N2004-04: Fenretinide LXS in Treating Patients With Recurrent, Refractory, or Persistent Neuroblastoma
- Conditions
- Neuroblastoma
- Interventions
- Other: laboratory biomarker analysisOther: pharmacological study
- Registration Number
- NCT00295919
- Lead Sponsor
- Children's Hospital Los Angeles
- Brief Summary
RATIONALE: Drugs used in chemotherapy, such as fenretinide LXS, 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 fenretinide LXS in treating patients with recurrent, refractory, or persistent neuroblastoma.
- Detailed Description
OBJECTIVES:
Primary
* Determine the maximum tolerated dose of fenretinide (4-HPR) Lym-X-Sorb™ (LXS) oral powder (4-HPR/LXS oral powder) in patients with recurrent, refractory, or persistent neuroblastoma.
* Define the toxicities of 4-HPR/LXS oral powder in these patients.
* Determine the plasma pharmacokinetics of 4-HPR/LXS oral powder and its metabolites in these patients.
* Determine the tolerability of the combination of ketoconazole and 4-HPR/LXS oral powder in these patients.
Secondary
* Determine the response rate in patients treated with 4-HPR/LXS oral powder.
* Determine the level of 4-HPR/LXS oral powder in normal peripheral blood mononuclear cells (PBMC) as a tumor cell surrogate tissue.
* Determine plasma levels of 4-HPR/LXS oral powder when given in combination with ketoconazole.
* Determine whether ketoconazole increases 4-HPR/LXS oral powder plasma levels.
OUTLINE: This is a dose-escalation study of fenretinide (4-HPR) Lym-X-Sorb™ (LXS) oral powder, followed by an open-label study. Patients are sequentially assigned to 1 of 2 intervention groups.
* Group I: Patients receive 4-HPR/LXS oral powder 3 times daily on days 0-6.
* Group II: Patients receive 4-HPR/LXS oral powder as in group I and oral ketoconazole once daily on days 0-6.
In both groups, treatment repeats every 21 days for at least 6 courses in the absence of disease progression or unacceptable toxicity. Patients in complete remission at study enrollment may receive up to 12 courses (9 months) of therapy.
Blood samples are collected at baseline and during courses 1, 2, and 6 for pharmacokinetic and correlative studies.
After completion of study treatment, patients are followed periodically.
PROJECTED ACCRUAL: A total of 32 patients will be accrued for the dose-escalation portion and 36 will be accrued for the open-label portion of this study.
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- All
- Target Recruitment
- 80
Not provided
Not provided
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SINGLE_GROUP
- Arm && Interventions
Group Intervention Description Single Group fenretinide lipid matrix - Single Group pharmacological study - Single Group laboratory biomarker analysis - Single Group ketoconazole -
- Primary Outcome Measures
Name Time Method Toxicity of HPR/LXS oral powder Day 1 of therapy to 16 days after last day of therapy Plasma pharmacokinetics of 4-HPR/LXS oral powder and its metabolites Day 0 of protocol therapy through Day 6 of Course 6 Maximum tolerated dose Day 1 of therapy to 16 days after last day of therapy Tolerability of the combination of ketoconazole and 4-HPR/LXS oral powder Day 1 of therapy to 16 days after last day of therapy
- Secondary Outcome Measures
Name Time Method
Trial Locations
- Locations (11)
Childrens Hospital Los Angeles
🇺🇸Los Angeles, California, United States
Children's Hospital of Philadelphia
🇺🇸Philadelphia, Pennsylvania, United States
Children's Hospital Boston
🇺🇸Boston, Massachusetts, United States
Children's Hospital and Regional Medical Center - Seattle
🇺🇸Seattle, Washington, United States
Lucile Packard Children's Hospital at Stanford University Medical Center
🇺🇸Palo Alto, California, United States
UCSF Helen Diller Family Comprehensive Cancer Center
🇺🇸San Francisco, California, United States
AFLAC Cancer Center and Blood Disorders Service of Children's Healthcare of Atlanta - Scottish Rite Campus
🇺🇸Atlanta, Georgia, United States
University of Chicago Comer Children's Hospital
🇺🇸Chicago, Illinois, United States
C.S. Mott Children's Hospital at University of Michigan Medical Center
🇺🇸Ann Arbor, Michigan, United States
Cincinnati Children's Hospital Medical Center
🇺🇸Cincinnati, Ohio, United States
Cook Children's Medical Center - Fort Worth
🇺🇸Fort Worth, Texas, United States