"Wait \& see" is currently the standard of care of recently diagnosed desmoid tumors (DT). In case of progression or symptomatic disease, medical therapy is nowadays widely used including chemotherapy.
Cryoablation has proven to be beneficial for the treatment of large, progressive and symptomatic DT.
This randomized phase II trial aims to compare cryoablation versus medical therapy in DT patients progressing after the "wait \& see" period. Moreover, a cross-over design has been anticipated to allow all patients to undergo cryoablation if necessary.
Subject (male or female) with extra-peritoneal desmoid tumor (confirmed by prior biopsy by an experienced pathologist within the RRePS network)
13 years of age or older
Measurable viable tumor (according to the mRECIST criteria using MRI. The baseline MRI imaging is mandatory in the 2 months prior to treatment initiation (D0).
Progressive disease (according to the mRECIST criteria) after the watchful waiting period or significant increase in symptoms requiring an active therapy, as advised in a multidisciplinary sarcoma tumor board
Intra-peritoneal or multifocal desmoid tumor
Concurrent or prior use of any antitumor agent for the current desmoid tumor
Relapse after surgery for desmoid tumor
Any contraindication including known hypersensitivity to vinorelbine or other vinca-alkaloids, or other constituents to navelbine, to vinblastine, to methotrexate or any excipients, such as, but not limited to current or recent (within 2 weeks) severe infection, severe renal failure, severe hepatic injury, chronic respiratory failure
Crossover
Cryoablation
Procedure/SurgeryPercutaneous imaging-guided cryoablation
Chemotherapy drug
DrugEither : methotrexate 30mg/m² + vinblastine 6mg/m² (IV infusion) once/week for 6 months, then every other week from months 7 to 12, or vinorelbine : for adults : 90mg/week (per os: 3x30mg, soft capsules) for 12 months; for adolescents: 60mg/m2/week (capped at 90mg/week) for 12 months
Rate of non-progressive disease
Rates of non-progressive disease (sum of complete response (CR), partial response (PR), and stable disease (SD) according to modified RECIST criteria)
Progression-free survival 1
Progression-free survival 1 (PFS1: from the beginning of cryoablation or medical therapy to first disease progression) in cryoablation and medical therapy group
Rate of patients who cross over to the other arm
Rate of complete response
Rate of complete response at 12 months in cryoablation and medical therapy group with and without a cross-over treatment
Secondary Progression-free survival 2
Secondary Progression-free survival 2 (PFS 2: from the cross-over to second disease progression) in cryoablation and medical therapy group
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University Hospital, Strasbourg, France
Lead sponsor
Direction Générale de l'Offre des Soins (DGOS)
Collaborator
