NCT00295919

N2004-04: Fenretinide LXS in Treating Patients With Recurrent, Refractory, or Persistent Neuroblastoma

Phase I Study of Fenretinide (4-HPR, NSC 374551) Lym-X-Sorb™ (LXS) Oral Powder in Patients With Recurrent or Resistant Neuroblastoma

• Status: Completed
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 80 (actual)
• Sponsor: New Approaches to Neuroblastoma Therapy Consortium · Academic / Other
• Sex: All
• Age: 0 Years – 30 Years
• Healthy volunteers: Not accepted

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.

Conditions

• Neuroblastoma

Interventions

Type	Name	Description
DRUG	fenretinide lipid matrix
DRUG	ketoconazole
OTHER	laboratory biomarker analysis
OTHER	pharmacological study

Timeline

• Start date: 2005-12-01
• Primary completion: 2014-03-01
• Completion: 2015-05-01
• First posted: 2006-02-24
• Last updated: 2026-04-14

Locations

11 sites across 1 country: United States