Valproic Acid in Childhood Progressive Brain Tumors

Medical University of South Carolina (MUSC)

Status and phase: Withdrawn; Phase 1

Conditions: Glioblastoma; Glioma; Pediatric Brain Tumor; Medulloblastoma; Anaplastic Astrocytoma

Treatments: Drug: Valproic Acid

Study type: Interventional

Funder types: Other

Identifiers

NCT01861990

VA2013

IRG-97-219-14 (Other Grant/Funding Number)

Details and patient eligibility

About

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

Full 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.

Sex: All

Ages: 1+ year old

Volunteers: No Healthy Volunteers

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)

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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0 participants in 1 patient group

Treatment arm

Experimental group

Description:

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.

Treatment:

Drug: Valproic Acid