Study of Antineoplaston Therapy + Radiation vs. Radiation Only in Diffuse, Intrinsic, Brainstem Glioma

NCT02887040 | Not Yet Recruiting Phase 3 DMC FDA Drug

• 1 other identifier: BRI-BT-52
• Study Type: interventional
• Target: 92
• Locations: 0 countries
• Sites: N/A

Brief Summary

Patients ≥ 3 years of age with newly-diagnosed, diffuse, intrinsic pontine glioma will be enrolled in this study. However, the primary objectives of this study are to 1) compare overall survival, the time from randomization to death from any cause, for study subjects 3-21 years of age with newly-diagnosed, diffuse, intrinsic pontine glioma who receive Antineoplaston therapy (Atengenal + Astugenal) + radiation therapy vs. radiation therapy alone and 2) describe the toxicity profile (all subjects) for Antineoplaston therapy + radiation therapy vs. radiation therapy alone. A secondary objective is to compare progression-free survival for study subjects 3-21 years of age with newly-diagnosed, diffuse, intrinsic pontine glioma treated with Antineoplaston therapy + radiation therapy vs. radiation therapy alone.

Conditions

Brain Stem Glioma

Keywords

Diffuse, intrinsic brain stem glioma; Antineoplaston therapy; Radiation

Outcome Measures

Primary Outcomes (1)

• Change in Percentage of Participants Who Survived (Overall Survival)

  The Kaplan-Meier nonparametric method is used to evaluate overall survival in the two therapy groups. Survival difference is evaluated using a log-rank test, which compares the two therapy groups. A Cox proportional hazards model is used as a supportive analysis to assess the magnitude of difference between the two therapy groups. The median survival rate in the two therapy groups (and 95% confidence intervals) is evaluated. Hazard ratio and its 95% confidence interval are estimated.

  6 months, 12 months, 24 months, 36 months, 48 months, 60 months

Secondary Outcomes (1)

• Change in Percentage of Participants Who Survived (Progression-free Survival)

  6 months, 12 months, 24 months, 36 months, 48 months, 60 months

Study Arms (2)

Radiation

EXPERIMENTAL

Study subjects receive a single daily radiation fraction of 180cGy, 5 days a week for 6 weeks overall, to a total radiation dose of 5400cGy.

Radiation: Radiation

Antineoplaston therapy + Radiation

EXPERIMENTAL

Antineoplaston therapy (Atengenal + Astugenal) by IV infusion every four hours for 104 weeks. Study subjects receive increasing dosages of Atengenal and Astugenal until the maximum tolerated dose is reached. Study subjects also receive a single daily radiation fraction of 180cGy, 5 days a week for 6 weeks overall, to a total radiation dose of 5400cGy.

Radiation: Radiation; Drug: Atengenal; Drug: Astugenal

Interventions

Radiation RADIATION

Subjects ≥ 3 years of age with a diffuse, intrinsic brain stem glioma will receive radiation.

Antineoplaston therapy + Radiation; Radiation

Atengenal DRUG

Subjects ≥ 3 years of age with a diffuse, intrinsic brain stem glioma will receive Atengenal in combination with Astugenal (Antineoplaston therapy) and radiation

Also known as: A10

Antineoplaston therapy + Radiation

Astugenal DRUG

Subjects ≥ 3 years of age with a diffuse, intrinsic brain stem glioma will receive Astugenal in combination with Atengenal (Antineoplaston therapy) and radiation

Also known as: AS2-1

Antineoplaston therapy + Radiation

Eligibility Criteria

• Age: 3 Years - 99 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• Subjects with Diffuse, Intrinsic Pontine Glioma as defined by the following criteria are eligible:
• A characteristic MRI appearance, including variable contrast enhancement after gadolinium administration, diffuse T2/FLAIR signal, and involvement of more than 50% of the pons.
• Confirmation of anaplastic glioma (i.e., oligodendroglioma, astrocytoma, oligoastrocytoma) or GBM histology if there is less than 50% involvement of the pons.
• Screening evaluation requires a MRI performed within 14 days prior to the start of ANP therapy. Study subjects must be on a fixed dose of steroids for at least five days prior to the screening MRI. If the steroid dose is changed between the date of imaging and the start of treatment, a new baseline MRI is required. All MRIs must be performed at an accredited radiology center. All MRIs should include at a minimum: T1-weighted images pre/post gadolinium administration, fluid attenuated inversion recovery (FLAIR), and T-2 weighted images.
• Subjects 3-21 years of age must have a clinical history of disease of less than 6 months and at least two of the following clinical findings: cranial nerve deficit, long tract signs (i.e. hemiparesis) and ataxia are eligible. Subjects \> 21 years of age do not need to meet these criteria.
• Subjects must be ≥ 3 years of age. RT is not recommended for subjects less than 3 years of age.
• Subjects ≤ 16 years of age with a Lansky performance status of \> 40 are eligible. Subjects \> 16 years of age with a Karnofsky performance status of \> 40 are eligible.
• Subjects with organ and marrow function (as defined below) are eligible.
• Hemoglobin ≥ 9 g/dL
• Leukocytes \> 2000/mm3
• Absolute neutrophil count \>1,000/ mm3
• Serum Na+ ≤ 150 mmol/L
• Serum K+ ≤ 5.5 mmol/L
• Serum creatinine ≤ 1.5 times institutional upper limit
• Platelets \>50,000/ mm3

You may not qualify if:

• No type of prior therapy, including other investigational agents, is allowable. A prior diagnostic biopsy or surgical shunt for hydrocephalus is permitted.
• Subjects with disseminated disease, multicentric tumors, leptomeningeal disease, or the history of retrotumoral bleeding are not eligible. The screening / baseline MRI includes the spinal cord to rule out leptomeningeal disease.
• Subjects with a known history of ganglioglioma are not eligible.
• Subjects with a current diagnosis or family history of neurofibromatosis I or II are not eligible. - Subjects with a current diagnosis or family history of neurofibromatosis are not eligible.
• Subjects with an uncontrolled intercurrent illness including, but not limited to, ongoing or active infection, uncontrolled hypertension despite maximal medical management (three supine blood pressure measurements ≥ 150/99 taken at least one hour apart) or psychiatric illness/social situations that would limit compliance with protocol study requirements are not eligible.
• Subjects with a history of New York Heart Association Class II congestive heart failure are not eligible.
• Pregnant women are not eligible because the teratogenic and abortifacient effects of ANP therapy in humans are unknown. Because there is an unknown but potential risk for adverse events in nursing infants secondary to the mother receiving ANP therapy, breastfeeding is discontinued if the mother receives ANP therapy.

Sponsors & Collaborators

• Burzynski Research Institute: lead

Related Publications (4)

• Burzynski SR, 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. doi: 10.4236/jct.2015.64036

  BACKGROUND

• Burzynski SR, Janicki TJ, Burzynski GS, Marszalek A. The response and survival of children with recurrent diffuse intrinsic pontine glioma based on phase II study of antineoplastons A10 and AS2-1 in patients with brainstem glioma. Childs Nerv Syst. 2014 Dec;30(12):2051-61. doi: 10.1007/s00381-014-2401-z. Epub 2014 Apr 10.

  PMID: 24718705 BACKGROUND

• Burzynski SR. Recent clinical trials in diffuse intrinsic brainstem glioma. Cancer Therapy 5:379-390, 2007. Epub 2007 Nov

  BACKGROUND

• Burzynski SR, Janicki TJ, Weaver RA, Burzynski B. Targeted therapy with antineoplastons A10 and AS2-1 of high-grade, recurrent, and progressive brainstem glioma. Integr Cancer Ther. 2006 Mar;5(1):40-7. doi: 10.1177/1534735405285380.

  PMID: 16484713 BACKGROUND

Related Links

• Burzynski Research Institute
• Burzynski Clinic

MeSH Terms

Interventions

Radiation; antineoplaston A10

Intervention Hierarchy (Ancestors)

Physical Phenomena

Study Officials

• Stanislaw R. Burzynski, MD, PhD

  Burzynski Research Institute

  STUDY CHAIR

Central Study Contacts

Stanislaw R Burzynski, M.D., Ph.D.

CONTACT

713-335-5697; srb@burzynskiclinic.com

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: SINGLE
• Who Masked: OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: August 9, 2016
• First Posted: September 1, 2016
• Study Start: October 1, 2025
• Primary Completion (Estimated): June 1, 2026
• Study Completion (Estimated): June 1, 2026
• Last Updated: September 16, 2025

Record last verified: 2025-09

Data Sharing

• IPD Sharing: Will not share