A Study of RGX-104 in Patients With Advanced Solid Malignancies and Lymphoma
Status:
Recruiting
Trial end date:
2022-03-01
Target enrollment:
Participant gender:
Summary
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).