Alternate Title
Basic Trial Information
Objectives
Entry Criteria
Expected Enrollment
Outcomes
Outline
Trial Contact Information
Related Information
Registry Information

Alternate Title

Trabectedin in Treating Young Patients With Solid Tumors That Have Relapsed or Not Responded to Treatment

Basic Trial Information

Phase	Type	Status	Age	Sponsor	Protocol IDs
Phase I	Treatment	Active	4 to 16	NCI	NCI-07-C-0054 NCT00437047

Special Category: NIH Clinical Center trial, NCI Web site featured trial

Objectives

1. Determine the maximum tolerated dose of trabectedin in pediatric patients with relapsed or refractory solid tumors.
2. Determine the toxicity profile of this drug in these patients.

Entry Criteria

Disease Characteristics:

• Histologically confirmed* solid tumor, including, but not limited to, any of the following:
  • Rhabdomyosarcoma or other soft tissue sarcomas
  • Ewing's sarcoma family of tumors
  • Osteosarcoma
  • Neuroblastoma
  • Wilms' tumor
  • Hepatic tumors
  • Germ cell tumors
  • Brain tumors
    • Must be on a stable or tapering dose of corticosteroids for 7 days before the baseline scan
   [Note: *Patients with optic or brainstem gliomas do not require histologic confirmation]
• Measurable or evaluable disease
• Relapsed disease OR failed to respond to front-line curative therapy and no other potentially curative treatment options are available

Prior/Concurrent Therapy:

• See Disease Characteristics
• Recovered from all prior therapy
• At least 3 weeks since prior myelosuppressive chemotherapy
• At least 1 week since prior growth factors (e.g., filgrastim [G-CSF] or epoetin alfa)
• At least 2 weeks since prior long-acting colony-stimulating factors (e.g., pegfilgrastim)
• At least 30 days since prior anticancer investigational agents
• At least 1 week since prior nonmyelosuppressive biologic therapy (e.g., retinoids)
• At least 4 weeks since prior radiotherapy to > 25% of marrow-containing bones (e.g., pelvis, spine, skull)
• At least 4 months since prior total-body irradiation or craniospinal radiotherapy
• At least 2 weeks since prior palliative radiotherapy
• At least 2 months since prior autologous stem cell transplantation
• No prior trabectedin
• No prior allogeneic stem cell transplantation
• No other concurrent investigational agents
• No other concurrent anticancer therapy, including chemotherapy, radiotherapy, or immunotherapy

Patient Characteristics:

• Karnofsky performance status (PS) 60-100% (patients > 10 years of age) OR Lansky PS 60-100% (patients ≤ 10 years of age)
• Absolute neutrophil count ≥ 1,500/mm³
• Platelet count ≥ 75,000/mm³ (transfusion independent)
• Hemoglobin ≥ 8 g/dL (transfusion allowed)
• ALT and AST ≤ 2.5 times upper limit of normal (ULN)
• Bilirubin normal (unless elevation is due to Gilbert's syndrome and approved by the principal investigator)
• Alkaline phosphatase normal OR 5' nucleotidase normal OR gamma-glutamyl transpeptidase (GGT) ≤ 2.5 times ULN
• Creatine kinase ≤ 2.5 times ULN
• Creatinine clearance ≥ 60 mL/min OR age-adjusted creatinine meeting the following criteria:
  • No greater than 0.8 mg/dL (≤ 5 years of age)
  • No greater than 1.0 mg/dL (6-10 years of age)
  • No greater than 1.2 mg/dL (11-15 years of age)
  • No greater than 1.5 mg/dL (≥ 15 years of age)
• No severe uncontrolled infections or other unrelated systemic illnesses that would preclude compliance with study requirements
• No known history of xeroderma pigmentosum or other diseases with reduced DNA repair
• Not pregnant or nursing
• Negative pregnancy test
• Fertile patients must use effective contraception

Expected Enrollment

A total of 24 patients will be accrued for this study.

Outcomes

Maximum tolerated dose
Toxicity

Outline

This is a dose-escalation study.

Patients receive trabectedin IV over 24 hours on day 1. Patients also receive filgrastim (G-CSF) subcutaneously (SC) beginning on day 3 and continuing until blood counts recover OR pegfilgrastim SC once on day 3. Treatment repeats every 21 days in the absence of disease progression or unacceptable toxicity.

Cohorts of 3-12 patients receive escalating doses of trabectedin until the maximum tolerated dose (MTD) is determined. The MTD is defined as the dose preceding that at which 2 of 6 or 4 of 12 patients experience dose-limiting toxicity.

Trial Contact Information

Trial Lead Organizations

NCI - Center for Cancer Research

Elizabeth Fox, MD, Principal investigator		Ph: 301-402-6641 Email: foxb@mail.nih.gov

U.S.A.
Maryland
	Bethesda
	Warren Grant Magnuson Clinical Center - NCI Clinical Trials Referral Office
		Clinical Trials Office - Warren Grant Magnusen Clinical Center - NCI Clinical Trials Referral Office	Ph:  888-NCI-1937

Related Information

Featured trial article

Registry Information
Official Title		A Phase I Trial and Pharmacokinetic Study of Trabectedin (Yondelis®, ET-743) in Children and Adolescents with Relapsed or Refractory Solid Tumors
Trial Start Date		2008-06-08
Registered in ClinicalTrials.gov		NCT00437047
Date Submitted to PDQ		2007-01-04
Information Last Verified		2009-07-05

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