Phase II Trial of Ribociclib and Everolimus in Advanced Dedifferentiated Liposarcoma (DDL) and Leiomyosarcoma (LMS)

Phase 2

Active, not recruiting

• Conditions: Soft Tissue Sarcoma
• Interventions: Drug: Ribociclib; Drug: Everolimus

• Registration Number: NCT03114527
• Lead Sponsor: Fox Chase Cancer Center

Brief Summary

This is a two center, 2 arm, Phase II study evaluating the combination of Ribociclib and Everolimus in patients with advanced DDL and LMS who have had at least 1 prior systemic therapy. Patients will be enrolled by sarcoma histology into DDL (Arm A) and LMS (Arm B). The purpose of this study is to determine the anti-tumor activity of this doublet therapy in these patient cohorts. Ribociclib will be administered orally at 300 mg/day 3 weeks on/1 week off. Everolimus will be administered 2.5 mg orally on a continuous 28 day cycle. Clinical and laboratory assessments will be made on day 1, d15 of cycle 1 and 2, and day 1 of each subsequent cycle. Tumor response will be assessed by RECIST 1.1 at (CT or MRI) at week 8, 16, 24 and every 12 weeks thereafter. Study drug administration will continue until disease progression, unacceptable toxicity or withdrawal of consent. Patients will be followed until death or are lost to follow-up for analysis of secondary endpoints.

There will be a 1 step registration process for dedifferentiated liposarcoma patients while patients with leiomyosarcoma will require a 2 step registration process. For step 1 of registration, patients must meet all the eligibility criteria necessary for step 1. For step 2 registration, patients must meet the inclusion criteria necessary for step 2 to be enrolled into the study.

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2017-03-23
• Study First Submitted QC: 2017-04-10
• Study First Posted: 2017-04-14
• Study Start: 2017-08-08
• Primary Completion: 2020-11-12
• Status Verified: 2025-04
• Last Update Submitted: 2025-04-03
• Last Update Posted: 2025-04-04
• Study Completion: 2025-12

Recruitment & Eligibility

• Status: ACTIVE_NOT_RECRUITING
• Sex: All
• Target Recruitment: 48

Inclusion Criteria

Not provided

Exclusion Criteria

Not provided

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
Leiomyosarcoma Arm	Ribociclib	Patients receive ribociclib orally at 300mg/day for 21 days of each 28 day cycle and everolimus 2.5mg orally on a continuous 28 day cycle.
Dedifferentiated Liposarcoma Arm	Ribociclib	Patients receive ribociclib orally at 300mg/day for 21 days of each 28 day cycle and everolimus 2.5mg orally on a continuous 28 day cycle.
Dedifferentiated Liposarcoma Arm	Everolimus	Patients receive ribociclib orally at 300mg/day for 21 days of each 28 day cycle and everolimus 2.5mg orally on a continuous 28 day cycle.
Leiomyosarcoma Arm	Everolimus	Patients receive ribociclib orally at 300mg/day for 21 days of each 28 day cycle and everolimus 2.5mg orally on a continuous 28 day cycle.

Primary Outcome Measures

Name	Time	Method
Antitumor activity of Ribociclib in combination with Everolimus in advanced LMS or DDL	16 weeks	Progression free rate

Secondary Outcome Measures

Name	Time	Method
Progression-free survival (PFS)	Through study completion	PFS will be measured by RECIST 1.1.
Objective response rate (ORR)	Through study completion	ORR will be measured by RECIST 1.1.
Overall survival (OS)	Through study completion	OS will be measured from the time of initiation of treatment until death
Number of participants with treatment-related adverse events	Up to 30 days after final treatment on study	Treatment-related adverse events will be assessed by CTCAE v4.03

Trial Locations

Locations (2)

Dana-Farber Cancer Institute; 🇺🇸 Boston, Massachusetts, United States

Fox Chase Cancer Center; 🇺🇸 Philadelphia, Pennsylvania, United States