Expanded Access Study of Fenretinide Lym-X-Sorb Plus Ketoconazole in Neuroblastoma
1 other identifier
expanded_access
N/A
1 country
3
Brief Summary
Currently there is no known effective treatment for recurrent/resistant neuroblastoma. Fenretinide is an anticancer agent that may work differently than standard chemotherapy medicines. It may cause the buildup of wax-like substances in neuroblastoma cancer cells, called "ceramides" or other chemicals, called 'reactive oxygen species'. In laboratory studies it was found that if too much ceramide or reactive oxygen species build up in neuroblastoma cells, they may die. In addition, researchers are testing to see if a drug called ketoconazole, commonly used to treat fungus infections, can increase fenretinide levels in the body by interfering with the body's ability to break down fenretinide. This study is being done: 1) to allow patients with recurrent/refractory neuroblastoma patients who would otherwise not be able to access fenretinide/LXS oral powder for treatment to do so; 2) to further describe the side effects of fenretinide and ketoconazole when given by mouth for seven days every three weeks; 3) to determine if a patient's tumor gets smaller after treatment with fenretinide oral powder plus ketoconazole or fenretinide oral powder alone.
Trial Health
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Trial Relationships
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Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
February 27, 2014
CompletedFirst Posted
Study publicly available on registry
March 3, 2014
CompletedMarch 21, 2022
March 1, 2022
February 27, 2014
March 17, 2022
Conditions
Keywords
Interventions
Fenretinide Lym-X-Sorb 1500 mg/m2/day, daily for 7 days every 3 weeks
Ketoconazole 6 mg/kg/day, daily for 7 days every 3 weeks.
Eligibility Criteria
You may qualify if:
- diagnosis of neuroblastoma either by histologic verification and/or demonstration of tumor cells in the bone marrow with increased urinary catecholamines.
- must have high risk neuroblastoma with one of the following: 1) recurrent/progressive disease at any time, 2) refractory disease, 3) persistent disease after at least a partial response to frontline therapy, or 4) Second or greater complete remission after definitive disease progression.
- must have at least one of the following sites of disease: 1) measurable tumor on MRI, CT scan, or X-Ray; 2)MIBG scan with positive uptake in at least one site; 3) bone marrow with tumor cells seen on routine morphology.
- must have an ECOG performance status of 0, 1, or 2
- must have a life expectancy of greater than or equal to 8 weeks
- must have fully recovered from the acute toxic effects of all prior chemotherapy, immunotherapy, or radiotherapy
- hemoglobin greater than or equal to 8.0 (may transfuse to achieve this level)
- ANC greater than or equal to 500 (must be at least 7 days after last dose of growth factor)
- platelet count greater than or equal to 50,000 (must be transfusion independent, defined as at least 1 week since last platelet transfusion)
- age-adjusted serum creatinine less than or equal to 1.5 times normal for age
- normal cardiac function documented by: ejection fraction (greater than or equal to 55%) documented by echocardiogram or radionuclide MUGA evaluation OR fractional shortening (greater than or equal to 27%) documented by echocardiogram AND EKG must demonstrate no abnormality severe enough to justify cardiac medications AND baseline QTc interval greater than or equal to 450 msecs
- total bilirubin less than or equal to 1.5 times normal for age
- ALT and AST less than or equal to 3 times normal for age (for this study, the upper limit of normal of ALT is defined as 45 U/L)
- normal prothrombin time (PT) for age
- baseline hepatitis titers without evidence of acute/active hepatitis. Patients will need to have a negative Hep B Surface Antigen (HBsAg), Hep B e Antigen (HBeAg), Anti-Hep B core Antibody IgM (Anti-HBc IgM), Anti-HAV IgM, and Anti-HCV IgM.
- +9 more criteria
You may not qualify if:
- Pregnancy or breast feeding. Due to the potential teratogenic effects of retinoids, pregnant women are NOT eligible. Breast milk feeding by study patient is NOT allowed.
- Patients with history of organ and allogeneic stem cell transplantation.
- Patients with a known history of allergy to soy products.
- Patients with a known history of a severe allergy or sensitivity of wheat gluten.
- Patients requiring anti-arrhythmia cardiac medications are NOT eligible.
- Prior therapy with fenretinide, or fenretinide + ketoconazole, if DLT's were experienced.
- A known history of intolerance of ketoconazole.
- Patients on other essential medications for which an interaction with ketoconazole can be expected and for which dose reductions to other essential medications cannot be made in a manner adequate to ensure patient safety.
- Patients who, in the opinion of the investigator, may not be able to comply with safety monitoring requirements of the study.
- Active hepatitis.
- Baseline cardiac QTc interval \>450 msecs.
- Eligible for enrollment on other national or regional treatment protocols employing fenretinide/LXS oral power that are reasonably accessible to the patient.
- Patient must NOT receive other anti-cancer agents while on Study.
- Ceftriaxone (Rocephin®) is NOT permitted for 24 hours prior to the start of the oral fenretinide course, during the course, and for 24 hours after the completion of seven day fenretinide course due to concerns of possible adverse effects on the hepatic clearance of fenretinide. Alternative antibiotics should be used. Other cephalosporins are permitted.
- Acetaminophen (Tylenol®) is NOT permitted for 24 hours prior the start of the oral fenretinide course, during the course, and for 48 hours following the completion of the seven day fenretinide course due to concerns of possible hepatic interactions. Ibuprofen (Motrin®) should be used for antipyretic control during this time period.
- +11 more criteria
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (3)
University of Oklahoma Health Science Center
Oklahoma City, Oklahoma, 79104, United States
UT Southwestern Medical Center
Dallas, Texas, 75235, United States
Cook Children's Hospital
Fort Worth, Texas, 76104, United States
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Intervention Hierarchy (Ancestors)
Study Design
- Study Type
- expanded access
- Sponsor Type
- NETWORK
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
February 27, 2014
First Posted
March 3, 2014
Last Updated
March 21, 2022
Record last verified: 2022-03