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Valproic Acid in Childhood Progressive Brain Tumors

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT01861990
Recruitment Status : Withdrawn (Feasibility of the trial was proven to be absent.)
First Posted : May 24, 2013
Last Update Posted : May 22, 2015
Information provided by (Responsible Party):
Amy-Lee Bredlau, Medical University of South Carolina

Brief Summary:
The study investigates valproic acid added to radiation and temozolomide therapy (standard of care) for progressive or recurrent pediatric brain tumors.

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

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.

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 0 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Valproic Acid for Children With Recurrent and Progressive Brain Tumors
Study Start Date : May 2013
Actual Primary Completion Date : December 2013
Actual Study Completion Date : December 2013

Arm Intervention/treatment
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

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

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

Information from the National Library of Medicine

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, Learn About Clinical Studies.

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Ages Eligible for Study:   1 Year and older   (Child, Adult, Older Adult)
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)

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT01861990

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

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Responsible Party: Amy-Lee Bredlau, Director, Pediatric Brain Tumor Program, Medical University of South Carolina Identifier: NCT01861990    
Other Study ID Numbers: VA2013
IRG-97-219-14 ( Other Grant/Funding Number: American Cancer Society Institutional Research Grant )
First Posted: May 24, 2013    Key Record Dates
Last Update Posted: May 22, 2015
Last Verified: May 2015
Keywords provided by Amy-Lee Bredlau, Medical University of South Carolina:
Brain tumor
Additional relevant MeSH terms:
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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