Clinical trial NCT00003476: Antineoplaston Therapy in Treating Children With Primary Malignant Brain Tumors
Official title: Phase II Study of Antineoplastons A10 and AS2-1 in Children With Primary Malignant Brain Tumors
Study type: INTERVENTIONAL
Overall status: TERMINATED
Phase: PHASE2
Conditions: Childhood Brain Tumor
Keywords: childhood anaplastic astrocytoma
recurrent childhood anaplastic astrocytoma
childhood glioblastoma multiforme
recurrent childhood glioblastoma multiforme
childhood brainstem glioma
recurrent childhood brainstem glioma
childhood primitive neuroectodermal tumor
recurrent childhood primitive neuroectodermal tumor
Lead sponsor: Burzynski Research Institute
Sponsor class: OTHER
First posted: 2003-01-27
Enrollment: 8
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 primary malignant brain tumors provide limited benefit to the patient. The anti-cancer properties of Antineoplaston therapy suggest that it may prove beneficial in the treatment of children with primary malignant brain tumors.

PURPOSE: This study is being performed to determine the effects (good and bad) that Antineoplaston therapy has on children with primary malignant brain tumors.

Detailed description: OBJECTIVES:

* To determine the efficacy of Antineoplaston therapy in children with primary malignant brain tumors 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 primary malignant brain tumors.

OVERVIEW: This is a single arm, open-label study in which children with primary malignant brain tumors 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 to this study

Eligibility criteria: DISEASE CHARACTERISTICS:

* Histologically confirmed incurable primary malignant brain tumor that has progressed, recurred, or persisted after initial therapy (must have failed standard therapy).
* Evidence of residual tumor by MRI performed within two weeks prior to study entry

PATIENT CHARACTERISTICS:

Age:

* 6 months to 17 years

Performance status:

* Karnofsky 60-100%

Life expectancy:

* At least 2 months

Hematopoietic:

* WBC at least 1,500/mm\^3
* Platelet count greater than 50,000/mm\^3

Hepatic:

* No hepatic failure
* Bilirubin no greater than 2.5 mg/dL
* SGOT/SGPT no greater than 5 times upper limit of normal

Renal:

* Creatinine no greater than 2.5 mg/dL

Cardiovascular:

* No severe heart disease
* No uncontrolled hypertension

Pulmonary:

* No severe lung disease

Other:

* Not pregnant or nursing
* Fertile patients must use effective contraception during and for 4 weeks after study participation
* No serious active infections or fever
* No other serious concurrent disease

PRIOR CONCURRENT THERAPY:

Biologic therapy:

* At least 4 weeks since prior immunotherapy and recovered
* No concurrent immunomodulating agents

Chemotherapy:

* At least 4 weeks since prior chemotherapy (6 weeks for nitrosoureas) and recovered
* No concurrent antineoplastic agents

Endocrine therapy:

* Recovered from prior endocrine therapy
* Concurrent corticosteroids for cerebral edema allowed

Radiotherapy:

* At least 8 weeks since prior radiotherapy (unless clear radiological evidence of progression) and recovered

Surgery:

* At least 4 weeks since prior surgery (unless clear radiological evidence of progression) and recovered

Other:

* No prior antineoplaston therapy

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
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:
citation: Burzynski SR, Janicki TJ, Burzynski GS, Marszalek A, Brookman, S. A phase II study of Antineoplastons A10 and AS2-1 in children with recurrent, refractory or progressive primary brain tumors - Final Report (Protocol BT-22). Journal of Cancer Therapy, 5:977-988, 2014.