Clinical trial NCT00417807: Imatinib Mesylate in Patients With Refractory Desmoplastic Small Round Cell Tumors
Official title: Open-label Trial of Imatinib Mesylate in Patients With Refractory Desmoplastic Small Round Cell Tumors (DSRCT) Expressing PDGF-R
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE1
PHASE2
Conditions: Refractory Desmoplastic Small Round Cell Tumors
Keywords: refractory desmoplastic small round cell tumors
PDGF-R
Lead sponsor: Novartis
Sponsor class: INDUSTRY
First posted: 2007-01-04
Enrollment: 9
Sex: ALL
Minimum age (years): 16
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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


Eligibility criteria: 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/exclusion criteria may apply.

Primary outcomes:
measure: Overall tumor response rates
timeFrame: Assessed every 3 months
Secondary outcomes:
measure: Explore how Imatinib cooperated with other treatment modatilities
timeFrame: Survival data was collected until death
measure: Safety and tolerability
timeFrame: Safety data collected until patients were no longer in study
measure: Conversion rate to surgical resectability
timeFrame: After best tumor response.
measure: Mutational analysis of molecular targets of imatinib (at any time during the study)
timeFrame: At any time during the study
Other outcomes:
Locations:
city: Ivrea
country: Italy
facility: Novartis Investigative Site
city: Milano
country: Italy
facility: Novartis Investigative Site
city: Napoli
country: Italy
facility: Novartis Investigative Site
city: Padova
country: Italy
facility: Novartis Investigative Site
city: Ravenna
country: Italy
facility: Novartis Investigative Site
city: Rozzano
country: Italy
facility: Novartis Investigative Site
city: Torino
country: Italy
facility: Novartis Investigative Site
city: Vatania
country: Italy
facility: Novartis Investigative Stie
Study officials:
affiliation: Novartis Pharmeceuticals
name: Novartis Pharmaceuticals
role: STUDY_DIRECTOR
References: