Antineoplaston Therapy in Treating Children With Visual Pathway Glioma

Study Overview
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Brief Summary
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RATIONALE: Current therapies for children with visual pathway gliomas, which are not amenable to or have not responded to standard therapy, 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 visual pathway gliomas, which are not amenable to or have not responded to standard therapy. PURPOSE: This study is being performed to determine the effects (good and bad) that Antineoplaston therapy has on children with visual pathway gliomas, which are not amenable to or have not responded to standard therapy.

Detailed Description
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OBJECTIVES: To determine the efficacy of Antineoplaston therapy in children with visual pathway gliomas, which are not amenable to or have not responded to standard therapy, 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 visual pathway gliomas, which are not amenable to or have not responded to standard therapy. OVERVIEW: This is a single arm, open-label study in which children with visual pathway gliomas, which are not amenable to or have not responded to standard therapy, 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: Approximately 20-40 patients will be accrued to this study.

Official Title
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Phase II Study of Antineoplastons A10 and AS2-1 Infusions in Children With Visual Pathway Glioma

Conditions
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Visual Pathway Glioma

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 (unless medically contraindicated) visual pathway glioma, which is not amenable to standard therapy or did not respond to standard therapy. Evidence of tumor by MRI scan performed within 2 weeks prior to the study entry Tumor must be at least 5 mm No brain stem tumors PATIENT CHARACTERISTICS: Age: 6 months to 17 years Performance status: Karnofsky 60-100% Life expectancy: At least 2 months Hematopoietic: WBC at least 2000/mm3 Platelet count greater than 50,000/mm3 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 renal insufficiency 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 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 and recovered (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 prior to study) Radiotherapy: At least 8 weeks since prior radiotherapy and recovered Surgery: Not specified Other: No prior antineoplaston therapy

Ages Eligible for Study
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Minimum Age: 6 Months
Maximum Age: 17 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 visual pathway glioma, which is not amenable to standard therapy or has not responded to standard therapy, 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 in the sum of the products of of the greatest perpendicular diameters of all measurable enhancing lesions and no Progressive Disease, sustained for at least four 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 | 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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visual pathway glioma not amenable to standard therapy, visual pathway glioma not responding to standard therapy