Clinical trial NCT00003479: Antineoplaston Therapy in Treating Patients With Ependymoma
Official title: Phase II Study of Antineoplastons A10 and AS2-1 Infusions in Patients With Ependymoma
Study type: INTERVENTIONAL
Overall status: TERMINATED
Phase: PHASE2
Conditions: Ependymoma
Keywords: Ependymoma
Anaplastic Ependymoma
Lead sponsor: Burzynski Research Institute
Sponsor class: OTHER
First posted: 2003-01-27
Enrollment: 9
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 patients with ependymoma provide limited benefit to the patient. The anti-cancer properties of Antineoplaston therapy suggest that it may prove beneficial in the treatment of patients with ependymoma .

PURPOSE: This study is being performed to determine the effects (good and bad) that Antineoplaston therapy has on patients with ependymoma.

Detailed description: OBJECTIVES:

* To determine the efficacy of Antineoplaston therapy in patients with ependymoma 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 ependymoma

OVERVIEW: This is a single arm, open-label study in which patients with ependymoma 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 ependymoma that is unlikely to respond to existing therapy and for which no curative therapy exists
* Evidence of residual tumor (\>= 5mm) by MRI scan performed within two weeks prior to study entry
* No brain stem tumors

PATIENT CHARACTERISTICS:

Age:

* 6 months and over

Performance status:

* Karnofsky 60-100%

Life expectancy:

* At least 2 months

Hematopoietic:

* WBC at least 2,000/mm3
* Platelet count greater than 50,000/mm3

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 renal failure
* 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 history of 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
* 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 and recovered (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 prior to study)

Radiotherapy:

* At least 8 weeks since prior radiotherapy and recovered

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: