Antineoplaston Therapy in Treating Children With Brain Tumors

• Sponsor: Burzynski Research Institute (Other)
• Overall Status: Completed
• CT.gov ID: NCT00003458
• Collaborator: (none)
• Enrollment: 34
• Location: 1
• Arm: 1
• Duration (Months): 216
• Patients Per Site Per Month: 0.2

Study Details

Study Description

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.

Condition or Disease	Intervention/Treatment	Phase
Childhood Brain Tumors	Drug: Antineoplaston therapy (Atengenal + Astugenal)	Phase 2

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.

Study Design

• Study Type: Interventional
• Actual Enrollment: 34 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 in Children With Brain Tumors
• Study Start Date: Sep 1, 1996
• Actual Primary Completion Date: Sep 1, 2014
• Actual Study Completion Date: Sep 1, 2014

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 brain tumor 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.

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 18 Years
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No

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

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, Ania Marszalek. A Phase II Study of Antineoplastons A10 and AS2-1 in Children with Brain Tumors. Final Report (Protocol BT-10). Journal of Cancer Therapy 8: 173-187, 2017

• Responsible Party: Burzynski Research Institute
• ClinicalTrials.gov Identifier: NCT00003458
• Other Study ID Numbers: CDR0000066490; BC-BT-10
• First Posted: Jan 27, 2003
• Last Update Posted: Mar 7, 2022
• Last Verified: Mar 1, 2022

• Individual Participant Data (IPD) Sharing Statement: No
• Plan to Share IPD: No
• Keywords provided by Burzynski Research Institute: 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
• Additional relevant MeSH terms: Brain Neoplasms

Study Results

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

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. Children with a brain tumor will receive Antineoplaston therapy (Atengenal + Astugenal).
Period Title: Overall Study
STARTED	34
COMPLETED	30
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 brain tumor will receive Antineoplaston therapy (Atengenal + Astugenal).
Overall Participants	34
Age (Years) [Median (Full Range) ]
Median (Full Range) [Years]	10.4
Sex: Female, Male (Count of Participants)
Female	21 61.8%
Male	13 38.2%

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.
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. Antineoplaston therapy (Atengenal + Astugenal): Children with a brain tumor will receive Antineoplaston therapy (Atengenal + Astugenal).
Measure Participants	30
Complete Response	1 2.9%
Partial Response	5 14.7%
Stable Disease	6 17.6%
Progressive Disease	18 52.9%

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. Children with a brain tumor will receive Antineoplaston therapy (Atengenal + Astugenal).
Measure Participants	34
6 months overall survival	64.7 190.3%
12 months overall survival	47.1 138.5%
24 months overall survival	35.3 103.8%
36 months overall survival	32.4 95.3%
48 months overall survival	20.6 60.6%
60 months overall survival	20.6 60.6%

Adverse Events

Time Frame	18 years, 5 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. Children with a brain tumor 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	21/34 (61.8%)
Blood and lymphatic system disorders
Hemoglobin	1/34 (2.9%)
Gastrointestinal disorders
Vomiting	1/34 (2.9%)
General disorders
Fever	1/34 (2.9%)
Infections and infestations
Central Venous Catheter Infection	2/34 (5.9%)
Infection (documented clinically): Lung (pneumonia)	1/34 (2.9%)
Infection (documented clinically): Skin (cellulitis)	1/34 (2.9%)
Investigations
Hyperntremia	1/34 (2.9%)
Nervous system disorders
Hemorrhage, CNS	2/34 (5.9%)
Hydrocephalus	1/34 (2.9%)
Neuropathy: motor	1/34 (2.9%)
Seizure	4/34 (11.8%)
Somnolence/depressed level of consciousness	9/34 (26.5%)
Pain: Head/headache	3/34 (8.8%)
Respiratory, thoracic and mediastinal disorders
Dyspnea (shortness of breath)	1/34 (2.9%)
Skin and subcutaneous tissue disorders
Hematoma	1/34 (2.9%)
Other (Not Including Serious) Adverse Events
	Antineoplaston Therapy
	Affected / at Risk (%)	# Events
Total	34/34 (100%)
Blood and lymphatic system disorders
Hemoglobin	15/34 (44.1%)
Leukocytes (total WBC)	12/34 (35.3%)
Lymphopenia	10/34 (29.4%)
Neutrophils/granulocytes (ANC/AGC)	7/34 (20.6%)
Platelets	2/34 (5.9%)
Ear and labyrinth disorders
Pain: Middle ear	2/34 (5.9%)
Endocrine disorders
Cushingoid appearance	5/34 (14.7%)
Eye disorders
Diplopia	3/34 (8.8%)
Gastrointestinal disorders
Anorexia	3/34 (8.8%)
Constipation	4/34 (11.8%)
Diarrhea	9/34 (26.5%)
Distension/bloating, abdominal	2/34 (5.9%)
Dry mouth/salivary gland (xerostomia)	3/34 (8.8%)
Heartburn/dyspepsia	4/34 (11.8%)
Nausea	15/34 (44.1%)
Taste alteration (dysgeusia)	2/34 (5.9%)
Vomiting	21/34 (61.8%)
Pain: Abdomen NOS	6/34 (17.6%)
Pain: Stomach	2/34 (5.9%)
General disorders
Non-functional central venous catheter	13/34 (38.2%)
Thrombosis/embolism - Central venous catheter	2/34 (5.9%)
Fatigue (asthenia, lethargy, malaise)	24/34 (70.6%)
Fever	11/34 (32.4%)
Rigors/chills	3/34 (8.8%)
Weight gain	3/34 (8.8%)
Edema/Fluid retention	8/34 (23.5%)
Infections and infestations
Central venous catheter infection	7/34 (20.6%)
Infection (documented clinically): Lung (pneumonia)	2/34 (5.9%)
Infection (documented clinically): Mucosa	3/34 (8.8%)
Infection (documented clinically): Upper airway NOS	5/34 (14.7%)
Opportunistic infection	4/34 (11.8%)
Investigations
Albumin, serum-low (hypoalbuminemia)	3/34 (8.8%)
Alkaline phosphatase	6/34 (17.6%)
Bicarbonate, serum-low	4/34 (11.8%)
GGT (gamma-Glutamyl transpeptidase)	4/34 (11.8%)
Hypercalcemia	3/34 (8.8%)
Hypercholesteremia	8/34 (23.5%)
Hyperglycemia	13/34 (38.2%)
Hypermagnesemia	2/34 (5.9%)
Hypernatremia	12/34 (35.3%)
Hypertriglyceridemia	4/34 (11.8%)
Hypocalcemia	4/34 (11.8%)
Hypoglycemia	11/34 (32.4%)
Hypokalemia	27/34 (79.4%)
Hypomagnesemia	3/34 (8.8%)
Hyponatremia	4/34 (11.8%)
Hypophosphatemia	5/34 (14.7%)
Proteinuria	10/34 (29.4%)
SGOT	7/34 (20.6%)
SGPT	10/34 (29.4%)
Uric acid, serum-high (hyperuricemia)	5/34 (14.7%)
Musculoskeletal and connective tissue disorders
Pain: Back	3/34 (8.8%)
Pain: Chest wall	2/34 (5.9%)
Pain: Extremity-limb	4/34 (11.8%)
Pain: Joint	5/34 (14.7%)
Pain: Muscle	3/34 (8.8%)
Pain: Neck	4/34 (11.8%)
Nervous system disorders
Hemorrhage, CNS	2/34 (5.9%)
Ataxia (incoordination)	10/34 (29.4%)
Confusion	4/34 (11.8%)
Dizziness	6/34 (17.6%)
Memory impairment	2/34 (5.9%)
Neuropathy: CN III Pupil, upper eyelid, extra ocular movements	2/34 (5.9%)
Neuropathy: cranial: CN VI Lateral deviation of eye	2/34 (5.9%)
Neuropathy: cranial: CN VII Motor-face; Sensory-taste	2/34 (5.9%)
Neuropathy: motor	7/34 (20.6%)
Neuropathy: sensory	2/34 (5.9%)
Seizure	6/34 (17.6%)
Somnolence/depressed level of consciousness	22/34 (64.7%)
Speech impairment	5/34 (14.7%)
Tremor	6/34 (17.6%)
Pain: Head/headache	21/34 (61.8%)
Renal and urinary disorders
Hemorrhage, GU: Bladder	3/34 (8.8%)
Hemorrhage, GU: Urinary NOS	3/34 (8.8%)
Incontinence, urinary	2/34 (5.9%)
Urinary frequency/urgency	6/34 (17.6%)
Respiratory, thoracic and mediastinal disorders
Hemorrhage, pulmonary: Nose	5/34 (14.7%)
Cough	5/34 (14.7%)
Dyspnea (shortness of breath)	6/34 (17.6%)
Skin and subcutaneous tissue disorders
Bruising (in absence of Grade 3 or 4 thrombocytopenia)	2/34 (5.9%)
Pruritus/itching	3/34 (8.8%)
Rash/desquamation	5/34 (14.7%)
Petechiae	3/34 (8.8%)

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.