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Exatecan Mesylate in Treating Children With Relapsed or Refractory Rhabdomyosarcoma

This study has been completed.
Information provided by (Responsible Party):
Daiichi Sankyo Inc. Identifier:
First received: March 6, 2003
Last updated: May 15, 2012
Last verified: May 2012

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.

Condition Intervention Phase
Drug: exatecan mesylate
Phase 2

Study Type: Interventional
Study Design: Allocation: Non-Randomized
Masking: Open Label
Primary Purpose: Treatment
Official Title: 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

Resource links provided by NLM:

Further study details as provided by Daiichi Sankyo Inc.:

Study Start Date: January 2003
Study Completion Date: April 2006
Primary Completion Date: April 2006 (Final data collection date for primary outcome measure)
Detailed Description:


  • 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.


Ages Eligible for Study:   Child, Adult, Senior
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No


  • 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
  Contacts and Locations
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Please refer to this study by its identifier: NCT00055939

United States, Colorado
University of Colorado Cancer Center at University of Colorado Health Sciences Center
Denver, Colorado, United States, 80218
United States, Florida
Nemours Children's Clinic
Jacksonville, Florida, United States, 32207
United States, New Jersey
Cancer Institute of New Jersey
New Brunswick, New Jersey, United States, 08903
United States, New York
Memorial Sloan-Kettering Cancer Center
New York, New York, United States, 10021
United States, Tennessee
St. Jude Children's Research Hospital
Memphis, Tennessee, United States, 38105
United States, Texas
Medical City Dallas Hospital
Dallas, Texas, United States, 75230
Simmons Cancer Center at University of Texas Southwestern Medical Center - Dallas
Dallas, Texas, United States, 75390-9063
University of Texas - MD Anderson Cancer Center
Houston, Texas, United States, 77030-4009
Canada, Ontario
Hospital for Sick Children
Toronto, Ontario, Canada, M5G 1X8
Sponsors and Collaborators
Daiichi Sankyo Inc.
Study Chair: Robert L. DeJager, MD, FACP Daiichi Sankyo Inc.
  More Information

Responsible Party: Daiichi Sankyo Inc. Identifier: NCT00055939     History of Changes
Other Study ID Numbers: CDR0000271888
Study First Received: March 6, 2003
Last Updated: May 15, 2012

Keywords provided by Daiichi Sankyo Inc.:
previously treated childhood rhabdomyosarcoma
recurrent childhood rhabdomyosarcoma

Additional relevant MeSH terms:
Neoplasms, Muscle Tissue
Neoplasms, Connective and Soft Tissue
Neoplasms by Histologic Type
Topoisomerase I Inhibitors
Topoisomerase Inhibitors
Enzyme Inhibitors
Molecular Mechanisms of Pharmacological Action
Antineoplastic Agents
Antineoplastic Agents, Phytogenic processed this record on April 25, 2017