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Antineoplaston Therapy in Treating Patients With Brain Stem Glioma

This study has been completed.
Information provided by (Responsible Party):
Burzynski Research Institute Identifier:
First received: November 1, 1999
Last updated: August 18, 2016
Last verified: August 2016

RATIONALE: Current therapies for a brain stem glioma provide very limited benefit to the patient. The anti-cancer properties of Antineoplaston therapy suggest that it may prove beneficial in the treatment of brain stem gliomas.

PURPOSE: This study is being performed to determine the effects (good and bad) that Antineoplaston therapy has on children (> 6 months of age) and adults with newly-diagnosed or recurrent brain stem gliomas.

Condition Intervention Phase
Brain Stem Gliomas
Drug: Antineoplaston therapy (Atengenal + Astugenal)
Phase 2

Study Type: Interventional
Study Design: Endpoint Classification: Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: Phase II Study of Antineoplastons A10 and AS2-1 In Patients With Brain Stem Glioma

Resource links provided by NLM:

Further study details as provided by Burzynski Research Institute:

Primary Outcome Measures:
  • Number of Participants With Objective Response [ Time Frame: 12 months ] [ Designated as safety issue: No ]
    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 Outcome Measures:
  • Percentage of Participants Who Survived [ Time Frame: 6 months, 12 months, 24 months, 36 months, 48 months, 60 months ] [ Designated as safety issue: No ]
    6 months, 12 months, 24 months, 36 months, 48 months, 60 months overall survival

Enrollment: 40
Study Start Date: March 1996
Study Completion Date: September 2007
Primary Completion Date: September 2007 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
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 dosage is reached.
Drug: Antineoplaston therapy (Atengenal + Astugenal)
Patients with a brain stem glioma will receive Antineoplaston therapy (Atengenal + Astugenal)
Other Name: A10 (Atengenal); AS2-1 (Astugenal)

Detailed Description:


  • To determine the efficacy of Antineoplaston therapy in patients with a brain stem glioma, 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 patients with a brain stem glioma.

OVERVIEW: This is a single arm, open-label study in which patients 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 in measured utilizing MRI scans, which are performed every 8 weeks for the first 2 years, every 3 months for the 3rd and 4th years, every 6 months for the 5th and 6th years, and annually thereafter.

PROJECTED ACCRUAL: A total of 20-40 patients will be accrued for this study.


Ages Eligible for Study:   6 Months to 99 Years   (Child, Adult, Senior)
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No


  • Histologically confirmed (except if medically contraindicated) brain stem glioma that is unlikely to respond to existing therapy and for which no curative therapy exists.
  • Tumor must be at least 5 mm in maximum diameter



  • 6 months or greater

Performance status:

  • Karnofsky 60-100%

Life expectancy:

  • At least 2 months


  • WBC at least 2,000/mm^3
  • Platelet count greater than 50,000/mm^3


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


  • Creatinine no greater than 2.5 mg/dL
  • No history of renal conditions that contraindicate high dosages of sodium


  • No severe heart disease
  • No uncontrolled hypertension
  • No history of congestive heart failure
  • No other cardiovascular conditions that contraindicate high dosages of sodium


  • No severe lung disease


  • 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


Biologic therapy:

  • At least 4 weeks since prior immunotherapy
  • No concurrent immunomodulating agents


  • At least 4 weeks since prior chemotherapy (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 before study entry)


  • At least 8 weeks since prior radiotherapy


  • Not specified


  • No prior Antineoplaston therapy
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its identifier: NCT00003459

United States, Texas
Burzynski Clinic
Houston, Texas, United States, 77055-6330
Sponsors and Collaborators
Burzynski Research Institute
Principal Investigator: Stanislaw R. Burzynski, MD, PhD Burzynski Research Institute
  More Information

Additional Information:
Burzynski, S.R., Janicki, J., Burzynski, G., Marszalek, A. A Phase II Study of Antineoplastons A10 and AS2-1 in Patients with Brainstem Gliomas. The Report on Non-Diffuse Intrinsic Pontine Glioma (Protocol BT-11). Journal of Cancer Therapy, 6: 334-344, 2015

Responsible Party: Burzynski Research Institute Identifier: NCT00003459     History of Changes
Other Study ID Numbers: CDR0000066491  BC-BT-11 
Study First Received: November 1, 1999
Results First Received: May 25, 2016
Last Updated: August 18, 2016
Health Authority: United States: Food and Drug Administration
Individual Participant Data  
Plan to Share IPD: No

Keywords provided by Burzynski Research Institute:
recurrent adult brain stem glioma
untreated adult brain stem glioma
untreated childhood brain stem glioma
recurrent childhood brain stem glioma

Additional relevant MeSH terms:
Neoplasms, Neuroepithelial
Neuroectodermal Tumors
Neoplasms, Germ Cell and Embryonal
Neoplasms by Histologic Type
Neoplasms, Glandular and Epithelial
Neoplasms, Nerve Tissue processed this record on October 25, 2016