- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT00003469
Antineoplaston Therapy in Treating Children With Rhabdoid Tumor of the Central Nervous System
Phase II Study of Antineoplastons A10 and AS2-1 in Children With Rhabdoid Tumor of the Central Nervous System
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.
Study Overview
Status
Conditions
Intervention / Treatment
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.
Study Type
Enrollment (Actual)
Phase
- Phase 2
Contacts and Locations
Study Locations
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Texas
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Houston, Texas, United States, 77055-6330
- Burzynski Clinic
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Participation Criteria
Eligibility Criteria
Ages Eligible for Study
Accepts Healthy Volunteers
Genders Eligible for Study
Description
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
Study Plan
How is the study designed?
Design Details
- Primary Purpose: Treatment
- Allocation: N/A
- Interventional Model: Single Group Assignment
- Masking: None (Open Label)
Arms and Interventions
Participant Group / Arm |
Intervention / Treatment |
---|---|
Experimental: 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.
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Children with a Rhabdoid tumor will receive Antineoplaston therapy (Atengenal + Astugenal).
Other Names:
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What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Number of Participants With Objective Response
Time Frame: 12 months
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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.
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12 months
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Secondary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Percentage of Participants Who Survived
Time Frame: 6 months, 12 months, 24 months
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6 months, 12 months, 24 months overall survival
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6 months, 12 months, 24 months
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Collaborators and Investigators
Sponsor
Publications and helpful links
Helpful Links
Study record dates
Study Major Dates
Study Start
Primary Completion (Actual)
Study Completion (Actual)
Study Registration Dates
First Submitted
First Submitted That Met QC Criteria
First Posted (Estimate)
Study Record Updates
Last Update Posted (Actual)
Last Update Submitted That Met QC Criteria
Last Verified
More Information
Terms related to this study
Keywords
Additional Relevant MeSH Terms
Other Study ID Numbers
- CDR0000066505
- BC-BT-14 (Other Identifier: Burzynski Research Institute, Inc.)
Plan for Individual participant data (IPD)
Plan to Share Individual Participant Data (IPD)?
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
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Susan Chi, MDBristol-Myers Squibb; Epizyme, Inc.RecruitingChordoma | Epithelioid Sarcoma | Atypical Teratoid Rhabdoid Tumor | INI1 (SMARCB1)-Deficient Primary CNS Malignant Tumors | SMARCA4-deficient Primary CNS Malignant Tumors | Malignant Rhabdoid Tumor (MRT) | Rhabdoid Tumor of the Kidney (RTK)United States
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Dana-Farber Cancer InstituteGateway for Cancer ResearchRecruitingEpithelioid Sarcoma | Atypical Teratoid/Rhabdoid Tumor | Malignant Rhabdoid Tumor | Rhabdoid Tumor of the Kidney | Chordoma (Poorly Differentiated or De-differentiated) | Other INI1 Negative Tumors (With PI Approval) | Other SMARCA4-deficient Malignant Tumors (With PI Approval)United States
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Epizyme, Inc.Active, not recruitingSynovial Sarcoma | Epithelioid Sarcoma | Renal Medullary Carcinoma | Malignant Rhabdoid Tumors (MRT) | Rhabdoid Tumors of the Kidney (RTK) | Atypical Teratoid Rhabdoid Tumors (ATRT) | Selected Tumors With Rhabdoid Features | INI1-negative Tumors | Malignant Rhabdoid Tumor of Ovary | Poorly Differentiated... and other conditionsFrance, United States, Australia, Taiwan, Belgium, United Kingdom, Canada, Germany, Italy
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Epizyme, Inc.CompletedSynovial Sarcoma | INI1-negative Tumors | Malignant Rhabdoid Tumor of Ovary | Rhabdoid TumorsUnited States, France, Canada, Italy, Germany, United Kingdom, Australia, Denmark, Netherlands
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