Rhabdoid Neoplasm of CNS
Conditions
Keywords
childhood rhabdoid tumor
Brief summary
RATIONALE: Current therapies for childhood Rhabdoid 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 Rhabdoid 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 Rhabdoid tumors.
Detailed description
OVERVIEW: This is a single arm, open-label study in which children with Rhabdoid 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 Rhabdoid 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 Rhabdoid 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 Rhabdoid 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 17 Years
Healthy volunteers
No
Inclusion criteria
DISEASE CHARACTERISTICS: * Histologically confirmed Rhabdoid tumor of the central nervous system that is unlikely to respond to existing therapy and 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 at least 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 uncontrolled hypertension * No severe heart disease * 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 participation * No serious active infections or fever * No other serious concomitant 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 (6 weeks for nitrosoureas) and recovered * No concurrent antineoplastic agents Endocrine therapy: * Concurrent corticosteroids for cerebral edema allowed (must be on a stable dose for at least 1 week before study entry) Radiotherapy: * At least 8 weeks since prior radiotherapy and recovered Surgery: * Not specified Other: * No prior antineoplaston treatment
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 | 6 months, 12 months, 24 months overall survival |
Countries
United States
Participant flow
Recruitment details
Four patients were recruited between February 1996 and May 2003. 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 Rhabdoid tumor will receive Antineoplaston therapy (Atengenal + Astugenal). | 4 |
| Total | 4 |
Baseline characteristics
| Characteristic | Antineoplaston Therapy |
|---|---|
| Age, Continuous | 3.7 Years |
| Sex: Female, Male Female | 3 Participants |
| Sex: Female, Male Male | 1 Participants |
Adverse events
| Event type | EG000 affected / at risk |
|---|---|
| deaths Total, all-cause mortality | — / — |
| other Total, other adverse events | 4 / 4 |
| serious Total, serious adverse events | 3 / 4 |
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 | Partial Response | 1 Participants |
| Antineoplaston Therapy | Number of Participants With Objective Response | Stable Disease | 1 Participants |
| Antineoplaston Therapy | Number of Participants With Objective Response | Progressive Disease | 2 Participants |
Secondary
Percentage of Participants Who Survived
6 months, 12 months, 24 months overall survival
Time frame: 6 months, 12 months, 24 months
Population: All study subjects receiving any Antineoplaston therapy
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| Antineoplaston Therapy | Percentage of Participants Who Survived | 6 months overall survival | 75.0 Percentage of participants |
| Antineoplaston Therapy | Percentage of Participants Who Survived | 12 months overall survival | 50.0 Percentage of participants |
| Antineoplaston Therapy | Percentage of Participants Who Survived | 24 months overall survival | 0.0 Percentage of participants |