Clinical trial NCT00048984: Diagnostic Study of Tumor Characteristics in Patients With Ewing's Sarcoma
Official title: A Groupwide Biology and Banking Study for Ewing Sarcoma
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Localized Ewing Sarcoma/Peripheral Primitive Neuroectodermal Tumor
Metastatic Ewing Sarcoma/Peripheral Primitive Neuroectodermal Tumor
Recurrent Ewing Sarcoma/Peripheral Primitive Neuroectodermal Tumor
Lead sponsor: Children's Oncology Group
Sponsor class: NETWORK
Collaborators: name: National Cancer Institute (NCI)
First posted: 2003-01-27
Enrollment: 637
Sex: ALL
Maximum age (years): 50
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Time perspective: PROSPECTIVE
Sampling method: PROBABILITY_SAMPLE
Study population: Patients who have Ewing's sarcoma

Brief summary: Diagnostic trial to study genetic differences in patients who have Ewing's sarcoma. Genetic testing may help predict how cancer will respond to treatment and allow doctors to plan more effective therapy.

Detailed description: PRIMARY OBJECTIVES:

I. To develop a mechanism to collect and distribute tumor specimens to various investigators, and a system to prioritize and develop quality-control measures for central data reporting of studies undertaken.

II. To determine the prognostic significance of translocation subtype in Ewing sarcoma; to determine the prognostic significance of translocation negative Ewing sarcoma.

III. To determine the prognostic significance of MRD detection in bone marrow specimens by RT-PCR determination of EWS-ETS fusion genes.

IV. To determine whether serum levels of IGF1, IGFBP3 are of significance in the outcome of patients with Ewing sarcoma.

V. To determine whether RNA expression profiles performed on diagnostic specimens will allow for the identification of newer prognostic categories and potentially new molecular targets for treatment in Ewing sarcoma.

VI. To identify new treatment targets for therapy. Further testing of these potential targets will be carried out in hopes of expediting translation of these findings to the clinic.

VII. To establish a bank of Ewing sarcoma xenografts in SCID/Beige mice. VIII. To establish clinical proteomics as a resource for investigations of altered signaling molecules in the pathogenesis of Ewing sarcoma.

OUTLINE: This is a multicenter study.

Patients undergo various specimen collections, including bone marrow aspirate, paraffin-embedded blocks of tumor tissue or slides of tumor tissue, and blood specimens. These specimens are collected before, during, and after any chemotherapy regimens, during follow-up, and at time of recurrence. Translocation studies are performed on specimens to identify fusion genes, specifically EWS-ETS. Serum IGF1 and IFGBP3 levels are determined. Bone marrow is assessed for minimal residual disease using reverse-transcriptase polymerase chain reaction.

Eligibility criteria: Inclusion Criteria:

* Newly diagnosed or recurrent Ewing's sarcoma
* Availability of the following specimens:

  * Paraffin-embedded block or 20 unstained slides and 1-3 thick (50 micron) sections from initial biopsy
  * Pretreatment serum and whole blood
* Concurrent therapy is not required

Primary outcomes:
measure: Event-free survival
timeFrame: 1 year
Secondary outcomes:
measure: Success rate in which biomarker analyses can be carried out
timeFrame: Up to 5 years
measure: Percent of the population on which biomarker analysis could be successfully conducted
timeFrame: Up to 5 years
measure: Percent of submissions on which biomarker analysis could be successfully conducted
timeFrame: Up to 5 years
measure: Relation to known prognostic factors including the presence or absence of metastatic disease, the site of disease, and other known risk factors
timeFrame: Up to 5 years
Other outcomes:
Locations:
city: Arcadia
country: United States
facility: Children's Oncology Group
state: California
Study officials:
affiliation: Children's Oncology Group
name: Daniel West
role: PRINCIPAL_INVESTIGATOR
References: