Fenretinide in Treating Children With Recurrent or Resistant Neuroblastoma
Other: pharmacological study
|Study Design:||Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
|Official Title:||A Phase II Study of Fenretinide (NSC# 374551, IND# 40294) in Children With Recurrent/Resistant High Risk Neuroblastoma|
- Response rate [ Time Frame: Up to 8 courses of therapy ] [ Designated as safety issue: No ]A responder is defined to be a patient who achieves a best overall response of complete response (CR), very good partial response (VGPR) or partial response (PR).
- Toxicity as assessed by the National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) version 3.0 [ Time Frame: Up to 5 years ] [ Designated as safety issue: Yes ]Assessed via a descriptive tabulation of the toxicity rates, overall and by stratum.
- Levels of fenretinide [ Time Frame: At baseline and during courses 1, 2, and 5 ] [ Designated as safety issue: No ]Assessed via descriptive analysis of the steady state levels of fenretinide overall and by stratum
- Plasma retinol levels [ Time Frame: At baseline and during courses 1, 2, and 5 ] [ Designated as safety issue: No ]Assessed via descriptive analysis of the plasma retinol levels overall and by stratum.
- Minimal residual disease (MRD) (Stratum 3) [ Time Frame: Up to 5 years ] [ Designated as safety issue: No ]Assessed by descriptive calculation of the proportion of responders.
|Study Start Date:||May 2003|
|Primary Completion Date:||March 2006 (Final data collection date for primary outcome measure)|
Experimental: Treatment (fenretinide)
Patients receive oral fenretinide 3 times daily (or 2 times daily if over 18 years of age) on days 1-7. Treatment repeats every 3 weeks for up to 30 courses in the absence of disease progression or unacceptable toxicity.
Given orallyOther: pharmacological study
Optional correlative studies
Determine the response rate in pediatric patients with recurrent or resistant high-risk neuroblastoma treated with fenretinide.
Determine the toxic effects of this drug in these patients. Determine the proportion of patients with disease detected only by bone marrow immunocytology, who clear all evidence of disease during treatment with this drug.
Determine minimal residual disease response by marrow and meta-iodobenzylguanidine (MIBG) I 123 scan in patients treated with this drug.
OUTLINE: Patients are stratified according to presence of measurable disease on CT scan/MRI (yes vs no). A third stratum of patients with tumor cells in bone marrow by immunocytology only is enrolled but is not evaluated for response.
Patients receive oral fenretinide 3 times daily (or 2 times daily if over 18 years of age) on days 1-7. Treatment repeats every 3 weeks for up to 30 courses in the absence of disease progression or unacceptable toxicity. Patients in stratum III who fail to achieve a complete response after 8 courses of therapy are removed from study.
Patients are followed monthly until blood counts and visual acuity are stable or normalized and then every 6 months for 2 years and annually for 3 years.
PROJECTED ACCRUAL: A total of 70 patients (25 each for strata I and II, 20 for stratum III) will be accrued for this study within 1-2 years.
Please refer to this study by its ClinicalTrials.gov identifier: NCT00053326
|United States, California|
|Children's Oncology Group|
|Arcadia, California, United States, 91006-3776|
|Principal Investigator:||Judith Villablanca||Children's Oncology Group|