List All Neuroblastoma Trials


Intravenous Fenretinide for Young Patients With Recurrent or Resistant Neuroblastoma (NANT 2004-03)
A Phase I Study of Intravenous (Emulsion) Fenretinide (4-HPR, NSC 374551) in Children with Recurrent or Resistant Neuroblastoma (IND #70,058)
Status Conditions Phase Study ID
Closed Neuroblastoma Phase I NANT 2004-03

RATIONALE: Drugs used in chemotherapy, such as fenretinide, work in different ways to stop the growth of tumor cells, either by killing the cells or by stopping them from dividing.

PURPOSE: This phase I trial is studying the side effects and best dose of intravenous fenretinide in treating young patients with recurrent or resistant neuroblastoma.

Julie Park, MD
Seattle Cancer Care Alliance 800-804-8824  
Eligibility Criteria (must meet the following to participate in this study)

Ages Eligible for Study:   up to 30 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No



  • Diagnosis of neuroblastoma either by histological confirmation and/or demonstration of tumor cells in the bone marrow with increased urinary catecholamines

    • Differentiating ganglioneuroblastoma allowed

      • No histological evidence only of ganglioneuroma by tumor biopsy or bone marrow biopsy
  • High-risk disease meeting at least one of the following criteria:

    • Recurrent/progressive disease at any time
    • Refractory disease (i.e., less than a partial response to front-line therapy that included ≥ 4 courses of chemotherapy)
    • Persistent disease after at least a partial response to front-line therapy (i.e., still has residual disease by MIBG, CT/MRI, or bone marrow biopsy)

      • Biopsy of at least one residual site demonstrating viable neuroblastoma required (tumor by bone marrow morphology is considered adequate documentation of disease)
  • Measurable disease meeting at least one of the following criteria:

    • Measurable tumor on MRI or CT scan, defined as at least one unidimensionally measurable lesion ≥ 20 mm by conventional techniques or ≥ 10 mm by spiral CT scan

      • For patients with persistent disease, a biopsy* of bone marrow or bone or soft tissue site must have demonstrated viable neuroblastoma
    • MIBG scan with positive uptake at a minimum of one site

      • For patients with persistent disease, a biopsy* of a MIBG positive site must have demonstrated viable neuroblastoma
    • Bone marrow with tumor cells seen on routine morphology (not by NSE staining only) of bilateral aspirate and/or biopsy of one bone marrow sample NOTE: *If the lesion was irradiated, the biopsy must have been done at least 4 weeks after completion of radiotherapy
  • No CNS parenchymal or meningeal-based lesions

    • Skull-based tumor lesions with or without intracranial extension are allowed provided there are no neurologic signs or symptoms or hydrocephalus related to the lesion
    • Patients with a history of complete surgical resection of CNS lesions are eligible provided there is no evidence of CNS lesions by MRI or CT scan at study entry
    • Patients with a history of CNS lesions must be off corticosteroid therapy for CNS lesions for ≥ 4 weeks


  • Performance status 0-2
  • Life expectancy ≥ 2 months
  • ANC ≥ 500/mm³
  • Platelet count ≥ 50,000/mm³ (transfusion independent)
  • Hemoglobin ≥ 8.0 g/dL (transfusion independent)
  • Serum creatinine ≤ 1.5 times normal for age
  • Total bilirubin ≤ 1.5 times normal for age
  • ALT and AST ≤ 3 times normal for age
  • Serum triglycerides < 300 mg/dL
  • Serum calcium < 11.6 mg/dL
  • Lipase normal for age
  • PT/PTT ≤ 1.5 times upper limit of normal for age (without fresh frozen plasma support; vitamin K allowed)
  • Not pregnant or nursing
  • Negative pregnancy test
  • Fertile patients must use effective contraception prior to, during, and for 2 months after completion of study treatment
  • LVEF ≥ 55% by ECHO or MUGA scan OR fractional shortening ≥ 27% by ECHO
  • No EKG abnormality
  • No dyspnea at rest or requirement for oxygen
  • No hematuria and/or proteinuria > 1+ on urinalysis
  • No known history of allergy to egg products
  • No known history of allergy to soy bean oil
  • No skin toxicity > grade 1 per CTCAE v3
  • Seizure disorder allowed if seizures are controlled on anticonvulsants and the anticonvulsant(s) is not contraindicated
  • Known genetic, metabolic, or psychiatric conditions, or other ongoing serious medical issues must be approved by the study chair prior to study registration


  • Recovered from all prior chemotherapy, immunotherapy, or radiotherapy
  • More than 3 weeks since prior myelosuppressive chemotherapy (4 weeks for nitrosoureas) and/or biologic therapy without stem cell support
  • More than 7 days since prior hematopoietic growth factors
  • No prior radiotherapy to the only site of measurable disease unless there has been subsequent disease progression at that site or a biopsy of that site showed viable neuroblastoma ≥ 4 weeks after completion of radiotherapy
  • Prior CNS irradiation allowed
  • At least 2 weeks since prior small field (focal) radiotherapy
  • At least 6 weeks since prior large field radiotherapy (i.e., total-body irradiation, craniospinal radiotherapy, whole abdominal or total lung radiotherapy, or radiotherapy to > 50% of marrow space)
  • At least 56 days since prior myeloablative autologous stem cell transplantation
  • At least 4 weeks since prior myelosuppressive therapy with stem cell support
  • At least 6 weeks since prior MIBG therapy
  • Prior oral fenretinide therapy allowed
  • At least 3 weeks since prior retinoid therapies
  • No prior organ transplantation
  • No prior myeloablative allogeneic stem cell transplantation unless stem cells were from an identical twin sibling
  • No concurrent systemic corticosteroids, including corticosteroids for emesis control

    • Concurrent inhaled corticosteroids for asthma control or steroids for metabolic deficiency states allowed
  • Concurrent palliative radiotherapy allowed provided the irradiated sites will not be used to measure response
  • No concurrent parenteral intralipids
  • No other concurrent chemotherapy or immunomodulating agents
  • No concurrent drugs suspected of causing pseudotumor cerebri, including tetracycline, nalidixic acid, nitrofurantoin, phenytoin, sulfonamides, lithium, or amiodarone
  • No concurrent vitamin A, C, or E supplements (except as part of routine total parenteral nutrition [TPN] supplements or as part of a single daily standard dose of oral multivitamin supplement)
  • No concurrent medications that may potentially act as modulators of intracellular ceramide levels or ceramide cytotoxicity, sphingolipid transport, or p-glycoprotein (MDR1) or MDR1 drug/lipid transporters, including cyclosporine or analogue, verapamil, tamoxifen or analogue, ketoconazole, chlorpromazine, mifepristone (RU486), indomethacin, or sulfinpyrazone
  • No other concurrent anticancer agents
  • No concurrent herbal supplements or other alternative therapy medications
  • No concurrent anti-arrhythmia or inotropic cardiac medications
Last Updated
April 09, 2012
See this trial at
Access protocol and consent forms at Fred Hutchinson Cancer Research Center
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