NCT02922764

A Study of RGX-104 in Patients With Advanced Lung & Endometrial Cancer

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 Expansion in Select Malignancies

• Status: Completed
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 146 (actual)
• Sponsor: Inspirna, Inc. · Industry
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

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.

Detailed description

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 endometrial cancer, 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).

Conditions

• Endometrial Cancer
• Endometrial Cancer Recurrent
• Lung Cancer Recurrent
• Lung Cancer
• Non-small Cell Lung Cancer Metastatic
• Non-small Cell Carcinoma

Interventions

Type	Name	Description
DRUG	RGX-104
DRUG	Ipilimumab
DRUG	Docetaxel
DRUG	Pembrolizumab
DRUG	Carboplatin
DRUG	Pemetrexed

Timeline

• Start date: 2016-11-01
• Primary completion: 2025-01-22
• Completion: 2025-01-22
• First posted: 2016-10-04
• Last updated: 2025-03-26

Locations

21 sites across 1 country: United States