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RECRUITING
NCT07774975
PHASE1

Safety Evaluation Study of Pitavastatin PLGA Nano-particle for Retinitis Pigmentosa

Sponsor: Kyushu University

View on ClinicalTrials.gov

Summary

Retinitis pigmentosa (RP) is a rare inherited retinal degenerative disease that causes progressive visual field loss and vision impairment, often leading to blindness. Currently, there are limited treatment options capable of slowing disease progression for the majority of patients with RP. Preclinical studies have suggested that retinal inflammation, particularly the activity of inflammatory monocytes and macrophages, may contribute to photoreceptor degeneration. ULREA-PVS-NP is a novel intravenous formulation consisting of pitavastatin encapsulated in poly(lactic-co-glycolic acid) (PLGA) nanoparticles, developed to target inflammatory pathways associated with retinal degeneration. Preclinical studies demonstrated suppression of inflammatory monocyte/macrophage activity and preservation of photoreceptors in animal models of RP. This is a single-center, open-label, investigator-initiated Phase 1 study designed to evaluate the safety of intravenous ULREA-PVS-NP in adults with RP. The study consists of two parts. In Part 1, participants will receive a single intravenous infusion of ULREA-PVS-NP at escalating dose levels (2 mg, 4 mg, or 8 mg) to evaluate safety and tolerability. Following review of safety data, Part 2 will evaluate repeated administration of the highest dose considered safe in Part 1, given once every four weeks for a total of three administrations. The primary objective is to evaluate the safety of ULREA-PVS-NP by assessing the incidence of adverse events. Secondary objectives include characterization of pharmacokinetic profiles, evaluation of changes in clinical laboratory tests and vital signs, and exploratory assessment of ophthalmologic outcomes and inflammatory biomarkers.

Official title: Investigator-initiated Phase 1 Clinical Trial to Evaluate the Safety of Pitavastatin PLGA Nano-particle in Patients With Retinitis Pigmentosa

Key Details

Gender

All

Age Range

18 Years - 70 Years

Study Type

INTERVENTIONAL

Enrollment

21

Start Date

2026-10

Completion Date

2028-03-31

Last Updated

2026-10-07

Healthy Volunteers

No

Interventions

DRUG

ULREA-PVS-NP

ULREA-PVS-NP is a poly(lactic-co-glycolic acid) (PLGA) nanoparticle formulation containing pitavastatin and administered intravenously. Part 1 is a single ascending-dose study in which participants receive a single intravenous administration of ULREA-PVS-NP at dose levels equivalent to 2 mg, 4 mg, or 8 mg of pitavastatin. Part 2 is a multiple-dose study in which participants receive repeated intravenous administrations at the highest dose level shown to be safe in Part 1.

Locations (1)

Kyushu University Hospital

Fukuoka, Japan