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A Randomized Trial of Filgrastim-SD/01 vs. Filgrastim in Newly Diagnosed Children and Young Adults With Sarcoma Treated With Dose-Intensive Chemotherapy

Phase 1
25 Years
Not Enrolling
Ewing's Sarcoma, Rhabdomyosarcoma, Sarcoma, Synovial Sarcoma

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Trial Information

A Randomized Trial of Filgrastim-SD/01 vs. Filgrastim in Newly Diagnosed Children and Young Adults With Sarcoma Treated With Dose-Intensive Chemotherapy


- Filgrastim (granulocyte colony-stimulating factor), which is administered by daily
subcutaneous injection after cytotoxic chemotherapy, shortens the duration of
chemotherapy-induced neutropenia and lowers the risk of infection.

- In children treated with dose-intensive chemotherapy, Filgrastim reduces the duration
of severe neutropenia and, as a result, has become a standard component of the
treatment regimen.

- Filgrastim-SD/01 (AMGEN), which is produced by PEGylation of the amino-terminus of
Filgrastim, is a sustained duration form of granulocyte colony-stimulating factor.

- In phase I and phase II trials in adults, a single dose of Filgrastim-SD/01 appears to
be equivalent to daily dosing of Filgrastim in enhancing neutrophil recovery and has a
comparable adverse event profile.

- Dose-intensive vincristine/cyclophosphamide/doxorubicin (VDoxC) alternating with
ifosfamide/etoposide (IE) has become standard therapy for children and adolescents with
Ewing's sarcoma and other sarcomas treated at the POB/NCI and other cancer centers
within the US.


- Compare the tolerance, toxicity, and therapeutic effects of Filgrastim-SD/01 given as a
single injection after chemotherapy to daily subcutaneous Filgrastim in patients with
newly diagnosed sarcoma receiving multi-agent, dose intensive chemotherapy.

- The pharmacokinetics of Filgrastim-SD/01 will also be compared to the pharmacokinetics
of Filgrastim.

- This trial will also be a platform for performing biological studies of these tumors
study neutrophil function and CD34 mobilization, and for detailed cardiac studies.


- Children and young adults (less than 25 years) with previously untreated high-risk
sarcomas (Ewing sarcoma, rhabdomyosarcoma, MPNST, and synovial sarcoma).

- No evidence of tumor infiltration of the bone marrow.


- Participants will be randomized (1:1) to receive a single dose of Filgrastim-SD/01 or
daily filgrastim as a SQ injection after each cycle of chemotherapy.

- Standard 5 drug dose-intensive chemotherapy with vincristine, doxorubicin,
cyclophosphamide alternating with ifosfamide and etoposide will be administered.

- Surgery or radiation for the primary tumor will occur after cycle 5.

- A total of 34 patients (17 patients per treatment arm) will be entered onto the trial.

Inclusion Criteria


Newly diagnosed histologically proven: Ewing's sarcoma family of tumors, including
peripheral neuroectodermal tumors; Alveolar rhabdomyosarcoma; Stage 3 or 4 embryonal
rhabdomyosarcoma; Malignant peripheral nerve sheath tumor that is unresectable,
incompletely resected with bulk residual disease or metastatic; Synovial cell sarcoma that
is unresectable, incompletely resected with bulk residual disease, or metastatic.

Age equal to or less than 25 years at the time of diagnosis.

Normal cardiac function (ejection fraction by MUGA or ECHO that is within the
institutional normal range).

Normal serum creatinine for age or creatinine clearance greater than 60 ml/min/1.73m(2).

Normal liver function (SGPT less than 5 times the upper limit of normal and bilirubin less
than 2.5 times the upper limit of normal).

Normal hematologic function (absolute neutrophil count equal to or greater than
1500/microL, hemoglobin equal to or greater than 9.0 g/dl and platelet count equal to or
greater than 100,000/microL).

Subjects of childbearing or child-fathering potential must be willing to use a medically
acceptable form of birth control, which includes abstinence, while being treated on this


Previous chemotherapy or radiotherapy.

Pregnant or breast feeding.

Histological evidence of tumor infiltration of bone marrow.

Stage 1 or 2 embryonal rhabdomyosarcomas.

Type of Study:


Study Design:

Primary Purpose: Treatment

Outcome Measure:

Duration of neutropenia

Principal Investigator

Crystal L Mackall, M.D.

Investigator Role:

Principal Investigator

Investigator Affiliation:

National Cancer Institute (NCI)


United States: Federal Government

Study ID:




Start Date:

March 2000

Completion Date:

May 2009

Related Keywords:

  • Ewing's Sarcoma
  • Rhabdomyosarcoma
  • Sarcoma
  • Synovial Sarcoma
  • Ewing's Sarcoma
  • G-CSF
  • Neutropenia
  • Rhabdomyosarcoma
  • Synovial Sarcoma
  • Sarcoma
  • Tumor
  • Rhabdomyosarcoma
  • Sarcoma, Synovial
  • Sarcoma, Ewing's
  • Neuroectodermal Tumors, Primitive, Peripheral
  • Sarcoma



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