VPG: Antineoplaston Therapy in Treating Children With Visual Pathway Glioma

• Sponsor: Burzynski Research Institute (Other)
• Overall Status: Completed
• CT.gov ID: NCT00003477
• Collaborator: (none)
• Enrollment: 12
• Location: 1
• Arm: 1
• Duration (Months): 143
• Patients Per Site Per Month: 0.1

Study Details

Study Description

Brief Summary

RATIONALE: Current therapies for children with visual pathway gliomas, which are not amenable to or have not responded to standard therapy, provide limited benefit to the patient. The anti-cancer properties of Antineoplaston therapy suggest that it may prove beneficial in the treatment of children with visual pathway gliomas, which are not amenable to or 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 with visual pathway gliomas, which are not amenable to or have not responded to standard therapy.

Condition or Disease	Intervention/Treatment	Phase
Visual Pathway Glioma	Drug: Antineoplaston therapy (Atengenal + Astugenal)	Phase 2

Detailed Description

OBJECTIVES:

• To determine the efficacy of Antineoplaston therapy in children with visual pathway gliomas, which are not amenable to or have 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 visual pathway gliomas, which are not amenable to or have not responded to standard therapy.

OVERVIEW: This is a single arm, open-label study in which children with visual pathway gliomas, which are not amenable to or 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: Approximately 20-40 patients will be accrued to this study.

Study Design

• Study Type: Interventional
• Actual Enrollment: 12 participants
• Allocation: N/A
• Intervention Model: Single Group Assignment
• Masking: None (Open Label)
• Primary Purpose: Treatment
• Official Title: Phase II Study of Antineoplastons A10 and AS2-1 Infusions in Children With Visual Pathway Glioma
• Study Start Date: Jun 1, 1996
• Actual Primary Completion Date: May 1, 2008
• Actual Study Completion Date: May 1, 2008

Arms and Interventions

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.	Drug: Antineoplaston therapy (Atengenal + Astugenal); Children with a visual pathway glioma, which is not amenable to standard therapy or has not responded to standard therapy, will receive Antineoplaston therapy (Atengenal + Astugenal).; Other Names: A10 (Atengenal); AS2-1 (Astugenal)

Outcome Measures

Primary Outcome Measures

1. 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. Stable Disease (SD): <50% decrease in the sum of the products of of the greatest perpendicular diameters of all measurable enhancing lesions and no Progressive Disease, sustained for at least four weeks. Progressive Disease (PD): >=25% increase in the sum of the products of of the greatest perpendicular diameters of all measurable enhancing lesions.

Secondary Outcome Measures

1. 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

Eligibility Criteria

Criteria

• Ages Eligible for Study: 6 Months to 17 Years
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No

DISEASE CHARACTERISTICS:

• Histologically confirmed (unless medically contraindicated) visual pathway glioma, which is not amenable to standard therapy or did not respond to standard therapy.

• Evidence of tumor by MRI scan performed within 2 weeks prior to the study entry

• Tumor must be at least 5 mm

• No brain stem tumors

PATIENT CHARACTERISTICS:

Age:

• 6 months to 17 years

Performance status:

• Karnofsky 60-100%

Life expectancy:

• At least 2 months

Hematopoietic:

• WBC at least 2000/mm3

• Platelet count greater than 50,000/mm3

Hepatic:

• Bilirubin no greater than 2.5 mg/dL

• SGOT/SGPT no greater than 5 times upper limit of normal

• No hepatic failure

Renal:

• Creatinine no greater than 2.5 mg/dL

• No renal insufficiency

• No history of renal conditions that contraindicate high dosages of sodium

Cardiovascular:

• No severe heart disease

• No uncontrolled hypertension

• 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

• No serious active infections or fever

• No other serious concurrent 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 and recovered (6 weeks for nitrosoureas)

• No concurrent antineoplastic agents

Endocrine therapy:

• Concurrent corticosteroids for cerebral edema allowed (must be on stable dose for at least 1 week prior to study)

Radiotherapy:

• At least 8 weeks since prior radiotherapy and recovered

Surgery:

• Not specified

Other:

• No prior antineoplaston therapy

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	Burzynski Clinic	Houston	Texas	United States	77055-6330

Sponsors and Collaborators

• Burzynski Research Institute

Investigators

• Principal Investigator: Stanislaw R. Burzynski, MD, PhD, Burzynski Research Institute

Study Documents (Full-Text)

None provided.

More Information

Additional Information:

• Burzynski Research Institute
• Burzynski Clinic

Publications

• Stanislaw R. Burzynski, Tomasz J. Janicki, Gregory S. Burzynski. Antineoplastons A10 and AS2-1 in the Treatment of Children with Optic Pathway Glioma: Final Report for BT-23. Cancer and Clinical Oncology 6(1): 25-35, 2017

• Responsible Party: Burzynski Research Institute
• ClinicalTrials.gov Identifier: NCT00003477
• Other Study ID Numbers: CDR0000066514; BC-BT-23
• First Posted: Jan 27, 2003
• Last Update Posted: Aug 24, 2017
• Last Verified: Jul 1, 2017

• Individual Participant Data (IPD) Sharing Statement: No
• Plan to Share IPD: No
• Keywords provided by Burzynski Research Institute: visual pathway glioma not amenable to standard therapy; visual pathway glioma not responding to standard therapy
• Additional relevant MeSH terms: Glioma; Optic Nerve Glioma

Study Results

Participant Flow

Recruitment Details	Twelve patients were recruited between June1996 and May 2004. All study subjects were seen at the Burzynski Clinic in Houston TX

Arm/Group Title	Antineoplaston Therapy
Arm/Group 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. Antineoplaston therapy (Atengenal + Astugenal): Children with a visual pathway glioma, which is not amenable to standard therapy or has not responded to standard therapy, will receive Antineoplaston therapy (Atengenal + Astugenal).
Period Title: Overall Study
STARTED	12
COMPLETED	8
NOT COMPLETED	4

Baseline Characteristics

Arm/Group Title	Antineoplaston Therapy
Arm/Group 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. Antineoplaston therapy (Atengenal + Astugenal): Children with a visual pathway glioma, which is not amenable to standard therapy or has not responded to standard therapy, will receive Antineoplaston therapy (Atengenal + Astugenal).
Overall Participants	12
Age (Years) [Median (Full Range) ]
Median (Full Range) [Years]	4.5
Sex: Female, Male (Count of Participants)
Female	3 25%
Male	9 75%

Outcome Measures

1. Primary Outcome

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. Stable Disease (SD): <50% decrease in the sum of the products of of the greatest perpendicular diameters of all measurable enhancing lesions and no Progressive Disease, sustained for at least four weeks. Progressive Disease (PD): >=25% increase in the sum of the products of of the greatest perpendicular diameters of all measurable enhancing lesions.
Time Frame	12 months

Outcome Measure Data

Analysis Population Description

[Not Specified]

Arm/Group Title	Antineoplaston Therapy
Arm/Group 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.
Measure Participants	8
Complete Response	2 16.7%
Partial Response	2 16.7%
Stable Disease	3 25%
Progressive Disease	1 8.3%

2. Secondary Outcome

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

Outcome Measure Data

Analysis Population Description

All study subjects receiving any Antineoplaston therapy

Arm/Group Title	Antineoplaston Therapy
Arm/Group 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.
Measure Participants	12
6 months overall survival	91.7 764.2%
12 months overall survival	83.3 694.2%
24 months overall survival	75.0 625%
36 months overall survival	58.3 485.8%
48 months overall survival	50.0 416.7%
60 months overall survival	50.0 416.7%

Adverse Events

Time Frame	8 years, 2 months
Adverse Event Reporting Description	Adverse event data was collected through regular patient assessment and regular laboratory testing
Arm/Group Title	Antineoplaston Therapy
Arm/Group 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. Antineoplaston therapy (Atengenal + Astugenal): Patients with a visual pathway glioma will receive Antineoplaston therapy (Atengenal + Astugenal)
All Cause Mortality
	Antineoplaston Therapy
	Affected / at Risk (%)	# Events
Total	/ (NaN)
Serious Adverse Events
	Antineoplaston Therapy
	Affected / at Risk (%)	# Events
Total	9/12 (75%)
Gastrointestinal disorders
Vomiting	1/12 (8.3%)
General disorders
Fever	1/12 (8.3%)
Infections and infestations
Central venous catheter infection	3/12 (25%)
Nervous system disorders
Seizure	2/12 (16.7%)
Somnolence/depressed level of consciousness	3/12 (25%)
Hemorrhage, CNS	1/12 (8.3%)
Other (Not Including Serious) Adverse Events
	Antineoplaston Therapy
	Affected / at Risk (%)	# Events
Total	12/12 (100%)
Blood and lymphatic system disorders
Hemoglobin	5/12 (41.7%)
Leukocytes (total WBC)	2/12 (16.7%)
Lymphopenia	3/12 (25%)
Neutrophils/granulocytes (ANC/AGC)	2/12 (16.7%)
Platelets	1/12 (8.3%)
Ear and labyrinth disorders
Auditory/Ear - Other	1/12 (8.3%)
Endocrine disorders
Cushingoid appearance	2/12 (16.7%)
Hot flashes/flushes	1/12 (8.3%)
Eye disorders
Nystagmus	1/12 (8.3%)
Ocular/Visual - Other	2/12 (16.7%)
Gastrointestinal disorders
Anorexia	2/12 (16.7%)
Dehydration	2/12 (16.7%)
Diarrhea	4/12 (33.3%)
Distension/bloating, abdominal	1/12 (8.3%)
Dry mouth/salivary gland (xerostomia)	3/12 (25%)
Heartburn/dyspepsia	2/12 (16.7%)
Nausea	7/12 (58.3%)
Vomiting	8/12 (66.7%)
Pain: Abdomen NOS	2/12 (16.7%)
General disorders
Central Venous Catheter Non-functional	3/12 (25%)
Central Venous Catheter - Other	2/12 (16.7%)
Fatigue (asthenia, lethargy, malaise)	6/12 (50%)
Fever	5/12 (41.7%)
Rigors/chills	3/12 (25%)
Central Venous Catheter Pain	1/12 (8.3%)
Immune system disorders
Allergic reaction/hypersensitivity (including drug fever)	4/12 (33.3%)
Allergic rhinitis (including sneezing, nasal stuffiness)	3/12 (25%)
Infections and infestations
Central Venous Catheter Infection	5/12 (41.7%)
Infection (documented clinically): Abdomen NOS	1/12 (8.3%)
Infection (documented clinically): Bladder (urinary)	1/12 (8.3%)
Infection (documented clinically): Lung (pneumonia)	1/12 (8.3%)
Infection (documented clinically): Middle ear (otitis	1/12 (8.3%)
Infection (documented clinically): Pharynx	1/12 (8.3%)
Infection (documented clinically): Sinus	2/12 (16.7%)
Infection (documented clinically): Soft tissue NOS	1/12 (8.3%)
Infection (documented clinically): Upper airway NOS	3/12 (25%)
Infection (documented clinically): Urinary tract NOS	1/12 (8.3%)
Opportunistic infection	1/12 (8.3%)
Viral hepatitis	1/12 (8.3%)
Investigations
Albumin, serum-low (hypoalbuminemia)	1/12 (8.3%)
Alkaline phosphatase	2/12 (16.7%)
Bicarbonate, serum-low	2/12 (16.7%)
Hypercholesteremia	1/12 (8.3%)
Hyperglycemia	2/12 (16.7%)
Hypernatremia	7/12 (58.3%)
Hypertriglyceridemia	1/12 (8.3%)
Hypocalcemia	3/12 (25%)
Hypokalemia	10/12 (83.3%)
Hypomagnesemia	3/12 (25%)
Hypoglycemia	4/12 (33.3%)
Hyponatremia	2/12 (16.7%)
Metabolic/Laboratory - Other	2/12 (16.7%)
Proteinuria	4/12 (33.3%)
SGOT	2/12 (16.7%)
SGPT	3/12 (25%)
Uric acid, serum-high (hyperuricemia)	2/12 (16.7%)
Musculoskeletal and connective tissue disorders
Muscle weakness: Left-sided	1/12 (8.3%)
Pain: Back	1/12 (8.3%)
Pain: Joint	1/12 (8.3%)
Pain: Muscle	2/12 (16.7%)
Pain: Neck	1/12 (8.3%)
Nervous system disorders
Hemorrhage, CNS	1/12 (8.3%)
Ataxia (incoordination)	1/12 (8.3%)
Dizziness	1/12 (8.3%)
Mood alteration	2/12 (16.7%)
Neuropathy: motor	1/12 (8.3%)
Seizure	2/12 (16.7%)
Somnolence/depressed level of consciousness	8/12 (66.7%)
Tremor	1/12 (8.3%)
Pain: Head/headache	6/12 (50%)
Renal and urinary disorders
Hemorrhage, GU: Urinary NOS	1/12 (8.3%)
Urinary frequency/urgency	4/12 (33.3%)
Respiratory, thoracic and mediastinal disorders
Pain: Chest/thorax NOS	1/12 (8.3%)
Pain: Throat/pharynx/larynx	1/12 (8.3%)
Cough	3/12 (25%)
Pulmonary/Upper Respiratory - Other	1/12 (8.3%)
Skin and subcutaneous tissue disorders
Rash/desquamation	1/12 (8.3%)

Limitations/Caveats

[Not Specified]

More Information

Certain Agreements

Principal Investigators are NOT employed by the organization sponsoring the study.

There is NOT an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.