Clinical trial NCT00003459: Antineoplaston Therapy in Treating Patients With Brain Stem Glioma
Official title: Phase II Study of Antineoplastons A10 and AS2-1 In Patients With Brain Stem Glioma
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Brain Stem Gliomas
Keywords: recurrent adult brain stem glioma
untreated adult brain stem glioma
untreated childhood brain stem glioma
recurrent childhood brain stem glioma
Lead sponsor: Burzynski Research Institute
Sponsor class: OTHER
First posted: 2003-01-27
Enrollment: 40
Sex: ALL
Minimum age (years): 6
Maximum age (years): 99
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: RATIONALE: Current therapies for a brain stem glioma provide very limited benefit to the patient. The anti-cancer properties of Antineoplaston therapy suggest that it may prove beneficial in the treatment of brain stem gliomas.

PURPOSE: This study is being performed to determine the effects (good and bad) that Antineoplaston therapy has on children (\> 6 months of age) and adults with newly-diagnosed or recurrent brain stem gliomas.

Detailed description: OBJECTIVES:

* To determine the efficacy of Antineoplaston therapy in patients with a brain stem glioma, 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 patients with a brain stem glioma.

OVERVIEW: This is a single arm, open-label study in which patients 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 in measured utilizing MRI scans, which are performed every 8 weeks for the first 2 years, every 3 months for the 3rd and 4th years, every 6 months for the 5th and 6th years, and annually thereafter.

PROJECTED ACCRUAL: A total of 20-40 patients will be accrued for this study.

Eligibility criteria: DISEASE CHARACTERISTICS:

* Histologically confirmed (except if medically contraindicated) brain stem glioma that is unlikely to respond to existing therapy and for which no curative therapy exists.
* Tumor must be at least 5 mm in maximum diameter

PATIENT CHARACTERISTICS:

Age:

* 6 months or greater

Performance status:

* Karnofsky 60-100%

Life expectancy:

* At least 2 months

Hematopoietic:

* WBC at least 2,000/mm\^3
* Platelet count greater than 50,000/mm\^3

Hepatic:

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

Renal:

* Creatinine no greater than 2.5 mg/dL
* No history of renal conditions that contraindicate high dosages of sodium

Cardiovascular:

* No severe heart disease
* No uncontrolled hypertension
* No history of congestive heart failure
* No other cardiovascular conditions that contraindicate high dosages of sodium

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 concomitant disease

PRIOR CONCURRENT THERAPY:

Biologic therapy:

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

Chemotherapy:

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

Endocrine therapy:

* Concurrent corticosteroids for cerebral edema allowed (must be on stable dose for at least 1 week before study entry)

Radiotherapy:

* At least 8 weeks since prior radiotherapy

Surgery:

* Not specified

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, 24 months, 36 months, 48 months, 60 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, S.R., Janicki, J., Burzynski, G., Marszalek, A. A Phase II Study of Antineoplastons A10 and AS2-1 in Patients with Brainstem Gliomas. The Report on Non-Diffuse Intrinsic Pontine Glioma (Protocol BT-11). Journal of Cancer Therapy, 6: 334-344, 2015
citation: Burzynski SR, Janicki TJ, Burzynski GS, Marszalek A. The response and survival of children with recurrent diffuse intrinsic pontine glioma based on phase II study of antineoplastons A10 and AS2-1 in patients with brainstem glioma. Childs Nerv Syst. 2014 Dec;30(12):2051-61. doi: 10.1007/s00381-014-2401-z. Epub 2014 Apr 10.