Lurbinectedin in FET-Fusion Tumors (LIFFT)
Children's Hospital of Philadelphia
Summary
The purpose of this study is to find out if a drug called lurbinectedin (the "study drug") is safe and effective at treating people with recurrent or relapsed solid tumors, including Ewing sarcoma.
Description
In this study, the investigators will test the activity of lurbinectedin as a targeted therapy for FET (FUS, Ewing Sarcoma Breakpoint Region 1 (EWRS1), TATA-Box-Binding Protein Associated Factor 15 (TAF15)). Ewing sarcoma is driven by the Ewing Sarcoma-Friend Leukemia Integration 1 Transcription Factor (EWS-FLI1). Lurbinectedin has been shown to inhibit EWS-FLI1 and Ewing Sarcoma-Wilms' Tumor Gene 1 (EWS-WT1) in preclinical models. Therefore, the goal of this study is to see if Lurbinectedin can be used to inhibit EWS-FLI1, EWS-WT1, or other FET fusion proteins to drive tumor responses in pati…
Eligibility
- Age range
- 10+ years
- Sex
- All
- Healthy volunteers
- No
Inclusion Criteria: 1. Age ≥ 10 years. 2. Phase 1: Histological confirmed diagnosis of recurrent or relapsed solid tumor failing primary therapy. Patients must have a known FET fusion (fusion that contains EWSR1, FUS, or TAF15) as documented by next generation sequencing, polymerase chain reaction (PCR) or Fluorescence in situ hybridization (FISH). Patients with a histological diagnosis of Ewing sarcoma with EWS-FLI1 are eligible for dose escalation but not for the exploratory cohort. Please note patients with Ewing sarcoma and alternative FET-ETS fusions (including but not limited to EWS-ERG…
Interventions
- DrugLurbinectedin
Lurbinectedin will be administered on a Day 1, Day 4 schedule every 21 days. Doses will be determined in the phase 1 portion of the trial.
Locations (6)
- Cedars-Sinai Medical CenterLos Angeles, California
- University of Iowa Hospitals and ClinicsIowa City, Iowa
- Dana-Farber Cancer InstituteBoston, Massachusetts
- University of MichiganAnn Arbor, Michigan
- Memorial Sloan Kettering Cancer CenterNew York, New York
- Children's Hospital of PhiladelphiaPhiladelphia, Pennsylvania