Antineoplaston Therapy in Treating Children With Primary Malignant Brain Tumors

Study Overview
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Brief Summary
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RATIONALE: Current therapies for children with primary malignant 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 children with primary malignant brain tumors. PURPOSE: This study is being performed to determine the effects (good and bad) that Antineoplaston therapy has on children with primary malignant brain tumors.

Detailed Description
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OBJECTIVES: To determine the efficacy of Antineoplaston therapy in children with primary malignant brain tumors 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 primary malignant brain tumors. OVERVIEW: This is a single arm, open-label study in which children with primary malignant 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. After 12 months, patients with a complete or partial response or with stable disease may continue treatment. 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 Primary Malignant Brain Tumors

Conditions
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Childhood Brain Tumor

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 incurable primary malignant brain tumor that has progressed, recurred, or persisted after initial therapy (must have failed standard therapy). Evidence of residual tumor by MRI performed within two weeks prior to study entry PATIENT CHARACTERISTICS: Age: 6 months to 17 years Performance status: Karnofsky 60-100% Life expectancy: At least 2 months Hematopoietic: WBC at least 1,500/mm^3 Platelet count greater than 50,000/mm^3 Hepatic: No hepatic failure Bilirubin no greater than 2.5 mg/dL SGOT/SGPT no greater than 5 times upper limit of normal Renal: Creatinine no greater than 2.5 mg/dL Cardiovascular: No severe heart disease 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 and recovered No concurrent immunomodulating agents Chemotherapy: At least 4 weeks since prior chemotherapy (6 weeks for nitrosoureas) and recovered No concurrent antineoplastic agents Endocrine therapy: Recovered from prior endocrine therapy Concurrent corticosteroids for cerebral edema allowed Radiotherapy: At least 8 weeks since prior radiotherapy (unless clear radiological evidence of progression) and recovered Surgery: At least 4 weeks since prior surgery (unless clear radiological evidence of progression) and recovered 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 primary malignant 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; Stable Disease (SD), <50% decrease and <25% increase in the sum of the products of of the greatest perpendicular diameters of all measurable enhancing lesions, sustained for at least eight weeks; Progressive Disease (PD), >=25% increase in the sum of the products of of the greatest perpendicular diameters of all measurable enhancing lesions. | 12 months | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Percentage of Participants Who Survived | Six months and Twelve months overall survival | 6 months, 12 months | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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childhood anaplastic astrocytoma, recurrent childhood anaplastic astrocytoma, childhood glioblastoma multiforme, recurrent childhood glioblastoma multiforme, childhood brainstem glioma, recurrent childhood brainstem glioma, childhood primitive neuroectodermal tumor, recurrent childhood primitive neuroectodermal tumor