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New Therapeutic Strategies for Patients With Ewing's Sarcoma Family of Tumors, High Risk Rhabdomyosarcoma, and Neuroblastoma

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT00001335
Recruitment Status : Completed
First Posted : November 4, 1999
Last Update Posted : March 4, 2008
Sponsor:
Information provided by:
National Institutes of Health Clinical Center (CC)

Study Description
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Brief Summary:
The prognosis for patients with metastatic Ewing's sarcoma family of tumors (ESF), rhabdomyosarcoma (RMS), and neuroblastoma (NBL) remains dismal, with less than 25% long-term disease-free survival. Though less grave, the prognosis for cure for other high-risk patients is approximately 50%. New treatment strategies, including the identification of highly active new agents, maximizing the dose intensity of the most active standard drugs, and the development of improved methods of consolidation to eradicate microscopic residual disease, are clearly needed to improve the outcome of these patients. This protocol will address these issues by commencing with a Phase II window, for the highest risk patients, to evaluate a series of promising drugs with novel mechanisms of action. All patients will then receive 5 cycles of dose-intensive "best standard therapy" with doxorubicin (adriamycin), vincristine, and cyclophosphamide (VAdriaC). Patients at high risk of relapse will continue onto a phase I consolidation regimen consisting of three cycles of dose-escalated Melphalan, Ifosfamide, Mesna, and Etoposide (MIME). Peripheral blood stem cell transfusions (PBSCT) and recombinant human G-CSF will be used as supportive care measures to allow maximal dose-escalation of this combination regimen.

Condition or disease Intervention/treatment Phase
Ewing's Sarcoma Neuroblastoma Rhabdomyosarcoma Drug: ADR-529 Drug: Topotecan Drug: G-CSF Phase 2

Detailed Description:
The prognosis for patients with metastatic Ewing's sarcoma family of tumors (ESF), rhabdomyosarcoma (RMS), and neuroblastoma (NBL) remains dismal, with less than 25% long-term disease-free survival. Though less grave, the prognosis for cure for other high-risk patients is approximately 50%. New treatment strategies, including the identification of highly active new agents, maximizing the dose intensity of the most active standard drugs, and the development of improved methods of consolidation to eradicate microscopic residual disease, are clearly needed to improve the outcome of these patients. This protocol will address these issues by commencing with a Phase II window, for the highest risk patients, to evaluate a series of promising drugs with novel mechanisms of action. All patients will then receive 5 cycles of dose-intensive "best standard therapy" with doxorubicin (adriamycin), vincristine, and cyclophosphamide (VAdriaC). Patients at high risk of relapse will continue onto a phase I consolidation regimen consisting of three cycles of dose-escalated Melphalan, Ifosfamide, Mesna, and Etoposide (MIME). Peripheral blood stem cell transfusions (PBSCT) and recombinant human G-CSF will be used as supportive care measures to allow maximal dose-escalation of this combination regimen.
Study Design
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Study Type : Interventional  (Clinical Trial)
Enrollment : 90 participants
Primary Purpose: Treatment
Official Title: New Therapeutic Strategies for Patients With Ewing's Sarcoma Family of Tumors, High Risk Rhabdomyosarcoma, and Neuroblastoma
Study Start Date : April 1993
Study Completion Date : January 2002

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Arms and Interventions
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Outcome Measures
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Eligibility Criteria
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Ages Eligible for Study:   Child, Adult, Older Adult
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

The patient must fall into one of the following diagnostic categories:

Ewing's sarcoma family of tumors (ESF): Includes Ewing's sarcoma (classic, atypical, extra-osseous), primitive sarcoma of bone, ectomesenchymoma, or peripheral primitive neuroectodermal tumor (peripheral neuroepithelioma).

Rhabdomyosarcoma: The patient must have either

High-risk arm: Metastatic disease at diagnosis (any site, any histology);

OR

Moderate-risk arm: Incompletely resected ( Clinical Group III) Stage II tumors and ALL stage III tumors (regardless of degree of surgical resection).

Neuroblastoma: Any patient with metastatic disease at diagnosis (POG stage D or Evans' stage IV); or, patients with loco-regional metastatic disease (POG stage C) or tumor infiltrating across the midline (Evans' stage III) IF they have an elevated serum ferritin (greater than 142 ng/ml), an amplified N-myc copy number (greater than 10 copies on Southern analysis), or a DNA index of greater than 1.1.

The patient must not have been previously treated with chemotherapy or radiation therapy.

Patients must be greater than or equal to 1 year of age but less than or equal to 25 years of age. Patients weight must be greater than or equal to 15 kg.

The patient (or his/her guardian if less than 18 years of age) must sign a document indicating that he/she is aware of the investigational nature of this treatment protocol and the potential risks and benefits that may be expected.

Potentially fertile female patients must have a documented negative urine or serum pregnancy test.

Patients must have a documented negative HIV serologic evaluation (Western Blot and/or ELISA).

Patients must not have abnormal cardiac function (left ventricular ejection fraction less than 45% as measured by gated equilibrium radionuclide angiography [MUGA scan] and confirmed by echocardiography).

Patients must not have impaired renal function (serum creatinine greater than or equal to twice the upper limit of normal for age).

Patients with a total bilirubin of greater than 4.0 mg/dl (or a direct bilirubin of greater than 2.0 mg/dl) or SGOT/SGPT greater than five times the upper limits of normal (NOT on the basis of hepatic involvement by tumor) will be excluded.

Patients with a second malignancy following previous therapy will be excluded.

Patients previously treated with chemotherapy or radiation therapy (other than limited, emergency radiation therapy) will be excluded.

Patients who are HIV-infected will be excluced.

Contacts and Locations
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To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00001335


Locations
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United States, Maryland
National Cancer Institute (NCI)
Bethesda, Maryland, United States, 20892
Sponsors and Collaborators
National Cancer Institute (NCI)
More Information
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ClinicalTrials.gov Identifier: NCT00001335    
Other Study ID Numbers: 930125
93-C-0125
First Posted: November 4, 1999    Key Record Dates
Last Update Posted: March 4, 2008
Last Verified: January 2002
Keywords provided by National Institutes of Health Clinical Center (CC):
Peripheral Stem Cell Transfusion
Up-Front Phase II Window
Dose-Intensive Vincristine
Dose-Intensive Doxorubicin
 Dose-Intensive Cyclophosphamide
ADR-529 for Cardioprotection
BID Radiation Therapy for Local Control
Additional relevant MeSH terms:
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Sarcoma
Neuroblastoma
Rhabdomyosarcoma
Sarcoma, Ewing
Neoplasms, Connective and Soft Tissue
Neoplasms by Histologic Type
Neoplasms
Neuroectodermal Tumors, Primitive, Peripheral
Neuroectodermal Tumors, Primitive
Neoplasms, Neuroepithelial
Neuroectodermal Tumors
Neoplasms, Germ Cell and Embryonal
Neoplasms, Glandular and Epithelial
 Neoplasms, Nerve Tissue
Myosarcoma
Neoplasms, Muscle Tissue
Osteosarcoma
Neoplasms, Bone Tissue
Neoplasms, Connective Tissue
Topotecan
Topoisomerase I Inhibitors
Topoisomerase Inhibitors
Enzyme Inhibitors
Molecular Mechanisms of Pharmacological Action
Antineoplastic Agents