Desmoid-type FibromatosisNCT02547831
Tailored Beta-catenin Mutational Approach in Extra-abdominal Sporadic Desmoids Tumor Patients
An observational study of Desmoid-type Fibromatosis, run by Fondazione IRCCS Istituto Nazionale dei Tumori, Milano at 2 sites in Italy.
Trial 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… Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Desmoid-type Fibromatosis
- What is being tested?
- Observational approach
- How many people
- 100 participants
- Where
- 2 sitesItaly
- Study dates
- Jul 2013to Aug 2016
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Study population: Patients affected by extra-abdominal primary fibromatosis
Who may be able to join
- 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
Who cannot join
- 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
Show all 9Show fewer
- 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
Trial locations and contacts
2 sites in 1 country.
Italy2 sites
Fondazione del Piemonte per l' Oncologia - IRCCS
Candiolo, Torino, 10060
Fondazione IRCCS Istituto Tumori Milano
Milan, 20133
About this study
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.
Read more
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.
Read the detailed description
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)
- Combination 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
- Being tested
- Observational approach
- Medical subject headings
- Desmoid Tumors
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Observational approachOther
Also called Wait and see approach
Patients will be placed under wait and see approach without any specific treatment
| Study group | Role | What they receive |
|---|---|---|
| Observational approach | Patients will be placed on wait and see approach and then shifted to specific treatment in case of progression Receives: Other: Observational approach |
What is being measured
The primary outcome is the main question the study is designed to answer.
Progression free survival
Measured: 3 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted11 Sep 2015 (estimated)
- Study startJul 2013
- Primary completionDec 2015
- Study completionAug 2016
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Desmoid-type FibromatosisStudies of the same condition, open and under way.
- More trials run by Fondazione IRCCS Istituto Nazionale dei Tumori, MilanoThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Case only
- Time perspective
- Prospective
- Enrolment
- 100 participants (actual)
Sponsor and investigators
- Sponsor type
- University, hospital or other
- Collaborators
- Ministero della Salute, Italy
- Responsible party
- Sponsor
- Investigators
- Alessandro Gronchi, MD, Principal investigator, Fondazione IRCCS Istituto Tumori Milano
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02547831
- Sponsor's protocol ID
- INT 13/12
- Status last verified
- Sep 2015
- First submitted
- 28 Jan 2015
- Last updated
- 11 Sep 2025
- Results posted
- No
- Data monitoring committee
- No
Publications
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.
PubMed 35247923
Study at a glance
From the registry record, last updated by the sponsor on 11 Sep 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Tailored Beta-catenin Mutational Approach in Extra-abdominal Sporadic Desmoids Tumor Patients
- ClinicalTrials.gov ID
- NCT02547831
- Acronym / sponsor ID
- INT 13/12
- Conditions
- Desmoid-type Fibromatosis
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 100 participants (actual)
- Lead sponsor
- Fondazione IRCCS Istituto Nazionale dei Tumori, MilanoUniversity, hospital or other
- Collaborators
- Ministero della Salute, Italy
- Study start
- Jul 2013 (actual)
- Primary completion
- Dec 2015 (actual)
- Study completion
- Aug 2016 (actual)
- First posted
- 11 Sep 2015 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 description
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)
- Combination 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.
Study design
- Observational model
- Case only
- Time perspective
- Prospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Observational approach | – | Other: Observational approachPatients will be placed on wait and see approach and then shifted to specific treatment in case of progression |
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- Patients affected by extra-abdominal primary fibromatosis
Key inclusion criteria (7 listed)
- 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)
Key exclusion criteria (9 listed)
- Controindication to MRI
- Familial-type desmoid
- Recurrence
- Extraabdominal primary fibromatosis resected with R0/R1 margins
- Patients on treatment for desmoid tumor
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Observational approachAlso: Wait and see approachPatients will be placed under wait and see approach without any specific treatment | Other | Observational approach |
Primary outcome
Progression free survival
Time frame: 3 years
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 11 Sep 2025.
- Derived from this study: PubMed 35247923Colombo 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.
Related trials for Desmoid-type Fibromatosis
RecruitingPhase 2
A Study in Adults With Desmoid Tumors
Desmoid Tumor
RecruitingPhase 4
Nirogacestat in Premenopausal Females With Desmoid Tumor/Aggressive Fibromatosis (DT/AF)
Desmoid Tumor, Aggressive Fibromatosis
RecruitingPhase 1/2
FOG-001 in Locally Advanced or Metastatic Solid Tumors
Cancer, Colorectal Cancer
Active, not recruitingPhase 2
A Study of a New Drug, Nirogacestat, for Treating Desmoid Tumors That Cannot be Removed by Surgery
Desmoid Fibromatosis, Recurrent Desmoid Fibromatosis
Enrolling by invitationObservational study
Patient-reported Outcomes Following Percutaneous Ablation of Desmoid Tumors
Desmoid Tumor, Aggressive Fibromatoses
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Alessandro Gronchi, MD principal investigator
Fondazione IRCCS Istituto Tumori Milano
2 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Desmoid-type Fibromatosis, run by Fondazione IRCCS Istituto Nazionale dei Tumori, Milano at 2 sites in Italy.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 7 inclusion and 9 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 11 Sep 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites in Italy. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Progression free survival (time frame: 3 years). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT02547831, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 11 Sep 2025.