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A Phase I Study of AdV-tk + Prodrug Therapy in Combination With Radiation Therapy for Pediatric Brain Tumors

This study is ongoing, but not recruiting participants.
Boston Children’s Hospital
Information provided by (Responsible Party):
Advantagene, Inc. Identifier:
First received: March 5, 2008
Last updated: December 30, 2016
Last verified: December 2016
This study will evaluate the administration of AdV-tk followed by valacyclovir in children with malignant glioma, including glioblastoma multiforme (GBM) and anaplastic astrocytoma (AA), as well as recurrent ependymomas in combination with radiation therapy. The primary objective is to determine if this approach is safe and can be effectively delivered without disturbing standard therapy.

Condition Intervention Phase
Malignant Glioma
Recurrent Ependymoma
Biological: AdV-tk
Drug: valacyclovir
Radiation: Radiation
Phase 1

Study Type: Interventional
Study Design: Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: A Phase I Study of AdV-tk + Prodrug Therapy in Combination With Radiation Therapy for Pediatric Brain Tumors

Resource links provided by NLM:

Further study details as provided by Advantagene, Inc.:

Primary Outcome Measures:
  • Safety based on standard laboratory and clinical adverse event monitoring [ Time Frame: 2 months ]

Secondary Outcome Measures:
  • Overall survival [ Time Frame: 5 years ]
  • Progression-free survival [ Time Frame: 5 years ]
  • Objective tumor response [ Time Frame: 5 years ]
  • Immunologic function [ Time Frame: 3 months ]

Estimated Enrollment: 12
Study Start Date: April 2010
Estimated Study Completion Date: December 2020
Primary Completion Date: December 2015 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: AdV-tk
AdV-tk + valacyclovir in combination with standard of care radiation
Biological: AdV-tk Drug: valacyclovir
Other Name: Prodrug
Radiation: Radiation
Other Name: Radiation therapy

Detailed Description:
This is an Open label, Phase I, dose escalation study. Patients will receive an injection of AdV-tk into the tumor or tumor bed during the surgical procedure followed by 14 days of anti-herpetic prodrug, valacyclovir, starting 1-3 days after vector injection. Standard radiotherapy will begin 3-7 days after AdV-tK injection. Standard chemotherapy may begin after completion of valacyclovir at the discretion of the treating physician and family. Two dose levels of AdV-tk will be evaluated with a fixed dose of valacyclovir.

Ages Eligible for Study:   3 Years to 21 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  • Patients must be 3 years of age or older
  • Patients must be planning to undergo standard of care treatment with surgery and radiation therapy.
  • Patients must have malignant glioma or recurrent ependymoma
  • Tumor must be accessible for injection and must not be located in the brainstem or deep midbrain
  • Performance Score: Karnofsky ≥60% if >10y/o, Lansky ≥60 if ≤10y/o
  • Bone Marrow Function: Patients must have adequate bone marrow function defined as a peripheral absolute neutrophil count ≥ 1000/µl, platelet count ≥ 100,000/µl (transfusion independent) and hemoglobin ≥ 8.0 gm/dL
  • Renal Function: Patients must have serum creatinine ≤ 1.5 times upper limit of institutional normal for age and/or GFR ≥ 70 mL/min/1.73 m2.
  • Hepatic Function: Bilirubin ≤ 1.5 times institutional normal; SGPT (ALT) < 3 times institutional normal
  • Serum electrolyte values (sodium, potassium, magnesium, calcium) must be checked prior to enrollment and clinically significant abnormalities corrected prior to surgery/AdV-tk injection
  • Patients with seizure disorder may be enrolled if well controlled
  • Signed informed consent according to institutional guidelines must be obtained

Exclusion Criteria:

  • Prior or ongoing liver disease including known cirrhosis, hepatitis B or C infection but not to exclude patients with a distant history of resolved hepatitis A infection
  • Patients on immunosuppressive drugs (with exception of corticosteroid)
  • Known history of HIV or underlying immunodeficiency
  • Patients with acute infections (viral, bacterial or fungal infections requiring therapy)
  • Pregnant or breast-feeding patients. Female patients of childbearing age must have negative serum or urine pregnancy test within 1 week of beginning therapy
  • Other serious co-morbid illness or compromised organ function
  • No other investigational anti-tumor agents within 30 days prior to study entry or during active participation in the study (defined as from study entry until tumor progression)
  Contacts and Locations
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Please refer to this study by its identifier: NCT00634231

United States, Illinois
Ann & Robert H. Lurie Children's Hospital of Chicago (Formerly Children's Memorial Hospital)
Chicago, Illinois, United States, 60611
United States, Massachusetts
Dana-Farber Cancer Institute
Boston, Massachusetts, United States, 02115
Sponsors and Collaborators
Advantagene, Inc.
Boston Children’s Hospital
Study Chair: Mark W Kieran, MD, PhD Dana-Farber Cancer Institute
  More Information

Responsible Party: Advantagene, Inc. Identifier: NCT00634231     History of Changes
Obsolete Identifiers: NCT00729105
Other Study ID Numbers: 07-098
Study First Received: March 5, 2008
Last Updated: December 30, 2016

Keywords provided by Advantagene, Inc.:
Malignant glioma
Glioblastoma multiforme
Anaplastic astrocytoma
Recurrent ependymoma
Gene therapy

Additional relevant MeSH terms:
Brain Neoplasms
Neoplasms, Neuroepithelial
Neuroectodermal Tumors
Neoplasms, Germ Cell and Embryonal
Neoplasms by Histologic Type
Neoplasms, Glandular and Epithelial
Neoplasms, Nerve Tissue
Central Nervous System Neoplasms
Nervous System Neoplasms
Neoplasms by Site
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Antiviral Agents
Anti-Infective Agents processed this record on April 27, 2017