Valproic Acid in Treating Young Patients With Recurrent or Refractory Solid Tumors or CNS Tumors

Not Recruiting

Trial ID: NCT00107458


RATIONALE: Drugs used in chemotherapy, such as valproic acid, work in different ways to stop the growth of tumor cells, either by killing the cells or by stopping them from dividing. Valproic acid may also stop the growth of solid tumors or CNS tumors by blocking blood flow to the tumor. PURPOSE: This phase I trial is studying the side effects and best dose of valproic acid in treating patients with recurrent or refractory solid tumors or CNS tumors.

Official Title

A Phase I Study of Valproic Acid in Children With Recurrent/Progressive Solid Tumors Including CNS Tumors

Stanford Investigator(s)



   - Histologically confirmed* malignant solid tumor, including CNS tumors, at original
   diagnosis or relapse

      - Recurrent or refractory disease NOTE: *Histologic confirmation not required for
      intrinsic brain stem or optic pathway tumors

   - Measurable or evaluable disease, defined by 1 of the following criteria:

      - Any unidimensionally measurable lesion ≥ 10 mm by standard MRI or CT scan for
      either solid or CNS tumors

      - At least 1 nonmeasurable lesion that is evaluable by nuclear medicine,
      immunocytochemistry, tumor markers, cerebrospinal fluid cytology, or other
      reliable measures

   - No known curative therapy exists

   - No documented tumor involvement in the bone marrow



   - 2 to 21

Performance status*

   - Lansky 50-100% (for patients ≤ 10 years of age)

   - Karnofsky 50-100% (for patients > 10 years of age)

Life expectancy

   - Not specified


   - Absolute neutrophil count ≥ 1,000/mm^3

   - Platelet count ≥ 100,000/mm^3 (transfusion independent)

   - Hemoglobin ≥ 8.0 g/dL (transfusions allowed)


   - Bilirubin ≤ 1.5 times upper limit of normal (ULN)

   - ALT ≤ 110 (ULN for this study is 45 U/L)

   - Albumin ≥ 2 g/dL


   - Creatinine clearance or radioisotope glomerular filtration rate ≥ 70 mL/min OR

   - Creatinine based on age as follows:

      - No greater than 0.8 mg/dL (for patients ≤ 5 years of age)

      - No greater than 1.0 mg/dL (for patients 6 to 10 years of age)

      - No greater than 1.2 mg/dL (for patients 11 to 15 years of age)

      - No greater than 1.5 mg/dL (for patients over 15 years of age)


   - Not pregnant or nursing

   - Negative pregnancy test

   - Fertile patients must use effective contraception

   - Neurologic deficits in patients with CNS tumors must be stable for ≥ 1 week before
   study entry

   - No uncontrolled infection

   - No known urea cycle disorders or other metabolic disorders

   - No other condition that would preclude study compliance


Biologic therapy

   - Recovered from prior immunotherapy

   - At least 7 days since prior hematopoietic growth factors that support platelet or WBC
   number or function

   - At least 7 days since prior antineoplastic biologic agents

   - At least 3 months since prior stem cell transplantation or rescue without total body

      - No evidence of active graft vs host disease

   - No other concurrent anticancer biologic therapy or immunotherapy


   - More than 3 weeks since prior myelosuppressive chemotherapy (6 weeks for nitrosoureas)
   and recovered

   - No other concurrent anticancer chemotherapy

Endocrine therapy

   - Patients with CNS tumors must be on a stable or decreasing dose of dexamethasone for
   the past 7 days


   - See Biologic therapy

   - Recovered from prior radiotherapy

   - At least 6 months since prior total body irradiation, craniospinal radiotherapy, or
   radiotherapy to ≥ 50% of the pelvis

   - At least 6 weeks since other prior substantial bone marrow radiotherapy

   - At least 2 weeks since prior local palliative small port radiotherapy

   - No concurrent anticancer radiotherapy


   - Not specified


   - No other concurrent investigational agents

   - No other concurrent anticancer agents

   - No other concurrent anticonvulsants

      - Patients receiving valproic acid (VPA) before study entry must have a total
      trough VPA concentration < 100 mcg/mL within the past 7 days


drug: valproic acid

Not Recruiting

Contact Information

Stanford University
School of Medicine
300 Pasteur Drive
Stanford, CA 94305
LPCH New Patient Coordinator

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