NCT06528769

Study of All-Trans Retinoic Acid (ATRA) and Cemiplimab in Patients With Advanced Leiomyosarcoma

A Phase II Study of All-Trans Retinoic Acid (ATRA) and Cemiplimab in Patients With Advanced Leiomyosarcoma

• Status: Recruiting
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 16 (estimated)
• Sponsor: Gabriel Tinoco · Academic / Other
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

A phase 2 study assessing the efficacy of all-trans retinoic acid (ATRA) and Cemiplimab in patients with metastatic/locally advanced - unresectable leiomyosarcoma (LMS) who have progressed standard-of-care therapy. Patients will be enrolled in cohorts according to a Bayesian Optimal Phase II design (BOP2). Study treatment will consist of ATRA at a starting dose of 150 mg/m2/day for 3 days orally prior to each cycle of Cemiplimab 350 mg IV q3 weeks for three cycles and then Cemiplimab monotherapy until the progress of disease or unacceptable toxicities develops.

Detailed description

PRIMARY OBJECTIVES I. To determine the efficacy of the combination of ATRA and Cemiplimab in patients with metastatic/locally advanced -unresectable LMS by assessing objective response (ORR), Response Evaluation Criteria in Solid Tumors (RECIST) version 1.1 and immune mediated Response Evaluation Criteria for Solid Tumors (iRECIST). SECONDARY OBJECTIVES I. To study the efficacy of the combination of ATRA and Cemiplimab in patients with advanced LMS, including progression-free survival (PFS), disease control rate (DCR), and overall survival (OS) based on modified RECIST version 1.1 and iRECIST. II. To determine the safety and tolerability of the combination of ATRA and Cemiplimab in patients with metastatic/locally advanced -unresectable LMS based upon the Common Terminology Criteria for Adverse Events (CTCAE) version 5 criteria. EXPLORATORY OBJECTIVES I. To study the effect of ATRA on the levels of myeloid-derived suppressor cells (MDSCs) in the peripheral blood of study patients OUTLINE: Patients receive ATRA orally (PO) on days -2 to 0 followed by cemiplimab intravenously (IV) on day 1 of each cycle. ATRA treatment is given for 3 cycles. Cycles repeat every 21 days in the absence of disease progression or unacceptable toxicity. Patients undergo urine sample collection during screening, and computed tomography (CT) scan/magnetic resonance imaging (MRI) and blood sample collection throughout the study. After completion of study treatment, patients are followed up every 12-16 weeks. .

Conditions

• Leiomyosarcoma
• Sarcoma

Interventions

Type	Name	Description
DRUG	all-trans retinoic acid	Given orally
DRUG	Cemiplimab	Given intravenously (IV)
PROCEDURE	Computed Tomography	Undergo CT
PROCEDURE	Magnetic resonance imaging	Undergo MRI
PROCEDURE	Biospecimen Collection	Undergo blood and urine sample collection

Timeline

• Start date: 2025-09-10
• Primary completion: 2027-12-31
• Completion: 2027-12-31
• First posted: 2024-07-30
• Last updated: 2025-09-30

Locations

1 site across 1 country: United States

Regulatory

• FDA-regulated drug study