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

ConditionsLeiomyosarcoma Sarcoma

Trial statusRecruiting

Trial phasePhase 2

Trial typeInterventional

Biological sexAll

Age18+

SponsorGabriel Tinoco

About this trial

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.

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

Age ≥18 years

Confirmed metastatic or locally advanced - unresectable Leiomyosarcoma (LMS)

Measurable disease based on RECIST 1.1. (At least one target lesion)

Patients must have received standard of care chemotherapy. No limits to prior lines of therapy.

Disqualifiers

Is currently participating and receiving study therapy or has participated in a study of an investigational agent and received study therapy or used an investigational device within 4 weeks of the first dose of treatment.

Patients with a history of autoimmune-related hypothyroidism who are stable on thyroid-replacement hormone are eligible for the study

Patients with controlled Type 1 diabetes mellitus who are on an insulin regimen are eligible for the study.

Rash must cover ≤ 10% of body surface area

Trial design

Design model

Single group

Treatments tested in this trial

all-trans retinoic acid
Drug
Given orally
Cemiplimab
Drug
Given intravenously (IV)
Computed Tomography
Procedure/Surgery
Undergo CT
Magnetic resonance imaging
Procedure/Surgery
Undergo MRI
Biospecimen Collection
Procedure/Surgery
Undergo blood and urine sample collection

Treatment groups

16 Participants

are divided into 1 treatment group

Group A: Treatment (all-trans retinoic acid [ATRA] and cemiplimab)Experimental treatment 5 interventions

Trial outcomes

Primary outcomes

Overall Response Rate (ORR)

Overall response rate (ORR) will be calculated based on modified RECIST version 1.1 and iRECIST with a 95% confidence interval (CI).

Time frame

Up to 24 months

Secondary outcomes

Progression-free survival (PFS)

Progression free survival (PFS) will be defined as the time from initiation of therapy to the time of RECIST progression or death and will be calculated based on modified RECIST version 1.1 and iRECIST. Kaplan-Meier methods will be used to estimate PFS with 95% CI.

Time frame

Up to 24 months

Disease control rate (DCR)

Percentage of patients whose disease shrinks or remains stable. DCR will be calculated based on modified RECIST version 1.1 and iRECIST with a 95% confidence interval (CI).

Time frame

Up to 24 months

Overall survival (OS)

Overall survival (OS) will be defined as time from initiation of therapy to death, or censored at last follow-up date if the Patient is alive and will be calculated based on modified RECIST version 1.1 and iRECIST. Kaplan-Meier methods will be used to estimate OS with 95% CI.

Time frame

Up to 24 months

Dose limiting toxicity

The maximum grade for each type of toxicity will be recorded for each patient, and frequency tables will be reviewed to determine toxicity patterns.

Time frame

From the first dose of study medication, up to 2 years

Other outcomes

Effect of ATRA on myeloid-derived suppressor cells (MDSCs)

MDSCs in peripheral blood from study patients will be summarized using mean +/- SEM, range, and median. Changes in these measurements from baseline to after treatment, or baseline to progression will be assessed using paired t-test or Wilcoxon signed rank test.

Time frame

Up to 24 months

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

Gabriel Tinoco

Lead sponsor

Ohio State University Comprehensive Cancer Center

Sponsor institution

Source ClinicalTrials.gov

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