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

Phase 2
Not Enrolling
Ewing's Sarcoma, Neuroblastoma, Rhabdomyosarcoma

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Trial Information

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

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.

Inclusion 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);


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

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.

Type of Study:


Study Design:

Endpoint Classification: Safety/Efficacy Study, Primary Purpose: Treatment


United States: Federal Government

Study ID:




Start Date:

April 1993

Completion Date:

January 2002

Related Keywords:

  • Ewing's Sarcoma
  • Neuroblastoma
  • Rhabdomyosarcoma
  • 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
  • Neuroblastoma
  • Rhabdomyosarcoma
  • Sarcoma, Ewing's
  • Neuroectodermal Tumors, Primitive, Peripheral
  • Sarcoma



National Cancer Institute (NCI) Bethesda, Maryland  20892