massachusetts
- Hospital of the University of Pennsylvania, 3 West Gates Building, 3400 Spruce Street — Philadelphia, Pennsylvania
- Royal Prince Alfred Hospital, Brain & Mind Research Institute, 94 Mallet Street — Camperdown, New South Wales
- UZ Leuven, Neurologie Herestraat 49 — Leuven,
- AP-HM Hôpital de La Timone — Saint-Pierre, Marseille
- Centre Mémoire de Ressources — Lille,
- Le Ber, Institut du Cerveau et de la Moelle Epinière — Paris,
- Hospital Clinic de Barcelona, Villaroel 170 Servicio de Neurología — Barcelona,
- Hospital Universitario de Donostia, Servicio De Neurologia, Consultas Externas Neurologia, San Sebastian, Guipúzcoa — Donostia / San Sebastian,
- University College London,Queen Square, Dementia Research Building, London, — London,
💰 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
- 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.