A Phase II Study of Fenretinide (NSC # 374551, IND #40294) in Children With Recurrent/Resistant High Risk Neuroblastoma
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.
Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
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).
Up to 8 courses of therapy
Children's Oncology Group
United States: Food and Drug Administration
|Children's Oncology Group||Arcadia, California 91006-3776|