NCT00528437

Brief Summary

The purpose of this study is to: Find out how safe and effective (by monitoring the good and/or bad effects) treatment with high dose temozolomide, thiotepa and carboplatin with stem cell rescue followed by 13-cis-retinoic acid has on children and adolescents with recurrent/refractory brain tumors Find out how the body uses 13-cis-retinoic acid by studying the your blood levels and proteins in the blood that break down the 13-cis-retinoic acid Determine how well 13-cis-retinoic acid penetrates into the spinal fluid.

Trial Health

90
On Track

Trial Health Score

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

Enrollment
46

participants targeted

Target at P25-P50 for phase_2

Timeline
Completed

Started Oct 2005

Longer than P75 for phase_2

Geographic Reach
2 countries

17 active sites

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

Study Start

First participant enrolled

October 1, 2005

Completed
1.9 years until next milestone

First Submitted

Initial submission to the registry

September 10, 2007

Completed
2 days until next milestone

First Posted

Study publicly available on registry

September 12, 2007

Completed
9.8 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

June 30, 2017

Completed
6 months until next milestone

Study Completion

Last participant's last visit for all outcomes

December 30, 2017

Completed
3.5 years until next milestone

Results Posted

Study results publicly available

July 8, 2021

Completed
Last Updated

July 8, 2021

Status Verified

June 1, 2021

Enrollment Period

11.8 years

First QC Date

September 10, 2007

Results QC Date

May 7, 2021

Last Update Submit

June 17, 2021

Conditions

Brain Tumors

Keywords

Recurrent or refractory medulloblastoma/PNETCNS germ cell tumorsEpendymomasAT/RTHigh grade gliomaOther malignant brain tumors

Outcome Measures

Primary Outcomes (2)

  • Event-free Survival (EFS)

    EFS will be reported in patients with recurrent or refractory medulloblastoma/ primitive neuroectodermal tumors. EFS is defined as the length of time after primary treatment for a cancer ends that the patient remains free of certain complications or events that the treatment was intended to prevent or delay.

    Day +42

  • Overall Survival (OS)

    OS will be reported in patients with recurrent or refractory medulloblastoma/ primitive neuroectodermal tumors. OS is defined as the length of time from the start of treatment that patients diagnosed with disease are still alive.

    Day +77

Secondary Outcomes (1)

  • Toxicity of of 13-cis-retinoic Acid

    One year

Study Arms (1)

Myeloablative Chemo-Temozolomide, Thiotepa, and Carboplatin.

OTHER
Drug: temozolomide, thiotepa, carboplatin, 13-cis-retinoic acid

Interventions

temozolomide, thiotepa, carboplatin, 13-cis-retinoic acidDRUG

13-cis-retinoic acid, when absorbed, may be subject to first-pass metabolism and subsequent plasma (and tumor) concentrations will depend on the rate of metabolism to the inactive 4-oxo metabolite.

Myeloablative Chemo-Temozolomide, Thiotepa, and Carboplatin.

Eligibility Criteria

Age6 Months - 21 Years
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64)

You may qualify if:

  • Patients with recurrent or refractory medulloblastoma/PNET, CNS germ cell tumors, ependymomas, AT/RT, high grade glioma and other malignant brain tumors. Brainstem gliomas are eligible if residual disease is \< 1.5cc and if the patient is off decadron.
  • Patients must have recurrent or refractory disease following at least one prior course of therapy and must have minimal residual disease defined as \< 1.5 cm2 of enhancement. Patients with + CSF cytology, linear or fine nodular leptomeningeal disease are eligible.
  • Adequate hematologic, renal, liver, and cardiac function as demonstrated by laboratory values performed within 21 days, inclusive, prior to administration of temozolomide.
  • Patients must have an adequate number of autologous stem cells available defined as a minimum of 2 x 106 CD 34+ cells/kg and preferably at least 5 x 106 CD 34+ cells/kg.

You may not qualify if:

  • Previous myeloablative therapy
  • Frequent vomiting or medical condition that could interfere with oral medication intake (e.g., partial bowel obstruction)
  • Previous or concurrent malignancies at other sites with the exception of surgically cured carcinoma in-situ of the cervix and basal or squamous cell carcinoma of the skin. Patients with prior malignancies which have not required anti-tumor treatment within the preceding 24 months are eligible.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (17)

Phoenix Children's Hospital

Phoenix, Arizona, 85016, United States

Location

Emory University

Atlanta, Georgia, 30322, United States

Location

Hawaii Pacific Health

Lihue, Hawaii, 96766, United States

Location

Children's Memorial Hospital

Chicago, Illinois, 60614, United States

Location

Riley Hospital for Children

Indianapolis, Indiana, 46202, United States

Location

Children's Hospital and Clinics of Minnesota

Minneapolis, Minnesota, 55404, United States

Location

Roswell Park Cancer Institute

Buffalo, New York, 14263, United States

Location

Steven and Alexandra Cohen Children's Medical Center of New York- North Shore LIJ

New Hyde Park, New York, 11040, United States

Location

NYU Hassenfeld Center

New York, New York, 10016, United States

Location

Nationwide Children's Hospital

Columbus, Ohio, 43205, United States

Location

Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, 19104, United States

Location

Medical Univ. of South Carolina

Charleston, South Carolina, 29425, United States

Location

Vanderbilt Univ.

Nashville, Tennessee, 37240, United States

Location

Children's Medical Center of Dallas

Dallas, Texas, 75235, United States

Location

MD Anderson Cancer Center (MDACC)

Houston, Texas, 77030, United States

Location

Virginia Commonwealth Univ.

Richmond, Virginia, 23284, United States

Location

Princess Margaret Hospital

Toronto, Ontario, M5G 2M9, Canada

Location

MeSH Terms

Conditions

Brain NeoplasmsRecurrenceEpendymomaGlioma

Interventions

TemozolomideThiotepaCarboplatinIsotretinoin

Condition Hierarchy (Ancestors)

Central Nervous System NeoplasmsNervous System NeoplasmsNeoplasms by SiteNeoplasmsBrain DiseasesCentral Nervous System DiseasesNervous System DiseasesDisease AttributesPathologic ProcessesPathological Conditions, Signs and SymptomsNeoplasms, NeuroepithelialNeuroectodermal TumorsNeoplasms, Germ Cell and EmbryonalNeoplasms by Histologic TypeNeoplasms, Glandular and EpithelialNeoplasms, Nerve Tissue

Intervention Hierarchy (Ancestors)

DacarbazineTriazenesOrganic ChemicalsImidazolesAzolesHeterocyclic Compounds, 1-RingHeterocyclic CompoundsPhosphoramidesOrganophosphorus CompoundsTriethylenephosphoramideAziridinesAzirinesCoordination ComplexesRetinoidsCarotenoidsPolyenesAlkenesHydrocarbons, AcyclicHydrocarbonsCyclohexenesCyclohexanesCycloparaffinsHydrocarbons, AlicyclicHydrocarbons, CyclicTerpenesPigments, BiologicalBiological Factors

Results Point of Contact

Title
Dr. Sharon Gardner
Organization
NYU Langone
Sharon.Gardner@nyulangone.org
212-263-9913

Study Officials

  • Sharon L Gardner, MD

    NYU Langone Health

    PRINCIPAL INVESTIGATOR

Publication Agreements

PI is Sponsor Employee
Yes

Study Design

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

Study Record Dates

First Submitted

September 10, 2007

First Posted

September 12, 2007

Study Start

October 1, 2005

Primary Completion

June 30, 2017

Study Completion

December 30, 2017

Last Updated

July 8, 2021

Results First Posted

July 8, 2021

Record last verified: 2021-06

Locations

US(16)CA(1)