Refractory Desmoplastic Small Round Cell Tumors
Conditions
Keywords
refractory desmoplastic small round cell tumors, PDGF-R
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
Interventions
Sponsors
Novartis Pharmaceuticals
Study design
Allocation
NON_RANDOMIZED
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
16 Years to No maximum
Healthy volunteers
No
Inclusion criteria
1. Patients \> 16 years of age. 2. Histologically documented diagnosis of DSRCT, unresponsive or in no complete remission after any conventional multimodality approach. 3. Immunohistochemical documentation of activated PDGF-R expression by tumor 4. At least one measurable site of disease 5. Performance status 0,1, 2 or 3 (Eastern Cooperative Oncology Group) 6. Adequate end organ function
Exclusion criteria
1. Patient has received any other investigational agents within 28 days of first day of study drug dosing, unless the disease is rapidly progressing. 2. Patient is \< 5 years free of another primary malignancy 3. Patient with congestive heart failure or myocardial infarction within 6 months of study 4. Female patients who are pregnant or breast-feeding. 5. Severe and/or uncontrolled medical disease 6. Known brain metastasis. 7. Chronic active hepatitis or cirrhosis 8. Known diagnosis of human immunodeficiency virus (HIV) infection. 9. Chemotherapy within 4 weeks prior to study entry, unless the disease is rapidly progressing. 10. Previous radiotherapy to \> 25 % of the bone marrow 11. Major surgery within 2 weeks prior to study entry. Other protocol defined inclusion/
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Overall tumor response rates | Assessed every 3 months | An imaging technique (CT scan or MRI) determined tumor response according to RECIST criteria. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Explore how Imatinib cooperated with other treatment modatilities | Survival data was collected until death | Recorded survival after treatment and other associated treatment modalities. |
| Safety and tolerability | Safety data collected until patients were no longer in study | — |
| Conversion rate to surgical resectability | After best tumor response. | Assessed actual resectability rate after therapy compared to resectability before therapy. |
| Mutational analysis of molecular targets of imatinib (at any time during the study) | At any time during the study | — |
Countries
Italy
Outcome results
None listed