Phase 1 Study of Gemcitabine and Nab-Sirolimus IV Combination for Advanced Leiomyosarcoma or Soft-Tissue Sarcomas with TSC1 or TSC2 Mutations

What this Trial Is About

Researchers are studying the combination of gemcitabine and nab-sirolimus in patients with advanced leiomyosarcomas or soft-tissue sarcomas that have specific genetic mutations called TSC2 or TSC1 loss-of-function mutations or deletions. This Phase I trial aims to find the highest dose of this drug combination that patients can tolerate safely. The study also looks at the side effects, tumor response, and survival outcomes, as well as exploring genetic and molecular markers related to treatment activity. Participants will receive gemcitabine and nab-sirolimus given intravenously. They will be assigned to different dose levels based on when they join the study. This trial focuses on patients with advanced or metastatic disease who meet specific genetic criteria. The study also includes an optional genomic research project to further understand rare tumors. During the study, participants will be closely monitored for safety and side effects throughout treatment, which lasts about one year on average. Researchers will use imaging tests to measure tumor size and check blood samples for tumor DNA changes. They will also assess molecular markers in tumor samples to understand the treatments effects. Participants must agree to regular testing and follow-up visits during the study period.

A Phase I Trial of Combination Gemcitabine and Nab-Sirolimus in Advanced Leiomyosarcomas or Advanced Soft-Tissue Sarcomas With TSC2 or TSC1 Loss-of-function Mutations or Deletions