Clinical trial NCT00055952: Exatecan Mesylate in Treating Patients With Ewing's Sarcoma, Primitive Neuroectodermal Tumor, or Desmoplastic Small Round Cell Tumor
Official title: A Phase II Study Of Intravenous DX-8951f (EXATECAN MESYLATE) Administered Daily For Five Days Every Three Weeks To Pediatric And Young Adult Patients With Ewing's Sarcoma (ES), Primitive Neuroectodermal Tumor (PNET), Or Desmoplastic Small Round Cell Tumor (DSRCT)
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Sarcoma
Keywords: childhood desmoplastic small round cell tumor
metastatic childhood soft tissue sarcoma
nonmetastatic childhood soft tissue sarcoma
recurrent childhood soft tissue sarcoma
localized Ewing sarcoma/peripheral primitive neuroectodermal tumor
metastatic Ewing sarcoma/peripheral primitive neuroectodermal tumor
recurrent Ewing sarcoma/peripheral primitive neuroectodermal tumor
Lead sponsor: Daiichi Sankyo
Sponsor class: INDUSTRY
First posted: 2003-03-07
Sex: ALL
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Primary purpose: TREATMENT

Brief summary: 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 patients who have relapsed or refractory Ewing's sarcoma or peripheral primitive neuroectodermal tumor or desmoplastic small round cell tumor.

Detailed description: OBJECTIVES:

* Determine the objective response rate in patients with Ewing's sarcoma, primitive neuroectodermal tumor, or desmoplastic small round cell tumor treated with exatecan mesylate.
* Determine the time to tumor progression in patients treated with this drug.
* Determine median survival and 6- and 12-month survival of patients treated with this drug.
* Determine the pain response in patients treated with this drug.
* Determine the qualitative and quantitative toxic effects of this drug in these patients.
* Determine the pharmacokinetics of this drug in these patients.

OUTLINE: This is an open-label, non-randomized, multicenter study. Patients are stratified according to disease (relapsed or refractory localized or metastatic Ewing's sarcoma or primitive neuroectodermal tumor vs desmoplastic small round cell tumor).

Patients receive exatecan mesylate IV over 30 minutes on days 1-5. Treatment repeats every 21 days in the absence of disease progression or unacceptable toxicity for a maximum of 12 courses, or 6 courses beyond maximal response (whichever is longer).

Patients are followed every 3 months for 1 year after withdrawal from study.

PROJECTED ACCRUAL: A total of 13-27 patients will be accrued for stratum I within 12 months. A total of 9-17 patients will be accrued for stratum II within 15 months.

Eligibility criteria: DISEASE CHARACTERISTICS:

* One of the following histologically confirmed diagnoses:

  * Relapsed or refractory Ewing's sarcoma or primitive neuroectodermal tumor
  * Desmoplastic small round cell tumor
* 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 of age)
* Lansky 60-100% (10 years of age 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
* AST or ALT no greater than 2.5 times upper limit of normal (ULN) (5 times ULN if liver metastases are present)
* Albumin at least 2.8 g/dL

Renal

* Creatinine less than 1.5 times ULN

Other

* Not pregnant or nursing
* Negative pregnancy test
* Fertile patients must use effective contraception
* No active serious infection
* No other malignancy within the past 5 years except nonmelanoma skin cancer or carcinoma in situ of the cervix
* No overt psychosis or mental disability that would preclude informed consent
* No other life-threatening illness within the past 6 months

PRIOR CONCURRENT THERAPY:

Biologic therapy

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

Chemotherapy

* Recovered from prior systemic chemotherapy
* Prior topoisomerase I inhibitor therapy allowed
* No other concurrent chemotherapy

Endocrine therapy

* Not specified

Radiotherapy

* More than 4 weeks since prior cranial, spinal, or whole pelvis radiotherapy
* More than 4 weeks since prior radiotherapy to 25% of bone marrow reserve
* No concurrent radiotherapy

Surgery

* At least 4 weeks since prior major surgery and recovered
* No concurrent surgery

Other

* More than 28 days since prior investigational drugs (including analgesics or antiemetics)
* No more than 2 prior treatment regimens for this disease
* No other investigational drugs during and for 28 days after study therapy
* No other concurrent anticancer therapy
* No concurrent grapefruit or grapefruit juice

Primary outcomes:
Secondary outcomes:
Other outcomes:
Locations:
city: Denver
country: United States
facility: University of Colorado Cancer Center at University of Colorado Health Sciences Center
state: Colorado
city: Jacksonville
country: United States
facility: Nemours Children's Clinic
state: Florida
city: New Brunswick
country: United States
facility: Cancer Institute of New Jersey
state: New Jersey
city: New York
country: United States
facility: Memorial Sloan-Kettering Cancer Center
state: New York
city: Memphis
country: United States
facility: St. Jude Children's Research Hospital
state: Tennessee
city: Dallas
country: United States
facility: Medical City Dallas Hospital
state: Texas
city: Dallas
country: United States
facility: Simmons Cancer Center at University of Texas Southwestern Medical Center - Dallas
state: Texas
city: Houston
country: United States
facility: University of Texas - MD Anderson Cancer Center
state: Texas
city: Toronto
country: Canada
facility: Hospital for Sick Children
state: Ontario
Study officials:
affiliation: Daiichi Sankyo
name: Robert L. DeJager, MD, FACP
role: STUDY_CHAIR
References: