Clinical trial · Interventional
Valproic Acid in Treating Young Patients With Recurrent or Refractory Solid Tumors or CNS Tumors
A Phase I Study of Valproic Acid in Children With Recurrent/Progressive Solid Tumors Including CNS Tumors
- Source
- ClinicalTrials.gov
- Retrieved
- Sep 8, 2026
- Layer
- normalized (units and labels harmonized; values unchanged)
- Run
- ING-CLINICALTRIALS-20260908-000001
Summary
Brief summary (as posted)
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.
Conditions
Conditions (2)
Free-text conditions as registered, with the CancerIndex entity they were reconciled to and the match type.
| Condition (as posted) | Mapped entity | Match | Confidence |
|---|---|---|---|
| Brain and Central Nervous System Tumors | — | UNRESOLVED | — |
| Unspecified Childhood Solid Tumor, Protocol Specific | Childhood Solid Neoplasm | ALIAS | 0.85 |
Interventions
Interventions (1)
| Intervention | Type | Mapped drug | Match |
|---|---|---|---|
| valproic acid | Drug | Valproic Acid | ALIAS |
Design
Arms and outcomes
Arms (3)
- type
- EXPERIMENTAL
- label
- Treatment 1
- description
- VPA Target Trough Concentration 75-100 mcg/mL, week 1 VPA dose: 15 mg/kg/day, divided tid
- interventionNames
- Drug: valproic acid
- type
- EXPERIMENTAL
- label
- Treatment 10
- description
- VPA Target Trough Concentration 100-150 mcg/mL, week 1 VPA dose: 15 mg/kg/day, divided tid
- interventionNames
- Drug: valproic acid
- type
- EXPERIMENTAL
- label
- Treatment 20
- description
- VPA Target Trough Concentration 150-200 mcg/mL
Eligibility
Eligibility (as posted)
- Sex
- All
- Minimum age
- 2 Years
- Maximum age
- 21 Years
Show eligibility criteria text
DISEASE CHARACTERISTICS: * 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 PATIENT CHARACTERISTICS: Age * 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 Hematopoietic * Absolute neutrophil count ≥ 1,000/mm\^3 * Platelet count ≥ 100,000/mm\^3 (transfusion independent) * Hemoglobin ≥ 8.0 g/dL (transfusions allowed) Hepatic * Bilirubin ≤ 1.5 times upper limit of normal (ULN) * ALT ≤ 110 (ULN for this study is 45 U/L) * Albumin ≥ 2 g/dL Renal * 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) Other * 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 PRIOR CONCURRENT THERAPY: 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 irradiation * No evidence of active graft vs host disease * No other concurrent anticancer biologic therapy or immunotherapy Chemotherapy * 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 Radiotherapy * 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 Surgery * Not specified Other * 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
References
Publications (1)
- RESULTSu JM, Li XN, Thompson P, Ou CN, Ingle AM, Russell H, Lau CC, Adamson PC, Blaney SM. Phase 1 study of valproic acid in pediatric patients with refractory solid or CNS tumors: a children's oncology group report. Clin Cancer Res. 2011 Feb 1;17(3):589-97. doi: 10.1158/1078-0432.CCR-10-0738. Epub 2010 Nov 29. PMID 21115653