Imatinib Mesylate in Patients With Refractory Desmoplastic Small Round Cell Tumors

Study Overview
=================
Brief Summary
-----------------
An open-label, non-comparative study conducted to investigate the activity and safety of imatinib mesylate in refractory desmoplastic small round cell tumors expressing PDGF-R. Patients will be treated up to 12 months, or to disease progression. Tumor will be evaluated according to Recist criteria

Official Title
-----------------
Open-label Trial of Imatinib Mesylate in Patients With Refractory Desmoplastic Small Round Cell Tumors (DSRCT) Expressing PDGF-R

Conditions
-----------------
Refractory Desmoplastic Small Round Cell Tumors

Intervention / Treatment
-----------------
* Drug: Imatinib mesilate


Participation Criteria
=================
Eligibility Criteria
-----------------
Inclusion criteria: Patients > 16 years of age. Histologically documented diagnosis of DSRCT, unresponsive or in no complete remission after any conventional multimodality approach. Immunohistochemical documentation of activated PDGF-R expression by tumor At least one measurable site of disease Performance status 0,1, 2 or 3 (Eastern Cooperative Oncology Group) Adequate end organ function Exclusion criteria: Patient has received any other investigational agents within 28 days of first day of study drug dosing, unless the disease is rapidly progressing. Patient is < 5 years free of another primary malignancy Patient with congestive heart failure or myocardial infarction within 6 months of study Female patients who are pregnant or breast-feeding. Severe and/or uncontrolled medical disease Known brain metastasis. Chronic active hepatitis or cirrhosis Known diagnosis of human immunodeficiency virus (HIV) infection. Chemotherapy within 4 weeks prior to study entry, unless the disease is rapidly progressing. Previous radiotherapy to > 25 % of the bone marrow Major surgery within 2 weeks prior to study entry. Other protocol defined inclusion/exclusion criteria may apply.

Ages Eligible for Study
-----------------
Minimum Age: 16 Years

Sexes Eligible for Study
-----------------
All

Accepts Healthy Volunteers
-----------------
No

Study Plan
=================
How is the study designed?
-----------------

Design Details

Primary Purpose: Treatment
Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Gleevec/Glivec<br> | Drug: Imatinib mesilate<br> <br> * Other names: STI571;|

What is the study measuring?
-----------------
Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Overall tumor response rates | An imaging technique (CT scan or MRI) determined tumor response according to RECIST criteria. | Assessed every 3 months | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Explore how Imatinib cooperated with other treatment modatilities | Recorded survival after treatment and other associated treatment modalities. | Survival data was collected until death | 
| Safety and tolerability |  | Safety data collected until patients were no longer in study | 
| Conversion rate to surgical resectability | Assessed actual resectability rate after therapy compared to resectability before therapy. | After best tumor response. | 
| Mutational analysis of molecular targets of imatinib (at any time during the study) |  | At any time during the study | 

 Terms related to the study
=================
Keywords Provided by Centre Hospitalier Valida
-----------------
refractory desmoplastic small round cell tumors, PDGF-R