N2004-04: Fenretinide LXS in Treating Patients With Recurrent, Refractory, or Persistent Neuroblastoma
|ClinicalTrials.gov Identifier: NCT00295919|
Recruitment Status : Completed
First Posted : February 24, 2006
Last Update Posted : February 15, 2016
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.
|Condition or disease||Intervention/treatment||Phase|
|Neuroblastoma||Drug: fenretinide lipid matrix Drug: ketoconazole Other: laboratory biomarker analysis Other: pharmacological study||Phase 1|
- 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.
- 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.
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||68 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||Phase I Study of Fenretinide (4-HPR, NSC 374551) Lym-X-Sorb™ (LXS) Oral Powder in Patients With Recurrent or Resistant Neuroblastoma|
|Study Start Date :||December 2005|
|Actual Primary Completion Date :||March 2014|
|Actual Study Completion Date :||May 2015|
- Maximum tolerated dose a
- Plasma pharmacokinetics of 4-HPR/LXS oral powder and its metabolites
- Tolerability of the combination of ketoconazole and 4-HPR/LXS oral powder
- Disease response
- Plasma level of 4-HPR/LXS oral powder in PBMC as a tumor cell surrogate tissue
- Plasma levels of 4-HPR/LXS oral powder when given in combination with ketoconazole
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00295919
|United States, California|
|Childrens Hospital Los Angeles|
|Los Angeles, California, United States, 90027-0700|
|Lucile Packard Children's Hospital at Stanford University Medical Center|
|Palo Alto, California, United States, 94304|
|UCSF Helen Diller Family Comprehensive Cancer Center|
|San Francisco, California, United States, 94143|
|United States, Georgia|
|AFLAC Cancer Center and Blood Disorders Service of Children's Healthcare of Atlanta - Scottish Rite Campus|
|Atlanta, Georgia, United States, 30342|
|United States, Illinois|
|University of Chicago Comer Children's Hospital|
|Chicago, Illinois, United States, 60637|
|United States, Massachusetts|
|Children's Hospital Boston|
|Boston, Massachusetts, United States, 02115|
|United States, Michigan|
|C.S. Mott Children's Hospital at University of Michigan Medical Center|
|Ann Arbor, Michigan, United States, 48109-0286|
|United States, Ohio|
|Cincinnati Children's Hospital Medical Center|
|Cincinnati, Ohio, United States, 45229-3039|
|United States, Pennsylvania|
|Children's Hospital of Philadelphia|
|Philadelphia, Pennsylvania, United States, 19104-4318|
|United States, Texas|
|Cook Children's Medical Center - Fort Worth|
|Fort Worth, Texas, United States, 76104|
|United States, Washington|
|Children's Hospital and Regional Medical Center - Seattle|
|Seattle, Washington, United States, 98105|
|Study Chair:||Barry J. Maurer, MD, PhD||Texas Tech University Health Sciences Center|