Antineoplaston Therapy in Treating Children With Rhabdoid Tumor of the Central Nervous System

Overview

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

  • 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

Detailed Description

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.

Interventions

  • 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

Primary 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.

Secondary Measures

  • 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

DISEASE CHARACTERISTICS:

  • 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

Investigator Details

  • Lead Sponsor
    • Burzynski Research Institute
  • Provider of Information About this Clinical Study
    • Sponsor
  • Overall Official(s)
    • Stanislaw R. Burzynski, MD, PhD, Principal Investigator, Burzynski Research Institute

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