NCT01319838

Brief Summary

Neuroblastoma is a cancer of the nervous system and accounts for 15% of cancer related deaths in children. With the advancement of treatment therapies, the long term survival rate has progressed to approximately 50%. The therapy used for treatment, however, is very toxic and associated with serious long-term side effects. Treatment for neuroblastoma typically includes chemotherapy, surgery, stem cell transplantation, radiation therapy, and immunotherapy. At the end of this treatment, children with neuroblastoma commonly take the drug isotretinoin for 6 months. Isotretinoin maintains the response to previous treatments and helps turn the remaining cancer cells into normal nerve cells. Most patients often respond to this treatment at first but are at a high-risk for the cancer coming back. The majority of the children who relapse after treatment or develop recurrent disease do so in the first two years following the completion of therapy and there are no current treatments to cure those who relapse. This study will explore whether or not extending the therapy with isotretinoin from 6 months to 24 months will help prevent the cancer from coming back without causing severe side effects.

Trial Health

30
At Risk

Trial Health Score

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

Trial has exceeded expected completion date
Timeline
Completed

Started Mar 2011

Typical duration for phase_1

Geographic Reach
1 country

1 active site

Status
withdrawn

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

Study Start

First participant enrolled

March 1, 2011

Completed
17 days until next milestone

First Submitted

Initial submission to the registry

March 18, 2011

Completed
4 days until next milestone

First Posted

Study publicly available on registry

March 22, 2011

Completed
2.7 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

December 1, 2013

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

December 1, 2013

Completed
Last Updated

December 10, 2013

Status Verified

December 1, 2013

Enrollment Period

2.8 years

First QC Date

March 18, 2011

Last Update Submit

December 9, 2013

Conditions

Neuroblastoma

Keywords

neuroblastomaisotretinoin

Outcome Measures

Primary Outcomes (2)

  • Progression free survival

    To determine the progression free survival in patients with high risk neuroblastoma who receive a prolonged course of biologic therapy with isotretinoin

    5 years after treatment completed

  • Isotretinoin toxicity

    To determine the toxicity and tolerability of a prolonged course of isotretinoin biologic therapy

    5 years after treatment completed

Secondary Outcomes (3)

  • Bone growth effect

    5 years after treatment completed

  • Isotretinoin pharmacokinetic profile

    1 year after treatment completed

  • Neurologic or psychologic sequelae

    1 year after treatment completed

Study Arms (1)

isotretinoin prolongation

EXPERIMENTAL

Prolonged treatment with isotretinoin, extending standard 6 month duration to 2 years

Drug: Isotretinoin

Interventions

IsotretinoinDRUG

\>12 kg: 160 mg/m2/day, given PO, divided BID \<=12 kg: 5.33 mg/kg/day, given PO, dividied BID doses given days 1-14 of 28 day cycle for 24 consecutive cycles

isotretinoin prolongation

Eligibility Criteria

AgeUp to 30 Years
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64)

You may qualify if:

  • \<=30 years of age
  • histologic verification of neuroblastoma
  • no active measurable disease on CT/MRI
  • ultra high risk status by having mixed response, no response or stable disease following initial treatment or by having recurrent neuroblastoma
  • Karnofsky \>=50% for patients \>16 years and Lansky \>=50% for patients \<=16 years
  • patients must have completed high risk therapy
  • organ function as defined in protocol

You may not qualify if:

  • patients with active measurable disease
  • patients who are pregnant or breast-feeding
  • concomitant medications stopped as indicated in protocol
  • patients with uncontrolled infection
  • patients with history of depression or psychotic disorder requiring medication

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

Children's Healthcare of Atlanta

Atlanta, Georgia, 30322, United States

Location

Related Links

MeSH Terms

Conditions

Neuroblastoma

Interventions

Isotretinoin

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

  • Howard Katzenstein, MD

    Children's Healthcare of Atlanta/Emory University

    PRINCIPAL INVESTIGATOR
0

Study Design

Study Type
interventional
Phase
phase 1
Allocation
NA
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
OTHER
Responsible Party
PRINCIPAL INVESTIGATOR
PI Title
Principal Investigator

Study Record Dates

First Submitted

March 18, 2011

First Posted

March 22, 2011

Study Start

March 1, 2011

Primary Completion

December 1, 2013

Study Completion

December 1, 2013

Last Updated

December 10, 2013

Record last verified: 2013-12

Locations

US(1)