Antineoplaston Therapy in Treating Patients With Brain Stem Glioma

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

Detailed Description
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OBJECTIVES: To determine the efficacy of Antineoplaston therapy in patients with a brain stem glioma, 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 patients with a brain stem glioma. OVERVIEW: This is a single arm, open-label study in which patients 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 in measured utilizing MRI scans, which are performed every 8 weeks for the first 2 years, every 3 months for the 3rd and 4th years, every 6 months for the 5th and 6th 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 Patients With Brain Stem Glioma

Conditions
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Brain Stem Gliomas

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) brain stem glioma that is unlikely to respond to existing therapy and for which no curative therapy exists. Tumor must be at least 5 mm in maximum diameter PATIENT CHARACTERISTICS: Age: 6 months or greater Performance status: Karnofsky 60-100% Life expectancy: At least 2 months Hematopoietic: WBC at least 2,000/mm^3 Platelet count greater than 50,000/mm^3 Hepatic: Bilirubin no greater than 2.5 mg/dL SGOT/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 uncontrolled hypertension No history of congestive heart failure No other cardiovascular conditions that contraindicate high dosages of sodium 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 concomitant 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) 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) Radiotherapy: At least 8 weeks since prior radiotherapy Surgery: Not specified Other: No prior Antineoplaston therapy

Ages Eligible for Study
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Minimum Age: 6 Months
Maximum Age: 99 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 dosage is reached. | Drug: Antineoplaston therapy (Atengenal + Astugenal)<br>* Patients with a brain stem 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, 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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recurrent adult brain stem glioma, untreated adult brain stem glioma, untreated childhood brain stem glioma, recurrent childhood brain stem glioma