Desmoid Type-fibromatosisPhase 2NCT02353429
Toremifene in Desmoid Tumor: Prospective Clinical Trial and Identification of Potential Molecular Targets
A Phase 2 study testing Toremifene in people with Desmoid Type-fibromatosis, run by Fondazione IRCCS Istituto Nazionale dei Tumori, Milano at 1 site in Italy.
Trial summary
This is a prospective study evaluating the activity and the safety of toremifene in patients with primary or recurrent sporadic DTs. Patients will be enrolled after the histological confirmation of DTs on biopsy Patients will start at 60 mg daily and dose-escalate to 180 mg upon progression. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Desmoid Type-fibromatosis
- What is being tested?
- Toremifene
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 25 participants
- Where
- 1 siteItaly
- Study dates
- Nov 2012to Dec 2015
- Phase
- Phase 2
- 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 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Adult patients (age > 18 years) with primary or locally recurrent, sporadic or FAP associated, desmoid fibromatosis
- Histologically documented diagnosis of DF
- At least one measurable site of disease at CT or MRI scans, which has not been previously embolised or irradiated
- Progressive disease demonstrated at contrast-enhanced MRI or CT scan by Response Evaluation Criteria in Solid Tumors (RECIST)
- Radiologic or clinical evidence of PD in the previous 6 months. Radiologic PD will be defined according to RECIST
- ECOG Performance status: 0-2
Show all 11Show fewer
- Prior hormonal therapy, chemotherapy, or molecular targeted therapies are allowed
- Adequate end organ function, defined as the following: total bilirubin < 1.5 x ULN, SGOT and SGPT < 2.5 x UNL (or < 5 x ULN if hepatic metastases are present), creatinine < 1.5 x ULN, ANC > 1.5 x 109/L, platelets > 100 x 109/L
- Female patients of child-bearing potential must have negative pregnancy test within 7 days before initiation of study drug dosing. Post menopausal women must be amenorrheic for at least 12 months to be considered of non-childbearing potential. Male and female patients of reproductive potential must agree to employ an effective barrier method of birth control throughout the study and for up to 3 months following discontinuation of study drug
- Life expectancy of at least 6 months
- Written, voluntary, informed consent.
Who cannot join
- Previous history of deep vein thrombosis
- Evidence of prolonged QTc >480 msec (using Bazetts correction, for which the formula is: QTc = QT/√RR) or history of familial long QT syndrome
- Previous arrhythmia
- Clinically significant bradycardia
- Endometrial hyperplasia
- Hepatic insufficiency
Show all 12Show fewer
- Other concurrent hormonal therapy, including hormonal contraceptives
- Patient has received any other investigational agents within 28 days of first day of study drug dosing. - Female patients who are pregnant or breast-feeding
- Patient has a severe and/or uncontrolled medical disease
- Patient has a known diagnosis of human immunodeficiency virus (HIV) infection
- Patient received chemotherapy within 4 weeks prior to study entry
- Patient had a major surgery within 2 weeks prior to study entry.
Trial locations and contacts
1 site in 1 country.
Lorella Rusi, MD contact
Italy1 site, 1 recruiting
- Recruiting
Fondazione IRCCS Istituto Tumori Milano
Milan, 20133
Chiara Colombo, MD, +39023903234, chiara.colombo@istitutotumori.mi.it
About this study
This is a prospective study evaluating the activity and the safety of toremifene in patients with primary or recurrent sporadic DTs.
Patients will be enrolled after the histological confirmation of DTs on biopsy Patients will start at 60 mg daily and dose-escalate to 180 mg upon progression. Disease assessment will be performed by contrast-enhanced MRI or CT scan, pain evaluation by a visual analog scale (VAS) every 3 months for the first and second year, twice yearly thereafter. Response will be evaluated either by RECIST and/or symptomatic relief.
Read the detailed description
This is a prospective study evaluating the activity and the safety of toremifene in patients with primary or recurrent sporadic DTs.
Patients will be enrolled after the histological confirmation of DTs on biopsy performed at the investigators institution or after the pathological review of tissue specimen obtained via needle biopsy or surgical excision (in case of recurrence) performed elsewhere. A new biopsy will be performed if the amount of tissue will not be sufficient for immunohistochemical analysis. Patients will start at 60 mg daily and dose-escalate to 180 mg upon progression. Disease assessment will be performed by contrast-enhanced MRI or CT scan, pain evaluation by a visual analog scale (VAS) every 3 months for the first and second year, twice yearly thereafter. Response will be evaluated either by RECIST and/or symptomatic relief.
- Conditions
- Desmoid Type-fibromatosis
- Being tested
- Toremifene
- Medical subject headings
- Desmoid Tumors
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ToremifeneDrug
Patients will receive 60 mg daily and then 180 daily in case of progression
| Study group | Role | What they receive |
|---|---|---|
| Toremifene treatment | Experimental | Patients will receive 60 mg daily of Toremifene and the dose will be escalated to 180 mg daily in case of progression Receives: Drug: Toremifene |
What is being measured
The primary outcome is the main question the study is designed to answer.
Time to progression
This study evaluates clinical benefit by comparing sequentially measured paired failure times within each treated patient, namely time to progression after the 60 mg toremifene dose (TTP1) versus the (possibly censored) time to progression after the 180 mg toremifene dose (TTP2)
Measured: 2 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted2 Feb 2015 (estimated)
- Study startNov 2012
- Primary completionJun 2015 (estimated)
- Study completionDec 2015 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (2)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
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 25 participants (estimated)
Sponsor and investigators
- Sponsor type
- University, hospital or other
- Collaborators
- Associazione Italiana per la Ricerca sul Cancro
- Responsible party
- Sponsor
- Investigators
- Chiara Colombo, MD, Principal investigator, Fondazione IRCCS Istituto Tumori Milano
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02353429
- Sponsor's protocol ID
- INT 112/11
- Status last verified
- Jan 2015
- First submitted
- 28 Jan 2015
- Last updated
- 2 Feb 2015
- Results posted
- No
- Data monitoring committee
- No
Publications
Colombo C, Tine G, Palassini E, Belfiore A, Marrari A, Collini P, Fiore M, Radaelli S, Perrone F, Miceli A, Gronchi A. Toremifene in desmoid fibromatosis: Prospective phase 2 study of two dose levels. Eur J Cancer. 2026 May 15;239:116673. doi: 10.1016/j.ejca.2026.116673. Epub 2026 Mar 20.
PubMed 41903360Fiore M, Colombo C, Radaelli S, Callegaro D, Palassini E, Barisella M, Morosi C, Baldi GG, Stacchiotti S, Casali PG, Gronchi A. Hormonal manipulation with toremifene in sporadic desmoid-type fibromatosis. Eur J Cancer. 2015 Dec;51(18):2800-7. doi: 10.1016/j.ejca.2015.08.026. Epub 2015 Nov 18.
PubMed 26602014
Study at a glance
From the registry record, last updated by the sponsor on 2 Feb 2015. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Toremifene in Desmoid Tumor: Prospective Clinical Trial and Identification of Potential Molecular Targets
- ClinicalTrials.gov ID
- NCT02353429
- Acronym / sponsor ID
- INT 112/11
- Conditions
- Desmoid Type-fibromatosis
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 25 participants (estimated)
- Lead sponsor
- Fondazione IRCCS Istituto Nazionale dei Tumori, MilanoUniversity, hospital or other
- Collaborators
- Associazione Italiana per la Ricerca sul Cancro
- Study start
- Nov 2012
- Primary completion
- Jun 2015 (estimated)
- Study completion
- Dec 2015 (estimated)
- First posted
- 2 Feb 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 study evaluating the activity and the safety of toremifene in patients with primary or recurrent sporadic DTs.
Patients will be enrolled after the histological confirmation of DTs on biopsy Patients will start at 60 mg daily and dose-escalate to 180 mg upon progression. Disease assessment will be performed by contrast-enhanced MRI or CT scan, pain evaluation by a visual analog scale (VAS) every 3 months for the first and second year, twice yearly thereafter. Response will be evaluated either by RECIST and/or symptomatic relief.
Detailed description
This is a prospective study evaluating the activity and the safety of toremifene in patients with primary or recurrent sporadic DTs.
Patients will be enrolled after the histological confirmation of DTs on biopsy performed at the investigators institution or after the pathological review of tissue specimen obtained via needle biopsy or surgical excision (in case of recurrence) performed elsewhere. A new biopsy will be performed if the amount of tissue will not be sufficient for immunohistochemical analysis. Patients will start at 60 mg daily and dose-escalate to 180 mg upon progression. Disease assessment will be performed by contrast-enhanced MRI or CT scan, pain evaluation by a visual analog scale (VAS) every 3 months for the first and second year, twice yearly thereafter. Response will be evaluated either by RECIST and/or symptomatic relief.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Toremifene treatment | Experimental | Drug: ToremifenePatients will receive 60 mg daily of Toremifene and the dose will be escalated to 180 mg daily in case of progression |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (11 listed)
- Adult patients (age > 18 years) with primary or locally recurrent, sporadic or FAP associated, desmoid fibromatosis
- Histologically documented diagnosis of DF
- At least one measurable site of disease at CT or MRI scans, which has not been previously embolised or irradiated
- Progressive disease demonstrated at contrast-enhanced MRI or CT scan by Response Evaluation Criteria in Solid Tumors (RECIST)
- Radiologic or clinical evidence of PD in the previous 6 months. Radiologic PD will be defined according to RECIST
Key exclusion criteria (12 listed)
- Previous history of deep vein thrombosis
- Evidence of prolonged QTc >480 msec (using Bazetts correction, for which the formula is: QTc = QT/√RR) or history of familial long QT syndrome
- Previous arrhythmia
- Clinically significant bradycardia
- Endometrial hyperplasia
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 |
|---|---|---|
| ToremifenePatients will receive 60 mg daily and then 180 daily in case of progression | Drug | Toremifene treatment |
Primary outcome
Time to progression
Time frame: 2 years
This study evaluates clinical benefit by comparing sequentially measured paired failure times within each treated patient, namely time to progression after the 60 mg toremifene dose (TTP1) versus the (possibly censored) time to progression after the 180 mg toremifene dose (TTP2)
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 2 Feb 2015.
- Derived from this study: PubMed 41903360Colombo C, Tine G, Palassini E, Belfiore A, Marrari A, Collini P, Fiore M, Radaelli S, Perrone F, Miceli A, Gronchi A. Toremifene in desmoid fibromatosis: Prospective phase 2 study of two dose levels. Eur J Cancer. 2026 May 15;239:116673. doi: 10.1016/j.ejca.2026.116673. Epub 2026 Mar 20.
- Derived from this study: PubMed 26602014Fiore M, Colombo C, Radaelli S, Callegaro D, Palassini E, Barisella M, Morosi C, Baldi GG, Stacchiotti S, Casali PG, Gronchi A. Hormonal manipulation with toremifene in sporadic desmoid-type fibromatosis. Eur J Cancer. 2015 Dec;51(18):2800-7. doi: 10.1016/j.ejca.2015.08.026. Epub 2015 Nov 18.
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.
Lorella Rusi, MD contact
Chiara Colombo, MD principal investigator
Fondazione IRCCS Istituto Tumori Milano
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Toremifene in people with Desmoid Type-fibromatosis, run by Fondazione IRCCS Istituto Nazionale dei Tumori, Milano at 1 site 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 11 inclusion and 12 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: status not confirmed, as of the sponsor's last update on 2 Feb 2015. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site 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.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Chiara Colombo, MD (+39022390 ext. 3234, chiara.colombo@istitutotumori.mi.it), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Time to progression (time frame: 2 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 NCT02353429, which also lists 2 linked publications.
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 2 Feb 2015.