NCT05918640

Lurbinectedin in FET-Fused Tumors

Lurbinectedin in FET-Fusion Tumors (LIFFT)

• Status: Recruiting
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 63 (estimated)
• Sponsor: Children's Hospital of Philadelphia · Academic / Other
• Sex: All
• Age: 10 Years
• Healthy volunteers: Not accepted

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.

Detailed 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 patients.

Conditions

• Ewing Sarcoma
• Desmoplastic Small Round Cell Tumor
• Pediatric Cancer
• Undifferentiated Sarcoma

Interventions

Type	Name	Description
DRUG	Lurbinectedin	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.

Timeline

• Start date: 2023-07-27
• Primary completion: 2027-07-30
• Completion: 2028-07-30
• First posted: 2023-06-26
• Last updated: 2026-03-31

Locations

6 sites across 1 country: United States

Regulatory

• FDA-regulated drug study