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 refractoryrhabdomyosarcoma.

Condition	Treatment or Intervention	Phase
previously treated childhood rhabdomyosarcoma recurrent childhood rhabdomyosarcoma	Drug: exatecan mesylate  Procedure: chemotherapy	Phase II

Further Study Details: 

OBJECTIVES:

• 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.

Genders Eligible for Study:  Both

Criteria

DISEASE CHARACTERISTICS:

• 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

PATIENT CHARACTERISTICS: Age

• 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

Hematopoietic

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

Hepatic

• 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)

Renal

• Creatinine no greater than 1.5 times ULN

Other

• 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

PRIOR CONCURRENT THERAPY: Biologic therapy

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

Chemotherapy

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

Endocrine therapy

• Not specified

Radiotherapy

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

Surgery

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

Other

• 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

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

Study chairs or principal investigators

Robert L. DeJager, MD, FACP,  Study Chair,  Daiichi Pharmaceuticals   

Study ID Numbers:  CDR0000271888; DAIICHI-8951A-PRT033; SJCRH-DXRMS
Record last reviewed:  March 2004
Last Updated:  October 13, 2004
Record first received:  March 6, 2003
ClinicalTrials.gov Identifier:  NCT00055939
Health Authority: Unspecified
ClinicalTrials.gov processed this record on 2005-04-08