Clinical Trial: Tailored Beta-catenin Mutational Approach in Extra-abdominal Sporadic Desmoids Tumor Patients

Study Status: Recruiting
Recruit Status: Recruiting
Study Type: Observational

Official Title: Tailored Beta-catenin Mutational Approach in Extra-abdominal Sporadic Desmoids Tumor Patients

Brief Summary:

This is a prospective, multicenter observational study under the umbrella of the ISG (Italian Sarcoma Group) evaluating local progression-free survival at 3 years of patients affected by extra-abdominal primary fibromatosis managed with front-line conservative approach and treated only in case of demonstrated progressive disease.

All patients included will be placed on wait and see approach and then shifted to treatment in case of documented radiological progressive disease.

For patient primarily evaluated for suspected desmoid tumor, a core-needle biopsy (eventually under CT/ultrasound guide) will be obtained for histological diagnosis and mutational analysis of CTNNB1 exon 3 (gene encoding Beta-catenin). If incisional biopsy or surgical procedure has done elsewhere, samples will be requested for histological confirmation and mutational analysis (centralization at the investigators Institution).

In case of progression at 3 months, defined as tumor growth documented radiologically (by contrast enhanced MRI) by Response Evaluation Criteria in Solid Tumors (RECIST), administered treatments will be proposed and then registered in the clinical database.The choice of the treatment and eventually the possibility of continuation of " surveillance only" will be at the discretion of Institution's Multidisciplinary Sarcoma Committee or as part of clinical trials with the consent of patient.


Detailed Summary:

This is a prospective, multicenter observational study under the umbrella of the ISG (Italian Sarcoma Group) evaluating local progression-free survival at 3 years of patients affected by extra-abdominal primary fibromatosis managed with front-line conservative approach and treated only in case of demonstrated progressive disease.

All patients included will be placed on wait and see approach and then shifted to treatment in case of documented radiological progressive disease.

For patient primarily evaluated for suspected desmoid tumor, a core-needle biopsy (eventually under CT/ultrasound guide) will be obtained for histological diagnosis and mutational analysis of CTNNB1 exon 3 (gene encoding Beta-catenin). If incisional biopsy or surgical procedure has done elsewhere, samples will be requested for histological confirmation and mutational analysis (centralization at our Institution) and in selected cases a new biopsy will be obtained.

In case of progression at 3 months, defined as tumor growth documented radiologically (by contrast enhanced MRI) by Response Evaluation Criteria in Solid Tumors (RECIST), administered treatments will be proposed and then registered in the clinical database (radiological evaluation will be centralized at Istituto Rizzoli, Bologna- Dr. Vandel).

Therapy will include the following options:

  • Surgery
  • Radiotherapy
  • Medical treatment including hormonal therapy (e.g., tamoxifen, toremifene), low-dose chemotherapy (e.g.,methotrexate and vinorelbine/vinblastine), NSAIDs (e.g., celecoxib), and target therapy (Glivec)
  • Combinat
    Sponsor: Fondazione IRCCS Istituto Nazionale dei Tumori, Milano

    Current Primary Outcome: Progression free survival [ Time Frame: 3 years ]

    Original Primary Outcome: Same as current

    Current Secondary Outcome:

    Original Secondary Outcome:

    Information By: Fondazione IRCCS Istituto Nazionale dei Tumori, Milano

    Dates:
    Date Received: January 28, 2015
    Date Started: July 2013
    Date Completion: August 2016
    Last Updated: September 10, 2015
    Last Verified: September 2015