Clinical trial NCT00003475: Antineoplaston Therapy in Treating Patients With Primary Malignant Brain Tumors
Official title: Phase II Study of Antineoplastons A10 and AS2-1 in Adult Patients With Primary Malignant Brain Tumors
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Malignant Brain Tumors
Keywords: adult glioblastoma multiforme
adult anaplastic astrocytoma
adult anaplastic astrocytoma/Mixed
Lead sponsor: Burzynski Research Institute
Sponsor class: OTHER
First posted: 2003-01-27
Enrollment: 40
Sex: ALL
Minimum age (years): 18
Maximum age (years): 99
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: RATIONALE: Current therapies for adults with primary malignant brain tumors that have not responded to standard therapy provide very limited benefit to the patient. The anti-cancer properties of Antineoplaston therapy suggest that it may prove beneficial in the treatment of adults with primary malignant brain tumors that have not responded to standard therapy.

PURPOSE: This study is being performed to determine the effects (good and bad) that Antineoplaston therapy has on adults with primary malignant brain tumors that have not responded to standard therapy.

Detailed description: OBJECTIVES:

* To determine the efficacy of Antineoplaston therapy in patients with primary malignant brain tumors that have not responded to standard therapy, 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 primary malignant brain tumor.

OVERVIEW: This is a single arm, open-label study in which adults with malignant brain tumors that have not responded to standard therapy 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 or cytologically confirmed incurable adult primary malignant brain tumor
* Evidence of progressive or recurrent tumor by MRI scan performed within 2 weeks prior to study entry
* Must have received and failed standard therapy
* Tumor must be at least 5 mm
* No brain stem tumors

PATIENT CHARACTERISTICS:

Age:

* 18 and over

Performance status:

* Karnofsky 60-100%

Life expectancy:

* At least 2 months

Hematopoietic:

* Hematological function normal
* WBC at least 2000/mm\^3
* Platelet count at least 50,000/mm\^3

Hepatic:

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

Renal:

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

Cardiovascular:

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

Pulmonary:

* No serious lung disease, such as severe chronic obstructive pulmonary disease

Other:

* Not pregnant or nursing
* Fertile patients must use effective contraception during and for 4 weeks after study participation
* No medical or psychiatric illness that would preclude study treatment
* No active infection

PRIOR CONCURRENT THERAPY:

Biologic therapy:

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

Chemotherapy:

* At least 4 weeks since prior chemotherapy, except in patients with disease progression during initial therapy
* At least 6 weeks since prior nitrosoureas, except in patients with disease progression during initial therapy
* No concurrent antineoplastic agents

Endocrine therapy:

* Corticosteroids allowed

Radiotherapy:

* See Disease Characteristics
* At least 8 weeks since prior radiotherapy, except in patients with disease progression during initial therapy

Surgery:

* At least 4 weeks since prior surgery, except in patients with disease progression during initial therapy

Other:

* Recovered from prior therapy
* Cytodifferentiating agents allowed

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 SR, Janicki TJ, Burzynski GS. A phase II study of antineoplastons A10 and AS2-1 in adult patients with recurrent glioblastoma multiforme: Final report (protocol BT-21). Journal of Cancer Therapy 5:946-956, 2014. DOI: http://dx.doi.org/10.4236/jct.2014.510100