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Imatinib Mesylate in Treating Patients With Relapsed or Refractory Solid Tumors of Childhood

Primary Purpose

Childhood Desmoplastic Small Round Cell Tumor, Childhood Synovial Sarcoma, Gastrointestinal Stromal Tumor

Status
Completed
Phase
Phase 2
Locations
United States
Study Type
Interventional
Intervention
imatinib mesylate
laboratory biomarker analysis
pharmacological study
Sponsored by
National Cancer Institute (NCI)
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Childhood Desmoplastic Small Round Cell Tumor

Eligibility Criteria

undefined - 30 Years (Child, Adult)All SexesDoes not accept healthy volunteers

Inclusion Criteria: Histologically confirmed solid tumors including the following: Ewing's sarcoma Bone or soft tissue primitive neuroectodermal tumor Osteosarcoma Neuroblastoma Desmoplastic small round cell tumor Synovial cell sarcoma Gastrointestinal stromal tumor (GIST) Metastatic pulmonary disease eligible No pleural effusion of any size or definite radiologic evidence of pleural-based disease Recurrent or refractory to conventional therapy GIST eligible at initial presentation Tumor tissue blocks must be available At least 1 measurable lesion At least 20 mm by conventional techniques At least 10 mm by spiral CT scan Lesions assessable only by radionuclide scan are not considered measurable Performance status - Lansky 50-100% (≤ 10 years of age) Performance status - Karnofsky 50-100% (> 10 years of age) At least 2 months Absolute neutrophil count ≥ 1,000/mm^3* Platelet count ≥ 75,000/mm^3* (transfusion independent) Hemoglobin ≥ 8.0 g/dL* (RBC transfusions allowed) Bilirubin ≤ 1.5 times upper limit of normal (ULN) ALT ≤ 2.5 times ULN INR < 1.5 PTT ≤ ULN Fibrinogen ≥ lower limit of normal Creatinine normal for age Glomerular filtration rate ≥ 70 mL/min No uncontrolled infection Not pregnant or nursing Negative pregnancy test Fertile patients must use effective barrier contraception At least 1 week since prior biologic therapy or immunotherapy and recovered At least 1 week since prior growth factors No concurrent immunomodulating agents At least 2 weeks since prior myelosuppressive chemotherapy (4 weeks for nitrosoureas) and recovered No concurrent chemotherapy No concurrent steroids Recovered from prior radiotherapy At least 2 weeks since prior local palliative radiotherapy (small port) At least 3 months since prior craniospinal radiotherapy or radiotherapy to 50% or more of pelvis At least 6 weeks since other prior substantial bone marrow radiation No concurrent radiotherapy during first course of treatment Concurrent palliative radiotherapy to local painful lesions allowed after first course of treatment provided there is no evidence of disease progression and at least 1 measurable lesion remains outside radiation port No concurrent therapeutic doses of warfarin No concurrent anticonvulsants that induce the cytochrome p450 enzyme system (e.g., phenytoin, carbamazepine, and phenobarbital) Concurrent benzodiazepines and gabapentin allowed Concurrent low-molecular weight heparin allowed

Sites / Locations

  • Children's Oncology Group

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Treatment (imatinib mesylate)

Arm Description

Patients receive oral imatinib mesylate once or twice daily on days 1-28. Courses repeat every 28 days for up to 2 years in the absence of disease progression or unacceptable toxicity.

Outcomes

Primary Outcome Measures

Response rate, determined using the RECIST criteria
95% confidence interval will be computed.
Toxicity reported using the CTC version 2.0

Secondary Outcome Measures

Time to disease progression
Calculated by the method of Kaplan and Meier.

Full Information

First Posted
February 14, 2002
Last Updated
April 14, 2015
Sponsor
National Cancer Institute (NCI)
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1. Study Identification

Unique Protocol Identification Number
NCT00030667
Brief Title
Imatinib Mesylate in Treating Patients With Relapsed or Refractory Solid Tumors of Childhood
Official Title
A Phase II Study of Gleevec (Imatinib Mesylate, NSC 716051 Formerly STI571) in Children With Refractory or Relapsed Solid Tumors
Study Type
Interventional

2. Study Status

Record Verification Date
December 2013
Overall Recruitment Status
Completed
Study Start Date
May 2002 (undefined)
Primary Completion Date
December 2005 (Actual)
Study Completion Date
December 2005 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
National Cancer Institute (NCI)

4. Oversight

5. Study Description

Brief Summary
Phase II trial to study the effectiveness of imatinib mesylate in treating patients who have relapsed or refractory solid tumors of childhood. Imatinib mesylate may stop the growth of tumor cells by blocking the enzymes necessary for their growth.
Detailed Description
OBJECTIVES: I. Determine the response rate of patients with relapsed or refractory pediatric solid tumors treated with imatinib mesylate. II. Determine the toxicity of this drug in these patients. III. Determine the time to progression in patients treated with this drug. IV. Determine the pharmacokinetics of this drug in these patients. V. Correlate response with c-kit and platelet-derived growth factor receptor expression in patients treated with this drug. OUTLINE: This is a multicenter study. Patients are stratified according to disease (Ewing's sarcoma/primitive neuroectodermal tumor vs osteosarcoma vs neuroblastoma vs other). Patients receive oral imatinib mesylate once or twice daily on days 1-28. Courses repeat every 28 days for up to 2 years in the absence of disease progression or unacceptable toxicity.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Childhood Desmoplastic Small Round Cell Tumor, Childhood Synovial Sarcoma, Gastrointestinal Stromal Tumor, Lung Metastases, Recurrent Childhood Soft Tissue Sarcoma, Recurrent Ewing Sarcoma/Peripheral Primitive Neuroectodermal Tumor, Recurrent Neuroblastoma, Recurrent Osteosarcoma

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
100 (Actual)

8. Arms, Groups, and Interventions

Arm Title
Treatment (imatinib mesylate)
Arm Type
Experimental
Arm Description
Patients receive oral imatinib mesylate once or twice daily on days 1-28. Courses repeat every 28 days for up to 2 years in the absence of disease progression or unacceptable toxicity.
Intervention Type
Drug
Intervention Name(s)
imatinib mesylate
Other Intervention Name(s)
CGP 57148, Gleevec, Glivec
Intervention Description
Given orally
Intervention Type
Other
Intervention Name(s)
laboratory biomarker analysis
Intervention Description
Correlative studies
Intervention Type
Other
Intervention Name(s)
pharmacological study
Other Intervention Name(s)
pharmacological studies
Intervention Description
Correlative studies
Primary Outcome Measure Information:
Title
Response rate, determined using the RECIST criteria
Description
95% confidence interval will be computed.
Time Frame
Up to 2 years
Title
Toxicity reported using the CTC version 2.0
Time Frame
Up to 2 years
Secondary Outcome Measure Information:
Title
Time to disease progression
Description
Calculated by the method of Kaplan and Meier.
Time Frame
Up to 2 years

10. Eligibility

Sex
All
Maximum Age & Unit of Time
30 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: Histologically confirmed solid tumors including the following: Ewing's sarcoma Bone or soft tissue primitive neuroectodermal tumor Osteosarcoma Neuroblastoma Desmoplastic small round cell tumor Synovial cell sarcoma Gastrointestinal stromal tumor (GIST) Metastatic pulmonary disease eligible No pleural effusion of any size or definite radiologic evidence of pleural-based disease Recurrent or refractory to conventional therapy GIST eligible at initial presentation Tumor tissue blocks must be available At least 1 measurable lesion At least 20 mm by conventional techniques At least 10 mm by spiral CT scan Lesions assessable only by radionuclide scan are not considered measurable Performance status - Lansky 50-100% (≤ 10 years of age) Performance status - Karnofsky 50-100% (> 10 years of age) At least 2 months Absolute neutrophil count ≥ 1,000/mm^3* Platelet count ≥ 75,000/mm^3* (transfusion independent) Hemoglobin ≥ 8.0 g/dL* (RBC transfusions allowed) Bilirubin ≤ 1.5 times upper limit of normal (ULN) ALT ≤ 2.5 times ULN INR < 1.5 PTT ≤ ULN Fibrinogen ≥ lower limit of normal Creatinine normal for age Glomerular filtration rate ≥ 70 mL/min No uncontrolled infection Not pregnant or nursing Negative pregnancy test Fertile patients must use effective barrier contraception At least 1 week since prior biologic therapy or immunotherapy and recovered At least 1 week since prior growth factors No concurrent immunomodulating agents At least 2 weeks since prior myelosuppressive chemotherapy (4 weeks for nitrosoureas) and recovered No concurrent chemotherapy No concurrent steroids Recovered from prior radiotherapy At least 2 weeks since prior local palliative radiotherapy (small port) At least 3 months since prior craniospinal radiotherapy or radiotherapy to 50% or more of pelvis At least 6 weeks since other prior substantial bone marrow radiation No concurrent radiotherapy during first course of treatment Concurrent palliative radiotherapy to local painful lesions allowed after first course of treatment provided there is no evidence of disease progression and at least 1 measurable lesion remains outside radiation port No concurrent therapeutic doses of warfarin No concurrent anticonvulsants that induce the cytochrome p450 enzyme system (e.g., phenytoin, carbamazepine, and phenobarbital) Concurrent benzodiazepines and gabapentin allowed Concurrent low-molecular weight heparin allowed
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Mason Bond
Organizational Affiliation
Children's Oncology Group
Official's Role
Principal Investigator
Facility Information:
Facility Name
Children's Oncology Group
City
Arcadia
State/Province
California
ZIP/Postal Code
91006-3776
Country
United States

12. IPD Sharing Statement

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Imatinib Mesylate in Treating Patients With Relapsed or Refractory Solid Tumors of Childhood

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