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

Phase 2

Terminated

• Conditions: High Grade Glioma
• Interventions: Drug: Antineoplaston therapy (Atengenal + Astugenal)

• Registration Number: NCT00003535
• Lead Sponsor: Burzynski Research Institute

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.

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 Timeline

• Study Start: 1994-04
• Primary Completion: 1998-01
• Study Completion: 1998-01
• Study First Submitted: 1999-11-01
• Study First Submitted QC: 2003-01-26
• Study First Posted: 2003-01-27
• Results First Submitted: 2017-08-01
• Results First Submitted QC: 2017-08-01
• Results First Posted: 2017-12-13
• Status Verified: 2018-03
• Last Update Submitted: 2018-03-07
• Last Update Posted: 2018-03-21

Recruitment & Eligibility

• Status: TERMINATED
• Sex: All
• Target Recruitment: 9

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

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Antineoplaston therapy	Antineoplaston therapy (Atengenal + Astugenal)	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.

Primary Outcome Measures

Name	Time	Method
Number of Participants With Objective Response	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

Name	Time	Method
Percentage of Participants Who Survived	6 months, 12 months, 24 months	6 months, 12 months, 24 months overall survival

Trial Locations

Locations (1)

Burzynski Clinic; 🇺🇸 Houston, Texas, United States