Clinical trial NCT00003515: Antineoplaston Therapy in Treating Patients With Metastatic, Recurrent, or Refractory Primitive Neuroectodermal Tumors
Official title: Phase II Study of Antineoplastons A10 and AS2-1 in Patients With Primitive Neuroectodermal Tumor Outside the Central Nervous System
Study type: INTERVENTIONAL
Overall status: TERMINATED
Phase: PHASE2
Conditions: Primitive Neuroectodermal Tumor
Keywords: Metastatic Primitive Neuroectodermal Tumor
Recurrent Primitive Neuroectodermal Tumor
Refractory Primitive Neuroectodermal Tumor
Lead sponsor: Burzynski Research Institute
Sponsor class: OTHER
First posted: 2003-01-27
Enrollment: 1
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: Current therapies for Metastatic, Recurrent, or Refractory Primitive Neuroectodermal Tumors provide very limited benefit to the patient. The anti-cancer properties of Antineoplaston therapy suggest that it may prove beneficial in the treatment of Metastatic, Recurrent, or Refractory Primitive Neuroectodermal Tumors.

PURPOSE: This study is being performed to determine the effects (good and bad) that Antineoplaston therapy has on patients with Metastatic, Recurrent, or Refractory Primitive Neuroectodermal Tumors.

Detailed description: OVERVIEW: This is a single arm, open-label study in which patients with Metastatic, Recurrent, or Refractory Primitive Neuroectodermal Tumors receive gradually escalating doses of intravenous Antineoplaston therapy (Atengenal + Astugenal) until the maximum tolerated dose is reached. Treatment continues up to12 months in the absence of disease progression or unacceptable toxicity.

OBJECTIVES:

* To determine the efficacy of Antineoplaston therapy in patients with Metastatic, Recurrent, or Refractory Primitive Neuroectodermal 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 patients with Metastatic, Recurrent, or Refractory Primitive Neuroectodermal Tumors.
* 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.

Eligibility criteria: DISEASE CHARACTERISTICS:

* Histologically confirmed incurable primitive neuroectodermal tumors outside the central nervous system that are unlikely to respond to existing therapy, meeting 1 of the following criteria:

  * Metastatic disease
  * Progressive, recurrent, or refractory disease after initial therapy, including surgery, chemotherapy, and/or radiotherapy
* Measurable disease by MRI or CT scan
* Tumor must be at least 2 cm

PATIENT CHARACTERISTICS:

Age:

* 6 months and over

Performance status:

* Karnofsky 60-100%

Life expectancy:

* At least 2 months

Hematopoietic:

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

Hepatic:

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

Renal:

* No 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 history of other cardiovascular conditions that contraindicate high dosages of sodium

Pulmonary:

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

Other:

* Not pregnant or nursing
* Fertile patients must use effective contraception during and for 4 weeks after study participation
* No active infection
* No nonmalignant systemic disease
* Not a high medical or psychiatric risk

PRIOR CONCURRENT THERAPY:

Biologic therapy:

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

Chemotherapy:

* See Disease Characteristics
* At least 4 weeks since prior chemotherapy (6 weeks for nitrosoureas)

Endocrine therapy:

* Concurrent corticosteroids allowed

Radiotherapy:

* See Disease Characteristics
* At least 8 weeks since prior radiotherapy

Surgery:

* See Disease Characteristics
* Recovered from prior surgery

Other:

* Prior cytodifferentiating agents allowed
* No prior antineoplastons
* No other concurrent antineoplastic agents

Primary outcomes:
Secondary outcomes:
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: