Antineoplaston Therapy in Treating Children With Primary Malignant Brain Tumors

July 24, 2017 updated by: Burzynski Research Institute

Phase II Study of Antineoplastons A10 and AS2-1 in Children With Primary Malignant Brain Tumors

RATIONALE: Current therapies for children with primary malignant brain tumors 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 primary malignant brain tumors.

PURPOSE: This study is being performed to determine the effects (good and bad) that Antineoplaston therapy has on children with primary malignant brain tumors.

Study Overview

Status

Terminated

Detailed Description

OBJECTIVES:

  • To determine the efficacy of Antineoplaston therapy in children with primary malignant brain tumors 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 primary malignant brain tumors.

OVERVIEW: This is a single arm, open-label study in which children with primary malignant brain 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. 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 to this study

Study Type

Interventional

Enrollment (Actual)

8

Phase

  • Phase 2

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

    • Texas
      • Houston, Texas, United States, 77055-6330
        • Burzynski Clinic

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

6 months to 18 years (Child, Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

DISEASE CHARACTERISTICS:

  • Histologically confirmed incurable primary malignant brain tumor that has progressed, recurred, or persisted after initial therapy (must have failed standard therapy).
  • Evidence of residual tumor by MRI performed within two weeks prior to study entry

PATIENT CHARACTERISTICS:

Age:

  • 6 months to 17 years

Performance status:

  • Karnofsky 60-100%

Life expectancy:

  • At least 2 months

Hematopoietic:

  • WBC at least 1,500/mm^3
  • Platelet count greater than 50,000/mm^3

Hepatic:

  • No hepatic failure
  • Bilirubin no greater than 2.5 mg/dL
  • SGOT/SGPT no greater than 5 times upper limit of normal

Renal:

  • Creatinine no greater than 2.5 mg/dL

Cardiovascular:

  • No severe heart disease
  • No uncontrolled hypertension

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 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 (6 weeks for nitrosoureas) and recovered
  • No concurrent antineoplastic agents

Endocrine therapy:

  • Recovered from prior endocrine therapy
  • Concurrent corticosteroids for cerebral edema allowed

Radiotherapy:

  • At least 8 weeks since prior radiotherapy (unless clear radiological evidence of progression) and recovered

Surgery:

  • At least 4 weeks since prior surgery (unless clear radiological evidence of progression) and recovered

Other:

  • No prior antineoplaston therapy

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
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.
Children with a primary malignant brain tumor will receive Antineoplaston therapy (Atengenal + Astugenal).
Other Names:
  • A10 (Atengenal); AS2-1 (Astugenal)

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
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; Stable Disease (SD), <50% decrease and <25% increase in the sum of the products of of the greatest perpendicular diameters of all measurable enhancing lesions, sustained for at least eight 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
Time Frame: 6 months, 12 months
Six months and Twelve months overall survival
6 months, 12 months

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

  • Burzynski SR, Janicki TJ, Burzynski GS, Marszalek A, Brookman, S. A phase II study of Antineoplastons A10 and AS2-1 in children with recurrent, refractory or progressive primary brain tumors - Final Report (Protocol BT-22). Journal of Cancer Therapy, 5:977-988, 2014.

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

March 1, 1996

Primary Completion (Actual)

January 1, 2012

Study Completion (Actual)

January 1, 2012

Study Registration Dates

First Submitted

November 1, 1999

First Submitted That Met QC Criteria

January 26, 2003

First Posted (Estimate)

January 27, 2003

Study Record Updates

Last Update Posted (Actual)

August 24, 2017

Last Update Submitted That Met QC Criteria

July 24, 2017

Last Verified

July 1, 2017

More Information

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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