Toremifene in Desmoid Tumor: Prospective Clinical Trial and Identification of Potential Molecular Targets

January 30, 2015 updated by: Fondazione IRCCS Istituto Nazionale dei Tumori, Milano

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.

Study Overview

Status

Unknown

Conditions

Intervention / Treatment

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 Type

Interventional

Enrollment (Anticipated)

25

Phase

  • Phase 2

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

14 years and older (Adult, Older Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

Inclusion Criteria:

  • 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
  • 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.

Exclusion Criteria:

  • 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
  • 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.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Toremifene treatment
Patients will receive 60 mg daily of Toremifene and the dose will be escalated to 180 mg daily in case of progression
Drug: Toremifene
Patients will receive 60 mg daily and then 180 daily in case of progression

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
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)
2 years

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Fondazione IRCCS Istituto Nazionale dei Tumori, Milano

Collaborators

Associazione Italiana per la Ricerca sul Cancro

Investigators

  • Principal Investigator: Chiara Colombo, MD, Fondazione IRCCS Istituto Tumori Milano

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

November 1, 2012

Primary Completion (Anticipated)

June 1, 2015

Study Completion (Anticipated)

December 1, 2015

Study Registration Dates

First Submitted

January 28, 2015

First Submitted That Met QC Criteria

January 30, 2015

First Posted (Estimate)

February 2, 2015

Study Record Updates

Last Update Posted (Estimate)

February 2, 2015

Last Update Submitted That Met QC Criteria

January 30, 2015

Last Verified

January 1, 2015

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • INT 112/11

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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