Tailored Beta-catenin Mutational Approach in Extra-abdominal Sporadic Desmoids Tumor Patients
Italy100 participantsStarted 2013-07
Plain-language 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.
Who can participate
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion Criteria:
* 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
Exclusion Criteria:
* 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
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
Progression free survival
Timeframe: 3 years
Trial details
NCT IDNCT02547831
SponsorFondazione IRCCS Istituto Nazionale dei Tumori, Milano