A Phase II Study of BMS-986504 for Treating Unresectable Malignant Peripheral Nerve Sheath Tumors with MTAP Gene Loss

What this Trial Is About

Malignant Peripheral Nerve Sheath Tumors MPNSTs are rare, aggressive cancers that develop in soft tissues and are a leading cause of serious illness and death in people with neurofibromatosis type 1. This research investigates whether a new drug called BMS-986504, which targets a specific gene mutation called homozygous MTAP deletion, may work better than existing treatments for patients with unresectable MPNST. The study focuses on evaluating the safety and effectiveness of this treatment in people whose tumors cannot be surgically removed and who have this genetic mutation confirmed by next-generation sequencing. Participants will receive BMS-986504 orally at a dose of 600 milligrams daily on Days 1 to 28 of each 28-day cycle. Treatment will continue until the disease worsens, unacceptable side effects occur, or participants choose to stop. Those experiencing dose-limiting toxicity may have their dose reduced or be taken off the trial. The study enrolls participants in two stages nine in the first stage, and if at least one shows response, eight more in the second stage. This design helps assess safety and tumor response over time. Throughout the study, participants will undergo regular assessments including scans to measure tumor size and blood tests to monitor organ function and side effects. Researchers will track objective response rates, progression-free survival, overall survival, adverse events, and treatment discontinuations for up to two years. Participants will be monitored closely for safety and treatment effects during and after therapy, with follow-up evaluations continuing to capture outcomes and ensure well-being.

Safety and Efficacy of BMS-986504 in Unresectable Malignant Peripheral Nerve Sheath Tumor