Childhood Brain Tumors
Conditions
Keywords
Anaplastic astrocytoma, Anaplastic glioma, Anaplastic oligodendroglioma, Astrocytoma, Astrocytoma pilocytic, Brain stem glioma, Craniopharyngioma, Ganglioglioma, Glioblastoma multiforme, Gliosarcoma, Medulloepithelioma, Neurocytoma, Oligodendroglioma, Oligodendroglioma-brainstem, Primitive neuroectodermal tumor
Brief summary
RATIONALE: Current therapies for childhood brain tumors provide limited benefit to the patient. The anti-cancer properties of Antineoplaston therapy suggest that it may prove beneficial in the treatment of childhood brain tumors. PURPOSE: This study is being performed to determine the effects (good and bad) that Antineoplaston therapy has on children (\> 6 months of age) with brain tumors.
Detailed description
OVERVIEW: This is a single arm, open-label study in which children with brain tumors 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. OBJECTIVES: * To determine the efficacy of Antineoplaston therapy in children with a brain tumor, 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 children with a brain tumor. * 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.
Interventions
DRUGAntineoplaston therapy (Atengenal + Astugenal)
Children with a brain tumor will receive Antineoplaston therapy (Atengenal + Astugenal).
Sponsors
Burzynski Research Institute
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
6 Months to 18 Years
Healthy volunteers
No
Inclusion criteria
DISEASE CHARACTERISTICS: * Histologically confirmed (except if medically contraindicated) childhood brain tumor for which no curative therapy exists * Measurable tumor by MRI scan performed within two weeks prior to study entry * Tumor must be at least 5 mm PATIENT CHARACTERISTICS: Age: * 6 months to 17 years Performance status: * Karnofsky 60-100% Life expectancy: * At least 2 months Hematopoietic: * WBC greater than 2000/mm\^3 * Platelet count greater than 50,000/mm\^3 Hepatic: * Bilirubin no greater than 2.5 mg/dL * SGOT and 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 history of heart failure * No history of other cardiovascular conditions that contraindicate high dosages of sodium * No uncontrolled hypertension 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 concurrent 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) and recovered * 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) Radiation therapy: * At least 8 weeks since prior radiotherapy and recovered Surgery: * Recovered from any prior surgery Other: * No prior antineoplaston therapy
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Number of Participants With Objective Response | 12 months | Objective response rate per Response Assessment in Neuro-Oncology (RANO) for target lesions and assessed by MRI: Complete Response (CR), disappearance of all disease sustained for at least four weeks; Partial Response (PR), \>=50% decrease in the sum of the products of of the greatest perpendicular diameters of all measurable enhancing lesions, sustained for at least four weeks. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Percentage of Participants Who Survived | 6 months, 12 months, 24 months, 36 months, 48 months, 60 months | 6 months, 12 months, 24 months, 36 months, 48 months, 60 months overall survival |
Countries
United States
Participant flow
Recruitment details
Thirty-four patients were recruited between September 1996 and July 2012. All study subjects were seen at the Burzynski Clinic in Houston TX
Participants by arm
| Arm | Count |
|---|---|
| Antineoplaston Therapy Antineoplaston therapy (Atengenal + Astugenal) by IV infusion every four hours for at least 12 months. Study subjects receive increasing dosages of Atengenal and Astugenal until the maximum tolerated dose is reached.
Antineoplaston therapy (Atengenal + Astugenal): Children with a brain tumor will receive Antineoplaston therapy (Atengenal + Astugenal). | 34 |
| Total | 34 |
Withdrawals & dropouts
| Period | Reason | FG000 |
|---|---|---|
| Overall Study | Not evaluable | 4 |
Baseline characteristics
| Characteristic | Antineoplaston Therapy |
|---|---|
| Age, Continuous | 10.4 Years |
| Sex: Female, Male Female | 21 Participants |
| Sex: Female, Male Male | 13 Participants |
Adverse events
| Event type | EG000 affected / at risk |
|---|---|
| deaths Total, all-cause mortality | — / — |
| other Total, other adverse events | 34 / 34 |
| serious Total, serious adverse events | 21 / 34 |
Outcome results
Primary
Number of Participants With Objective Response
Objective response rate per Response Assessment in Neuro-Oncology (RANO) for target lesions and assessed by MRI: Complete Response (CR), disappearance of all disease sustained for at least four weeks; Partial Response (PR), \>=50% decrease in the sum of the products of of the greatest perpendicular diameters of all measurable enhancing lesions, sustained for at least four weeks.
Time frame: 12 months
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| Antineoplaston Therapy | Number of Participants With Objective Response | Complete Response | 1 Participants |
| Antineoplaston Therapy | Number of Participants With Objective Response | Partial Response | 5 Participants |
| Antineoplaston Therapy | Number of Participants With Objective Response | Stable Disease | 6 Participants |
| Antineoplaston Therapy | Number of Participants With Objective Response | Progressive Disease | 18 Participants |
Secondary
Percentage of Participants Who Survived
6 months, 12 months, 24 months, 36 months, 48 months, 60 months overall survival
Time frame: 6 months, 12 months, 24 months, 36 months, 48 months, 60 months
Population: All study subjects receiving any Antineoplaston therapy
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| Antineoplaston Therapy | Percentage of Participants Who Survived | 60 months overall survival | 20.6 Percentage of participants |
| Antineoplaston Therapy | Percentage of Participants Who Survived | 6 months overall survival | 64.7 Percentage of participants |
| Antineoplaston Therapy | Percentage of Participants Who Survived | 12 months overall survival | 47.1 Percentage of participants |
| Antineoplaston Therapy | Percentage of Participants Who Survived | 24 months overall survival | 35.3 Percentage of participants |
| Antineoplaston Therapy | Percentage of Participants Who Survived | 36 months overall survival | 32.4 Percentage of participants |
| Antineoplaston Therapy | Percentage of Participants Who Survived | 48 months overall survival | 20.6 Percentage of participants |