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O6-Benzylguanine-Mediated Tumor Sensitization With Chemoprotected Autologous Stem Cell in Treating Patients With Malignant Gliomas

Phase 1
Terminated
Conditions
Gliosarcoma
Glioblastoma
Interventions
Radiation: 3-Dimensional Conformal Radiation Therapy
Procedure: Autologous Hematopoietic Stem Cell Transplantation
Biological: Filgrastim
Procedure: In Vitro-Treated Peripheral Blood Stem Cell Transplantation
Radiation: Intensity-Modulated Radiation Therapy
Other: Laboratory Biomarker Analysis
Radiation: Proton Beam Radiation Therapy
Registration Number
NCT00669669
Lead Sponsor
Fred Hutchinson Cancer Center
Brief Summary

This phase I/II trial studies the side effects and best dose of temozolomide when given together with radiation therapy, carmustine, O6-benzylguanine, and patients' own stem cell (autologous) transplant in treating patients with newly diagnosed glioblastoma multiforme or gliosarcoma. Giving chemotherapy, such as temozolomide, carmustine, and O6-benzylguanine, and radiation therapy before a peripheral stem cell transplant stops the growth of cancer cells by stopping them from dividing or killing them. Giving colony-stimulating factors, such as filgrastim or plerixafor, and certain chemotherapy drugs, helps stem cells move from the bone marrow to the blood so they can be collected and stored. Chemotherapy or radiation therapy is then given to prepare the bone marrow for the stem cell transplant. The stem cells are then returned to the patient to replace the blood-forming cells that were destroyed by the chemotherapy and radiation therapy.

Detailed Description

PRIMARY OBJECTIVES:

I. Determine the safety and feasibility of infusing autologous granulocyte colony-stimulating factor (G-CSF) (filgrastim) mobilized stem cells transduced with a Phoenix-gibbon ape leukemia virus (GALV)-pseudotype vector expressing methylguanine methyltransferase (MGMT) (P140K).

II. Define the dose of BCNU (carmustine) that results in efficient engraftment of gene modified cells when given with peripheral blood stem cell support.

SECONDARY OBJECTIVES:

I. Determine the engraftment of gene-modified cells after conditioning with BCNU.

II. Determine the ability to select gene-modified cells in vivo with this regimen.

III. Evaluate the molecular and clonal composition of gene-modified cells after chemotherapy with temozolomide.

IV. Observe patients for clinical anti-tumor response.

V. Determine the correlation of the level of MGMT (P140K) marking with toxicity, temozolomide dose achieved, and response.

VI. Characterize the toxicity associated with this regimen.

OUTLINE: This is a phase I, dose-escalation study of temozolomide followed by a phase II study.

PART I: Within 35 days of surgery, patients undergo 3 dimensional (3D) conformal intensity-modulated radiation therapy (IMRT) or proton beam radiation therapy daily 5 days per week for 6 weeks. Patients receive filgrastim subcutaneously (SC) on days -7 to -3 and begin stem cell collection on the 4th day of filgrastim administration (up to 3 apheresis). Patients may also receive plerixafor SC on days -5 to -3. The CD34+ stem cells are separated from the patient's stem cells and they are transduced with Phoenix-RD114 pseudotype vector (retrovirus). One day after apheresis is completed, patients receive carmustine intravenously (IV) over 3 hours followed 2 hours later by temozolomide orally (PO). At least twenty-four hours after completion of carmustine and temozolomide, patients undergo reinfusion of genetically-modified stem cells.

PART II: Beginning approximately 4 weeks after completion of Phase 1 of the study, patients receive O6-benzylguanine IV continuously over 48 hours followed by temozolomide PO within 1 hour. Treatment may repeat at least every 28 days for a total of 24 courses in the absence of disease progression or unacceptable toxicity.

After completion of study treatment, patients are followed every 1-3 months for 2 years, every 3-6 months for 3 years, and then annually thereafter for 10 years.

Recruitment & Eligibility

Status
TERMINATED
Sex
All
Target Recruitment
12
Inclusion Criteria
  • Patients with glioblastoma multiforme or gliosarcoma
  • The patient or legal guardian must be able to comprehend the informed consent form and sign prior to patient enrollment
  • Karnofsky performance status at time of study entry must be >= 70%
  • Life expectancy of >= 3 months
  • Patients must agree to undergo repeat clinical neurological examinations and brain magnetic resonance imaging (MRI) with appropriate contrast after every other cycle of chemotherapy
  • White blood cell (WBC) > 3000/ul
  • Absolute neutrophil count (ANC) > 1500/ul
  • Platelets > 100,000/ul
  • Hemoglobin > 10 gm/100ml
  • Total and direct bilirubin < 1.5 times upper limit of laboratory normal
  • Serum glutamic oxaloacetic transaminase (SGOT) and serum glutamate pyruvate transaminase (SGPT) =< 3 times upper limit of laboratory normal
  • Alkaline phosphatase =< 3 times upper limit of laboratory normal
  • Blood urea nitrogen (BUN) < 1.5 times upper limit of laboratory normal
  • Serum creatinine < 1.5 times upper limit of laboratory normal
  • Left ventricular ejection fraction (LVEF) >= 40%, however, subjects with a LVEF in the range of 40-49% should have cardiology clearance prior to intervention
  • MGMT promoter methylation analysis of surgically resected tumor or tumor biopsy must demonstrate an unmethylated or hypomethylated MGMT promoter status
Exclusion Criteria
  • Patients with cardiac insufficiency and a LVEF of < 40%; history of coronary artery disease or arrhythmia, which has required or requires ongoing treatment
  • Patients with active pulmonary infection and/or pulse oximetry < 90% and a corrected diffusion capacity of the lung for carbon monoxide (DLCO) < 70% of predicted
  • Active systemic infection
  • Patients who are human immunodeficiency virus (HIV) positive
  • Pregnant or lactating women; a beta-human chorionic gonadotropin (HCG) level will be obtained from women of childbearing potential; fertile men and women should use effective contraception
  • Previous chemotherapy for any malignancy including temozolomide, dacarbazine (DTIC) or prior nitrosourea
  • Diabetes mellitus
  • Bleeding disorder
  • Methylated or hypermethylated MGMT promoter status within tumor tissue
  • Medical or psychiatric condition which in the opinion of the protocol chairman would compromise the patient's ability to tolerate this protocol
  • Prior interstitial radiotherapy, stereotactic or gamma knife surgery or implanted BCNU-wafers

Study & Design

Study Type
INTERVENTIONAL
Study Design
SINGLE_GROUP
Arm && Interventions
GroupInterventionDescription
Treatment (chemotherapy, autologous stem cell transplant)Laboratory Biomarker AnalysisSee Detailed Description
Treatment (chemotherapy, autologous stem cell transplant)Autologous Hematopoietic Stem Cell TransplantationSee Detailed Description
Treatment (chemotherapy, autologous stem cell transplant)3-Dimensional Conformal Radiation TherapySee Detailed Description
Treatment (chemotherapy, autologous stem cell transplant)Intensity-Modulated Radiation TherapySee Detailed Description
Treatment (chemotherapy, autologous stem cell transplant)Proton Beam Radiation TherapySee Detailed Description
Treatment (chemotherapy, autologous stem cell transplant)FilgrastimSee Detailed Description
Treatment (chemotherapy, autologous stem cell transplant)In Vitro-Treated Peripheral Blood Stem Cell TransplantationSee Detailed Description
Treatment (chemotherapy, autologous stem cell transplant)O6-BenzylguanineSee Detailed Description
Treatment (chemotherapy, autologous stem cell transplant)TemozolomideSee Detailed Description
Treatment (chemotherapy, autologous stem cell transplant)CarmustineSee Detailed Description
Treatment (chemotherapy, autologous stem cell transplant)PlerixaforSee Detailed Description
Primary Outcome Measures
NameTimeMethod
Number of Participants Dose-limiting Toxicity (DLT)Up to 6 weeks after infusion

Defined as any grade 4 nonhematopoietic toxicity that is likely related to the investigational procedures (Part I)

Number of Participants With Retrovirus or LeukemiaUp to 2 years after infusion

Replication competent retrovirus or diagnosis of leukemia

Secondary Outcome Measures
NameTimeMethod
Response RateUp to 66 months

Number of patients with reduction in tumor burden of a predefined amount

Number of Participants With ChemoprotectionUp to 66 months

assessed by the ability to increase the Temozolomide dose beyond 472 mg/m\^2

Number of Participants That SurvivedUp to 74 months

From the first day of treatment until death, assessed up to 74 months.

Time to ProgressionUp to 66 months.

From the first day of treatment (transplant) until unequivocal progression is documented, assessed up to 66 months.

Number of Participants With ChemoselectionUp to 59 months

assessed by the increase in peripheral blood Vector Copy Number (VCN), the average copies of integrated transgene per cell, after chemotherapy

Duration of ResponseUp to 65 months

From the onset of temozolomide to the date at which unequivocal disease progression, assessed up to 65 months.

Gene Transfer EfficiencyUp to 59 months

Assessed by gene marking in peripheral blood prior to chemoselection. Gene marking is assessed in whole blood by quantitative PCR and reported as a vector copy number (VCN) or the average copies of integrated transgene per cell. The units here will be reported as copies/cell.

Gene Transfer Efficiency After ChemotherapyUp to 59 months

Assessed by gene marking in peripheral blood after chemoselection. Gene marking is assessed in whole blood by quantitative PCR and reported as a vector copy number (VCN) or the average copies of integrated transgene per cell. The units here will be reported as copies/cell.

Trial Locations

Locations (1)

Fred Hutch/University of Washington Cancer Consortium

🇺🇸

Seattle, Washington, United States

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