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This study evaluates the drug Trabectedin (Yondelis) in children and adolescents with treatment-resistant solid tumors. It included patients aged 4-17 with confirmed tumors such as rhabdomyosarcoma, Ewing sarcoma, neuroblastoma, and Wilms' tumor. It was a Phase 1 study and has been completed.
The summary above is a plain-language rendering of the official record. The original English title is shown for reference.
Criteria are reproduced from the ClinicalTrials.gov record in the original English. Only the trial team can determine eligibility.
* INCLUSION CRITERIA: AGE: greater than or equal to 4 years and less than 17 years of age. DIAGNOSIS: Histologically confirmed solid tumors, which may include but are not limited to rhabdomyosarcoma and other soft tissue sarcomas, Ewing's sarcoma family of tumors, osteosarcoma, neuroblastoma, Wilms' tumor, hepatic tumors, germ cell tumors, and brain tumors with the exception that histologic confirmation is not required for patients with optic or brainstem gliomas. MEASURABLE/EVALUABLE DISEASE: Patients must have measurable or evaluable disease. PRIOR THERAPY: The patient's cancer must have relapsed after or failed to respond to frontline curative therapy and there must not be other potentially curative treatment options available. * Patients must have fully recovered to less than or equal to grade 1 from the acute toxic effects of all prior chemotherapy, immunotherapy, or radiotherapy prior to entering this study. * Myelosuppressive chemotherapy: The last dose of all myelosuppressive anticancer drugs must be at least 3 weeks prior to study entry. * Growth factors: The last dose of growth factors such as filgrastim and epoetin must be at least one week prior to study entry, the last dose of long-acting colony stimulating factors, such as pegfilgrastim, must be 2 weeks prior to study entry. * Investigational anti-cancer agents: The last dose of all investigational agents must be at least 30 days prior to study entry. * Biologic anti-cancer agents: The last dose of non-myelosuppressive biologic agents for the treatment of the patient's cancer (example, retinoids) must be at least 7 days prior to study entry. * Radiation therapy: The last dose of radiation to more than 25% of marrow containing bones (pelvis, spine, skull) must be at least 4 weeks prior to study entry, TBI and craniospinal radiation must be completed at least 4 months prior to study entry. The last dose of all other local palliative radiation must be at least 2 weeks prior to study entry. * Stem Cell Transplantation. Patients must be at least 2 months post-autologous transplant and recovered from treatment-related toxicities. Patients who have received an allogeneic transplant are excluded. CONCOMITANT MEDICATIONS: -Patients with brain tumors must be on a stable or tapering dose of corticosteroids for 7 days prior to the date of the baseline scan performed for the purpose of assessing response to therapy on this study. PERFORMANCE STATUS: Patients must have a Lansky (less than or equal to 10 years old) or Karnofsky (greater than 10 years old) score of greater than or equal to 60%. HEMATOLOGIC FUNCTION: Peripheral absolute neutrophil count greater than or equal to 1,500/microliter and a platelet count greater than or equal to 75,000/microliter independent of transfusion, and a hemoglobin of greater than or equal to 8 gm/dl (transfusion permitted to achieve this level). HEPATIC FUNCTION: * ALT (SGPT) and AST (SGOT) must be less than or equal to 2.5 x the upper limit of normal (ULN). * Bilirubin must be less than or equal to ULN. If the patient has Gilbert's syndrome, normal bilirubin is not required but should be discussed with the PI or study chair. * Alkaline phosphatase must be less than or equal to ULN for age and sex or if alkaline phosphatase greater than ULN then 5' nucleotidase must be less than or equal to ULN or the gamma-glutamyl transpeptidase (GGT) must be less than or equal to 2.5 times the ULN to be eligible. Normal Values for Alkaline Phosphatase at the NIH Clinical Center (U/L): Age between 4 to 6 years with a normal value (U/L) in Males 93 to 309 and a normal value (U/L) in Females 96 to 297. Age between 7 to 9 years with a normal value (U/L) in Males 86 to 315 and a normal value (U/L) in Females 69 to 325. Age between 10 to 12 years with a normal value (U/L) in Males 42 to 362 and a normal value (U/L) in Females 51 to 332. Age between 13 to 15 years with a normal value (U/L) in Males 74 to 390 and a normal value (U/L) in Females 50 to 162. Age between 16 to 18 years with a normal value (U/L) in Males 52 to 171 and a normal value (U/L) in Females 47 to 119. OTHER: creatine kinase less than or equal to 2.5 times the ULN. RENAL FUNCTION: Age-adjusted serum creatinine OR a creatinine clearance greater than or equal to 60 mL/min/1.73 m(2). Age less than or equal to 5 with a serum creatinine less than or equal to 0.8 (mg/dl). Age greater than 5 years and less than or equal to 10 with a serum creatinine of less than or equal to 1.0 (mg/dl). Age greater than 10 and less than or equal to 15 with a serum creatinine less than or equal to 1.2 (mg/dl). Age greater than 15 with a serum creatinine less than or equal to 1.5 (mg/dl). EXCLUSION CRITERIA: Patients with severe uncontrolled infections or other unrelated systemic illnesses, which in the judgment of the Principal or Associate Investigator would compromise the patient's ability to tolerate trabectedin or are likely to interfere with the study procedures or results. Patients with known history of xeroderma pigmentosum or other diseases with reduced DNA repair. Pregnant or breast-feeding females. Sexually active patients must be willing to use an effective form of birth control. Patients currently receiving other investigational agents. Patients who have received allogeneic stem cell transplants. Patients who have had prior therapy with trabectedin (ET-743, Yondelis).