This site became the new on June 19th. Learn more.
Show more Menu IMPORTANT: Listing of a study on this site does not reflect endorsement by the National Institutes of Health. Talk with a trusted healthcare professional before volunteering for a study. Read more... Menu IMPORTANT: Talk with a trusted healthcare professional before volunteering for a study. Read more... Menu
Give us feedback

Valproic Acid in Childhood Progressive Brain Tumors

This study has been withdrawn prior to enrollment.
(Feasibility of the trial was proven to be absent.)
Information provided by (Responsible Party):
Amy-Lee Bredlau, Medical University of South Carolina Identifier:
First received: May 22, 2013
Last updated: May 21, 2015
Last verified: May 2015
The study investigates valproic acid added to radiation and temozolomide therapy (standard of care) for progressive or recurrent pediatric brain tumors.

Condition Intervention Phase
Pediatric Brain Tumor Glioma Anaplastic Astrocytoma Medulloblastoma Glioblastoma Drug: Valproic Acid Phase 1

Study Type: Interventional
Study Design: Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Valproic Acid for Children With Recurrent and Progressive Brain Tumors

Resource links provided by NLM:

Further study details as provided by Amy-Lee Bredlau, Medical University of South Carolina:

Primary Outcome Measures:
  • Completion of protocol [ Time Frame: 2 months ]
    Number of participants completing the protocol will be measured.

Secondary Outcome Measures:
  • Time to progression [ Time Frame: 2 months ]
    Participants will be evaluated for progression every 2 months while on the study.

Enrollment: 0
Study Start Date: May 2013
Study Completion Date: December 2013
Primary Completion Date: December 2013 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Treatment arm
All participants will be enrolled on to one, open-label arm. Participants will be treated with valproic acid in addition to standard of care therapy.
Drug: Valproic Acid
All participants enrolled on valproic acid arm.
Other Name: Valproate, VPA, Depakote, Depakote ER, Depakene

Detailed Description:
Patients with progressive or recurrent pediatric brain tumors are administered valproic acid, an HDAC inhibitor, along with standard of care therapy (radiation and temozolomide) for induction therapy. Thereafter, patients will be able to continue on valproic acid and temozolomide therapy as long as the combination is well tolerated and the tumor is not progressing.

Ages Eligible for Study:   1 Year and older   (Child, Adult, Senior)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  • Subject, parent, or guardian willing and able to give informed consent
  • Recurrent or progressive pediatric brain tumor, with MRI evidence of disease
  • Age at first diagnosis of brain tumor 1-21 years old
  • Lansky or Karnofsky performance score of at least 50 at diagnosis

Exclusion Criteria:

  • Pregnancy
  • Prior intolerance to valproic acid
  • History of use of temozolomide
  • Use of enzyme inducing anticonvulsant medications (see appendix B)
  • Known urea cycle disorder (e.g. ornithine transcarbamylase deficiency)
  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: NCT01861990

United States, South Carolina
Medical University of South Carolina
Charleston, South Carolina, United States, 29425
Sponsors and Collaborators
Medical University of South Carolina
Principal Investigator: Amy L Bredlau, MD Medical University of South Carolina
  More Information

Responsible Party: Amy-Lee Bredlau, Director, Pediatric Brain Tumor Program, Medical University of South Carolina Identifier: NCT01861990     History of Changes
Other Study ID Numbers: VA2013
IRG-97-219-14 ( Other Grant/Funding Number: American Cancer Society Institutional Research Grant )
Study First Received: May 22, 2013
Last Updated: May 21, 2015

Keywords provided by Amy-Lee Bredlau, Medical University of South Carolina:
Brain tumor

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
Neuroectodermal Tumors, Primitive
Valproic Acid
Enzyme Inhibitors
Molecular Mechanisms of Pharmacological Action
GABA Agents
Neurotransmitter Agents
Physiological Effects of Drugs
Antimanic Agents
Tranquilizing Agents
Central Nervous System Depressants
Psychotropic Drugs processed this record on September 21, 2017