Antineoplaston Therapy in Treating Children With Recurrent or Refractory High-Grade Glioma

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

Detailed Description
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OBJECTIVES: To determine the efficacy of Antineoplaston therapy in children with recurrent/progressive high grade gliomas, 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 recurrent/progressive high grade gliomas. OVERVIEW: This is a single arm, open-label study in which children with recurrent/progressive high grade gliomas 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 for this study.

Official Title
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Phase II Study of Antineoplastons A10 and AS2-1 in Children With High Grade Glioma

Conditions
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High Grade Glioma

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


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Histologically confirmed high-grade glioma (glioblastoma multiforme or anaplastic astrocytoma) that is recurrent or progressive or with residual tumor after standard therapy, including radiotherapy Measurable tumor by MRI scan performed within two weeks prior to study entry Male or female patients Children 6 months to 17 years Performance status: Karnofsky 60-100% Life expectancy of at least 2 months WBC greater than 1,500/mm^3 Platelet count greater than 50,000/mm^3 No evidence of hepatic or renal insufficiency and a total bilirubin and serum creatinine no greater than 2.5 mg/dL and SGOT/SGPT no greater than 5 times upper limit of normal Must have recovered from adverse effect of previous therapy At least 8 weeks elapsed since last dose of radiation At least 4 weeks elapsed since last dose of chemotherapy (6 weeks for nitrosoureas) Corticosteroids permitted using the smallest dose that is compatible with preservation of optimal neurologic function Acceptable methods of birth control (in females of child-bearing potential or in sexually active males)during and up to four weeks following completion of study Exclusion Criteria: Prior A10 and AS2-1 treatment Severe heart disease Uncontrolled hypertension Lung disease Hepatic failure Serious active infections, fever or other serious concurrent disease that would interfere with the evaluation of the treatment drug. Pregnant or nursing Serious concurrent disease Concurrent antineoplastic or immunomodulatory agents

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 recurrent/progressive high grade glioma 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 overall survival | 6 months, 12 months, 24 months | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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childhood anaplastic astrocytoma, childhood glioblastoma multiforme