Antineoplaston Therapy in Treating Children With Brain Tumors

Study Overview
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Brief Summary
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RATIONALE: Current therapies for childhood brain tumors provide limited benefit to the patient. The anti-cancer properties of Antineoplaston therapy suggest that it may prove beneficial in the treatment of childhood brain tumors. PURPOSE: This study is being performed to determine the effects (good and bad) that Antineoplaston therapy has on children (> 6 months of age) with brain tumors.

Detailed Description
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OVERVIEW: This is a single arm, open-label study in which children with brain tumors receive gradually escalating doses of intravenous Antineoplaston therapy (Atengenal + Astugenal) until the maximum tolerated dose is reached. Treatment continues for at least 12 months in the absence of disease progression or unacceptable toxicity. OBJECTIVES: To determine the efficacy of Antineoplaston therapy in children with a brain tumor, as measured by an objective response to therapy (complete response, partial response or stable disease). To determine the safety and tolerance of Antineoplaston therapy in children with a brain tumor. To determine objective response, tumor size is measured utilizing MRI scans, which are performed every 8 weeks for the first two years, every 3 months for the third and fourth years, every 6 months for the 5th and sixth years, and annually thereafter. PROJECTED ACCRUAL: A total of 20-40 patients will be accrued to this study.

Official Title
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Phase II Study of Antineoplastons A10 and AS2-1 in Children With Brain Tumors

Conditions
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Childhood Brain Tumors

Intervention / Treatment
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* Drug: Antineoplaston therapy (Atengenal + Astugenal)


Participation Criteria
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Eligibility Criteria
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DISEASE CHARACTERISTICS: Histologically confirmed (except if medically contraindicated) childhood brain tumor for which no curative therapy exists Measurable tumor by MRI scan performed within two weeks prior to study entry Tumor must be at least 5 mm PATIENT CHARACTERISTICS: Age: 6 months to 17 years Performance status: Karnofsky 60-100% Life expectancy: At least 2 months Hematopoietic: WBC greater than 2000/mm^3 Platelet count greater than 50,000/mm^3 Hepatic: Bilirubin no greater than 2.5 mg/dL SGOT and SGPT no greater than 5 times upper limit of normal No hepatic failure Renal: Creatinine no greater than 2.5 mg/dL No history of renal conditions that contraindicate high dosages of sodium Cardiovascular: No severe heart disease No history of heart failure No history of other cardiovascular conditions that contraindicate high dosages of sodium No uncontrolled hypertension Pulmonary: No severe lung disease Other: Not pregnant or nursing Fertile patients must use effective contraception during and for 4 weeks after study participation No serious active infections or fever No other serious concurrent disease PRIOR CONCURRENT THERAPY: Biologic therapy: At least 4 weeks since prior immunotherapy No concurrent immunomodulating agents Chemotherapy: At least 4 weeks since prior chemotherapy (6 weeks for nitrosoureas) and recovered No concurrent antineoplastic agents Endocrine therapy: Concurrent corticosteroids for cerebral edema allowed (must be on stable dose for at least 1 week before study entry) Radiation therapy: At least 8 weeks since prior radiotherapy and recovered Surgery: Recovered from any prior surgery Other: No prior antineoplaston therapy

Ages Eligible for Study
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Minimum Age: 6 Months
Maximum Age: 18 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Antineoplaston therapy<br>Antineoplaston therapy (Atengenal + Astugenal) by IV infusion every four hours for at least 12 months. Study subjects receive increasing dosages of Atengenal and Astugenal until the maximum tolerated dose is reached. | Drug: Antineoplaston therapy (Atengenal + Astugenal)<br>* Children with a brain tumor will receive Antineoplaston therapy (Atengenal + Astugenal).<br>* Other names: A10 (Atengenal); AS2-1 (Astugenal);|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Number of Participants With Objective Response | Objective response rate per Response Assessment in Neuro-Oncology (RANO) for target lesions and assessed by MRI: Complete Response (CR), disappearance of all disease sustained for at least four weeks; Partial Response (PR), >=50% decrease in the sum of the products of of the greatest perpendicular diameters of all measurable enhancing lesions, sustained for at least four weeks. | 12 months | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Percentage of Participants Who Survived | 6 months, 12 months, 24 months, 36 months, 48 months, 60 months overall survival | 6 months, 12 months, 24 months, 36 months, 48 months, 60 months | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Anaplastic astrocytoma, Anaplastic glioma, Anaplastic oligodendroglioma, Astrocytoma, Astrocytoma pilocytic, Brain stem glioma, Craniopharyngioma, Ganglioglioma, Glioblastoma multiforme, Gliosarcoma, Medulloepithelioma, Neurocytoma, Oligodendroglioma, Oligodendroglioma-brainstem, Primitive neuroectodermal tumor