Exatecan Mesylate in Treating Children With Relapsed or Refractory Rhabdomyosarcoma

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. Identifier: NCT00055939
Recruitment Status : Completed
First Posted : March 7, 2003
Last Update Posted : May 16, 2012
Information provided by (Responsible Party):
Daiichi Sankyo, Inc.

March 6, 2003
March 7, 2003
May 16, 2012
January 2003
April 2006   (Final data collection date for primary outcome measure)
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Complete list of historical versions of study NCT00055939 on Archive Site
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Exatecan Mesylate in Treating Children With Relapsed or Refractory Rhabdomyosarcoma
A Phase II Study Of Intravenous DX-8951f (EXATECAN MESYLATE) Administered Daily For Five Days Every Three Weeks To Pediatric Patients With Relapsed Or Refractory Rhabdomyosarcoma

RATIONALE: Drugs used in chemotherapy use different ways to stop tumor cells from dividing so they stop growing or die.

PURPOSE: Phase II trial to study the effectiveness of exatecan mesylate in treating children who have relapsed or refractory rhabdomyosarcoma.


  • Determine the objective response rate (complete and partial responses) in pediatric patients with relapsed or refractory rhabdomyosarcoma treated with exatecan mesylate.
  • Determine the time to tumor progression in patients treated with this drug.
  • Determine the median survival and survival at 6 and 12 months in patients treated with this drug.
  • Assess pain in patients treated with this drug.
  • Evaluate the quantitative and qualitative toxic effects of this drug in these patients.
  • Evaluate the pharmacokinetics of this drug in these patients.

OUTLINE: This is an open-label, nonrandomized, multicenter study.

Patients receive exatecan mesylate IV over 30 minutes on days 1-5. Treatment repeats every 3 weeks for up to 12 courses in the absence of disease progression or unacceptable toxicity. Patients who achieve a complete response (CR) receive 6 additional courses beyond CR.

PROJECTED ACCRUAL: A total of 13-27 patients will be accrued for this study within 1 year.

Phase 2
Allocation: Non-Randomized
Masking: None (Open Label)
Primary Purpose: Treatment
Drug: exatecan mesylate
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*   Includes publications given by the data provider as well as publications identified by Identifier (NCT Number) in Medline.
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April 2006
April 2006   (Final data collection date for primary outcome measure)


  • Histologically confirmed recurrent or resistant rhabdomyosarcoma
  • Measurable disease

    • The following are not considered measurable disease:

      • Ascites
      • Pleural effusion
      • Lytic bone lesions
  • No symptomatic brain metastases



  • Any age

Performance status

  • ECOG 0-2 (over 10 years old)
  • Lansky 60-100% (10 years old and under)

Life expectancy

  • At least 12 weeks


  • Absolute neutrophil count at least 750/mm^3
  • Platelet count at least 75,000/mm^3
  • Hemoglobin at least 8.5 g/dL


  • Bilirubin no greater than 2.0 mg/dL
  • Albumin at least 2.8 g/dL
  • AST or ALT no greater than 2.5 times upper limit of normal (ULN) (5 times ULN if liver metastases present)


  • Creatinine no greater than 1.5 times ULN


  • Not pregnant or nursing
  • Negative pregnancy test
  • Fertile patients must use effective contraception
  • No life threatening illness (unrelated to tumor) within the past 6 months
  • No other malignancy within the past 5 years except nonmelanoma skin cancer or carcinoma in situ of the cervix
  • No concurrent active serious infection
  • No concurrent uncontrolled infection
  • No overt psychosis or other incompetency that would preclude study compliance or giving informed consent
  • No other concurrent noncancer-related illness that would preclude study participation or follow-up


Biologic therapy

  • At least 3 months since prior autologous bone marrow or stem cell transplantation
  • No concurrent anticancer biologic therapy


  • Recovered from prior adjuvant or systemic chemotherapy
  • Prior topoisomerase I inhibitor therapy allowed
  • No other concurrent anticancer chemotherapy

Endocrine therapy

  • Not specified


  • More than 4 weeks since prior extensive radiotherapy to the cranial-spinal axis, whole pelvis, or 25% of marrow reserve
  • No concurrent anticancer radiotherapy


  • At least 4 weeks since prior major surgery
  • Recovered from prior surgery
  • No concurrent anticancer surgery


  • At least 28 days since prior investigational drugs (including analgesics or antiemetics)
  • No more than 2 prior regimens for rhabdomyosarcoma
  • No concurrent grapefruit-containing beverages or foods
  • No other concurrent investigational drugs during and for 28 days after final dose of study drug
Sexes Eligible for Study: All
Child, Adult, Older Adult
Contact information is only displayed when the study is recruiting subjects
Canada,   United States
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Daiichi Sankyo, Inc.
Daiichi Sankyo, Inc.
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Study Chair: Robert L. DeJager, MD, FACP Daiichi Sankyo, Inc.
Daiichi Sankyo, Inc.
May 2012

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP