NCT00053326

Brief Summary

This phase II trial is studying how well fenretinide works in treating children with recurrent or resistant neuroblastoma. Drugs used in chemotherapy use different ways to stop tumor cells from dividing so they stop growing or die.

Trial Health

80
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
70

participants targeted

Target at P50-P75 for phase_2

Geographic Reach
1 country

1 active site

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

January 27, 2003

Completed
1 day until next milestone

First Posted

Study publicly available on registry

January 28, 2003

Completed
3 months until next milestone

Study Start

First participant enrolled

May 1, 2003

Completed
2.8 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

March 1, 2006

Completed
Last Updated

October 8, 2013

Status Verified

October 1, 2013

Enrollment Period

2.8 years

First QC Date

January 27, 2003

Last Update Submit

October 7, 2013

Conditions

Recurrent Neuroblastoma

Outcome Measures

Primary Outcomes (2)

  • Response rate

    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

  • Toxicity as assessed by the National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) version 3.0

    Assessed via a descriptive tabulation of the toxicity rates, overall and by stratum.

    Up to 5 years

Secondary Outcomes (3)

  • Levels of fenretinide

    At baseline and during courses 1, 2, and 5

  • Plasma retinol levels

    At baseline and during courses 1, 2, and 5

  • Minimal residual disease (MRD) (Stratum 3)

    Up to 5 years

Study Arms (1)

Treatment (fenretinide)

EXPERIMENTAL

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.

Drug: fenretinideOther: pharmacological study

Interventions

fenretinideDRUG

Given orally

Treatment (fenretinide)
pharmacological studyOTHER

Optional correlative studies

Treatment (fenretinide)

Eligibility Criteria

AgeUp to 21 Years
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64)

You may qualify if:

  • Diagnosis of recurrent or resistant/refractory high-risk neuroblastoma by one or both of the following:
  • Histological confirmation
  • Demonstration of tumor cells in bone marrow with increased urinary catecholamines
  • Stratum I:
  • At least 1 unidimensionally measurable lesion\*
  • At least 20 mm by MRI and/or CT scan OR at least 10 mm by spiral CT scan
  • Stratum II: Meets one or both of the following criteria:
  • At least 1 site with positive uptake on meta-iodobenzylguanidine (MIBG) I 123 scan
  • Tumor in bilateral bone marrow aspirate/biopsy by routine morphology (no NSE staining only)
  • Stratum III:
  • At least 5 tumor cells/10\^6 mononuclear cells in the bone marrow by immunocytology only (on 2 successive bone marrows performed from 1 day to 4 weeks apart)
  • Patients in first response (i.e., patients with persistent tumor at end of frontline therapy, but who have never had disease relapse or progression) must have histological\* or morphological (by bone marrow) confirmation\*\* of viable tumor on CT scan, MRI, or MIBG scan after completion of myeloablative therapy (for strata I and II)
  • No catecholamine elevation only
  • Performance status - 0-2
  • At least 2 months
  • +33 more criteria

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

Children's Oncology Group

Arcadia, California, 91006-3776, United States

Location

MeSH Terms

Conditions

Neuroblastoma

Interventions

Fenretinide

Condition Hierarchy (Ancestors)

Neuroectodermal Tumors, Primitive, PeripheralNeuroectodermal Tumors, PrimitiveNeoplasms, NeuroepithelialNeuroectodermal TumorsNeoplasms, Germ Cell and EmbryonalNeoplasms by Histologic TypeNeoplasmsNeoplasms, Glandular and EpithelialNeoplasms, Nerve Tissue

Intervention Hierarchy (Ancestors)

RetinoidsCarotenoidsPolyenesAlkenesHydrocarbons, AcyclicHydrocarbonsOrganic ChemicalsCyclohexenesCyclohexanesCycloparaffinsHydrocarbons, AlicyclicHydrocarbons, CyclicTerpenesPigments, BiologicalBiological Factors

Study Officials

  • Judith Villablanca

    Children's Oncology Group

    PRINCIPAL INVESTIGATOR

Study Design

Study Type
interventional
Phase
phase 2
Allocation
NA
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
NIH
Responsible Party
SPONSOR

Study Record Dates

First Submitted

January 27, 2003

First Posted

January 28, 2003

Study Start

May 1, 2003

Primary Completion

March 1, 2006

Last Updated

October 8, 2013

Record last verified: 2013-10

Locations

US(1)