Clinical trials

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.

Cohort A(GAIA-102 alone): Confirm the safety of GAIA-102 alone for refractory/relapse neuroblastoma or pediatric solid tumors with lung metastases, and decide recommended dose for Phase II. Cohort B(GAIA-102 with Dinutuximab): Confirm the safety of GAIA-102 with Dinutuximab, Filgrastim, Teceleukin combination for refractory/relapse neuroblastoma and decide recommended dose for Phase II. Cohort C(GAIA-102 with Nivolumab):Confirm the safety of GAIA-102(Follow the recommended doses in Cohort A) with Nivolumab. Cohort D(GAIA-102 with Nivolumab, Teceleukin): Confirm the safety of GAIA-102(Follow the recommended doses in Cohort A) with Nivolumab, Teceleukin.

The primary objective of this study is to evaluate the safety and tolerability of sivelestat sodium hydrate administered in combination with standard steroid pulse therapy in patients experiencing an acute NMOSD attack. Safety assessments will include adverse events, laboratory parameters, vital signs, and other clinically relevant findings. In addition, the study will explore whether the addition of sivelestat sodium hydrate to standard steroid pulse therapy improves neurological outcomes in patients with acute NMOSD. Participants will receive intravenous sivelestat sodium hydrate at a dose of 4.8 mg/kg/day administered as a continuous infusion (0.2 mg/kg/hour) for 5 consecutive days, receive steroid pulse therapy according to the study protocol, and be followed for 28 days after treatment initiation for safety and efficacy evaluations.

Phase I Part : Confirm the safety of GAIA-102 as a monotherapy or GAIA-102 and pembrolizumab in combination for advanced gastrointestinal cancer of microsatellite stable with malignant ascites, and determine the recommended number of doses for Phase II part. Phase II Part : Research the efficacy and safety of as a monotherapy or GAIA-102 and pembrolizumab for advanced gastrointestinal cancer of microsatellite stable with malignant ascites at the recommended dose of GAIA-102 decided in the Phase I part.