NCT00005796

Brief Summary

RATIONALE: Drugs used in chemotherapy use different ways to stop tumor cells from dividing so they stop growing or die. Combining more than one drug may kill more tumor cells. Inserting a specific gene into a person's peripheral stem cells may improve the body's ability to fight cancer or make the cancer more sensitive to chemotherapy. PURPOSE: Phase I trial to study the effectiveness of combination chemotherapy plus gene therapy in treating patients who have CNS tumors.

Trial Health

87
On Track

Trial Health Score

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

Enrollment
10

participants targeted

Target at below P25 for phase_1

Timeline
Completed

Started Feb 2000

Longer than P75 for phase_1

Geographic Reach
1 country

2 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

February 1, 2000

Completed
4 months until next milestone

First Submitted

Initial submission to the registry

June 2, 2000

Completed
3 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

June 1, 2003

Completed
11 months until next milestone

First Posted

Study publicly available on registry

April 28, 2004

Completed
7.6 years until next milestone

Study Completion

Last participant's last visit for all outcomes

December 1, 2011

Completed
Last Updated

March 25, 2015

Status Verified

March 1, 2015

Enrollment Period

3.3 years

First QC Date

June 2, 2000

Last Update Submit

March 23, 2015

Conditions

Bone Marrow SuppressionBrain and Central Nervous System TumorsDrug/Agent Toxicity by Tissue/Organ

Keywords

recurrent adult brain tumoradult brain stem gliomaadult ependymomaadult medulloblastomaadult glioblastomachildhood high-grade cerebral astrocytomachildhood oligodendrogliomaadult anaplastic astrocytomaadult anaplastic oligodendrogliomaadult mixed gliomadrug/agent toxicity by tissue/organbone marrow suppressionuntreated childhood brain stem gliomarecurrent childhood brain stem gliomauntreated childhood cerebellar astrocytomarecurrent childhood cerebellar astrocytomarecurrent childhood cerebral astrocytomarecurrent childhood medulloblastomanewly diagnosed childhood ependymomarecurrent childhood ependymomaadult giant cell glioblastomaadult gliosarcoma

Outcome Measures

Primary Outcomes (1)

  • Determine the toxicity (detection of replication competent retrovirus) associated with CD34+ cells transduced with a retroviral vector expressing human O6-methylguanine DNA methyltransferase in adult and pediatric patients with poor prognosis CNS tumors.

    Year 2

Study Arms (1)

Single arm

EXPERIMENTAL

PCV therapy

Procedure: filgrastimBiological: gene therapyDrug: lomustineDrug: procarbazine hydrochlorideDrug: vincristine sulfateProcedure: in vitro-treated peripheral blood stem cell transplantation

Interventions

filgrastimPROCEDURE

GCSF is given after chemo administration

Single arm
gene therapyBIOLOGICAL

stem cells are collected and given back to the patients after chemotherapy adminstration

Single arm
lomustineDRUG

chemotherapy is administered every 21 days

Single arm
procarbazine hydrochlorideDRUG

chemotherapy is administered every 21 days.

Single arm
vincristine sulfateDRUG

chemotherapy is administered every 21 days

Single arm
in vitro-treated peripheral blood stem cell transplantationPROCEDURE

stem cells are reinfused after chemotherapy administration

Single arm

Eligibility Criteria

Age5 Years+
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)
DISEASE CHARACTERISTICS: Histologically proven newly diagnosed CNS tumors Eligible tumor types: Glioblastoma multiforme (WHO grade IV) Anaplastic astrocytoma (WHO grade III) Anaplastic oligodendroglioma (WHO grade III) Mixed anaplastic oligoastrocytoma (WHO grade III) Incompletely resected ependymoma Diffusely intrinsic pontine or medullary glioma Histology requirement waived OR Histologically proven recurrent or progressive CNS tumors Eligible tumor types: Same as above plus oligodendroglioma (WHO grade II) No brainstem tumors arising from the cervicomedullary region or midbrain without histologic proof of malignancy No supratentorial low grade astrocytomas (WHO grade I or II) PATIENT CHARACTERISTICS: Age: 5 and over Performance status: ECOG 0-2 Life expectancy: At least 2 months Hematopoietic: Absolute neutrophil count greater than 1,000/mm3 Platelet count greater than 100,000/mm3 (transfusion independent) Hemoglobin greater than 10 g/dL at time of pulmonary function testing Hepatic: Bilirubin less than 1.2 mg/dL SGOT or SGPT less than 3 times normal Renal: Creatinine less than 1.5 mg/dL OR Creatinine clearance or radioisotope GFR greater than 70 mL/min Pulmonary: FEV1, FVC, and/or DLCO greater than 60% predicted Children who are uncooperative with pulmonary function tests must have the following: No evidence of dyspnea at rest No exercise intolerance Oxygen saturation (by pulse oximetry) greater than 94% on room air Other: Minimum weight of 10 kg Not pregnant or nursing No active infection PRIOR CONCURRENT THERAPY: Biologic therapy: No growth factors after completion of study chemotherapy Chemotherapy: No prior nitrosourea or procarbazine Endocrine therapy: No concurrent dexamethasone as antiemetic Radiotherapy: No prior craniospinal radiotherapy Surgery: Not specified

Contact the study team to discuss eligibility requirements. They can help determine if this study is right for you.

Sponsors & Collaborators

Study Sites (2)

Indiana University Cancer Center

Indianapolis, Indiana, 46202-5265, United States

Location

Dana-Farber Cancer Institute

Boston, Massachusetts, 02115, United States

Location

MeSH Terms

Conditions

Central Nervous System NeoplasmsDrug-Related Side Effects and Adverse ReactionsBrain NeoplasmsEpendymomaMedulloblastomaGlioblastomaAstrocytomaOligodendrogliomaGliomaFamilial ependymomaGliosarcoma

Interventions

FilgrastimGenetic TherapyLomustineProcarbazineVincristine

Condition Hierarchy (Ancestors)

Nervous System NeoplasmsNeoplasms by SiteNeoplasmsNervous System DiseasesChemically-Induced DisordersBrain DiseasesCentral Nervous System DiseasesNeoplasms, NeuroepithelialNeuroectodermal TumorsNeoplasms, Germ Cell and EmbryonalNeoplasms by Histologic TypeNeoplasms, Glandular and EpithelialNeoplasms, Nerve TissueNeuroectodermal Tumors, Primitive

Intervention Hierarchy (Ancestors)

Granulocyte Colony-Stimulating FactorColony-Stimulating FactorsGlycoproteinsGlycoconjugatesCarbohydratesHematopoietic Cell Growth FactorsCytokinesIntercellular Signaling Peptides and ProteinsPeptidesAmino Acids, Peptides, and ProteinsProteinsBiological FactorsBiological TherapyTherapeuticsGenetic EngineeringGenetic TechniquesInvestigative TechniquesNitrosourea CompoundsUreaAmidesOrganic ChemicalsNitroso CompoundsBenzamidesBenzoatesAcids, CarbocyclicCarboxylic AcidsBenzene DerivativesHydrocarbons, AromaticHydrocarbons, CyclicHydrocarbonsVinca AlkaloidsSecologanin Tryptamine AlkaloidsIndole AlkaloidsAlkaloidsHeterocyclic CompoundsIndolesHeterocyclic Compounds, 2-RingHeterocyclic Compounds, Fused-RingIndolizidinesIndolizines

Study Officials

  • James Croop, MD, PhD

    Riley's Children Cancer Center at Riley Hospital for Children

    STUDY CHAIR

Study Design

Study Type
interventional
Phase
phase 1
Allocation
NA
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
OTHER
Responsible Party
SPONSOR

Study Record Dates

First Submitted

June 2, 2000

First Posted

April 28, 2004

Study Start

February 1, 2000

Primary Completion

June 1, 2003

Study Completion

December 1, 2011

Last Updated

March 25, 2015

Record last verified: 2015-03

Locations

US(2)