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A Phase II Study Of Trabectedin (ET-743, Yondelis®) in Children With Recurrent Rhabdomyosarcoma, Ewing Sarcoma, or Nonrhabdomyosarcomatous Soft Tissue Sarcomas

Phase 2
1 Year
21 Years
Not Enrolling
Previously Treated Childhood Rhabdomyosarcoma, Recurrent Childhood Rhabdomyosarcoma, Recurrent Childhood Soft Tissue Sarcoma, Recurrent Ewing Sarcoma, Peripheral Primitive Neuroectodermal Tumor

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Trial Information

A Phase II Study Of Trabectedin (ET-743, Yondelis®) in Children With Recurrent Rhabdomyosarcoma, Ewing Sarcoma, or Nonrhabdomyosarcomatous Soft Tissue Sarcomas


I. Determine the response rate in pediatric patients with recurrent or refractory soft
tissue sarcomas or Ewing's sarcoma family of tumors treated with ecteinascidin 743

II. Determine the toxicity of this drug in these patients. III. Determine the
pharmacokinetics of this drug in these patients.


Patients receive trabectedin IV over 3 hours on day 1. Treatment repeats every 21days for up
to 26 courses in the absence of disease progression or unacceptable toxicity.

After completion of study treatment, patients are followed up for up to 5 years.

Inclusion Criteria:

- Histologically confirmed recurrent or refractory sarcoma tumors, including the

- Rhabdomyosarcoma

- Nonrhabdomyosarcomatous soft tissue sarcoma

- Ewing's sarcoma

- Measurable disease by imaging studies

- Lesions assessable only by radionuclide scans are not considered measurable

- If the only measurable lesion has been previously irradiated, then that lesion
must have shown evidence of an interim increase in size

- No significant amount of metastatic liver disease, defined as the following:

- Lesions occupying more than 25% of the liver by imaging and abnormal liver
function tests or abnormal synthetic liver function

- Performance status - Lansky 50-100% (10 years of age and under)

- Performance status - Karnofsky 50-100% (over 10 years of age)

- Absolute neutrophil count at least 1,000/mm^3

- Platelet count at least 100,000/mm^3 (transfusion independent)

- Hemoglobin at least 8.0 g/dL (transfusion allowed)

- No concurrent CYP3A4 inhibitors, including the following:

- Grapefruit juice

- Erythromycin

- Azithromycin

- Clarithromycin

- Rifampin and its analogs

- Fluconazole

- Ketoconazole

- Itraconazole

- Cimetidine

- Cannabinoids (marijuana or dronabinol)

- Leukotriene inhibitors used in asthma (e.g., zafirlukast, montelukast, or

- Bilirubin no greater than upper limit of normal (ULN)

- Total alkaline phosphatase no greater than ULN

- Hepatic fraction alkaline phosphatase and 5 nucleotidase no greater than ULN

- SGOT and SGPT ≤ 2.5 times ULN

- Albumin ≥ 2.5 g/dL

- Gamma-glutamyl transferase < 2.5 times ULN

- Maximum creatinine based on age as follows:

- 0.8 mg/dL (5 years of age and under)

- 1.0 mg/dL (6 to 10 years of age)

- 1.2 mg/dL (11 to 15 years of age)

- 1.5 mg/dL (over 15 years of age)

- Creatinine clearance or radioisotope glomerular filtration rate (GFR) at least 70

- No uncompensated congestive heart failure within the past 6 months

- Not pregnant or nursing

- Fertile patients must use effective contraception during and for 2 months after study

- No active uncontrolled infection

- Weight ≥ 15 kilograms

- More than 1 week since prior growth factors that support platelet or white blood cell
number or function

- At least 7 days since prior biologic agents and recovered

- No prior allogeneic stem cell transplantation

- No other concurrent immunomodulating agents

- More than 3 weeks since prior myelosuppressive chemotherapy (4 weeks for
nitrosoureas) and recovered

- No more than 2 prior multi-agent chemotherapy regimens

- No other concurrent anticancer chemotherapy

- Concurrent steroids allowed

- At least 6 weeks since prior since prior extended radiotherapy and recovered

- No prior total body radiotherapy

- Concurrent radiotherapy to localized painful lesions allowed provided at least 1
measurable lesion is not irradiated*

- At least 7 days since prior enzyme-inducing anticonvulsants (e.g., carbamazepine,
phenobarbital, or phenytoin)

- No concurrent enzyme-inducing anticonvulsants

- No other concurrent investigational agents

Type of Study:


Study Design:

Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Outcome Measure:

Response rate (complete response [CR] and partial response [PR]) using the Response Evaluation Criteria in Solid Tumors (RECIST)

Outcome Description:

Response rates and confidence intervals will be constructed according to the method of Chang and O'Brien. All patients who have two consecutive evaluations of stable disease, CR, or PR must have imaging studies reviewed centrally at the Children Oncology Group (COG).

Outcome Time Frame:

Up to 5 years after completion of study treatment

Safety Issue:


Principal Investigator

Sylvain Baruchel

Investigator Role:

Principal Investigator

Investigator Affiliation:

Children's Oncology Group


United States: Food and Drug Administration

Study ID:




Start Date:

January 2008

Completion Date:

Related Keywords:

  • Previously Treated Childhood Rhabdomyosarcoma
  • Recurrent Childhood Rhabdomyosarcoma
  • Recurrent Childhood Soft Tissue Sarcoma
  • Recurrent Ewing Sarcoma
  • Peripheral Primitive Neuroectodermal Tumor
  • Rhabdomyosarcoma
  • Neuroectodermal Tumors
  • Neuroectodermal Tumors, Primitive
  • Rhabdomyosarcoma, Embryonal
  • Sarcoma
  • Sarcoma, Ewing's
  • Neuroectodermal Tumors, Primitive, Peripheral



Roswell Park Cancer Institute Buffalo, New York  14263
University of Arkansas for Medical Sciences Little Rock, Arkansas  72205
St. Jude Children's Research Hospital Memphis, Tennessee  38105-2794
Children's Hospital Central California Madera, California  93638-8762
Nationwide Children's Hospital Columbus, Ohio  43205-2696
University Of Vermont Burlington,, Vermont  05403
Seattle Children's Hospital Seattle, Washington  98105
Wake Forest University Health Sciences Winston-Salem, North Carolina  27157
Childrens Memorial Hospital Chicago, Illinois  60614
New York University Langone Medical Center New York, New York  10016
Columbia University Medical Center New York, New York  10032