Antineoplaston Therapy in Treating Children With Brain Tumors

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

RATIONALE: Current therapies for childhood 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 childhood brain 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 brain tumors.

Study Overview

• Status: Completed
• Conditions: Childhood Brain Tumors
• Intervention / Treatment: Drug: Antineoplaston therapy (Atengenal + Astugenal)

Detailed Description

OVERVIEW: This is a single arm, open-label study in which children with 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.

OBJECTIVES:

• To determine the efficacy of Antineoplaston therapy in children with a brain 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 brain 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.

• Study Type: Interventional
• Enrollment (Actual): 34
• Phase: Phase 2

Contacts and Locations

Study Locations

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

Participation Criteria

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 (except if medically contraindicated) childhood brain tumor 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 greater than 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 severe heart disease
• No history of heart failure
• No history of other cardiovascular conditions that contraindicate high dosages of sodium
• 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
• 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 stable dose for at least 1 week before study entry)

Radiation therapy:

• At least 8 weeks since prior radiotherapy and recovered

Surgery:

• Recovered from any prior surgery

Other:

• No prior antineoplaston therapy

Study Plan

How is the study designed?

Design Details

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

Number of Arms

1

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.	Drug: Antineoplaston therapy (Atengenal + Astugenal); Children with a 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	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

Collaborators and Investigators

• Sponsor: Burzynski Research Institute

Publications and helpful links

General Publications

• Stanislaw R. Burzynski, Tomasz J. Janicki, Gregory S. Burzynski, Ania Marszalek. A Phase II Study of Antineoplastons A10 and AS2-1 in Children with Brain Tumors. Final Report (Protocol BT-10). Journal of Cancer Therapy 8: 173-187, 2017

Helpful Links

• Burzynski Research Institute
• Burzynski Clinic

Study record dates

Study Major Dates

• Study Start: September 1, 1996
• Primary Completion (Actual): September 1, 2014
• Study Completion (Actual): September 1, 2014

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): March 7, 2022
• Last Update Submitted That Met QC Criteria: March 3, 2022
• Last Verified: March 1, 2022

More Information

Terms related to this study

• Keywords: Anaplastic astrocytoma; Gliosarcoma; Glioblastoma multiforme; Astrocytoma; Craniopharyngioma; Oligodendroglioma; Brain stem glioma; Ganglioglioma; Medulloepithelioma; Anaplastic oligodendroglioma; Neurocytoma; Primitive neuroectodermal tumor; Anaplastic glioma; Astrocytoma pilocytic; Oligodendroglioma-brainstem
• Additional Relevant MeSH Terms: Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Neoplasms; Neoplasms by Site; Central Nervous System Neoplasms; Nervous System Neoplasms; Brain Neoplasms
• Other Study ID Numbers: CDR0000066490; BC-BT-10 (Other Identifier: Burzynski Research Institute, Inc.)

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: No