Clinical trial NCT00003513: Antineoplaston Therapy in Treating Patients With Metastatic, Recurrent, or Refractory Neuroblastoma
Official title: Phase II Study of Antineoplastons A10 and AS2-1 In Patients With Neuroblastoma
Study type: INTERVENTIONAL
Overall status: TERMINATED
Phase: PHASE2
Conditions: Neuroblastoma
Keywords: Metastatic Neuroblastoma
Recurrent Neuroblastoma
Refractory Neuroblastoma
Lead sponsor: Burzynski Research Institute
Sponsor class: OTHER
First posted: 2003-01-27
Enrollment: 3
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 Neuroblastoma 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 Neuroblastoma.

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

Detailed description: Metastatic, Recurrent, or Refractory Neuroblastoma patients 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 Neuroblastoma, 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 Neuroblastoma.
* 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 neuroblastoma that is unlikely to respond to existing therapy, meeting 1 of the following criteria:

  * Metastatic disease
  * Progressive, recurrent, or persistent 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: