Antineoplaston Therapy in Treating Children With Recurrent or Refractory High-Grade Glioma

• ClinicalTrials.gov Identifier: NCT00003535
• Recruitment Status: Terminated
• First Posted: January 27, 2003
• Results First Posted: December 13, 2017
• Last Update Posted: March 21, 2018
• Sponsor: Burzynski Research Institute
• Information provided by (Responsible Party): Burzynski Research Institute

Study Description

Brief Summary:

RATIONALE: Current therapies for children with recurrent/progressive high grade gliomas provide very limited benefit to the patient. The anti-cancer properties of Antineoplaston therapy suggest that it may prove beneficial in the treatment of children with recurrent/progressive high grade gliomas.

PURPOSE: This study is being performed to determine the effects (good and bad) that Antineoplaston therapy has on children (> 6 months of age) with recurrent/progressive high grade gliomas.

Condition or disease	Intervention/treatment	Phase
High Grade Glioma	Drug: Antineoplaston therapy (Atengenal + Astugenal)	Phase 2

Detailed Description:

OBJECTIVES:

• To determine the efficacy of Antineoplaston therapy in children with recurrent/progressive high grade gliomas, 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 recurrent/progressive high grade gliomas.

OVERVIEW: This is a single arm, open-label study in which children with recurrent/progressive high grade gliomas 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 for this study.

Study Design

• Study Type: Interventional (Clinical Trial)
• Actual Enrollment: 9 participants
• Allocation: N/A
• Intervention Model: Single Group Assignment
• Masking: None (Open Label)
• Primary Purpose: Treatment
• Official Title: Phase II Study of Antineoplastons A10 and AS2-1 in Children With High Grade Glioma
• Study Start Date: April 1994
• Actual Primary Completion Date: January 1998
• Actual Study Completion Date: January 1998

Arms and Interventions

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 recurrent/progressive high grade glioma will receive Antineoplaston therapy (Atengenal + Astugenal).; Other Name: A10 (Atengenal); AS2-1 (Astugenal)

Outcome Measures

Primary Outcome Measures :

1. 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.

Secondary Outcome Measures :

1. Percentage of Participants Who Survived [ Time Frame: 6 months, 12 months, 24 months ]
   6 months, 12 months, 24 months overall survival

Eligibility Criteria

• Ages Eligible for Study: 6 Months to 18 Years (Child, Adult)
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No

Criteria

Inclusion Criteria:

• Histologically confirmed high-grade glioma (glioblastoma multiforme or anaplastic astrocytoma) that is recurrent or progressive or with residual tumor after standard therapy, including radiotherapy
• Measurable tumor by MRI scan performed within two weeks prior to study entry
• Male or female patients
• Children 6 months to 17 years
• Performance status: Karnofsky 60-100%
• Life expectancy of at least 2 months
• WBC greater than 1,500/mm^3
• Platelet count greater than 50,000/mm^3
• No evidence of hepatic or renal insufficiency and a total bilirubin and serum creatinine no greater than 2.5 mg/dL and SGOT/SGPT no greater than 5 times upper limit of normal
• Must have recovered from adverse effect of previous therapy
• At least 8 weeks elapsed since last dose of radiation
• At least 4 weeks elapsed since last dose of chemotherapy (6 weeks for nitrosoureas)
• Corticosteroids permitted using the smallest dose that is compatible with preservation of optimal neurologic function
• Acceptable methods of birth control (in females of child-bearing potential or in sexually active males)during and up to four weeks following completion of study

Exclusion Criteria:

• Prior A10 and AS2-1 treatment
• Severe heart disease
• Uncontrolled hypertension
• Lung disease
• Hepatic failure
• Serious active infections, fever or other serious concurrent disease that would interfere with the evaluation of the treatment drug.
• Pregnant or nursing
• Serious concurrent disease
• Concurrent antineoplastic or immunomodulatory agents

Contacts and Locations

Locations

United States, Texas

Burzynski Clinic

Houston, Texas, United States, 77055-6330

Sponsors and Collaborators

Burzynski Research Institute

Investigators

• Principal Investigator: Stanislaw R. Burzynski, MD, PhD; Burzynski Research Institute

More Information

Additional Information:

Burzynski Research Institute 

Burzynski Clinic 

• Responsible Party: Burzynski Research Institute
• ClinicalTrials.gov Identifier: NCT00003535
• Other Study ID Numbers: CDR0000066582; BC-BT-06 ( Other Identifier: Burzynski Research Institute, Inc. )
• First Posted: January 27, 2003
• Results First Posted: December 13, 2017
• Last Update Posted: March 21, 2018
• Last Verified: March 2018

Individual Participant Data (IPD) Sharing Statement:

• Plan to Share IPD: No

Keywords provided by Burzynski Research Institute:

childhood anaplastic astrocytoma; childhood glioblastoma multiforme

Additional relevant MeSH terms:

Glioma; Neoplasms, Neuroepithelial; Neuroectodermal Tumors; Neoplasms, Germ Cell and Embryonal; Neoplasms by Histologic Type; Neoplasms; Neoplasms, Glandular and Epithelial; Neoplasms, Nerve Tissue