Randomized Phase II Study Comparing Cryoablation and Chemotherapy for Desmoid Tumors That Worsen After Watchful Waiting

What this Trial Is About

Researchers are evaluating treatments for desmoid tumors DT that progress after an initial period of watchful waiting, which is currently the standard care. This phase II randomized trial compares cryoablation, a procedure that freezes the tumor, with medical therapy using chemotherapy drugs. The trial also includes a cross-over design, allowing patients to receive cryoablation if needed after initial treatment. Participants are assigned to one of two groups one receives a single cryoablation procedure on Day 0, while the other receives chemotherapy chosen by the investigator. Chemotherapy options include methotrexate plus vinblastine given weekly by intravenous infusion for the first 6 months and less frequently afterward, or vinorelbine taken orally for 12 months, with doses adjusted for adults and adolescents. The study aims to assess treatment effects over several months, including the possibility of switching treatments. During the study, participants undergo regular assessments including MRI scans to measure tumor response, pain evaluations, quality of life questionnaires, and monitoring for side effects. Researchers will measure the rate of disease control at 12 months and track progression-free survival and treatment-related adverse events up to 39 months. Health economics and patient-reported outcomes are also evaluated, with ongoing follow-up to understand long-term effects.

Cryoablation Versus Medical Therapy in Desmoid Tumors Progressing After Watchful Waiting