A Study of RGX-104 in Patients With Advanced Solid Malignancies and Lymphoma


Study Number
51817
Phase
1
Purpose

Study RGX-104-001 is a Phase 1, first-in-human, dose escalation and expansion study of RGX-104, an oral small molecule targeting the liver X receptor (LXR), as a single agent and in combination with nivolumab, ipilimumab, docetaxel, or pembrolizumab plus carboplatin/pemetrexed. RGX-104 activates LXR, resulting in depletion of both myeloid-derived suppressor cells (MDSCs) as well as tumor blood vessels. MDSCs block the ability of T-cells and other cells of the immune system from attacking tumors. During the dose escalation stage, multiple doses and schedules of orally administered RGX-104 with or without nivolumab, ipilimumab, docetaxel, or pembrolizumab plus carboplatin/pemetrexed (single agent or combination therapy) will be evaluated in patients with advanced solid tumors and lymphoma (i.e., locally advanced and unresectable, or metastatic) who have had progressive disease (PD) on available standard systemic therapies or for which there are no standard systemic therapies of relevant impact. Dose escalation in combination with pembrolizumab plus carboplatin/pemetrexed will be restricted to patients with non-small cell lung cancer (NSCLC). In the expansion stage of the study, additional patients with epithelial ovarian carcinoma (EOC), NSCLC, or small cell lung cancer (SCLC)/high-grade neuroendocrine tumors (HG-NET) will be treated at the MTD (or maximum tested dose if no MTD is identified, or dose below the MTD if there is evidence suggesting a more favorable risk/benefit profile). This stage will provide further characterization of the safety, efficacy, PK, and pharmacodynamics, including biomarkers of immunologic activity and LXR target activation, of RGX-104 as a single agent (EOC), in combination with docetaxel (SCLC/HG-NET), and and in combination with pembrolizumab plus carboplatin/pemetrexed (NSCLC).

Full Title

RGX-104-001: A Phase 1 Study of RGX-104, a Small Molecule LXR
Agonist, as a Single Agent and as Combination Therapy in Patients
with Advanced Solid Malignancies and Lymphoma with an Expansion
in Select Malignancies

ClinicalTrials.Gov ID
NCT02922764

To inquire about participating in these studies, call 1-800-ROSWELL (1-800-767-9355) or e-mail askroswell@roswellpark.org.