Status:
COMPLETED
N2001-03: CEP-701 in Treating Young Patients With Recurrent or Refractory High-Risk Neuroblastoma
Lead Sponsor:
New Approaches to Neuroblastoma Therapy Consortium
Collaborating Sponsors:
National Cancer Institute (NCI)
Conditions:
Neuroblastoma
Eligibility:
All Genders
1-30 years
Phase:
PHASE1
Brief Summary
RATIONALE: CEP-701 may stop the growth of tumor cells by blocking the enzymes necessary for their growth. PURPOSE: This phase I trial is studying the side effects and best dose of CEP-701 in treating...
Detailed Description
OBJECTIVES: Primary * Determine the maximum tolerated dose of CEP-701 in pediatric patients with recurrent or refractory high-risk neuroblastoma. * Determine the dose-limiting toxicity of this drug ...
Eligibility Criteria
Inclusion
- DISEASE CHARACTERISTICS:
- Diagnosis of neuroblastoma confirmed by at least 1 of the following:
- Histology
- Demonstrates clumps of tumor cells in the bone marrow with elevated urinary catecholamine metabolites
- Recurrent or resistant/refractory disease
- Neuroblastoma metastatic to the bone marrow with granulocytopenia, anemia, and/or thrombocytopenia allowed
- High-risk disease
- Patients in first response after completion of a prior front-line myeloablative regimen OR who were medically ineligible to receive a front-line myeloablative regimen must meet at least 1 of the following criteria:
- Viable neuroblastoma determined by biopsy of a persistent lesion as seen on CT scan, MRI, or metaiodobenzylguanidine (MIBG) scan
- If lesion was irradiated, biopsy must be performed at least 4 weeks after completion of prior radiotherapy
- Morphologic evidence of tumor in bone marrow
- Second or greater response (without histologic confirmation) allowed
- Meets at least 1 of the following criteria:
- At least 1 unidimensionally measurable lesion on CT scan, MRI, or X-ray
- At least 20 mm by conventional techniques OR at least 10 mm by spiral CT scan
- MIBG scan with positive uptake at a minimum of 1 site
- Bone marrow with tumor cells on routine morphology (not by NSE staining only) of bilateral aspirate and/or biopsy AND/OR at least 5 tumor cells/10\^6 mononuclear cells in the bone marrow by immunocytologic analysis of 2 consecutive bone marrows performed at least 1 day but no more than 4 weeks apart
- PATIENT CHARACTERISTICS:
- Age
- 21 and under at diagnosis
- Performance status
- Karnofsky 50-100% (for patients \> 16 years of age)
- Lansky 50-100% (for patients ≤ 16 years of age)
- Life expectancy
- More than 2 months
- Hematopoietic
- See Disease Characteristics
- Absolute neutrophil count ≥ 1,000/mm\^3
- Platelet count ≥ 50,000/mm\^3 (transfusion independent)
- Hemoglobin ≥ 8.0 g/dL (red blood cell transfusions allowed)
- Hepatic
- ALT and AST ≤ 3.0 times upper limit of normal (ULN)
- Total bilirubin ≤ 1.5 times ULN
- Renal
- Creatinine ≤ 1.5 times normal OR
- Creatinine clearance or radioisotope glomerular filtration rate ≥ 60 mL/min
- Cardiovascular
- Ejection fraction ≥ 50% by echocardiogram or MUGA OR
- Fractional shortening ≥ 28% or above lower limit of normal by echocardiogram
- Pulmonary
- Lung function normal
- No dyspnea at rest
- No exercise intolerance
- No supplemental oxygen requirement
- Other
- Not pregnant
- Negative pregnancy test
- Fertile patients must use effective contraception
- No uncontrolled infection
- No other concurrent illness that would preclude study treatment
- PRIOR CONCURRENT THERAPY:
- Biologic therapy
- See Chemotherapy
- At least 2 weeks since prior biologic or non-myelosuppressive therapy and recovered
- More than 7 days since prior growth factors
- No prior allogeneic stem cell transplantation AND no extensive chronic graft-versus-host disease
- No concurrent growth factors except filgrastim (G-CSF) or sargramostim (GM-CSF) administered for neutropenia lasting for more than 7 days or for confirmed or clinical septicemia associated with neutropenia
- Chemotherapy
- At least 3 months since prior myeloablative chemotherapy with stem cell transplantation
- At least 2 weeks since prior chemotherapy and recovered
- Endocrine therapy
- No concurrent corticosteroid therapy except replacement therapy for adrenal insufficiency or treatment for increased intracranial pressure
- Radiotherapy
- See Disease Characteristics
- Recovered from prior radiotherapy
- At least 6 weeks since prior therapeutic-dose MIBG
- At least 6 weeks since prior craniospinal or other radiotherapy involving significant bone marrow (i.e., total pelvis or total abdomen)
- At least 4 weeks since prior radiotherapy to any site biopsied
- At least 2 weeks since prior local palliative radiotherapy (small port)
- Surgery
- Not specified
- Other
- No prior CEP-701
- No concurrent administration of any of the following CYP3A4 inhibitors:
- Cyclosporine
- Clotrimazole
- Ketoconazole
- Erythromycin
- Clarithromycin
- Troleandomycin
- HIV protease inhibitors
- Nefazodone
- Itraconazole
- Voriconazole
Exclusion
Key Trial Info
Start Date :
August 1 2003
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
February 1 2011
Estimated Enrollment :
47 Patients enrolled
Trial Details
Trial ID
NCT00084422
Start Date
August 1 2003
End Date
February 1 2011
Last Update
April 10 2023
Active Locations (12)
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1
Childrens Hospital Los Angeles
Los Angeles, California, United States, 90027-0700
2
Lucille Salter Packer Children's Hospital, Stanford University
Palo Alto, California, United States, 94305
3
UCSF Helen Diller Family Comprehensive Cancer Center
San Francisco, California, United States, 94143
4
AFLAC Cancer Center and Blood Disorders Service of Children's Healthcare of Atlanta - Egleston Campus
Atlanta, Georgia, United States, 30322