RATIONALE: Current therapies for childhood Rhabdoid 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 Rhabdoid 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 Rhabdoid tumors.
Full Title of Study: “Phase II Study of Antineoplastons A10 and AS2-1 in Children With Rhabdoid Tumor of the Central Nervous System”
- Study Type: Interventional
- Study Design
- Allocation: N/A
- Intervention Model: Single Group Assignment
- Primary Purpose: Treatment
- Masking: None (Open Label)
- Study Primary Completion Date: May 2003
OVERVIEW: This is a single arm, open-label study in which children with Rhabdoid 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 Rhabdoid 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 Rhabdoid 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.
- Drug: Antineoplaston therapy (Atengenal + Astugenal)
- Children with a Rhabdoid tumor will receive Antineoplaston therapy (Atengenal + Astugenal).
Arms, Groups and Cohorts
- Experimental: Antineoplaston therapy
- 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.
Clinical Trial Outcome Measures
- Number of Participants With Objective Response
- Time Frame: 12 months
- 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.
- Percentage of Participants Who Survived
- Time Frame: 6 months, 12 months, 24 months
- 6 months, 12 months, 24 months overall survival
Participating in This Clinical Trial
- Histologically confirmed Rhabdoid tumor of the central nervous system that is unlikely to respond to existing therapy and 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 at least 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 uncontrolled hypertension – No severe heart disease – No history of congestive heart failure – No history of 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 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: – Concurrent corticosteroids for cerebral edema allowed (must be on a stable dose for at least 1 week before study entry) Radiotherapy: – At least 8 weeks since prior radiotherapy and recovered Surgery: – Not specified Other: – No prior antineoplaston treatment
Gender Eligibility: All
Minimum Age: 6 Months
Maximum Age: 17 Years
Are Healthy Volunteers Accepted: No
- Lead Sponsor
- Burzynski Research Institute
- Provider of Information About this Clinical Study
- Overall Official(s)
- Stanislaw R. Burzynski, MD, PhD, Principal Investigator, Burzynski Research Institute
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