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Antineoplaston Therapy in Treating Children With Primitive Neuroectodermal Tumors (PNET)

Primary Purpose

Childhood CNS Primitive Neuroectodermal Tumor

Status
Completed
Phase
Phase 2
Locations
United States
Study Type
Interventional
Intervention
Antineoplaston therapy (Atengenal + Astugenal)
Sponsored by
Burzynski Research Institute
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Childhood CNS Primitive Neuroectodermal Tumor focused on measuring Recurrent primitive neuroectodermal tumor

Eligibility Criteria

6 Months - 17 Years (Child)All SexesDoes not accept healthy volunteers

DISEASE CHARACTERISTICS: Histologically confirmed (except if medically contraindicated) incurable primitive neuroectodermal tumor Evidence of progressive or recurrent tumor by MRI scan performed within 2 weeks prior to study entry Must have received and failed prior standard therapy 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 insufficiency 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 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 prior to study entry) Radiotherapy: At least 8 weeks since prior radiotherapy and recovered Surgery: Not specified Other: No prior antineoplaston treatment

Sites / Locations

  • Burzynski Clinic

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Antineoplaston therapy

Arm Description

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.

Outcomes

Primary Outcome Measures

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.

Secondary Outcome Measures

Percentage of Participants Who Survived
6 months, 12 months, 24 months, 36 months, 48 months, 60 months overall survival

Full Information

First Posted
November 1, 1999
Last Updated
July 24, 2017
Sponsor
Burzynski Research Institute
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1. Study Identification

Unique Protocol Identification Number
NCT00003460
Brief Title
Antineoplaston Therapy in Treating Children With Primitive Neuroectodermal Tumors
Acronym
PNET
Official Title
Phase II Study of Antineoplastons A10 and AS2-1 In Children With Primitive Neuroectodermal Tumors
Study Type
Interventional

2. Study Status

Record Verification Date
July 2017
Overall Recruitment Status
Completed
Study Start Date
April 1996 (undefined)
Primary Completion Date
February 2005 (Actual)
Study Completion Date
February 2005 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Burzynski Research Institute

4. Oversight

Data Monitoring Committee
No

5. Study Description

Brief Summary
RATIONALE: Current therapies for children with primitive neuroectodermal tumors that have not responded to standard therapy provide very limited benefit to the patient. The anti-cancer properties of Antineoplaston therapy suggest that it may prove beneficial in the treatment of children with primitive neuroectodermal tumors that have not responded to standard therapy. PURPOSE: This study is being performed to determine the effects (good and bad) that Antineoplaston therapy has on children (> 6 months of age) with primitive neuroectodermal tumors that has not responded to standard therapy.
Detailed Description
OBJECTIVES: To determine the efficacy of Antineoplaston therapy in children with primitive neuroectodermal tumors that has not responded to standard therapy, 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. OVERVIEW: This is a single arm, open-label study in which children with primitive neuroectodermal tumors that have not responded to standard therapy 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. After 12 months, patients with a complete or partial response or with stable disease may continue treatment. 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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Childhood CNS Primitive Neuroectodermal Tumor
Keywords
Recurrent primitive neuroectodermal tumor

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
13 (Actual)

8. Arms, Groups, and Interventions

Arm Title
Antineoplaston therapy
Arm Type
Experimental
Arm Description
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.
Intervention Type
Drug
Intervention Name(s)
Antineoplaston therapy (Atengenal + Astugenal)
Other Intervention Name(s)
A10 (Atengenal); AS2-1 (Astugenal)
Intervention Description
Children with a primitive neuroectodermal tumor that has not responded to standard therapy will receive Antineoplaston therapy (Atengenal + Astugenal).
Primary Outcome Measure Information:
Title
Number of Participants With Objective Response
Description
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
Secondary Outcome Measure Information:
Title
Percentage of Participants Who Survived
Description
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

10. Eligibility

Sex
All
Minimum Age & Unit of Time
6 Months
Maximum Age & Unit of Time
17 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
DISEASE CHARACTERISTICS: Histologically confirmed (except if medically contraindicated) incurable primitive neuroectodermal tumor Evidence of progressive or recurrent tumor by MRI scan performed within 2 weeks prior to study entry Must have received and failed prior standard therapy 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 insufficiency 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 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 prior to study entry) Radiotherapy: At least 8 weeks since prior radiotherapy and recovered Surgery: Not specified Other: No prior antineoplaston treatment
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Stanislaw R Burzynski, MD, PhD
Organizational Affiliation
Burzynski Research Institute
Official's Role
Principal Investigator
Facility Information:
Facility Name
Burzynski Clinic
City
Houston
State/Province
Texas
ZIP/Postal Code
77055-6330
Country
United States

12. IPD Sharing Statement

Plan to Share IPD
No
Links:
URL
http://www.burzynskiresearch.com
Description
Burzynski Research Institute
URL
http://www.burzynskiclinic.com
Description
Burzynski Clinic

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Antineoplaston Therapy in Treating Children With Primitive Neuroectodermal Tumors

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