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

NCT02547831 | Completed

• 1 other identifier: INT 13/12
• Study Type: observational
• Target: 100
• Locations: 1 country
• Sites: 2

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.

Conditions

Desmoid-type Fibromatosis

Outcome Measures

Primary Outcomes (1)

• Progression free survival

  3 years

Study Arms (1)

Observational approach

Patients will be placed on wait and see approach and then shifted to specific treatment in case of progression

Other: Observational approach

Interventions

Observational approach OTHER

Patients will be placed under wait and see approach without any specific treatment

Also known as: Wait and see approach

Observational approach

Eligibility Criteria

• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

Patients affected by extra-abdominal primary fibromatosis

You may qualify if:

• Sporadic forms
• No age limit (pediatric patients can be included)
• Extra abdominal fibromatosis primary or with previous inadequate resection (R2) of the extremities, chest/abdominal wall and head/neck
• Histological diagnosis according to the WHO criteria done on biopsy or surgical specimen by our pathologist
• Diagnostic radiological exam performed (contrast enhanced MRI- T1 and T2 weighted)
• Signed informed consent form
• Adequate compliance of the patients to the plan of follow-up

You may not qualify if:

• Controindication to MRI
• Familial-type desmoid
• Recurrence
• Extraabdominal primary fibromatosis resected with R0/R1 margins
• Patients on treatment for desmoid tumor
• Other malignancies within past 5 years, with exception of carcinoma in situ of cervix and basocellular skin cancers treated with eradicating intent
• Serious psychiatric disease that precludes informed consent or limits compliance
• Medical disease requesting treatment corresponding to one of the drugs currently use in desmoid tumor \[hormonal therapy (e.g., tamoxifen/toremifene, low-dose chemotherapy (e.g., methotrexate and vinorelbine/vinblastine), NSAIDs (e.g., celecoxib), and target therapy (Glivec)\]
• Impossibility to ensure adequate follow-up

Sponsors & Collaborators

• Fondazione IRCCS Istituto Nazionale dei Tumori, Milano: lead
• Ministero della Salute, Italy: collaborator

Study Sites (2)

Fondazione del Piemonte per l' Oncologia - IRCCS

Candiolo, Torino, 10060, Italy

Location

Fondazione IRCCS Istituto Tumori Milano

Milan, 20133, Italy

Location

Related Publications (1)

• Colombo C, Fiore M, Grignani G, Tolomeo F, Merlini A, Palassini E, Collini P, Stacchiotti S, Casali PG, Perrone F, Mariani L, Gronchi A. A Prospective Observational Study of Active Surveillance in Primary Desmoid Fibromatosis. Clin Cancer Res. 2022 Sep 15;28(18):4027-4032. doi: 10.1158/1078-0432.CCR-21-4205.

  PMID: 35247923 DERIVED

MeSH Terms

Conditions

Desmoid Tumors

Condition Hierarchy (Ancestors)

Fibroma; Neoplasms, Fibrous Tissue; Neoplasms, Connective Tissue; Neoplasms, Connective and Soft Tissue; Neoplasms by Histologic Type; Neoplasms

Study Officials

• Alessandro Gronchi, MD

  Fondazione IRCCS Istituto Tumori Milano

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: observational
• Observational Model: CASE ONLY
• Time Perspective: PROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: January 28, 2015
• First Posted: September 11, 2015
• Study Start: July 1, 2013
• Primary Completion: December 1, 2015
• Study Completion: August 1, 2016
• Last Updated: September 11, 2025

Record last verified: 2015-09

Locations

IT (2)