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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 currently recruiting participants. (see Contacts and Locations)
Verified November 2013 by Advantagene, Inc.
Children's Hospital Boston
Information provided by (Responsible Party):
Advantagene, Inc. Identifier:
First received: March 5, 2008
Last updated: November 19, 2013
Last verified: November 2013

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: Endpoint Classification: Safety Study
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 ] [ Designated as safety issue: Yes ]

Secondary Outcome Measures:
  • Overall survival [ Time Frame: 5 years ] [ Designated as safety issue: Yes ]
  • Progression-free survival [ Time Frame: 5 years ] [ Designated as safety issue: Yes ]
  • Objective tumor response [ Time Frame: 5 years ] [ Designated as safety issue: Yes ]
  • Immunologic function [ Time Frame: 3 months ] [ Designated as safety issue: No ]

Estimated Enrollment: 12
Study Start Date: April 2010
Estimated Primary Completion Date: December 2014 (Final data collection date for primary outcome measure)
Intervention Details:
    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
Genders Eligible for Study:   Both
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
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its identifier: NCT00634231

Contact: Lianne Greenspan, MS 617-632-6740

United States, Illinois
Ann & Robert H. Lurie Children's Hospital of Chicago (Formerly Children's Memorial Hospital) Recruiting
Chicago, Illinois, United States, 60611
Contact: Kelly Verel, BS,CCRP    312-227-4858   
Contact: Stewart Goldman, MD    312 227-4874   
Principal Investigator: Stewart Goldman, MD         
United States, Massachusetts
Dana-Farber Cancer Institute Recruiting
Boston, Massachusetts, United States, 02115
Contact: Mark W Kieran, MD, PhD    617-632-4907   
Contact: Lianne Greenspan, MS    617-632-6740   
Principal Investigator: Mark W Kieran, MD, PhD         
Sponsors and Collaborators
Advantagene, Inc.
Children's Hospital Boston
Study Chair: Mark W Kieran, MD, PhD Dana-Farber Cancer Institute
  More Information

No publications provided

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: November 19, 2013
Health Authority: United States: Food and Drug Administration

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

Additional relevant MeSH terms:
Brain Neoplasms
Brain Diseases
Central Nervous System Diseases
Central Nervous System Neoplasms
Neoplasms by Histologic Type
Neoplasms by Site
Neoplasms, Germ Cell and Embryonal
Neoplasms, Glandular and Epithelial
Neoplasms, Nerve Tissue
Neoplasms, Neuroepithelial
Nervous System Diseases
Nervous System Neoplasms
Neuroectodermal Tumors
Anti-Infective Agents
Antiviral Agents
Pharmacologic Actions
Therapeutic Uses processed this record on November 20, 2014