A Study of Atengenal and Astugenal in Diffuse, Intrinsic Pontine Glioma (DIPG)

NCT02742883 | Suspended Phase 2 FDA Drug

• 1 other identifier: BRI-BT-55
• Study Type: interventional
• Target: 1
• Locations: 1 country
• Sites: 1

Brief Summary

Current therapies for diffuse, intrinsic pontine glioma (DIPG) provide very limited benefit to the patient. The rationale for the use of Antineoplaston therapy in this protocol study derives from experience with subjects from prior Phase 2 studies and Compassionate Exemption patients treated with Antineoplaston therapy at the Burzynski Clinic. This study is designed to analyze the efficacy and safety of Antineoplaston therapy in five separate DIPG patient cohorts, which are defined by age and prior therapy. This is a two stage study with 20 patients in each cohort being enrolled in the first stage and an additional 20 patients being enrolled in the second stage, if pre-determined efficacy endpoints in the first stage are realized.

Conditions

Diffuse, Intrinsic Pontine Glioma

Outcome Measures

Primary Outcomes (1)

• Efficacy Measured by Objective Response Rate

  Objective Response Rate

  104 weeks

Secondary Outcomes (1)

• Efficacy Measured by Overall Survival

  6 months, 12 months, and 24 months

Study Arms (1)

Antineoplaston therapy

EXPERIMENTAL

Antineoplaston therapy (Atengenal + Astugenal) by IV infusion every four hours for up to 104 days. Study subjects receive increasing dosages of Atengenal and Astugenal until the maximum tolerated dosage is reached, but not exceeding 12.0 g/kg/d Atengenal or 0.4 mg/kg/d Astugenal.

Drug: Antineoplaston therapy (Atengenal + Astugenal)

Interventions

Antineoplaston therapy (Atengenal + Astugenal) DRUG

Patients with diffuse intrinsic pontine glioma will receive Antineoplaston therapy (Atengenal + Astugenal).

Also known as: A10 (Atengenal); AS2-1 (Astugenal)

Antineoplaston therapy

Eligibility Criteria

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

You may qualify if:

• Five cohorts of patients with diffuse, intrinsic pontine glioma will be studied:
• Patients age 3 months to \< 3 years;
• Patients age 3-21 years with progressive disease (PD) following radiation therapy (RT) ± chemotherapy and/or other therapies;
• Patients age 3-21 years with newly diagnosed DIPG who (or whose parents / guardians) have refused RT;
• Patients age \> 21 years with PD following RT ± chemotherapy and/or other therapies; and
• Patients age \> 21 years with newly diagnosed DIPG who have refused RT.

You may not qualify if:

• Disseminated disease, multicentric tumors, or leptomeningeal disease;
• Uncontrolled intercurrent illness;
• A history of New York Heart Association Class II congestive heart failure or above;
• Pregnancy.

Sponsors & Collaborators

• Burzynski Research Institute: lead

Study Sites (1)

Burzynski Clinic

Houston, Texas, 77055-6330, United States

Location

Related Links

• Burzynski Research Institute
• Burzynski Clinic

MeSH Terms

Conditions

Diffuse Intrinsic Pontine Glioma

Interventions

antineoplaston A10; antineoplaston AS 2-1

Condition Hierarchy (Ancestors)

Glioma; Neoplasms, Neuroepithelial; Neuroectodermal Tumors; Neoplasms, Germ Cell and Embryonal; Neoplasms by Histologic Type; Neoplasms; Neoplasms, Glandular and Epithelial; Neoplasms, Nerve Tissue; Brain Stem Neoplasms; Infratentorial Neoplasms; Brain Neoplasms; Central Nervous System Neoplasms; Nervous System Neoplasms; Neoplasms by Site; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases

Study Officials

• Stanislaw R. Burzynski, MD, PhD

  Burzynski Research Institute

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: April 12, 2016
• First Posted: April 19, 2016
• Study Start: April 13, 2016
• Primary Completion (Estimated): March 1, 2028
• Study Completion (Estimated): March 1, 2028
• Last Updated: September 16, 2025

Record last verified: 2025-09

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)