Clinical trial NCT00003535: Antineoplaston Therapy in Treating Children With Recurrent or Refractory High-Grade Glioma
Official title: Phase II Study of Antineoplastons A10 and AS2-1 in Children With High Grade Glioma
Study type: INTERVENTIONAL
Overall status: TERMINATED
Phase: PHASE2
Conditions: High Grade Glioma
Keywords: childhood anaplastic astrocytoma
childhood glioblastoma multiforme
Lead sponsor: Burzynski Research Institute
Sponsor class: OTHER
First posted: 2003-01-27
Enrollment: 9
Sex: ALL
Minimum age (years): 6
Maximum age (years): 18
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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.

Eligibility 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

Primary outcomes:
measure: Number of Participants With Objective Response
timeFrame: 12 months
Secondary outcomes:
measure: Percentage of Participants Who Survived
timeFrame: 6 months, 12 months, 24 months
Other outcomes:
Locations:
city: Houston
country: United States
facility: Burzynski Clinic
state: Texas
Study officials:
affiliation: Burzynski Research Institute
name: Stanislaw R. Burzynski, MD, PhD
role: PRINCIPAL_INVESTIGATOR
References: