Experimental IV drug shows promise for tough childhood cancer
NCT ID NCT00646230
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This early-phase study tested an intravenous form of the drug fenretinide in 17 children whose neuroblastoma had come back or didn't respond to standard treatments. The main goals were to find the safest dose and understand side effects. Researchers also looked at how the drug moved through the body and whether it could shrink tumors.
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Study facts
What this study's own registry entry says, in plain language.
- Phase
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Phase 1
The first testing in people. Mainly checks safety and dose, usually in a small group.
- Participants
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17 people
The number who actually took part.
- Start date
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Dec 2006
- Finished
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Mar 2012
- Lead sponsor
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Other sponsor
The registry's catch-all category, for sponsors it does not file as a company, a government agency, or a research network.
Who can take part
This study's own entry requirements. Only the study team can say for certain whether you qualify.
- Ages
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0 to 30 years
- Sex
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Anyone
- Healthy volunteers
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Not accepted
This study is not open to healthy volunteers. The entry requirements below say who it is open to.
Show the full entry requirements Hide the full entry requirements
Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.
DISEASE CHARACTERISTICS: * 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 PATIENT CHARACTERISTICS: * 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 PRIOR CONCURRENT THERAPY: * 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
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
Locations
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AFLAC Cancer Center and Blood Disorders Service of Children's Healthcare of Atlanta - Egleston Campus
Atlanta, Georgia, 30322, United States
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C.S. Mott Children's Hospital at University of Michigan Medical Center
Ann Arbor, Michigan, 48109-0286, United States
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Children's Hospital Boston
Boston, Massachusetts, 02115, United States
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Children's Hospital and Regional Medical Center - Seattle
Seattle, Washington, 98105, United States
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Childrens Hospital Los Angeles
Los Angeles, California, 90027-0700, United States
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Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, 45229-3039, United States
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Cook Children's Medical Center - Fort Worth
Fort Worth, Texas, 76104, United States
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Hospital for Sick Children
Toronto, Ontario, M5G 1X8, Canada
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Lucile Packard Children's Hospital at Stanford University Medical Center
Palo Alto, California, 94304, United States
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Morgan Stanley Children's Hospital of New York-Presbyterian
New York, New York, 10032, United States
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Texas Children's Cancer Center and Hematology Service at Texas Children's Hospital
Houston, Texas, 77030-2399, United States
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UCSF Helen Diller Family Comprehensive Cancer Center
San Francisco, California, 94115, United States
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University of Chicago Comer Children's Hospital
Chicago, Illinois, 60637, United States
More trials for these conditions
Other studies related to the condition(s) this trial covers.
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- Can a shorter chemo combo tame High-Risk neuroblastoma?
- Can a radioactive 'Smart Bomb' take down resistant childhood cancers?