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CompletedNCT00003958Updated Jun 17, 2013

Combination Chemotherapy in Treating Patients With Previously Untreated Rhabdomyosarcoma

A Phase 3 interventional study of dactinomycin and vincristine sulfate in Adult Malignant Mesenchymoma, Adult Rhabdomyosarcoma and Alveolar Childhood Rhabdomyosarcoma, sponsored by Children's Oncology Group. Completed at 1 site in United States. Open to participants aged Up to 49 Years. Per ClinicalTrials.gov, last updated 2013-06-17.

Sponsored by Children's Oncology Group · Phase 3, Interventional, and Treatment

Phase
Phase 3
Study type
Interventional
Enrollment
702
Allocation
Randomized
Ages
Up to 49 Years
Sex
All
01

Study summary

This randomized phase III trial is comparing two different combination chemotherapy regimens to see how well each works in treating patients with previously untreated rhabdomyosarcoma or sarcoma. Drugs used in chemotherapy, such as dactinomycin, cyclophosphamide, vincristine, and topotecan, use different ways to stop tumor cells from dividing so they stop growing or die. It is not yet known which combination chemotherapy regimen is more effective in treating rhabdomyosarcoma.

Read the detailed description

OBJECTIVES:

I. Compare the early response rates, failure-free survival, and survival of patients with intermediate-risk rhabdomyosarcoma treated with surgery, radiotherapy, and vincristine, dactinomycin, and cyclophosphamide (VAC) vs VAC alternating with vincristine, topotecan, and cyclophosphamide.

II. Compare the acute and late effects of these two treatment regimens in these patients.

III. Determine the rate of second-look surgery in selected patients with bulk residual tumor at diagnosis (i.e., Clinical Group III) and the proportion of these that render the patient tumor free or with microscopic tumor only.

IV. Determine the rate of local failure in selected patients with bulk residual tumors at diagnosis (i.e., Clinical Group III) who, after second-look resection, have response-adjusted radiotherapy dose reduction.

V. Determine if preoperative radiotherapy followed by second-look surgery is feasible for selected patients with bulk residual disease (i.e., Clinical Group III) who respond poorly to induction chemotherapy.

OUTLINE: This is a randomized, multicenter study. Patients are stratified according to disease (embryonal histology, stage II or III, Clinical Group III vs embryonal histology, Clinical Group IV, less than 10 years of age vs alveolar or undifferentiated sarcoma histology, stage I, Clinical Group I vs alveolar or undifferentiated sarcoma histology, stage II or III, Clinical Group II or III). Patients are randomized to 1 of 2 treatment arms.

Arm I: Patients receive vincristine IV over 5-10 minutes once a week on weeks 0-12, 15, 18-24, 27, 30-36, and 39. Dactinomycin IV is administered over 15-20 minutes once a week on weeks 0, 3, 6, 9, 12, 21, 24, 27, 30, 33, 36, and 39. Cyclophosphamide IV is administered over 30-60 minutes once a week on weeks 0, 3, 6, 9, 12, 15, 18, 21, 24, 27, 30, 33, 36, and 39. After the initial 12 weeks of chemotherapy, depending on tumor shrinkage, patients may undergo surgery. After recovery from surgery, patients receive radiotherapy once a day, 5 days a week, during weeks 12-18. For patients receiving radiotherapy during weeks 0-6, dactinomycin is omitted during weeks 3 and 6 and administered during weeks 15 and 18. For patients receiving radiotherapy during weeks 12-18, dactinomycin is omitted during weeks 15 and 18. Patients showing an adequate response at week 24 continue chemotherapy during weeks 24-39.

Patients with Clinical Group III tumors of a parameningeal site with documented evidence of intracranial extension receive radiotherapy within the first 2 weeks of the initiation of the first course of chemotherapy (day 0).

Patients with Clinical Group II parameningeal tumors and Clinical Group III parameningeal tumors with base of skull erosion and/or cranial nerve palsy without evidence of intracranial extension receive radiotherapy on week 12 (day 84) or immediately thereafter.

Patients with Clinical Group IV parameningeal tumors with distant metastases receive radiotherapy to the primary site on week 12 (day 84). Patients with distant metastases confined to one site may receive radiotherapy to the metastatic site concurrently with therapy to the primary site if it began within 2 weeks of the initiation of chemotherapy (day 0).

Arm II: Patients receive treatment as in arm I, except dactinomycin is replaced with topotecan IV over 15-30 minutes daily for 5 days during weeks 3, 9, 21, 27, 33, and 39.

All patients receive filgrastim (G-CSF) or sargramostim (GM-CSF) subcutaneously beginning 24 hours after completion of each course of chemotherapy and continuing 1 year, until hematopoietic recovery.

Patients are followed every 1-2 months for 1 year, every 3 months for 1 year, every 6 months for 1 year, and then annually thereafter.

02

Conditions studied

  • Adult Malignant Mesenchymoma
  • Adult Rhabdomyosarcoma
  • Alveolar Childhood Rhabdomyosarcoma
  • Childhood Malignant Mesenchymoma
  • Embryonal Childhood Rhabdomyosarcoma
  • Embryonal-botryoid Childhood Rhabdomyosarcoma
  • Nonmetastatic Childhood Soft Tissue Sarcoma
  • Previously Untreated Childhood Rhabdomyosarcoma
  • Stage I Adult Soft Tissue Sarcoma
  • Stage II Adult Soft Tissue Sarcoma
  • Stage III Adult Soft Tissue Sarcoma
03

In context

Sarcoma

1,667 studies on the registry are indexed under Sarcoma; 393 are open to participants now.

This study's enrollment of 702 is above the median of 40 across 1,283 interventional studies indexed under Sarcoma.

Browse Sarcoma studies →

Lead sponsor

Children's Oncology Group is the lead sponsor of 436 studies on the registry; 34 are open to participants now.

Of its 12 completed or terminated interventional studies of FDA-regulated products, 11 (92%) have results posted.

Counted across the registry records on this site, refreshed daily.

04

Who can participate

Ages eligible
Up to 49 Years
Sexes eligible
All
Accepts healthy volunteers
No

Eligibility criteria

Inclusion Criteria:

  • Histologically proven disease of any of the following types:

    • Non metastatic alveolar rhabdomyosarcoma

      • Stage I, II, or III; Clinical Group I, II, or III
    • Stage II or III, Clinical Group III embryonal rhabdomyosarcoma

      • Botryoid
      • Spindle cell
    • Under 10 years, stage IV, Clinical Group IV embryonal rhabdomyosarcoma

      • Botryoid
      • Spindle cell
    • Undifferentiated sarcoma

      • Stage I, II, or III; Clinical Group I, II, or III
    • Ectomesenchymoma

      • Stage I, II, or III; Clinical Group I, II, or III, with alveolar features
      • Under 10 years, Stage IV, Clinical Group IV, with embryonal features
  • No more than 6 weeks since initial surgical procedure (e.g., biopsy) giving the definitive diagnosis
  • No parameningeal rhabdomyosarcoma with positive CSF cytology or multiple intracranial metastases
  • Bilirubin no greater than 1.5 mg/dL
  • Creatinine normal* for age
  • Not pregnant or nursing
  • Fertile patients must use effective contraception
  • No prior chemotherapy
  • Prior steroids allowed
  • No prior radiotherapy
  • See Disease Characteristics
05

Study design

Phase
Phase 3
Primary purpose
Treatment
Allocation
Randomized
Intervention model
Parallel assignment
Masking
None (open label)
Enrollment
702 participants (actual)

Study arms

  • Experimental
    Arm I

    Vincristine sulfate IV once a wk on wks 0-12, 15, 18-24, 27, 30-36, and 39. Dactinomycin IV once a wk on wks 0, 3, 6, 9, 12, 21, 24, 27, 30, 33, 36, and 39. Cyclophosphamide IV once a wk on wks 0, 3, 6, 9, 12, 15, 18, 21, 24, 27, 30, 33, 36, and 39. After 12 weeks of chemotherapy, depending on tumor shrinkage, pts may undergo surgery. After recovery from therapeutic conventional surgery, patients receive radiation therapy once a day, 5 days a wk, during wks 12-18. For pt receiving radiotherapy during wks 0-6, dactinomycin is omitted during wks 3 and 6 and during wks 15 and 18. For patients receiving radiotherapy during wks 12-18, dactinomycin is omitted during wks 15 and 18. Patients with adequate response at wk 24 continue chemotherapy during wks 24-39. All pts receive filgrastim (G-CSF) or sargramostim (GM-CSF) subcutaneously beginning 24 hours after completion of each course of chemotherapy and continuing 1 year, until hematopoietic recovery.

    Biological: dactinomycin · Drug: vincristine sulfate · Drug: cyclophosphamide · Procedure: therapeutic conventional surgery · Radiation: radiation therapy · Biological: filgrastim · Biological: sargramostim · Other: laboratory biomarker analysis

  • Experimental
    Arm II

    Patients receive treatment as in arm I, except dactinomycin is replaced with topotecan hydrochloride IV over 15-30 minutes daily for 5 days during weeks 3, 9, 21, 27, 33, and 39. All patients receive filgrastim (G-CSF) or sargramostim (GM-CSF) subcutaneously beginning 24 hours after completion of each course of chemotherapy and continuing 1 year, until hematopoietic recovery.

    Drug: vincristine sulfate · Drug: cyclophosphamide · Procedure: therapeutic conventional surgery · Radiation: radiation therapy · Drug: topotecan hydrochloride · Biological: filgrastim · Biological: sargramostim · Other: laboratory biomarker analysis

Interventions

  • Biologicaldactinomycin

    Given IV

    Also known as: ACT-D, actinomycin C1, AD, Cosmegen, DACT

  • Drugvincristine sulfate

    Given IV

    Also known as: leurocristine sulfate, VCR, Vincasar PFS

  • Drugcyclophosphamide

    Given IV

    Also known as: CPM, CTX, Cytoxan, Endoxan, Endoxana

  • Proceduretherapeutic conventional surgery

    Undergo surgery

  • Radiationradiation therapy

    Undergo radiotherapy

    Also known as: irradiation, radiotherapy, therapy, radiation

  • Drugtopotecan hydrochloride

    Given IV

    Also known as: hycamptamine, Hycamtin, SKF S-104864-A, TOPO

  • Biologicalfilgrastim

    Given SC

    Also known as: G-CSF, Neupogen

  • Biologicalsargramostim

    Given SC

    Also known as: GM-CSF, Leukine, Prokine

  • Otherlaboratory biomarker analysis

    Correlative studies

06

What researchers measure

Primary outcomes

  1. Long-term failure-free survival (FFS) between the two treatment groups

    Time frame: Up to 5 years

Secondary outcomes

  1. Overall survival between treatments

    Time frame: Up to 5 years

  2. Rate of second look surgery

    Time frame: Week 12

  3. Proportion of patients rendered tumor-free or with microscopic tumor only

    Time frame: Week 12

  4. Estimation of the rate of local failure for the patients who undergo second look surgery

    Done using standard cumulative incidence curves.

    Time frame: Week 12

07

Study locations

1 site
  • Children's Oncology Group
    Arcadia, California 91006-3776, United States
08

Updates

Tracking since Sep 25, 2026
No changes since tracking began. The registry record was last updated on Jun 17, 2013, before this site started recording changes on Sep 25, 2026. Its history is on ClinicalTrials.gov ↗
09

Registry details

Key details

Study ID
NCT00003958
Lead sponsor
Children's Oncology Group
Collaborators
National Cancer Institute (NCI)
Responsible party
Sponsor
First posted
Jan 27, 2003
Start date
Sep 2002
Primary completion
Oct 2007
Last update
Jun 17, 2013

Study contacts

Carola Arndt
principal investigator · Children's Oncology Group

Oversight

Data monitoring committee
Yes
View the source record on ClinicalTrials.gov ↗

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