NCT04408625

Phase 1/2 Clinical Trial of LY3884963 in Patients With Frontotemporal Dementia With Progranulin Mutations (FTD-GRN)

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)

• Status: Active Not Recruiting
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 35 (estimated)
• Sponsor: Prevail Therapeutics · Industry
• Sex: All
• Age: 30 Years – 85 Years
• Healthy volunteers: Not accepted

Summary

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.

Conditions

• Frontotemporal Dementia

Interventions

Type	Name	Description
BIOLOGICAL	LY3884963	Participants will receive a single dose of LY3884963, administered intra cisterna magna
DRUG	Methylprednisolone	IV pulses every 2 weeks in the first 3 months.
DRUG	Optional Sirolimus	At the investigators discretion following steroid tolerability issues, patients may receive a loading dose, followed by maintenance dose, followed by dose tapering; administered as concomitant medication
DRUG	Optional Prednisone	If needed and at the investigator discretion, Oral Prednisone may be added to the immunosuppression regimen

Timeline

• Start date: 2020-11-09
• Primary completion: 2029-11-30
• Completion: 2029-11-30
• First posted: 2020-05-29
• Last updated: 2026-02-10

Locations

12 sites across 6 countries: United States, Australia, Belgium, France, Spain, United Kingdom

Regulatory

• FDA-regulated drug study